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TitreDateDurée
Exploring the Promise and Peril of CRISPR28 Aug 202400:57:36

The emergence of CRISPR, a powerful tool for gene editing, promises to correct devastating genetic diseases, but it also raises concerns about how it will be used and who will make those decisions. Neal Baer, in his new book “The Promise and Peril of CRISPR,” brings together a series of essays by bioethicists, geneticists, and others who explore ethical and policy considerations raised by the technology. We spoke to Baer, co-director of the master’s degree program in Media, Medicine, and Health at Harvard Medical School; about whether there are unique questions raised by CRISPR as a dual-use technology, the book’s recasting of the case of Chinese scientist He Jiankui, and why he thinks there needs to be public debate now over the use of the technology to make heritable changes to the genome.

Transforming Protein Engineering with Generative AI21 Aug 202400:35:47

Speeding Protein Engineering with Whether it’s the development of new biotherapeutics, or replacing hydrocarbons from their role in industrial manufacturing, engineering proteins suited for a task is a time consuming and expensive process. Cradle Bio has developed generative AI technology to accelerate protein engineering by reducing the number of experiments needed to arrive at a product candidate and enable the optimization of multiple properties at once. We spoke to Elise de Reus, co-founder of Cradle, about the company's generative AI technology, how it works, and how it’s changing the process of protein engineering.

Using Generative AI to Produce Precise Gene Editors19 Jun 202400:23:15

Earlier this year, Profluent announced it had edited the human genome with OpenCRISPR-1, the world’s first AI-created and open-source gene editor. Though the open-source aspect helped garner attention for Profluent, it also served as a demonstration of the company’s generative AI platform to create novel CRISPR gene editors. We spoke to Hilary Eaton, chief business officer for Profluent, about how the company’s generative AI engine works, its business model, and why its platform has the potential to create other protein therapeutics of varying modalities.

Targeting RNA Dysregulation in Cancer29 Sep 202200:25:57
Inspirna is putting RNA biology to work to address difficult to treat cancers that affect large numbers of people, such as colorectal cancer, small cell lung cancer, and non-small cell lung cancer. Its platform technology is being used to discover the underlying drivers of cancer that can be drugged by both small molecules and biologics to address RNA dysregulation. We spoke to Masoud Tavazoie, co-founder and CEO of Inspirna, about the role RNA dysregulation plays in certain cancers, Inspirna’s platform technology, and its clinical pipeline in development.
Building a Pipeline of Next-Generation Kinase Inhibitors22 Sep 202200:37:53
Kinase inhibitors have become staples in the arsenal to battle cancer. These medicines can disrupt the cell signaling that drives the growth and spread of tumors. There are more than 30 approved kinase inhibitors today, but they often have dose limiting toxicities, in part because they often lack specificity and can have activity against multiple kinases at once. Blueprint Medicines is seeking to develop next-generation kinase inhibitors that are highly selective to treat genomically-defined cancers and rare diseases. We spoke to Fouad Namouni, president of research and development for Blueprint, about the company’s discovery platform, the large unexplored territory of kinases, and what makes Blueprint’s kinase medicines next-generation.
Rethinking How Cell and Gene Therapies Are Made to Improve Accessibility15 Sep 202200:42:06
Cell and gene therapies offer great promise for treating, preventing, or curing serious health issues like cancer, genetic disorders, immunodeficiencies, and rare diseases. Nevertheless, the complexity and costs of producing and delivering these personalized medicines creates barriers to patients benefiting from their potential. Orgenesis is seeking to change the business model around cell and gene therapies through its Point-of-Care Platform, which it says can lower the cost, accelerate development, and expand access for patients. We spoke to Vered Caplan, CEO of Orgenesis, about the challenges of cell and gene therapy, how the company’s Point-of-Care Platform technology addresses those, and its network approach to building a pipeline of therapies across a wide range of indications.
