Neurogene Inc.

Cell and gene therapy leaders want the US Food and Drug Administration to loosen its regulatory oversight of early clinical trials to ensure the innovation will remain in the US and not move to China.

Early data from Neurogene’s Phase I/II trial of NGN-401 in Rett syndrome look promising, but it remains unclear how differentiated they are from competitor Taysha Gene Therapy’s TSHA-102, being develo

On 11 November, Neurogene investors reacted poorly to the serious adverse event the company reported in the mid-stage trial of its Rett syndrome gene therapy ; on 18 November they punished the compan

Two and a half months after its first US Food and Drug Administration approval for Incyte Corp.-partnered Niktimvo (axatilimab-csfr) in chronic graft-versus-host disease (GVHD), Syndax Pharmaceuticals