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TitreDateDurée
The power of tumor infiltrating lymphocytes to fight melanoma06 Sep 202400:38:03

Tumor infiltrating lymphocyte (TIL) therapy uses a person’s own immune cells to fight advanced melanoma, offering new hope for patients who have limited treatment options. 

This week, we have a conversation with Brian Gastman, EVP of medical affairs at Iovance Biotherapeutics, about TILs and the company’s pipeline.

Iovance recently submitted a marketing authorization application to the European Medicines Agency for lifileucel, a TIL cell therapy, for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. 

If approved, lifileucel will be the first and only approved therapy in this treatment setting in all European Union member states.

The submission is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced post-anti-PD1 melanoma.

Iovance’s Amtagvi is the first FDA-approved T cell therapy for a solid tumor indication. 

00:47-04:44: About Iovance Biotherapeutics
04:44-07:57: What is polyclonal tumor infiltrating lymphocyte treatment?
07:57-14:55: What is the production process for TILs?
14:55-18:32: Are there any limiting factors for TIL treatment?
18:32-20:59: Is early intervention important?
20:59-21:22: Does better psychology help?
21:22-22:06: Are other companies working on TILs?
22:06-27:25: Clinical trials 
27:25-29:25: How do you address cost?
29:25-34:21: Iovance’s pipeline
34:21-35:30: Can TILs be improved?
35:30-37:21: Where does the TIL space go from here?

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The future of combination vaccines30 Aug 202400:24:44

The development of combination vaccines could represent a significant advancement in the fight against infectious diseases. With the potential to streamline the vaccination process and provide broader protection, these vaccines could greatly improve public health preparedness.

Moderna is harnessing the power of its mRNA platform to develop vaccines that target multiple respiratory viruses at once, including COVID-19, RSV, and influenza. The breakthrough technology enables the immune system to combat multiple pathogens simultaneously, revolutionizing immunization efforts and enhancing public health preparedness.

To look at combination vaccines, their usefulness and potential for the future, we had an in-depth conversation with Cesar Sanz Rodriguez, vice president, Europe & Switzerland, medical affairs, at Moderna.

00:45-02:53: What is combination vaccine technology?
02:53-05:33: What are the advantages of targeting multiple respiratory diseases simultaneously?
05:33-06:02: Making vaccinations more friendly
06:02-07:57: How does the immune system handle tackling many pathogens at the same time?
07:57-10:23: Is there an effect of efficacy with combination vaccines?
10:23-12:50: How do you manage different times between boosters?
12:50-13:57: Are many companies working on combination vaccines?
13:57-17:45: How easy is it to add vaccines, for example to tackle avian flu, into combination vaccines?
17:45-19:06: Is there a limit to the number of vaccines in a combination vaccine? 
19:06-20:46: How easily can vaccines be updated to address variants?
20:46-22:30: What is the future of combination vaccines?
22:30-23:56: What is in Moderna’s pipeline related to vaccines?

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Priovant - finding options to treat autoimmune diseases28 Jun 202400:30:05

US-based biotech company Priovant Therapeutics is dedicated to developing novel therapies for autoimmune diseases with high morbidity and few available treatment options. 

The company's lead asset, brepocitinib, is a dual selective inhibitor of TYK2 and JAK1. Through dual TYK2/JAK1 inhibition, brepocitinib suppresses key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL6, IL12, and IL23—with a single, targeted therapy. 

Brepocitinib has generated positive data in seven phase 2 studies with oral once-daily administration. It is currently being evaluated in a phase 3 program for dermatomyositis and is entering a phase 3 program for non-infectious uveitis.

This week on the podcast we have a conversation with Priovant’s CEO Benjamin Zimmer about dermatomyositis, non-infectious uveitis, current treatments, potential options, and how brepocitinib is making a difference. 

00:55-02:34: About Priovant
02:34-06:38: What are dermatomyositis and non-infectious uveitis?
06:38-08:35: What are the current treatments?
08:35-12:32: What is brepocitinib?
12:32-15:36: Are there other treatments in development?
15:36-17:50: What is your relationship with Pfizer?
17:50-20:48: Brepocitinib clinical trials
20:48-24:06: Future plans and timeline
24:06-27:59: Finding patients for clinical trials
27:59-29:19: Future opportunities
 
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Beyond Biotech podcast 12: Gate Neurosciences, Oncolyze, 3Brain02 Sep 202200:56:29

2:48 Labiotech.eu news
5:22 Gate Neurosciences
16:18 3Brain
31:13 Oncolyze
50:22 JLL

This week’s podcast guests are Mike McCully, CEO of Gate Neurosciences; Steven Evans, CEO and co-founder of Oncolyze; and Philipp Markolin, scientific marketing specialist at 3Brain.

We also have our weekly chat with global commercial real estate services company JLL, and this week it’s a chat with  Amber Schiada, head of life sciences research in the Americas for the company.

Gate launches to develop CNS treatments

Gate Neurosciences has officially launched to develop its next-generation therapeutics addressing synaptic dysfunction in patients suffering from central nervous system (CNS) disorders.

Gate, a U.S. biotech company, was founded in 2019 by a team of neuroscience industry veterans to address the need for more effective treatments for CNS disorders, and to overcome long-standing challenges in CNS drug development. 

Since its founding, the company has acquired a portfolio of next-generation compounds from large pharma, along with corresponding data from thousands of patients dosed across numerous clinical trials that will inform and de-risk future development.

Oncolyze set for AML clinical trials

After successfully completing in vitro and in vivo experiments, which support the notion that its lead drug candidate OM-301 breaks down cancer cells and increases survival rates, Oncolyze now has its sights set on advancing OM-301 into clinical trials.

Oncolyze has designed a novel anti-cancer drug (OM-301) that targets a particular cell surface protein. This protein, HDM2, is known to exist at high levels inside cancer cells and is thought to be an important driver of cancer growth and metastasis. More recently, though, it was discovered that HDM2 is also found on the cell surface of many types of cancer cells, but not normal cells.

3D microchip gives insights into human brain

A microchip that allows scientists to study the complexity of 3D cellular networks at unrivaled scale and precision has been added to 3Brain AG’s brain-on-chip portfolio.

In collaboration with Swiss precision manufacturing experts, CSEM, 3Brain AG made the announcement last month.

The cell-electronic interface technology will also allow scientists to gain novel mechanistic insights into the inner workings of the most complex structure in the universe, the human brain.


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Beyond Biotech podcast 11: Ilya Pharma, IRB Barcelona, Krystal Biotech, Phase Genomics26 Aug 202200:58:34

2:15 Labiotech.eu news
5:12 Phase Genomics
16:44 IRB Barcelona
29:37 Ilya Pharma
43:00 Krystal Biotech
53:54   JLL

Our guests are Ilya Pharma CEO and co-founder Evelina Vågesjö and chief financial officer Oskar Lund; Miguel Martín-Álvarez, postdoctoral fellow in the cancer science unit at IRB Barcelona; Hubert Chen, head of clinical development at Krystal Biotech; and Ivan Liachko, CEO of Phase Genomics.

We also have a new weekly addition, as JLL will be joining us with a look at financial news. This week, we’re joined by Robert Coughlin, JLL managing director and New England Life Science Practice Group lead.

Study says gene editing with CRISPR/Cas9 can lead to cell toxicity and genome instability


CRISPR allows the introduction of a desired DNA sequence into (virtually) any spot of the genome, thus modifying or inactivating a gene. The technique is widely used in biomedical research and some CRISPR-based therapies are in clinical trials for the treatment of human blood disorders, some types of cancer and HIV.

Scientists at IRB Barcelona, led by ICREA researcher Fran Supek, have now reported that, depending on the targeted spot of the human genome, CRISPR gene editing can give rise to cell toxicity and genomic instability. 

Ilya Pharma finalizes global patent coverage for modified lactic acid bacteria to treat wounds 

Ilya Pharma, a Swedish clinical stage biopharma company focused on delivering local immunotherapies to patients, has announced the issue of four new patents.

These are: one in India, and continuation patents in China, Australia and the U.S. This means the company’s method for using modified bacteria for treatment of both mucosal and cutaneous wounds in humans and animals has patent protection in all major markets.

