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
Recent moves in the industry include changes at the top at Mitsubishi Tanabe Pharma and MC2 Therapeutics, plus MindMed acquires chief financial officer from Longboard Pharma.
In a turn of fortunes, Lyra Therapeutics has reported positive Phase III results for LYR-210 in chronic rhinosinusitis, boosting hopes for US approval. The company plans to submit an NDA and pursue further trials, but its cash position is precarious.
IGI CEO tells Scrip about the “very encouraging” feedback at ASCO to promising early data for the firm's first-in-class investigational trispecific antibody in multiple myeloma. Is a licensing deal in the offing?