Beyond Biotech - the podcast from Labiotech Labiotech

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...