Developing Next-Generation Radiopharmaceuticals08 Sep 202200:26:47
Radiopharmaceuticals have long been shown to be effective at killing cancers, but often damage healthy cells because they are so toxic. Convergent Therapeutics has developed platform technology that takes advantage of the power of radiopharmaceuticals, but links them to targeting mechanisms, such as monoclonal antibodies and ligands, to deliver them in a precise and highly targeted ways. Its lead experimental therapy is an actinium-linked monoclonal antibody that targets prostate-specific membrane antigen to treat prostate cancer. The ability to use dual targeting mechanisms offers the potential for powerful combinations of radiopharmaceuticals while limiting their toxicities. We spoke to Philip Kantoff, co-founder and CEO of Convergent, about the company’s platform technology for developing next-generation radiopharmaceuticals, how the company targets tumor surface molecules, and how its approach minimizes the toxicity of these therapies.
Why Great Science and Data Aren’t Enough01 Sep 202200:28:24
Whether it’s capturing the imagination of a venture capitalist, enticing a potential partner, or attracting talent, CEOs need to be able to tell their company’s story effectively. In his new book “Winners Have a Story,” Giuseppe Marzio discusses how CEOs can turn complex science and technology into compelling tales. Marzio, who worked as a scientist in biopharma before launching a communications agency, comes to his interest in storytelling honestly. Growing up with a love for the cinema, he had dreams of becoming a filmmaker and has studied the structure of stories. We spoke to Marzio, founder of the communications agency Chiaro, about his new book “Winners Have a Story,” why a good pitch shares a common structure with novels and movies, and why a company’s strategy and story are inextricably linked.
Targeting Sugars that Let Cancers Evade the Immune System25 Aug 202200:26:40
It’s been understood for more than 60 years that cancer cells overproduce sugars on their surface. More recently it’s been discovered that these sugars can help cancer cells evade the immune system. Palleon Pharmaceuticals is developing immunotherapies that work by targeting these cell surface sugars and make them vulnerable to attack. We spoke to David Feltquate, chief medical officer of Palleon, about the potential to harness glycobiology to treat cancer, how the company is working to modulate cell-surface sugars, and its emerging pipeline of glyco-immuno therapies.
Engineering Stem Cells to Withstand Targeted Therapies18 Aug 202200:18:52
Current treatment of acute myeloid leukemia generally consists of chemotherapy followed by a hematopoietic stem cell transplant to eliminate any remaining cancer cells from the bone marrow. Even though outcomes remain poor with around 40 percent of transplanted patients experiencing cancer relapse, and patients who do relapse having two-year survival rates of less than 20 percent, the approach has changed little in 40 years. One reason is that newly transplanted patients are unable to receive targeted cancer therapies since those therapies would be toxic to the fragile transplanted stem cells. Vor Bio’s solution is to edit the stem cells prior to transplanting them so they lack the receptors targeted therapies attack. We spoke to Robert Ang, president and CEO of Vor Bio, about the company’s platform technology, how it works, and why it has the potential to change outcomes for patients with AML and other hematologic malignancies.
Targeting a Convergence of Signaling Pathways that Drive the Growth and Spread of Cancer11 Aug 202200:24:15
In cancer, the rigorous translational machinery of the human body goes haywire, causing an overproduction of proteins that fuel the growth and spread of tumors, as well as enabling them to evade the immune system. Effector Therapeutics is developing a new class of cancer therapies called selective translation regulator inhibitors, or STRIs, that can inhibit the production of proteins that drive a cancer. We spoke to Steve Worland, president and CEO of Effector, about the company’s new class of therapies, how they target a central node where two major cancer signaling pathways converge, and how they pack the punch of a combination therapy in a single drug.