Ilya is currently developing a portfolio of three first-in-class immunotherapies for skin and mucosal diseases, including ILP100-Topical for treatment surgical wounds in diabetic, prediabetic and obese patients especially at risk, ILP100-Topical for diabetic ulcers and ILP100-Oral for patients with immune mediated enterocolitis resulting from cancer treatment with checkpoint inhibitors.

Krystal Biotech gets green light for cystic fibrosis trial

U.S. company Krystal Biotech is set to start a clinical trial for cystic fibrosis after the United States Food and Drug Administration (FDA) accepted its investigational new drug (IND) application.

The gene therapy company will be evaluating KB407, a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs. 

By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has the potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation.

Phase Genomics using ProxiMeta for phage-bacteria interactions

Phase Genomics, Inc., a developer of proximity ligation-based genomic solutions, recently received $5.5M in combined non-dilutive funding from the Bill and Melinda Gates Foundation and the National Institute of Allergy and Infectious Diseases. With the new funding, Phase Genomics will leverage its phage-bacteria interactome repository to power the development of an  AI-driven predictive engine for therapeutic phage discovery.

The new financing will allow the company to harness its proximity-guided metagenomics platform, ProxiMeta, to develop the world’s largest atlas of phage-bacteria interactions and use machine learning tools to identify phages that can be used in therapeutic settings such as C. difficile infections, ulcerative colitis, and Crohn’s disease. 
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Beyond Biotech podcast 10: Endpoint Health, Nevrargenics, Triastek19 Aug 202200:52:42

This week our guests on the podcast are Xianghao Zuo, deputy director of R&D at Triastek; Endpoint Health CEO Jason Springs; and Andy Whiting, CEO of Nevrargenics.

2:15 News
4:28 Nevrargenics
22:26 Triastek
37:14 Endpoint Health

Triastek and Lilly working together on 3D printing for drugs

A new research collaboration between Chinese company Triastek, Inc. and Eli Lilly and Company will leverage 3D printing technology to achieve precise targeting and programmed drug release in the intestine. 

Triastek has developed 3D printing technology to provide solutions for drug development. According to the agreement, Triastek and Lilly will focus on improving the bioavailability of drugs in the intestine. 

Triastek will first conduct in-depth studies of the excipient – inert ingredients added to a drug product for specific functional roles -- to ensure drug stability is maintained during development and 3D printing. Then, Triastek will identify the optimum 3D structure design for achieving targeted and programmed drug release.

Triastek has already received two investigational drug clearances from the U.S. Food and Drug Administration for its 3D printed drugs, T19 and T20. 

Endpoint Health receives funding for precision immunology pipeline

Endpoint Health, Inc., a therapeutics company dedicated to addressing unmet needs in immune-mediated acute and chronic diseases, recently recveived $52 million in equity and debt financing. 

Proceeds from the financing round are being used to extend the company’s Precision-First platform and expand its therapeutic pipeline to include programs for chronic immune-mediated diseases. 

In addition, proceeds will be used to advance Antithrombin III, the company’s first precision therapy, to a phase II clinical trial for the treatment of sepsis. The company expects to submit its Investigational New Drug application and Investigational Device Exemption to the US Food and Drug Administration for its Antithrombin III program later this year.

Completion of the Series A financing follows Endpoint Health’s announcement of a global collaboration and licensing agreement with Grifols to develop and commercialize Antithrombin III in sepsis.

Nevrargenics

Nevrargenics Ltd is a UK drug discovery and development company focused on innovative and novel medicines for neurodegenerative disease.

Developed through a commercial team, the research aims to bring a series of drug solutions to market and to service disease areas of major unmet need. The focus area of the company is the development of drugs to target neurodegenerative conditions.

Two research groups, based at Aberdeen and Durham universities, have been collaborating for several years, and through this collaboration, have designed, synthesized and identified a lead drug candidate with the potential for establishing new treatment approaches. 

The strategy is to employ new dual-acting retinoic acid receptor modulators (RAR-M) as the basis for developing a series of new rationally designed, effective treatments, meeting that most severe of unmet needs: neurodegeneration.

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Beyond Biotech podcast 9: ARCH Venture Partners, Micropep, PTC Therapeutics, Valneva12 Aug 202200:55:01

This week's guests are ARCH Venture Partners’ managing director Kristina Burow; founder and CEO of Micropep Thomas Laurent; Kylie O’Keefe, SVP, commercial & corporate strategy at PTC Therapeutics, and Juan Carlos Jaramillo, chief medical officer of Valneva.

1:40 News
3:47 PTC
18:32 Micropep
33:01 Valneva
42:12 ARCH

ARCH Venture Partners closes $2.97B biotech fund

ARCH Venture Partners has announced the closing of ARCH Venture Fund XII, with $2.975 billion to invest in the creation, development and funding of early stage biotechnology companies. 

ARCH Venture Partners is one of the largest early-stage technology venture firms in the U.S. It invests primarily in companies it co-founds with leading scientists and entrepreneurs, bringing innovations in life sciences and physical sciences to market.

Upstaza gets EC green light

PTC Therapeutics, Inc. says Upstaza (eladocagene exuparvovec) has been given marketing authorization by the European Commission. 

Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency, and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.

Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications.

The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.  

Micropep raises €8.75M to develop alternative crop-protection products

French ag-biotech company Micropep, which develops biological micropeptide-based solutions for agriculture, has completed an €8.75 million ($9.3 million) series A1 financing round. 

Based in Toulouse, Micropep has developed a technological platform that makes it possible to precisely regulate a plant’s gene expression, without altering its DNA, by the exogenous application of micropeptides, a family of proteins naturally produced by plants to respond to stress and regulate their development.

The new investment also marks the first step towards the commercialization of Micropep’s products. Micropep is using this funding to accelerate the development phase of its first micropeptide candidates in the U.S. and will build a locally-recruited team to deploy its solutions. 

Pfizer and Valneva to start phase 3 Lyme disease vaccine study

Pfizer Inc. and Valneva SE have initiated a phase 3 clinical study, Vaccine Against Lyme for Outdoor Recreationists (VALOR), to investigate the efficacy, safety and immunogenicity of their investigational Lyme disease vaccine candidate, VLA15.

“With increasing global rates of Lyme disease, providing a new option for people to help protect themselves from the disease is more important than ever,” said Annaliesa Anderson, senior vice president and head of vaccine research and development at Pfizer. 

Juan Carlos Jaramillo, chief medical officer of Valneva, said, “We are extremely pleased to reach this important milestone in the development of VLA15. Lyme disease continues to spread, representing a high unmet medical need that impacts the lives of many in the northern hemisphere. We look forward to further investigating the VLA15 candidate in phase 3, which will take us a step closer to potentially bringing this vaccine to both adults and children who would benefit from it.”

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Beyond Biotech podcast 8: Biotech Growth Trust, Deep Science Ventures, Endogena Therapeutics, eureKARE05 Aug 202201:00:51

This week’s podcast guests are Laura Fletcher, head of business development and strategic partnerships at Deep Science Ventures; Matthias Steger, CEO of Endogena Therapeutics; Rodolphe Besserve, CEO, and Georges Rawadi, chief of biotech studio development, at eureKARE; and Biotech Growth Trust portfolio manager, Geoff Hsu.

News: 02:30
Endogena Therapeutics 5:10
eureKARE 21:00
Deep Science Ventures 33:46
Biotech Growth Trust 47:44

Endogena Therapeutics Inc. has announced the first patient has been treated in a phase 1/2a clinical study of its lead product, EA-2353, a photoreceptor regeneration treatment for retinitis pigmentosa (RP).

RP is a group of inherited diseases causing slow and progressive retinal degeneration and loss of vision, for which there is currently no treatment for most patients. It is a leading cause of inherited blindness, with an estimated 1.5 million people worldwide presently affected.

eureKARE, an investment company focused on financing and building next generation biotech companies in the fields of synthetic biology, has launched its first synthetic biology studio in Belgium. 

eureKARE is developing a pan-European biotech studio network covering several areas where synthetic biology can create solutions and high market value products.

The synthetic biology studios will focus on biomedical and other thematic applications in different European innovation hotspots. The strategy is based on identifying, selecting, and nurturing high-quality European science. 

The venture builder Deep Science Ventures (DSV) has expanded an existing alliance with Cancer Research UK’s innovation-focused arm Cancer Research Horizons, with the mission to establish new oncology startups in the U.K.

