Gene therapy start-up GenSight Biologics Inc. will use its recent €32 million Series A financing to develop its preclinical gene therapies for rare and blinding ophthalmic diseases. The company will develop two therapies: one for Leber’s hereditary optic neuropathy, a degradation of the optic nerve that results from a mitochondrial DNA mutation, and an optogenetic therapy addressing retinitis pigmentosa, among the most common inherited causes of blindness in people below the age of 50.
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CEO Paul Stoffels said gaining US clearance for an IND for its novel CAR-T product was demanding, but now opens up a pathway towards a pivotal study starting in 2025.
A final rejection of Leqembi could also spell the same fate for Lilly’s rival drug but public outcry and demand for Alzheimer’s therapies might force the regulator’s hand
Ascentage will share at ASCO new clinical data on its Bcl-2 inhibitor lisaftoclax, part of the Chinese firm's novel pipeline, on which its CEO shared with Scrip plans and strategy, along with some views on the use of RWD in China.
AI-powered drug development was a major topic at the recent Bio Korea meeting, where experts discussed how the technology is changing R&D activities and the challenges being faced by Korean firms as they look to catch up with their global competitors.
Data from several closely watched solid tumor drug trials – some considered practice-changing – are scheduled for presentation at the meeting, including for AstraZeneca and Daiichi’s Enhertu.