Neurogene Halts Batten and Rett Gene Therapy Programs Amid FDA Setbacks and Stock Plunge

Neurogene recently faced a significant setback when the FDA denied their request for Regenerative Medicine Advanced Therapy (RMAT) designation for their CLN5 Batten disease gene therapy program, NGN-101. Despite being in Phase I/II trials and pioneering testing for CLN5, the FDA's denial, attributed to the rarity of the disease, rendered the continuation of the program unfeasible. This decision was compounded by the lack of a streamlined FDA pathway, prompting Neurogene to halt further advancements of NGN-101, which were initially supported by modest financial commitments[1][2].