Moving from Preservation to Restoration with Off-the-Shelf Cell Therapies04 Aug 202200:21:19
While cell therapies hold great promise for not only halting the progression of degenerative disease but potentially restoring the body, cost has remained a barrier. Lineage Cell Therapeutics is developing allogenic cell therapies that don’t rely on modifying a patient’s own cells, making them scalable and less costly than autologous ones. At the end of last year, the company entered into a partnership with Genentech to develop and commercialize OpRegen, its experimental cell therapy for dry age-related macular degeneration. We spoke to Brian Culley, CEO of Lineage Cell Therapeutics, about the company’s platform technology for off-the-shelf cell therapies, how its platform works, and what the collaboration with Genentech will do to advance the technology.
Using Gene Therapies to Treat Chronic, Inflammatory Conditions28 Jul 202200:21:27
A broad range of serious diseases involve chronic inflammation, which causes both pain and progressive damage. Xalud Therapeutics is developing a locally-delivered, non-viral gene therapy that is designed to harness the ability of interleukin-10 to regulate the immune system and restore homeostasis. The company’s lead indication is in osteoarthritis We spoke to Diem Nguyen, CEO of Xalud, about the role inteleukin-10 plays in regulating multiple pathways in the immune system, Xalud’s gene therapy, and the indications the company is pursuing.
Enabling the Nervous System to Repair Itself12 Jun 202400:17:22

About 500,000 people suffer from a spinal cord injury each year. Treatments can involve surgical procedures to stabilize the spine and physical rehabilitation, which can have limited benefits. There are currently no FDA-approved therapies that can promote repair and improve function following a spinal cord injury. NervGen Pharma is seeking to change that by developing therapies that allow the nervous system to repair itself. We spoke to Mike Kelly, CEO of NervGen, about the potential for using therapies designed to allow the nervous system to repair itself, how the company’s lead experimental candidate for spinal cord injury works, and why the same approach holds promise in treating a range of neurodegenerative diseases.

Unlocking the Full Potential of Antibody Therapies21 Jul 202200:38:27
Traditional antibody discovery depends, in part, on a bit of good fortune that banks on the hope that either screening antibody libraries or exposing an organism to an antigen will result in the discovery of a compelling therapy. Yanay Ofran, CEO of Biolojic Design, says the problem with this approach is that it ignores many of the performance-related aspects of an antibody beyond its ability to bind to a target. His company’s AI platform for antibody discovery seeks to capitalize on the full capabilities of antibodies to develop smarter therapies that can serve as molecular switches and act conditionally. We spoke to Ofran about Biolojic’s platform technology, how it mimics the way the immune system makes antibodies, and why its approach will lead to smarter therapies.
Combatting Superbugs and Emerging Viruses14 Jul 202200:22:04
The rise of antibiotic resistance and the threat of emerging viral pathogens have created global public health threats. Recce Pharmaceuticals is developing a new class of synthetic anti-infectives that it says can overcome the hyper-cellular mutation of bacteria and viruses. What’s more, the company says their potency doesn’t diminish even with repeated use. We spoke to James Graham, CEO of Recce, about its synthetic anti-infectives, how they work, and why they can be used repeatedly against a broad range of bacteria and viruses.
How a Plant-Derived Gel Halts Bleeding in Seconds07 Jul 202200:21:22
Whether in an operating room or in a trauma situation, the ability to quickly stop bleeding represents a critical need in healthcare. At the end of December, Cresilon filed for U.S. Food and Drug Administration approval to market its hemostatic gel to rapidly stop bleeding. The filing follows the launch of Vitigel, Cresilon’s hemostatic gel for the animal health market. We spoke to Joe Landolina, co-founder and CEO of Cresilon, about how he developed the plant-derived gel, the significant need he is seeking to address, and how the gel is able to halt bleeding in a matter of seconds.
Exploiting a Vulnerability in Cancers30 Jun 202200:27:54
In cancer, synthetic lethality refers to a state in which two genetic mutations that alone may allow a cancer cell to survive will kill it when they exist simultaneously. Cyteir Therapeutics is seeking to exploit that strategy with what it calls next-generation synthetically lethal therapies to treat a wide range of cancers. We spoke to Markus Renschler, president and CEO of Cyteir therapeutics, about synthetic lethality, Cyteir’s pipeline, and life as a small public biotech in 2022.