Just months after Cancer Research UK (CRUK) established its industry-orientated division Cancer Research Horizons, the research charity has doubled down on a collaboration with DSV, a U.K. venture builder operating in a range of sectors including biotech, agriculture and computation. 

According to the latest agreement, the partners will co-found startups working in up to 10 areas of oncology, such as improving the number of patients that can benefit from immuno-oncology treatments.

The Biotech Growth Trust PLC, based in the UK, seeks capital appreciation through investment in the worldwide biotech industry. It invests in a diversified portfolio of shares and related securities in companies on a worldwide basis.

Its portfolio is operated by U.S.-headquartered OrbiMed Capital LLC. 

Following the strong results of the previous year, the company’s NAV (net asset value) per share total return dropped by 33.8%, compared to a 55.1% increase in 2021, and the share price total return was down 37.0%. 

However, ​​overall, since OrbiMed’s appointment in 2005, and to the year end, The Biotech Growth Trust’s NAV has increased by 861.6% or 14.3% per annum.

In its annual report, the portfolio manager stated the company remains confident that there are a number of potential catalysts that could lead to a recovery in the biotechnology sector this year and into the future, and their overall investment strategy remains unchanged.

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Beyond Biotech podcast 7: World Hepatitis Day29 Jul 202200:45:18

This week's podcast features three interviews related to hepatitis.

Our guests are Ahmed Elsharkawy, consultant transplant hepatologist and honorary senior clinical lecturer at the University of Birmingham in the UK; Ziv Ben-Ari, director of the Liver Diseases Center at the Sheba Medical Center in Israel, and James McIlroy, chief executive officer at EnteroBiotix.

World Hepatitis Day

Taking place on July 28 every year, World Hepatitis Day unites the world under a single theme to raise awareness of the global burden of viral hepatitis with the intention of influencing real change. This year’s theme is ‘I Can’t Wait’.

Dr Ahmed Elsharkawy is a consultant transplant hepatologist and honorary senior clinical lecturer at the University of Birmingham and he is also chair of hepatitis B virus special interest group which is part of the British Association for the Study of the Liver. He is a strong supporter and advocate of World Hepatitis Day. 

Professor Ziv Ben-Ari is director of the Center for Liver Diseases and Liver Research Laboratory at the Sheba Medical Center in Israel and Professor of Medicine at Tel-Aviv University.

She has more than 25 years of experience in evaluating candidates for liver transplantation and monitoring liver transplants including five years as a fellow in Hepatology and Liver Transplantation at the Royal Free Hospital, London.  She has chaired the Israeli Liver Research Society, chairs the Liver Committee of the National Council of Gastroenterology and is an active member of the American and European Association for the Study of the Liver. She has published more than 175 studies in leading medical journals.

Dr James McIlroy is a qualified medical doctor and biopharmaceutical entrepreneur widely recognized as a pioneer and thought leader in the application of the microbiome to treat disease.

He founded EnteroBiotix with the vision of building one of the world’s leading microbiome drug development companies.

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Beyond Biotech podcast 6: Mogrify, +ND Capital, Poolbeg Pharma22 Jul 202200:46:05

On this week’s podcast, our guests are Jeremy Skillington, CEO of Poolbeg Pharma; Dani Bach, and Eric Moessinger, both partners at +ND Capital; and Louise Modis, chief scientific officer at Mogrify.

Poolbeg Pharma, a clinical stage infectious disease pharmaceutical company, has received ethics and competent authority approval to commence its lipopolysaccharide (LPS) human challenge clinical trial for POLB 001, a strain agnostic, small molecule immunomodulator which aims to address the unmet medical need for a treatment for severe influenza.

Biopharma company Mogrify Limited, and Japanese regenerative medicine firm Astellas Pharma Inc., have announced a collaborative research agreement on in vivo regenerative medicine approaches to address sensorineural hearing loss.

Belgian headquartered biotech Galapagos NV has acquired CellPoint and AboundBio, a move the Belgian company said propels it into next-generation cell therapy.

The deal saw the Lausanne-based team of US venture capital firm, +ND Capital, mark its second successful exit in the European biotech market. 

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Beyond Biotech podcast 5: ANeuroTech, ISA Pharmaceuticals, Life Length14 Jul 202200:58:17

This week’s guests are Erik Buntinx, CEO and founder of ANeuroTech; Stephen J. Matlin, CEO of Life Length; and Kees Melief, chief scientific officer at ISA Pharmaceuticals.

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Beyond Biotech podcast 4: CorePath, Rensselaer, Versameb14 Jul 202200:46:51

This week’s guests are Erik Buntinx, CEO and founder of ANeuroTech; Stephen J. Matlin, CEO of Life Length; and Kees Melief, chief scientific officer at ISA Pharmaceuticals.

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Beyond Biotech podcast 3: Ariceum Therapeutics, TWB, Unilever01 Jul 202200:41:30

On this week’s Labiotech.eu podcast, we have three guests.

We had conversations with Manfred Rüdiger, CEO of Ariceum Therapeutics; Jon Hague, Unilever’s R&D VP for homecare; and Olivier Rolland, executive director at TWB.

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How ADCs can deliver death cap mushroom toxins to treat cancer21 Jun 202400:28:09

Heidelberg Pharma is a clinical stage biotech company developing antibody drug conjugates (ADCs).

Its lead amanitin-based ADC product candidate, HDP-101, targets relapsed or refractory multiple myeloma. HDP-101 recently received orphan drug designation; it is currently in a phase I/IIa clinical trial, demonstrating first signs of clinical efficacy. 

The company is also expanding its therapeutic product pipeline with a further four programs across a variety of malignant hematologic and solid tumors.

To tell us about how HDP-101 works, and what Heidelberg Pharma is doing in the space, we had a conversation with the company’s CEO, Prof. Andreas Pahl.

00:55-02:05: About Heidelberg Pharma
02:05-03:41: What are antibody-drug conjugates?
03:41-05:27: Why are antibody-drug conjugates in the news currently?
05:27-06:15: What is big pharma’s involvement in the field?
06:15-07:04: Will there be more antibody-drug conjugate approvals?
07:04-08:42: What can antibody-drug conjugates be used to treat?
08:42-09:48: How do you avoid toxicity issues?
09:48-10:31: How important is the linker?
10:31-12:06: What is alpha-amanitin?
12:06-13:28: How do you make a toxin not toxic?
13:28-14:11: What tumor types can be targeted?
14:11-15:59: How do you discover a toxin can treat a disease?
15:59-17:38: How is alpha-amanitin delivered?
17:38-19:27: Is alpha-amanitin a monotherapy?
19:27-22:16: What clinical trials are you doing?
22:16-23:31: What are the next steps now you have orphan drug designation?
23:31-24:14: What are the timelines?
24:14-25:36: Where are antibody-drug conjugates headed?
25:36-27:14: Are AI and machine learning making an impact?

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Beyond Biotech podcast 2: Asabys, Asebio, MOA Foodtech24 Jun 202200:39:13

This week’s podcast is dedicated to Spain.

We have conversations with bioindustry association AseBio’s CEO, Ion Arocena; Clara Campàs, managing partner and co-founder of venture capital company Asabys, and Susana Sánchez Gómez, co-founder and chief scientific officer at MOA Foodtech.

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Beyond Biotech 01: June 17, 202217 Jun 202200:48:53

Three interviews on the show this week, with Ros Deegan, CEO of OMass; David Solomon, CEO of PharNext; and two guests from Forbion - Sander Slootweg, managing partner, and Dirk Kersten, general partner. 
And a quick look at the news from the past seven days.

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Beyond Biotech: Preview10 Jun 202200:06:09

A quick preview of the new, weekly podcast on what's new in the world of biotech, from Labiotech.eu

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Looking to cure Type 1 diabetes14 Jun 202400:23:40

Diamyd Medical develops precision medicine therapies for the prevention and treatment of Type 1 diabetes and LADA (latent autoimmune diabetes in adults). 

Diamyd is an antigen-specific immunomodulatory therapeutic for the preservation of endogenous insulin production. It has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation. 

DIAGNODE-3, a confirmatory phase 3 trial, uses injections into a superficial lymph node can to optimize the treatment response. 

