Neurogene Inc.

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

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

The US Food & Drug Administration’s inaugural class of applications for its START rare disease pilot program includes three early-stage gene therapy programs intended to treat pediatric diseases, incl