Democratizing AI23 Jun 202200:34:49
AI Dynamics is seeking to make AI accessible. Though its NeoPulse platform can be used across industries, the life sciences is one of the key markets the company is targeting with its technology being used to do everything from target identification for drug development to diagnosing and triaging TB patients by the sounds of their cough. We spoke to Rajeev Dutt, CEO and president of AI Dynamics, about the company’s core AI technology, how it seeks to make AI accessible, and why he believes it can transform drug development and healthcare by moving the needle on costs.
A Rapid Onset Nasal Spray to Treat Mental Health Conditions16 Jun 202200:30:54
Social anxiety disorder can cause debilitating physical and emotional manifestations when a person is doing such things as meeting new people, speaking in public, or interviewing for a job. It can cause sweating, a rapid heartbeat, shortness of breath, and dizziness. The fear of embarrassment and humiliation can lead to depression and even addiction. VistaGen Therapeutics is developing intranasally delivered therapies for CNS disorders that can provide rapid relief at the onset of an episode much like someone with asthma might use a rescue inhaler. We spoke to Shawn Singh, CEO of VistaGen, about social anxiety disorder, the need for innovative approaches to treat that and other CNS conditions, and the potential for rapid onset therapies to treat a range of mental health conditions.
A Regenerative Medicine Company Puts Its Off-the-Shelf Vessel Replacements to the Test in Ukraine09 Jun 202200:25:07
Humacyte’s human acellular vessels are experimental, engineered, off-the-shelf replacement vessels that are being developed for vascular repair, reconstruction, and replacement. The vessels are designed to overcome long-standing limitations in vessel tissue repair and replacement. Though not yet approved, the manufactured vessels are getting a real-world test as the company has been providing them to hospitals in Ukraine to treat wounded civilians and soldiers with vascular injuries. We spoke to Laura Niklason, CEO of Humacyte, about the company’s human acellular vessels, how they are produced, and the potential needs they can address.
Battling Resistance in Tumors with “Pan-Variant” Kinase Inhibitors02 Jun 202200:30:17
The emergence of tyrosine kinase inhibitors represented a major advance in the fight against cancer, but the ability of tumors to mutate and develop resistance to these therapies remains a challenge. Theseus Pharmaceuticals is developing what it calls “pan-variant” kinase inhibitors in the hopes of outsmarting tumors by anticipating the range of kinases that may drive their spread and growth as they change. We spoke to Tim Clackson, president and CEO of Theseus Pharmaceuticals, about its structural-based approach to drug development, how it determines the appropriate targets for its pan-variant kinase inhibitors, and why it believes its next-generation TKIs can overcome the challenge of drug resistance.
Treating Disease by Editing Genes in the Microbiome26 May 202200:42:33
The emerging understanding of the role the microbiome plays in wellness and disease is opening up a large number of potential therapeutic targets in the millions of genes that drive the microorganisms that live within the body. Eligo Bioscience is developing a new class of precision medicines that uses gene editing to address the expression of pathogenic genes in the microbiome to treat not only infectious disease, but other conditions such as inflammatory diseases and cancer as well. We spoke to Xavier Duportet, CEO of Eligo Bioscience, about the company’s use of synthetic DNA to target bacterial genes, its platform technology, and the wide range of conditions that can be addressed through this approach.
Embracing the Promise of Natural Killer Cells in the Fight Against Cancer19 May 202200:27:02
While cancer immunotherapies have been promising, CAR T cell therapies have been costly, effective on less than half of patients, and focused on hematologic cancers. Cytovia Therapeutics is enlisting natural killer cells, part of the innate immune system, to develop off-the-shelf immunotherapies to treat liquid and solid tumors. The company is leveraging a cluster of technologies to produce CAR NK cell therapies, edited NK cells to enhance their targeting, and NK engagers to redirect NK cells toward tumor targets. We spoke to Daniel Teper, co-founder and CEO of Cytovia, about the case for NK immunotherapies, Cytovia’s platform technologies, and the company’s pipeline of therapies in preclinical development.