This week on the podcast we have a conversation about treatments of type 1 diabetes, including the company’s development of Diamyd. Our guest is Anders Essen-Möller, founder and chairman of Diamyd Medical.

00:51-19:16: Diamyd Medical’s approach to Type 1 diabetes
19:16-21:03: Other approaches to tackling Type 1 diabetes
21:03-22:08: Keeping costs of Type 1 diabetes treatment low
22:08-22:53: Can Type 1 diabetes be cured?

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Developing treatments for rare pediatric diseases07 Jun 202400:29:17

Rare diseases affect around 1 in 2,000 people. Many of the thousands of rare diseases are difficult to diagnose. Of the known rare diseases, about 80% have genetic causes.  

Leukodystrophies are a group of rare genetic disorders for which there is currently no curative therapy.

This week on the podcast we are discussing novel treatments of rare pediatric diseases in a conversation with Dan Williams, CEO, executive director and co-founder of Synaptix Bio. Synaptix Bio is working on developing treatments for severe leukodystrophies. 

The company has partnered with the Children’s Hospital of Philadelphia to take a TUBB4a leukodystrophy therapy to the clinic and beyond. 

01:41-05:08: About Synaptix Bio
05:08-07:07: What are rare diseases?
07:07-09:36: How do you develop treatments for rare diseases?
09:36-11:15: How many rare diseases have treatments?
11:15-13:32: What is the space looking like for companies looking to develop rare disease treatments?
13:32-14:10: How does the fact that many rare diseases are genetic affect drug development?
14:10-16:21: Do the low numbers of people with rare diseases affect funding and research? 
16:21-17:55: Are the drugs being developed useful in treating other diseases?
17:55-22:44: What is H-ABC?
22:44-23:25: Are there any treatments for H-ABC? 
23:25-25:15: Where is Synaptix Bio with clinical trials?
25-15-27:40: How do you design clinical trials for H-ABC with the number of patients being low?

This week’s podcast was sponsored by KBI Biopharma. Visit their facility in Geneva, Switzerland, on June 27, to join the discussion on solving biotherapeutic challenges. Register at kbibiopharma.com/conferences.

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Can cell therapy tackle hearing loss?31 May 202400:31:00

Hearing loss is a huge – and growing – global challenge.

Using its OSPREY platform, UK-based Rinri Therapeutics is developing a portfolio of off-the-shelf regenerative cell therapy products to address sensorineural hearing loss, a significant global medical challenge. 

The company’s lead asset, Rincell-1, is designed to regenerate auditory neurons and provide a novel treatment for patients with severe-to-profound age-related hearing loss or auditory neuropathy. The company is looking to start first-in-human trials in 2025.

Today, on the podcast we have a conversation with Rinri CEO Simon Chandler about the different kinds of hearing loss, what’s being done to tackle them, and Rinri’s novel approach to treatments.

01:08-03:19: About Rinri Therapeutics
03:19-05:16: About hearing loss
05:16-07:31: Mental health and hearing loss
07:31-10:22: The different kinds of hearing loss
10:22-11:37: The importance of early intervention
11:37-13:46: What is Rinri doing to tackle hearing loss?
13:46-15:58: The difficulty of accessing the cochlea
15:58-19:12: Is your treatment a cure for hearing loss?
19:12-20:45: Would repeat treatment be necessary?
20:45-23:30: Are other companies working on hearing loss?
23:30-25:27: The cost of treating hearing loss
25:27-27:13: The economic costs of hearing loss
27:13-28:33: Rinri treatment timelines
28:33-30:15: The rest of Rinri’s pipeline

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How biotech is tackling myasthenia gravis24 May 202400:28:50

June is myasthenia gravis awareness month. Myasthenia gravis is a chronic autoimmune disorder where antibodies destroy the communication between nerves and muscle, and this results in weakness of the skeletal muscles. 

It especially affects the voluntary muscles of the eyes, mouth, throat and/or limbs. It is most frequent in women from 20 and 30 and men aged 50 and older. 

To talk about the disease, how it is being treated, and what is in the pipeline for future treatments, we had a conversation with Dana Vigier, head of Central Eastern and Northern Europe at Alexion.

01:07-04:26: Background on Dr Dana Vigier
04:26-07:07: What is myasthenia gravis?
07:07-09:43: What is the prognosis for someone diagnosed with myasthenia gravis?
09:43-11:33: How effective are current treatments for myasthenia gravis?
11:33-12:56: Do all the myasthenia gravis drugs work in the same way? 
12:56-18:35: What R&D is taking place in myasthenia gravis?
18:35-21:14: Alexion and its work on myasthenia gravis
21:14-22:01: Myasthenia gravis clinical trials
22:01-23:58: The importance of myasthenia gravis awareness month
23:58-25:31: Would early diagnosis make a difference?
25:31-27:54: Transformation through new treatments

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Antisense oligonucleotides17 May 202400:27:22

German-headquartered Isarna Therapeutics is developing a portfolio of antisense therapies targeting an emerging therapeutic field in human biology – transforming growth factor beta (TGF-B) signaling.

Precise modulation of TGF-B pathways using antisense therapy may result in safer and more effective treatment options for a broad range of indications.

The company’s lead compound, ISTH0036, is in clinical development in ophthalmology.

On the podcast this week we chat with René Rückert, chief operating officer at Isarna Therapeutics, who explains TGF-B and antisense oligonucleotides’ role in tackling disease.


00:59-02:45: About Isarna Therapeutics
02:45-05:05: What is transforming growth factor beta?
05:05-06:16: Why is TGF a good target?
06:16-09:00: What are antisense oligonucleotides?
09:00-10:02: What are antisense oligonucleotides useful for treating?
10:02-11:21: What advantages do antisense oligonucleotides have?
11:21-13:00: How can antisense oligonucleotides be delivered?
13:00-14:58: Are antisense oligonucleotides still being researched?
14:58-16:31: Are there challenges with using antisense oligonucleotides?
16:31-16:56: Are many companies working on antisense oligonucleotides?
16:56-18:06: Are antisense oligonucleotides best used as a monotherapy or with other treatments?
18:06-20:42: What is Isarna’s pipeline?
20:42-22:47: How does your AMD treatment work?
22:47-23:50: What is the timeline for your treatment?
23:50-26:34: What can patients expect?


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Making waves in cardiovascular disease treatment10 May 202400:21:02

Cytokinetics is a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which cardiac muscle performance is compromised.

This week, we have a conversation with the CEO of Cytokinetics, Robert Blum, about the company’s aficamten, its next-in-class cardiac myosin inhibitor, prior to results from a phase 3 clinical trial in obstructive hypertrophic cardiomyopathy. 


00:54-02:32: About Cytokinetics
02:32-04:11: What are the current treatments for cardiovascular conditions?
04:11-05:44: About cardiovascular disease
05:44-08:59: What is aficamten?
08:59-09:12: How is it administered?
09:12-10:04: Where is aficamten on the path to commercialization?
10:04-12:06: What does this mean for patients?
12:06-12:42: What will you be presenting in Portugal?
12:42-16:06: Could aficamten have other applications?
16:06-16:50: What is CK-136?
16:50-18:02: What is omecamtiv mecarbil?
18:02-20:26: Cytokinetics’ business model


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New ways to treat Duchenne muscular dystrophy03 May 202400:23:56

Duchenne muscular dystrophy (DMD) is a genetic disorder with progressive muscle degeneration and weakness due to changes in the protein dystrophin, which helps keep muscle cells intact. 

There are few treatments for DMD, although recent advances mean that males – who are primarily affected by DMD – can now sometimes survive into their 30s.

More drugs are being developed in the fight against DMD, and this week we had a conversation with Frank Gleeson, CEO of Satellos Bioscience, about recent work on addressing DMD, including the company’s own work in the field.


00:47-02:01: About Satellos Bioscience
02:01-04:33: What is Duchenne muscular dystrophy?
04:33-05:43: What are the challenges in treating Duchenne muscular dystrophy?
05:43-09:08: What are the new treatments for Duchenne muscular dystrophy?  
09:08-11:20: What is your treatment for Duchenne muscular dystrophy?
11:20-13:42: Are there different approaches to treating Duchenne muscular dystrophy?
13:42-14:06: How is your treatment delivered?
14:06-17:32: How important is early intervention?
17:32-18:56: Where is Satellos at with clinical trials?
18:56-20:41: Preclinical trial results
20:41-21:44: Outreach to the Duchenne muscular dystrophy community
21:44-23:12: Is a cure for Duchenne muscular dystrophy possible?


