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
Psychedelics appear to be in vogue after years of being relegated to alternative medicine. But investor pushback after the highly anticipated release of Phase III trial data by Compass Pathways reveals an increasingly high bar for development.
Emerging Company Profile: Early-stage biotech Eolo will seek cash and deals for its novel obesity drug SANA after positive Phase I results for weight loss, lean muscle mass preservation and fat mass reduction.