Overcoming Barriers to Delivering Large Molecules to the Brain05 Jun 202400:18:14

One of the challenges of treating neurodegenerative diseases is delivering therapies across the blood-brain barrier. Aliada Therapeutics is developing therapies that use its platform technology that enables the delivery of large molecules, like antibodies, across that barrier. Its lead program is in development to treat Alzheimer’s disease. We spoke to John Dunlop, chief scientific officer of Aliada, about its platform technology, its origins at Johnson & Johnson’s Janssen, and the company’s partnering strategy for leveraging the technology.

Addressing Neurodegenerative Disease by Suppressing a Hyper Immune Response12 May 202200:22:43
Neurodegenerative diseases such as ALS, Alzheimer’s disease, and frontotemporal dementia have been difficult conditions for drug developers to target. Coya Therapeutics is approaching these conditions as diseases of immune system dysfunction to address the neuroinflammation that underlies them We spoke to Adrian Hepner, president and chief medical officer of Coya, about the role a hyper immune response plays in the progress of neurodegenerative diseases, how the company’s experimental regulatory T cell therapy works to halt that, and its pipeline in development.
Bringing Precision Medicine to Psychiatry05 May 202200:33:41
The introduction of precision medicine has revolutionized the treatment of cancer and other diseases, but mental health conditions have not benefitted from the same type of treatment innovation. Alto Neuroscience is working to move psychiatry away from a trial-and-error approach to develop targeted medicines with the use of biomarkers to match the right drug to the right patient. We spoke to Amit Etkin, founder and CEO of Alto Neuroscience, about its efforts to develop precision medicines for mental health disorders, its AI platform for biomarker discovery, and its therapeutic pipeline in development.
Attacking Neurodegenerative Diseases with Protein Degraders28 Apr 202200:26:08
The use of protein degraders is a promising area of emerging small molecule therapies. Rather than inhibit disease-causing proteins, degraders use a natural cellular process to break them down. Origami Therapeutics is using its proprietary drug discovery platform to develop novel protein degraders to target neurodegenerative diseases. The approach allows the company to target proteins that are considered undruggable because they lack an identified binding site. We spoke to Beth Hoffman, founder and CEO of Origami, about its platform for developing degraders, its focus on neurodegenerative diseases, and how its looking to potential partners to exploit the full potential of its platform technology.
Restoring Immune Activity While Suppressing Cancer Proliferation21 Apr 202200:20:33
Cancer immunotherapies have shown great promise, but many tumors can develop resistance as the changing tumor microenvironment can help cancers evade detection and allow cancer cells to proliferate. Teon Therapeutics is developing immno-oncology therapies for difficult to treat cancers that are designed to have the dual effect of restoring immune activity while suppressing cancer cell proliferation. We spoke to Serge Messerlian, CEO of Teon Therapeutics, about what happens when immunotherapies fail, the unique approach Teon is taking, and how the dual activity of its cancer therapies can restore immune activity while suppressing cancer cell proliferation.
A New Model for Preventing Life-Threatening Blood Clots14 Apr 202200:19:18
Existing anticoagulants can prevent life-threatening blood clots for people with cardiovascular and metabolic diseases. The problem is that because of their mechanism of action, they also work to suppress the body’s ability to stop bleeding as it should. As a result, these therapies can be under utilized by patients who need them. Anthos Therapeutics is developing an experimental monoclonal antibody that can suppress coagulation without disrupting hemostasis, the biological process of stopping bleeding. We spoke to John Glasspool, CEO of Anthos, about the problems of existing anticoagulants, the novel target its monoclonal antibody acts on, and how it is able to uncouple the pathways for thrombosis and hemostasis.
