University of Pennsylvania

The FDA fast-tracked NeuroVive's NeuroSTAT for severe traumatic brain injury (TBI), aiming to protect mitochondria and prevent secondary brain damage. NeuroSTAT showed promising results in phase 2 trials, with potential to improve survival, quality of life, and neurological function. It also has orphan drug designation in the US and Europe, offering market exclusivity benefits.

Centessa Pharmaceuticals appointed Stephen Kanes MD PhD as Chief Medical Officer. Kanes, with over 30 years in neuroscience and psychiatry, will advance Centessa's OX2R agonists for treating sleep-wake and psychiatric disorders. ORX750, their lead agonist, is in Phase 2 trials for narcolepsy and idiopathic hypersomnia.

Solid Biosciences' SGT-212, a novel AAV-based gene therapy for Friedreich’s ataxia, targets CNS and cardiac symptoms via dual administration. FDA cleared its IND, with a Phase 1b trial expected in 2H 2025. The therapy aims to restore frataxin levels, addressing FA's neurological and cardiac manifestations.

Solid Biosciences received FDA IND clearance for SGT-212, a novel gene therapy for Friedreich’s ataxia, targeting both neurologic and cardiac symptoms via dual administration. A Phase 1b trial is set for 2H 2025, aiming to treat the disease's full spectrum.

WuXi AppTec sold its South Philly-based WuXi Advanced Therapies unit to Altaris LLC, addressing concerns over Chinese ownership in U.S. biotech. The sale, excluding other WuXi facilities, follows legislative pressure and aims to strengthen the U.S. supply chain. Altaris commits to growing the unit, while WuXi continues its broader biotech mission.

Solid Biosciences Inc. announced FDA clearance for SGT-212, a novel gene therapy for Friedreich’s ataxia, targeting CNS and cardiac symptoms via dual administration. A Phase 1b trial is expected in 2H 2025. SGT-212 aims to restore frataxin levels, addressing disease progression and symptoms.

Solid Biosciences Inc. announced FDA clearance for SGT-212, a novel AAV-based gene therapy for Friedreich’s ataxia (FA), targeting CNS and cardiac symptoms via dual administration. A Phase 1b trial is expected in 2H 2025. SGT-212 aims to restore frataxin levels, addressing FA's neurological and cardiac manifestations.

SGT-212, a novel AAV-based gene therapy by Solid Biosciences, targets Friedreich’s ataxia (FA) by delivering full-length frataxin to the cerebellum and heart via dual administration. A Phase 1b trial is set for 2H 2025, aiming to address FA's neurological and cardiac symptoms. The FDA has cleared its IND application, marking a significant step towards treating FA's full spectrum.

The CAR-T cell therapy market is rapidly growing, with 11 products commercialized globally, including 7 FDA-approved in the U.S. CAR-T therapy modifies patient T-cells to target and kill cancer cells, showing promise in treating blood cancers. Challenges include treating solid tumors, reducing 'vein-to-vein' time, and reimbursement issues. The industry is advancing through gene-transfer techniques, partnerships, and exploring allogeneic therapies.