Neurogene Faces Setbacks in Batten and Rett Syndrome Gene Therapy Programs

Neurogene faced a significant setback with its Batten disease gene therapy program recently, following a decision to halt the advancement of NGN-101 after being denied the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation[1][2]. Despite being the first to test a CLN5 therapy and completing Phase 1/2 trial enrollment, Neurogene determined that progressing with the trial was unviable due to the disease's rarity and lack of a streamlined FDA path for future development[1][2]. The program, originally licensed from the University of North Carolina at Chapel Hill, required only modest financial investments, yet the decision to halt it led to a considerable drop in the company's stock, highlighting the challenges Neurogene faces in pioneering gene therapies for rare diseases[2].