The Race to Live Forever07 Apr 202200:32:45
Though the search for eternal youth has long been the fodder for myths and legends, science has been pushing us closer toward extending healthy years of life and has set some people off on efforts to defy death altogether. Peter Ward, in his new book The Price of Immortality: The Race to Live Forever, explores a subculture of immortality seekers who have turned to cryonics, as well as efforts to merge man with technology as a way to escape death. The growing understanding of the biology of aging and advances in regenerative medicine, though, are creating the potential to alter notions of human lifespans. We spoke to Ward about his book, distinguishing science fact from science fiction, and the growing understanding of the biology of aging that offers the potential for extending healthy years of life.
A Model for Moving from Breakthrough to Blockbuster31 Mar 202200:25:29
About 15 years ago, Harvard Business School professor Gary Pisano took at look at how small entrepreneurial biotechs fared against large and mature pharmaceutical companies and found the two sectors were about the same when it came to R&D productivity. Now, a new book From Breakthrough to Blockbuster: The Business of Biotechnology finds that the biotechnology industry is far more effective at bringing innovative therapies to market than Big Pharma and offers a prescription for large drug companies to decentralize decision-making to improve their ability to innovate. We spoke to venture investor and former biotech executive Don Drakeman, co-author of the book, about its findings, how decentralized decision-making can produce greater innovation, and lessons from COVID-19.
Building the Next-Generation of Cell and Gene Therapies24 Mar 202200:21:36
Poseida Therapeutics is leveraging its set of platform technologies to develop a range of next-generation cell and gene therapies. By using these technologies, alone or in combination, the company said it’s able to overcome limitations of the current generation of cell and gene therapies. The company’s most advanced candidates in its pipeline include a set of allogenic CAR T therapies. We spoke to Mark Gergen, CEO of Poseida, about the company’s efforts to develop off-the-shelf CAR T therapies, its platform technologies, and why it believes its CAR T cells will provide advantages over competitors’ therapies.
Building Small Molecule Drugs that Are Superior to Biologics17 Mar 202200:28:21
Though structure-based drug discovery has been long been used, technologic advances have given this approach greater power. ShouTi believes its next-generation, computational, structure-based drug discovery platform will allow it to develop small molecule drugs that are superior in safety and efficacy to biologic and peptide therapies its seeking to replace. We spoke to Raymond Stevens, CEO of ShouTi, about the company’s structure-based drug discovery platform, how it works, and why he believes it will be able to produce small molecule drugs that will be superior to biologics.
A New Class of Cancer Therapies Targets Solid Tumors with a Dual Action10 Mar 202200:21:33
Virus-like drug conjugates, or VDCs, are a new class of cancer therapies Aura Biosciences is developing to target a broad range of solid tumors. These therapies have a dual mechanism of action. They deliver a toxic payload to cancer cells, but also activate a secondary immune mediated response to kill cancer cells. We spoke to Elisabet de los Pinos, CEO of Aura Biosciences, about the company’s VDCs, how they work, and its pipeline in development.
Developing Complex Therapies to Tackle Complex Diseases29 May 202400:23:14

SalubrisBio, rather than shy away from complexity, embraces it. The company’s lead experimental therapy is an antibody fusion protein in development for both heart failure and the rare, neurodevelopmental condition amyotrophic lateral sclerosis. We spoke to Sam Murphy, CEO of SalubrisBio, about the challenges of pursuing complex diseases with complex therapies, its pipeline, and how its China-based parent has provided it financial freedom from the vagaries of the capital markets.

A Pursuit of Off-The-Shelf CAR-T Therapies03 Mar 202200:30:51
While the emergence of CAR-T therapies have been promising, these autologous cellular therapies are costly to produce since they require taking T cells from a patient being treated, altering them, and then infusing them back into the patient. Cellectis is among a growing list of companies pursuing off-the-shelf CAR-T therapies. The company’s U-CARTs, or universal chimeric antigen receptor T cells, are allogenic products that can be standardized and carry both time and cost advantages. We spoke to André Choulika, CEO of Cellectis, about the company’s off-the-shelf CAR-T therapies, the platform technology behind them, and its programs in development.