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Overcoming clinical trial challenges26 Apr 202400:26:00

This week, we have a conversation about clinical trial design with Gen Li, the president and founder of Phesi, a global provider of patient-centric data analysis. 

Earlier this year, Phesi’s global analysis of all clinical trials conducted in 2023 revealed that more than a quarter (28%) of trials were cancelled during phase II – above the average attrition rate of 20% before 2020. 


00:39-01:31: About Phesi
01:31-01:49: Is your database global?
01:49-03:08: How successful are the different phases of clinical trials?
03:08-04:29: What are the biggest challenges for clinical trials currently?
04:29-06:23: Are clinical trials improving?
06:23-08:14: How can data improve clinical trials?
08:14-10:47: How does artificial intelligence affect clinical trial design?
10:47-12:53: Can clinical trial costs be reduced?
12:53-15:15: Can clinical trial times be shortened? 
15:15-18:21: Can data help with clinical trial diversity?
18:21-19:46: How can you fill knowledge gaps?
19:46-22:01: Do you have less data for rare diseases?
22:01-23:28: How does your company help with clinical trial design?
23:28-25:12: What kind of solutions can you provide?


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New German biotech spins out to develop radiotherapeutics23 Aug 202400:18:35

Pentixapharm is a clinical-stage radiopharmaceutical development company targeting a range of diseases. 

While it is currently owned by the Eckert & Ziegler Group, it will soon be spun off as a separate company. Pentixapharm’s clinical pipeline includes PENTIXATHER, am Yttrium-90 based therapeutic against CNS lymphoma, and PENTIXAFOR, a Gallium-68 based companion diagnostic. Additionally, PENTIXAFOR is being developed as a diagnostic tool for primary aldosteronism (PA), a significant cause of hypertension.

Recently, the company announced the acquisition of the target discovery business of Glycotope.

The deal includes a portfolio of preclinical antibodies against multiple oncology targets that can be developed into radiopharmaceuticals. It also includes Glycotope’s laboratories, cell banks, tumor target data base, and the equipment needed to exploit the discovery platform, along with a range of patents, licenses, and other tangible assets. 

This week, we had a conversation with Andreas Eckert, founder and chairman of the supervisory board at Pentixapharm.

00:57-03:07: About the Pentixapharm spinout
03:07-05:19: What is Pentixapharm’s pipeline?
05:19-06:01: What is the CXCR4 ligand approach?
06:01-08:51: What are the development plans for tackling primary aldosteronism? 
08:51-10:28: Is the process fast, and what about cost?
10:28-11:47: The bigger economic picture
11:47-14:12: About Glycotope
14:12-14:44: How synergistic are Pentixapharm and Glycotope?
14:44-15:32: Is the entire company being acquired?
15:32-16:27: Will outsourcing still be important to the company?
16:27-17:55: What does the future hold for Pentixapharm?

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The biotech investment landscape in 202419 Apr 202400:28:32

RTW is a global multi-strategy investment firm focused on supporting innovative biotech and biopharma companies across the US, Europe, and China. 

The company invests in both public and private companies across their entire life cycle, with a focus on those addressing next-generation gene and RNA therapies, rare diseases, targeted oncological, cardiovascular, and neurological disorders, and more.


To get an overview of what’s happening in financing for biotech companies currently, we had a conversation with Stephanie Sirota, chief business officer of RTW Investments.


01:11-04:18: About RTW Investments
04:18-06:45: What kind of biotech companies do you invest in?
06:45-08:31: How do you evaluate biotech companies?
08:31-11:36: How hands-on are you with companies you invest in?
11:36-14:08: How can companies raise funds currently?
14:08-16:05: Are there any undervalued sectors in biotech?
16:05-17:52: How important are M&As?
17:52-18:30: Is there a trend with M&As?
18:30-19:46: What is the state of the market with IPOs?
19:46-21:21: What are the major trends in biotech in 2024?
21:21-22:17: How could the US elections affect biotech funding?
22:17-24:41: What is the royalties market?
24:41-26:12: Are there regional differences?
26:12-27:52: Getting good biotech deals today


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Redefining chronic pain treatment12 Apr 202400:35:42

Doloromics is a precision neuropharmaceuticals company. It was set up four years ago, to use newer technologies, particularly in the omics space, to take advantage of different single-cell transcriptomic technologies or proteomic technologies to better understand the progression of neurological diseases like chronic pain.

The Doloromics pipeline is built upon a proprietary discovery and validation platform DOLOReS. 

Jackson Brougher, chief scientific officer and co-founder of Doloromics, is our guest on this week’s Beyond Biotech podcast.


00:46-04:41: About Doloromics
04:41-07:09: What is chronic pain?
07:09-08:30: How challenging is the subjectivity of pain?
08:30-11:04: What is nociception?
11:04-12:23: The challenges of treating pain
12:23-16:35: Is chronic pain a disease?
16:35-19:57: What is VX-548?
19:57-22:17: Monoclonal antibodies and CGRPs
22:17-26:22: How have recent advances in genomic technologies advanced the understanding of pain?
26:22-31:03: What is the DOLOReS platform?
31:03-32:37: Delivery methods
32:37-33:55: Keeping costs low
33:55-34:58: Next steps


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Colossal aims to bring back mammoth by 202805 Apr 202400:38:24

Based in Austin, Texas, biotech Colossal Biosciences is at the forefront of de-extinction, critically endangered species protection and the repopulation of critical ecosystems. 

Its mission is to use CRISPR technology to bring back extinct animals, such as the mammoth, the thylacine, and the dodo.

Since DNA was recovered in 2021 from mammoths frozen in the Arctic tundra, the company has been working on splicing bits of the recovered DNA into the genome of its closest relative, the Asian elephant, as it shares 99.6% of its DNA. 


Ben Lamm, co-founder and CEO of Colossal Biosciences spoke with us about the company’s plans.


01:03-02:57: Introduction to Ben Lamm
02:57-04:26: About Colossal Biosciences
04:26-09:35: What techniques are used to tackle de-extinction?
09:35-13:48: What are the biggest challenges?
13:48-14:57: What tools do you need to ‘build’ an extinct species? 
14:57-19:17: How do you address not adversely affecting ecosystems?
19:17-22:22: How do you take on negativity over your de-extinction work?
22:22-23:26: Reintroducing existing species into previous habitats?
23:26-24:06: Does this extend to critically endangered species?
24:06-25:54: Why is there a lot of interest in de-extinction?
25:54-28:10: How do you ensure we don’t repeat the same patterns of extinction?
28:10-29:38: Can you address the loss of current species?
29:38-30:35: How can you help solve the loss of species?
30:35-33:48: Addressing the challenges of introducing different species?
33:48-35:22: Transparency through documentaries
35:22-37:27: 2028 goal for mammoth calves


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New treatments being developed for schizophrenia22 Mar 202400:25:31

It would appear that 2024 is shaping up to be a big year for the development of treatments for schizophrenia, with several clinical trials under way.

Recently, Boehringer Ingelheim and Sosei Group Corporation (Sosei Heptares) announced a global collaboration and exclusive option-to-license agreement. This is to develop and commercialize Sosei Heptares’ portfolio of first-in-class GPR52 agonists, a novel G protein-coupled receptor (GPCR) target. The goal is to improve patient outcomes by addressing the positive, negative, and cognitive symptoms of schizophrenia.

Clíona MacSweeney, Sosei Heptares’ GPR52 program leader, spoke with us about that partnership, as well as other developments in the field of schizophrenia treatments.


