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...

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...

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...

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...

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...

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...

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...

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...

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...

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...

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...

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:...

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...

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...

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:...

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...

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...

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...

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...

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...

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...

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...

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...

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:...