Mapping the Human Proteome24 Feb 202200:25:33
Though it’s been nearly 20 years since the Human Genome Project provided a blueprint for human biology, it still left much work to be done to understand health and disease at a molecular level. The Sweden-based Human Protein Atlas, which is seeking to map human proteins in cells, tissues, and organs, recently published significant updates to the open-access resource. We spoke to Mathias Uhlén, director of the Human Protein Atlas, about how the atlas is changing the diagnosis and treatment of disease, what’s known about the human proteome to date, and how this understanding will be essential to brining about an era of precision medicine.
Gene Therapy Provides Hope to Hemophilia B Patients17 Feb 202200:23:41
In 2020, CSL Behring entered into a global license and commercialization agreement with UniQure for the company’s experimental hemophilia B gene therapy. The expected, one-and-done treatment carries the potential to free people with the genetic bleeding disease from reliance on regular infusions of clotting factor IX for which they are deficient. We spoke to Steve Pascoe, senior vice president and head of therapeutic areas and development strategy for R&D at CSL about hemophilia B, how the gene therapy fits into CSL’s broader therapeutic offerings, and the encouraging results from the recent pivotal study.
Making Data-Driven Medicine a Reality10 Feb 202200:20:26
The promise of data-driven medicine is that it can accelerate the diagnosis of disease, provide patients with the most effective treatments for their particular conditions, and improve drug development. Sophia Genetics is drawing on extensive information from its global, data-sharing network to make data-driven medicine a reality. We spoke to Emily Paul, product director of platform for Sophia Genetics, about Sophia’s vision for data-driven medicine, the challenges of managing vast amounts of data and turning it into actionable information, and how its changing patient care today.
A Neuroscientist Turned Venture Investor Discusses TechBio Investing03 Feb 202200:28:00
The growing convergence of information technology and biotechnology are creating a compelling new group of companies that live in both these worlds at once. Lux Capital, which has long invested in both sectors, has a growing portfolio of these emerging TechBio companies. We spoke to Adam Goulburn, partner at Lux Capital, about his investment process, how he tempers the promise of technology with management realities and market timelines, and the changing landscape for venture investing.
A Nonprofit Seeks to Make Cell and Gene Therapies Affordable Worldwide27 Jan 202200:21:22
Cell and gene therapies are among the most promising approaches to treating diseases because they carry the potential to cure chronic and progressive conditions. The problem is that the high cost of producing these therapies, which often need to be tailored to individual patients, limits access to them, particularly in low- and middle-income countries. Caring Cross is seeking to change that through its nonprofit model that focuses on enabling hospitals and health systems to manufacture advanced therapies locally and deliver them in a cost-effective manner. We spoke to Boro Dropulić, co-founder of Caring Cross, about how the nonprofit is seeking to lower the cost of these therapies, how it operates, and why its initially focused on HIV and sickle cell disease.
Disposing Toxic Proteins to Treat Neurodegenerative Diseases20 Jan 202200:21:19
KeifeRx is developing a pipeline of orally delivered tyrosine kinase inhibitors to treat neurodegenerative diseases. It has a portfolio of these drugs that it has optimized to penetrate the brain, clear damaged cells, and treat these conditions through the bulk disposal of disease-causing toxic proteins. It believes its approach offers the potential to significantly improve on current treatments, which it calls primarily palliative because they fail to adequately eliminate the toxic proteins at the root of these deadly diseases. We spoke to KeifeRx CEO Chris Hoyt and co-founder and head of the company’s scientific advisory board Charbel Moussa, about how it has repurposed and optimized a cancer therapy to treat neurodegenerative diseases, and why it may have broad applications across these conditions.