00:57-03:44: What is schizophrenia?
03:44-04:52: What are the current schizophrenia treatments?
04:52-06:27: Why is schizophrenia challenging to treat?
06:27-07:34: Why is 2024 a big year for schizophrenia treatments?
07:34-08:56: Seratonin and dopamine modulation
08:56-10:46: How does muscarinic modulation work?
10:46-12:35: Excitatory and inhibitory tone pipeline 
12:35-14:12: Digital therapeutic and epigenetic modulation
14:12-16:12: GPR52 agonists
16:12-17:53: How are GPR52 agonists different?
17:53-19:03: Are there lots of schizophrenia treatments coming?
19:03-20:10: Will some new schizophrenia treatments be used in conjunction with existing treatments?
20:10-20:49: Is a schizophrenia cure possible?
20:49-21:22: Treatment frequency
21:22-22:21: Where is Sosei Heptares with clinical trials for schizophrenia?
22:21-22:45: What else is in Sosei Heptares’ pipeline?
22:45-23:35: Sosei Heptares becoming Nxera
23:35-24:42: Schizophrenia conferences


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Reformulating psychedelics for neurodegenerative diseases15 Mar 202400:41:37

There is a clear demand for novel, neurological treatments. While it’s widely known psychedelics hold enormous therapeutic potential, there are a host of challenges, including dosing and potential side effects for the patient.

Our guest this week is Dr Sam Clark, who founded Terran Biosciences to develop a safe, effective portfolio of therapeutics and technologies for patients with neurological and psychiatric diseases. 


00:52-04:09: About Terran Biosciences
04:09-06:03: What are the challenges of using psychedelics?
06:03-07:32: Is there still a stigma attached to use of psychedelics? 
07:32-11:45: How do psychedelics work on the brain?
11:45-13:44: Why are psychedelics a good treatment?
13:44-16:51: What is Idazoxan XR?
16:51-18:01: How do you make changes to drugs?
18:01-21:19: How did you develop your business model?
21:19-22:40: What are Eplivanserin and Volinanserin?
22:40-24:49: How useful are repurposed drugs?
24:49-29:06: Keeping drugs affordable
29:06-30:31: Dealing with patents
30:31-32:41: What are empathogens?
32:41-33:12: Where is the company at with clinical trials?
33:12-35:45: What is NM-101?
35:45-37:47: How does Terran differ from other companies?
37:47-39:54: Are you looking to cure conditions?
39:54-40:48: Schizophrenia

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How AI immune system mapping can boost drug discovery08 Mar 202400:28:46

Immunai is mapping the immune system at unprecedented scale and granularity. The map, paired with machine learning, looks at how the immune system will respond to drug targets, offering an affordable way to prevent expensive drug failures. 

The ultimate goal is to market immune treatments for diseases like cancer faster than ever before. 

In this week’s conversation, Noam Solomon, CEO and co-founder of Immunai, covers the data gap in drug discovery and how machine learning (ML) can solve it, how to de-risk early-stage drug discovery, predictions for AI, and more.

00:41-01:05: About Immunai
01:05-01:37: Why map the immune system?
01:37-02:36: Are you taking a step back to study the problem in order to move forward?
02:36-03:41: How difficult is it to map the immune system?
03:41-05:21: What is your AMICA platform?
05:21-07:16: Where does your data come from? 
07:16-09:01: How do you account for differences between patients?
09:01-11:27: What are the biggest challenges to drug development?
11:27-13:59: How can AI improve drug development?
13:59-14:47: Will AI advances speed up drug development?
14:47-15:58: Is the use of AI applicable in all diseases and conditions?
15:58-17:40: What sets your approach apart from other companies using AI?
17:40-18:46: What partnerships does Immunai have? 
18:46-20:16: What are pharma companies looking for from Immunai?
20:16-23:09: How can AI help with clinical trials?
23:09-24:24: Can AI help with preventative care?
24:24-26:22: Google Maps for the immune system
26:22-27:10: What will we see from AI in drug discovery in the short term? 
27:10-27:58: What are the next steps for Immunai?

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Is Tome writing the final chapter in genomic medicine?01 Mar 202400:33:11

Tome Biosciences recently exited stealth mode with almost a quarter of a billion dollars in funding. The company has developed a new approach to gene editing, programmable genomic integration (PGI), which it says represents the final chapter in genomic medicines. 

We discussed the company’s formation and approach with the CEO and president of Tome, Rahul Kakkar. 


00:45-04:30: About Tome Biosciences
04:30-05:37: Is there still a role for CRISPR-Cas9?
05:37-07:41: Raising funds for Tome
07:41-11:15: What are the benefits of PGI?
11:15-12:54: How do you find and change the genetic code?
12:54-15:04: What is the difference between integrase-mediated PGI and ligase-mediated PGI? 
15:04-16:41: Are there dangers with these techniques? 
16:41-17:30: Is the editing reversible?
17:30-20:11: What diseases or conditions will you be addressing?
20:11-21:38: Is success considered to be curing diseases?
21:38-25:16: How will you address costs and scalability?
25:16-29:03: Can you explain this being the final chapter in genomic medicine?
29:03-30:24: How will you fine tune these techniques?
30:24-31:24: What was the reason for the takeover of Replace Therapeutics?
 31:24-32:26: What are the next steps?

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Transforming treatments for epilepsy23 Feb 202400:41:27

After decades of lagging nearly many other disease areas, neurology is finally seeing signs of significant innovation. 

One of the biggest reasons it’s trailed behind has been the difficulty in teasing out complex neurobiology – an understanding necessary to design novel medicines. Rapport Therapeutics is playing a significant role in turning the tide. 

Rapport creates precision neuromedicines that act only in the brain regions where disease originates, avoiding the side effects that affect many of today’s treatments.


This week, we have a conversation with Rapport CEO Abe Ceesay, about the company’s transformational approach, and about what’s new in neuromedicine.


00:45-02:43: About Rapport
02:43-05:01: What attracted you to Rapport?
05:01-09:13: What are the issues in developing drugs in neuroscience?
09:13-12:09: What are the areas with unmet needs?
12:09-16:24: What are receptor-associated proteins?
16:24-19:25: How do you avoid side-effects?
19:25-20:35: Are other companies working in this space?
20:35-23:49: How do you approach designing treatments for different conditions?
23:49-26:10: Where is Rapport currently with clinical trials?
26:10-28:26: How do you define success in your trials?
28:26-30:27: Is your treatment used in conjunction with others?
30:27-32:45: How is your treatment administered?
32:45-36:10: Why is there increased interest in developing drugs for neurological disorders?
36:10-38:20: Looking to the future
38:20-40:44: What are the next steps for Rapport?


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How does AI assist drug discovery?16 Feb 202400:38:26

Artificial intelligence (AI) is certainly in the news constantly; however, it’s been used in drug discovery for some time.

A new collaboration between artificial intelligence drug discovery company Insilico Medicine and University of Toronto biochemist and molecular geneticist Igor Stagljar will test AI-designed molecules against "undruggable" cancer targets. 


The research will test 15 to 20 undruggable targets - but are they undruggable? And how does AI work in the drug discovery process?


This week, we have a conversation with Kyle Tretina, Alliance Manager of AI Platforms at Insilico Medicine, on a wide range of subjects including drug discovery, undruggable targets, the collaboration with the University of Toronto, and more.


00:58-05:11: About Insilico Medicine
05:11-06:09: Why is AI in the news?
06:09-07:39: Helping people through AI
07:39-09:10: What is Insilico Medicine doing with AI?
09:10-10:15: Does Insilico Medicine take drugs from idea to trials?
10:15-11:32: How do your partnerships come about?
11:32-19:34: How does drug development start with AI?
19:34-24:43: Can AI address undruggable targets?
24:43-25:05: What do you need to do after finding a potential drug?
25:05-27:57: Can quantum computing aid drug development?
27:57-30:13: How can AI help reduce costs and save time?
30:13-32:56: What is your partnership with the University of Toronto?
32:56-36:24: What is the timescale for introducing drugs from AI?
36:24-37:29: What conditions are you working on?


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Biosimilars and what’s new in cell and gene therapies09 Feb 202400:37:17

This week on the podcast, we have a conversation with Fran Gregory, vice president of Emerging Therapies at Cardinal Health.

The discussion covers the company’s role in healthcare, biosimilars, the latest in cell and gene therapies, and what’s new at Advanced Therapies Week.

00:40-02:30: About Cardinal Health
02:30-05:12: What is Cardinal Health’s role in industry?
05:12-06:11: What are the challenges of logistics?
06:11-07:20: How important is innovation to Cardinal Health?
07:20-12:26: How can Cardinal Health help to reduce costs?
12:26-13:08: Keeping up with global healthcare
13:08-19:46: How do you stay in touch with what’s new in drug discovery?
19:46-22:07: Biosimilars Report
22:07-27:15: What is the current state of the cell and gene therapy space?
27:15-29:57: Are there interesting recent advances in the cell and gene therapy?
29:57-33:38: Advanced Therapies Week
33:38-36:40: What trends are you expecting in 2024?

