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.&...
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 developi...
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 st...
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 c...
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 opera...
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 clinica...
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 recen...
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 Phesi01:31-01:49: Is your database global?01:49-03:08: How successful are the differe...
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, i...
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 el...
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 addres...
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 Biosciences04:09-06:03: What are the challe...
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 so...
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 Biosciences04:30-05:37: Is there still a role for CRISPR-Cas9?05:37-07:41: Raising funds for Tome07...
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 ...
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 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 Health02:30-05:12: 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 C...
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...
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 add...