Repurposing a Drug for Parasitic Infections as a Targeted GI Therapy13 Jan 202200:20:44
When AzurRX BioPharma merged with First Wave Bio last year it renamed itself First Wave BioPharma. The company is developing targeted, non-systemic therapies for gastrointestinal diseases with its lead candidate niclosamide, an approved therapy to treat tapeworms that it believes has anti-viral and anti-inflammatory properties. The company is developing niclosamide in six GI-indications including COVID-19 related GI disease, Immune Checkpoint Inhibitor-associated colitis and diarrhea in advanced oncology patients, ulcerative proctitis/ proctosigmoiditis, ulcerative colitis, and Crohn’s disease. We spoke to James Sapirstein, CEO of First Wave BioPharma, about the merger, niclosamide, and its potential to treat a range of gastrointestinal disorders.
Targeting Chronic, Low-Grade Inflammation to Address Diseases of Aging06 Jan 202200:29:46
HCW Biologics is developing immunotherapies to target chronic inflammation associated with aging. The company’s therapeutics are designed to disrupt the link between chronic, low-grade inflammation and age-related disorders. The company believes this type of inflammation is a significant contributing factor to cancer and several chronic conditions, including cardiovascular disease, diabetes, neurodegenerative disease, and autoimmune disease. We spoke to Hing Wong, CEO of HCW Biologics, about the link between chronic inflammation and diseases of aging, the company’s immunotherapies, and its platform for developing therapies that target this unwanted inflammation to treat cancer and other diseases.
The Year in Biotech and What’s Ahead in 202230 Dec 202100:24:47
As 2021 fades into the history books, it will be remembered as one with exuberant IPOs, punishing aftermarkets, and a lingering pandemic that once again is turning the annual JPMorgan Healthcare Conference into a virtual event. We continue our annual tradition of sitting down with STAT Senior Writer Adam Feuerstein to discuss the year that was in biotech, the best and worst CEOs of 2021, and what’s ahead in the new year.
How Solving a Problem with Genetic Medicines May Solve Another with Infectious Diseases22 May 202400:25:25

One of the challenges facing genetic medicines is delivering payloads across the human cell membrane and inside cells both effectively and with high tolerability. Aegis Life is capitalizing on its parent Entos Pharmaceuticals’ nucleic acid delivery platform technology to address infectious diseases. It recently secured investment from the Bill & Melinda Gates Foundation to help in the fight against malaria, HIV, and other conditions. We spoke to John Lewis, founder and CEO of Aegis Life, about the need it is addressing, the company’s platform technology, and how it is leveraging that to address the barriers to the delivery of vaccines and antibody therapies in developing economies.

Restoring the Guardian of the Genome to Fight Tumors23 Dec 202100:20:26
The p53 protein is known as the “guardian of the genome.” It plays an essential role in suppressing tumors. Rain Therapeutics is targeting a regulator of p53 that is overexpressed in certain cancer and can inactivate it, allowing certain cancers to grow and progress. We spoke to Avanish Vellanki, chairman and CEO of Rain Therapeutics, about the company’s experimental precision therapy milademetan, how it works, and its potential to treat a range of cancers.
Advancing a Cell Therapy with the Potential to Cure HIV16 Dec 202100:49:28
Though HIV has fallen out of the headlines, the virus continues to represent a significant public health threat. American Gene Technologies is developing an experimental cell therapy that it says is potentially curative for HIV. We spoke to Jeff Galvin, CEO and founder of American Gene Technologies, about the state of HIV, the company’s experimental cell therapy for HIV, and why the one-time treatment has the potential to free patients from chronic use of antiviral therapies.
Harnessing More Efficient Organisms to Reshore BioManufacutring09 Dec 202100:28:09
The COVID pandemic has called attention to the United States’ reliance on a supply chain that makes access to critical medicines dependent on the ability to make them overseas and ship them in a timely manner. At the same time, harnessing new ways of making biologics, is making it possible to gain significant savings over traditional manufacturing approaches. rBIO is betting it will be able to cost-effectively produce biologics in the United States and its starting with insulin to prove its point. We spoke to Cameron Owen, co-founder of rBIO, about the how the company is engineering different organism to increase the efficiency of biomanufacturing, why it is starting with insulin, and why reshoring biomanufacturing should be viewed as a critical issue for the United States.
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