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Challenging the European biotech funding status quo16 Aug 202400:36:36

There are many questions when it comes to setting up a biotech company, and raising funds.

Currently, many companies are concerned about fundraising, but there are things that can be done to maximize efforts. Does a company’s location affect fundraising? What is the best approach to successfully raise funds? What are the current trends in biotech funding?

To look at questions surrounding the state of European biotech funding, and how it compares with the global scene, intellectual property, and how to challenge the status quo, we have a conversation with Dima Kuzmin, managing partner at 4BIO Capital.

4BIO is an international venture capital firm unlocking the treatments of the future by investing in advanced therapies and other emerging technologies.

00:45-02:36: About 4BIO
02:36-07:39: What are the trends in biotech investments currently?
07:39-09:30: Is being in a well-known biotech hub necessary to raise funds?
09:30-14:14: Is relocation necessary for companies starting up if they aren’t near a biotech hub?
14:14-17:57: What differences are there in investment patterns between Europe, the US and Asia?
17:57-21:35: How can funding match the research strength there is in Europe?
21:35-25:07: Are there any emerging hubs in Europe, or opportunities to create new hubs?
25:07-26:36: Are Spain and Italy good locations for biotech?
26:36-28:32: What are the best ways for European companies to attract US and Asian capital?
28:32-35:47: How do you see the European biotech space evolving?

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BioSenic developing severe knee osteoarthritis treatment02 Feb 202400:31:43

BioSenic, a Belgian clinical-stage company specializing in serious autoimmune and inflammatory diseases and cell therapy, will share data on its late-clinical asset JTA-004 at the Osteoarthritis Research Society International (OARSI) World Congress 2024 in Vienna, Austria. 

The post hoc analysis of a phase 3 study found that a single injection of JTA-004 was safe and efficacious for patients with a newly characterized severe inflammatory subtype of knee osteoarthritis (OA). 

This week on the podcast, we talk about the treatment with BioSenic chief scientific officer and chief operating officer, Dr Carole Nicco.  

00:52-03:19: About BioSenic
03:19-07:34: What is osteoarthritis?
07:34-11:45: What is ALLOB?
11:45-15:03: What is JTA-004?
15:04-17:14: What will you be presenting at the OARSI event? 
17:14-20:20: How useful is attending events for BioSenic?
20:20-21:04: How often will JTA-004 need to be administered?
21:04-24:08: Are other companies working on osteoarthritis?
24:08-26:26: What stage are your arsenic trioxide trails at?
26:26-29:50: Will this be useful in treating other autoimmune diseases?
29:50-31:08: What are the next steps?

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Boosting fertility by changing the vaginal microbiome26 Jan 202400:32:38

Freya Biosciences is a clinical-stage biotech company dedicated to reimagining women's health and redefining fertility for those previously deprived of options. 

With a focus on microbial immunotherapies, Freya addresses immune drivers underlying a range of reproductive health conditions.

The company recently received $38 million Series A financing to advance the clinical development of its lead drug candidate, an investigational vaginal microbial immunotherapeutic. The treatment is to address infertility in women with dysbiotic vaginal microbiota who are undergoing assisted reproductive technology (ART). 

This week’s conversation is with Freya chief science officer and co-founder Johan van Hylckama Vlieg.

00:58-01:55: About Freya Biosciences
01:55-07:12: What is the vaginal microbiome?
07:12-08:11: Are there differences between individuals’ vaginal microbiomes?
08:11-10:20: Does this make treatments challenging?
10:20-15:33: How does your platform use immunotherapy on the microbiome?
15:33-16:51: How does the vaginal microbiome change following treatment?
16:51-18:14: What happens to the vaginal microbiome of unsuccessful treatments?
18:14-20:10: How is success measured?
20:10-21:51: Could this lead to treatments of other conditions?
21:51-22:51: Does the vaginal microbiome change during menopause?
22:51-25:05: What are your clinical trials?
25:05-26:25: Does your treatment work better in combination with others?
26:25-27:36: Is it easy to find patients for trials?
27:36-30:14: How are the treatments manufactured?
30:14-31:55: How important is your recent funding?

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Epic Bio - gene editing without cutting DNA19 Jan 202400:32:43

Epic Bio is an epigenetic editing company, leveraging the power of CRISPR without cutting DNA.

The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. 

This week, we discuss epigenetic editing, why it’s reversible, and how it can treat FSHD and other conditions, with Epic Bio’s founder, Dr Stanley Qi, one of the original inventors of CRISPR.

01:15-02:51: Dr Xi’s background
02:51-05:11: The beginning of Epic Bio
05:12-09:23: What is epigenetic editing?
09:23-10:47: What is the Gene Expression Modulation System?
10:47-12:47: How is the editing reversible?
12:47-18:01: How will epigenetic editing advance treatments?
18:01-21:24: Tackling multiple conditions
21:24-22:46: How will your platform tackle FSHD?
22:46-24:18: FSHD clinical trial
24:18-25:51: How will you address costs?
25:51-27:18: Are other companies working on epigenetics?
27:18-29:27: What else is in the pipeline?
29:27-31:58: What are the next steps?

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Could natural killer cells transform Alzheimer’s treatment?12 Jan 202400:28:14

A US company, with its origins in South Korea, is using natural killer (NK) cells to treat Alzheimer’s disease, with remarkable results.

This week, we have a conversation with Dr Paul Song, CEO of NKGen Biotech, a company with its sights set on changing the Alzheimer’s treatment landscape.

00:58-02:55: About NKGen Biotech
 02:56-05:39: Why is Alzheimer’s so challenging to treat?
 05:39-07:28: Are there other treatments making a difference?
 07:28-13:47: What is NKGen Biotech’s approach to treating Alzheimer’s?
 13:47-15:38: Can you treat multiple conditions at the same time?
 15:38-15:55: Are there any side effects?
 15:55-16:33: What dose are you able to give?
 16:33-18:31: How can you keep the cost of treatments low?
 18:31-19:19: How, and how often, will it be administered?
 19:19-20:49: How are you approaching taking on later stage Alzheimer’s?
 20:49-22:21: Are you looking to cure Alzheimer’s?
 22:21-23:50: Can this approach be used for other diseases?
 23:50-24:47: Are you looking for partnerships?
 24:47-25:47: What stage are your trials at?
 25:47-26-19: What are the next steps for NKGen Biotech?
 26:19-26:35: How are you enrolling patients?
 26:35-27:32: What is your approximate timeline?

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Gene editing in the microbiome to treat acne05 Jan 202400:29:02

Eligo Bioscience is a gene-editing company focused on addressing diseases driven by the expression of bacterial genes from the microbiome. 

Its first target is moderate to severe acne vulgaris, an inflammatory disease that affects about 3% of the global population.

This week, we talk to Xavier Duportet, CEO of Eligo Bioscience, as the company looks to head to the clinic with its acne vulgaris treatment. 

00:53-02:42: About Eligo Bioscience
 02:42-06:07: How are you creating a new class of transformative genetic medicine?
 06:08-10:07: How important is the microbiome?
 10:07-11:48: How do you account for differences in the microbiome?
 11:48-13:17: What conditions are you looking to treat?
 13:17-18:42: What is your EB005 platform?
 18:42-21:28: How will your acne treatment be administered?
 21:28-22:06: How will Eligo keep the cost of the acne treatment low?
 22:06-24:36: Will your platform be applicable to other diseases?
 24:36-25:52: Will a combination of treatments of the microbiome make a difference?
 25:52-28:14: What will the recent funding mean to Eligo Bioscience?

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Tackling challenging diseases with vaccines15 Dec 202300:25:47

Vaxxinity, Inc. is a U.S.-based biotechnology company looking to develop vaccines for several hard-to-treat diseases such as Alzheimer’s and Parkinson’s. 

The company is pioneering a new class of medicines through its proprietary technology platform, which has enabled the innovation of novel synthetic peptide immunotherapy candidates to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia. 

In this week’s podcast, the company’s CEO, Mei Mei Hu, tells us about its work, and the aim of democratizing healthcare across the globe. 

