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Neil McFarlane, president and CEO of Zevra Therapeutics, discusses the rare lysosomal storage disorder Niemann-Pick disease type C, the FDA’s upcoming decision on whether to approve the drug, and its broader efforts to build itself into a rare disease therapeutics company. Global Genes #RARECast https://lnkd.in/gSv3YEE9

Mati Gill, CEO of AION Labs, sits down with Amar Drawid, Ph.D. to discuss how the AI venture studio works with its Big Pharma partners to define the biggest drug development hurdles the industry faces; and how it holds challenges to attract academics, entrepreneurs, and life sciences professionals with the best solutions to build new companies. #AI #drug development #startups #venturecapital #biotech Audio only versions are available at https://lnkd.in/d5sBSFxz or your preferred podcast platform. https://lnkd.in/d5HUY2Zy

Michael Kelly, CEO of NervGen Pharma Corp., discusses 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 neurodegenerative diseases. https://lnkd.in/gAMqqM-w

Rare disease focused M&A transactions maintained their momentum in May as total potential deal values year-to-date were 192 percent above the same period in 2023, according to data compiled by DealForma and Global Genes. Marie Daghlian crunches the numbers. https://lnkd.in/gC532tD9

Coming Thursday to the Life Sciences D’n’A podcast: Mati Gill, CEO of the AI venture studio AION Labs joins Amar Drawid, Ph.D. Here’s a preview. You can find past episodes on our YouTube channel https://lnkd.in/g4W3TF-g, or audio only versions at https://lsdna.podbean.com/ or your preferred podcast platform.

Giles Platford, president of the Plasma-Derived Therapies Business Unit at Takeda, discusses its work in plasma-derived therapies, its recently approved therapy for the rare neuromuscular condition CIDP, and what issues need to be addressed to ensure an adequate supply of human plasma for therapeutic applications. Global Genes #RARECast https://lnkd.in/gYrDxM95

John Dunlop, chief scientific officer of Aliada Therapeutics, discusses the company's platform technology, its origins at Johnson & Johnson’s Janssen, and the company’s partnering strategy for leveraging the technology. https://lnkd.in/gmPcpEPv

Here's what you May have missed in May from The Bio Report, RARECast, Life Sciences D'n'A, and more. https://lnkd.in/dqsNgaZu

Salvador Rico, MD, PhD, chief medical officer of Encoded Therapeutics Inc., discusses the company’s lead program in Dravet syndrome, its efforts to develop gene therapies with optimized regulatory elements to target specific organs, and why he believes its approach is a point of differentiation for the company. Global Genes #RARECast https://lnkd.in/gc8vh8YU

Noam Solomon founder and CEO of Immunai, sits down with Amar Drawid, Ph.D. to discuss how the company is using machine learning to take a multi-omics approach to understanding the complexities of the immune system, how it is constructing an atlas of immune cells, and how it is leveraging this to develop the next generation of immunomodulatory drugs. #machinelearning, #AI #immunomodulatory, #precisionmedicine, #drugdiscovery https://lnkd.in/g2uRX8g3