01:00-02:21: About Vaxxinity
02:21-03:39: How does Vaxxinity’s approach differ from other companies?
03:39-04:38: How important is being able to roll out products globally?
04:38-06:28: What is your new class of vaccines?
06:28-08:58: How are you tackling diseases that are difficult to treat?
08:58-10:10: How does your Vaxxine Platform work?
10:10-10:58: Do synthetic peptides make this more scalable?
10:58-12:02: How are you addressing safety?
12:02-13:03: What has the reaction been like to your company?
13:03-14:14: What diseases are you tackling?
14:14-15:40: What is your pipeline for taking on these conditions?
15:40-16:16: Do you vaccines need booster shots?
16:16-18:17: Are you looking to cure diseases?
18:17-20:02: Could your approach be applied to other diseases?
20:02-20:29: Are you looking to partnerships?
20:29-21:10: What are the next steps?
21:10-21:24: When do you expect to see data?
21:24-21:42: When do you hope to have products being delivered to patients?
21:42-24:37: How does your COVID vaccine differ from others?

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Using worms to treat diseases08 Dec 202300:21:37

Is there a connection between worms being eradicated from the gut in the early 20th Century and an increase in allergies and autoimmune disease?  

This week, we have a conversation with Holoclara CEO Andrea Choe, who not only believes there is a connection, but also sees secretions from worms as a way to tackle such diseases. 

01:31-06:05: About Holoclara
06:06-08:26: From discovery to setting up a company
08:26-10:40: Was this a symbiotic relationship?
10:40-12:30: As we are losing species for a variety of reasons, are there other species that may also be beneficial?
12:30-14:03: What are roundworms secreting?
14:03-15:28: How do you take these secretions and advance that to creating a drug?
15:28-15:44: Do you need the worms to make your product?
15:44-16:02: What conditions are you looking to address?
16:02-16:43: Are you looking to cures or disease maintenance?
16:43-16:58: How far down the path are you with this?
16:58-17:26: How important are partnerships and investment?
17:26-18:12: What is reaction like to the concept of using worms to tackle diseases?
18:12-19:25: What are the next steps?
19:25-20:44: Are there other cures to be discovered from other species?


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Ipsen advances liver disease treatment01 Dec 202300:21:47

Jennifer Schranz is senior vice president and global head of rare diseases in the R&D division at Ipsen.

On the podcast this week, she discusses a breakthrough treatment for primary biliary cholerangitis and cholestatic liver disease.

00:56-00:49: Background information
01:44-03:21: What are primary biliary cholerangitis (PBC) and cholestatic liver disease?
03:21-03:50: How rare is PBC?
03:50-04:45: How easy is PBC diagnosis?
04:45-05:45: What are the current treatments for the condition?
05:45-07:22: What are the challenges to innovation?
07:22-08:27: What is elafibranor?
08:27-08:37: How is it administered?
08:37-09:18: Is elafibranor taken for life?
09:18-11:08: What is the ELATIVE trial, and what were the results you presented at the AASLD Liver Meeting?
11:08-13:09: What was the reaction at the meeting?
13:09-13:55: A patient-centric approach
13:55-14:44: Is a cure for PBC a possibility?
14:44-17:20: How important are acquisitions to Ipsen’s pipeline?
17:20-18:33: Working on multiple diseases
18:33-19:58: How much impact can you have on liver diseases?
19:58-21:04: What are the next steps for elafibranor?

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New class of small molecule therapeutics introduced24 Nov 202300:26:35

On this week’s podcast we have a conversation with Jordi Mata-Fink, co-founder and chief executive officer of the newly-launched U.S. company Gate Bioscience.

00:45-01:06: About Gate Bioscience
01:06-02:09: What are disease-causing extracellular proteins?
02:09-04:44: How have diseases caused by extracellular proteins been treated in the past?
 04:44-05:47: Have there been diseases that haven’t been addressed?
 05:47-07:05: Tackling the different issues created by extracellular proteins
 07:05-09:41: What are molecular gates?
 09:41-12:56: How does your technique distinguish between good and bad extracellular proteins?
12:56-16:24: How did the idea become a company?
16:24-17:24: Which diseases will you focus on?
17:24-18;47: How do you address costs?
18:47-19:11: How will this be given to patients?
19:11-20:01: Disease cure or disease management?
20:01-21:35: How was the company created?
21:35-24:18: The rise of small molecules
24:18-25:58: What are the next steps?

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How to make vaccines affordable17 Nov 202300:40:36

Among other things, UK-headquartered company Phenotypeca has been working with the Bill & Melinda Gates Foundation to produce albumin for low and middle-income countries to reduce the costs of vaccines.

The company’s CEO, Johnny Cordiner, and research and development director, Professor Ed Louis, tell us about the work, as well as the company and its other projects.

01:01-11:57: About Phenotypeca
11:57-14:04: What is recombinant protein technology?
14:04-16:39: What are the issues around the cost of albumin?
16:39-20:48: How can you help improve affordability?
20:48-26:55: What is QTL technology?
26:55-30:18: What are the benefits of this technology?
30:19-34:22: Partnering with the Bill & Melinda Gates Foundation
34:22-36:37: What’s the timeline?
36:37-39:45: What else are you working on?

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[Rebroadcast] Epigenetic editing – the power of CRISPR without cutting DNA09 Aug 202400:32:57

While our podcast takes a brief break, Labiotech invites you to enjoy some of our favorite episodes. We will return with a brand-new episode on August 16, 2024! Have an awesome summer!

Epic Bio is an epigenetic editing company, leveraging the power of CRISPR without cutting DNA.

The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. 

This week, we discuss epigenetic editing, why it’s reversible, and how it can treat FSHD and other conditions, with Epic Bio’s founder, Dr Stanley Qi, one of the original inventors of CRISPR.

01:15-02:51: Dr Xi’s background
02:51-05:11: The beginning of Epic Bio
05:12-09:23: What is epigenetic editing?
09:23-10:47: What is the Gene Expression Modulation System?
10:47-12:47: How is the editing reversible?
12:47-18:01: How will epigenetic editing advance treatments?
18:01-21:24: Tackling multiple conditions
21:24-22:46: How will your platform tackle FSHD?
22:46-24:18: FSHD clinical trial
24:18-25:51: How will you address costs?
25:51-27:18: Are other companies working on epigenetics?
27:18-29:27: What else is in the pipeline?
29:27-31:58: What are the next steps?

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Mending a broken heart with biotech10 Nov 202300:29:10

French company CellProthera has seen some remarkable results treating people who have had a heart attack.  On this week’s podcast, the company’s CEO Matthieu de Kalbermatten tells us how it works.

01:00-03:29: About CellProthera
03:30-06:30: What are ProtheraCytes?
06:30-09:02: Is this a cure?
09:02-10:24: Is prevention the best option?
10:25-11:49: Is this therapy useful to treat other conditions?
11:50-13:55: How quickly do you need to treat a patient?
13:56-16:39: The field of regenerative therapy
16:39-17:52: Is regenerative therapy a growing sector?
17:53-18:26: Clinical trials
18:26-19:22: Upcoming phase 3 trials
19:22-20:36: The regulatory pathway
20:37-23:25: Cost and scale
23:25-24:20: The need for partnerships
24:21-24:57: Next steps
24:57-28:30: Is there excitement in the medical field for this treatment?

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How quantum computing can accelerate drug discovery03 Nov 202300:28:45

On today’s podcast, Jean-Philip Piquemal, the chief scientific officer and co-founder of Qubit Pharmaceuticals, explains how the company’s Atlas platform is leading the way to more effective and safer drugs.

00:54-01:52: The history of Qubit Pharmaceuticals

01:52-04:04: What is the Atlas software?

04:04-05:18: Failing quickly is the key

05:19-06:53: What are digital twins?

06:54-09:00: Improving safety, reducing costs, and boosting drug discovery

09:00-10:44: Areas of interest

10:44-13:21: Recent successes

13:23-14:57: Preparing for future pandemics

14:58-17:03: Staying ahead of other companies

17:04-18:29: Enough diseases to go around

18:30-20:11: Upgrading Atlas software

20:13-21:59: How Qubit works with pharma companies

21:59-24:41: The Quantum for Bio program

24:42-27:58: Looking to the future, and helping patients
 

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