ALS gene therapy

Columbia University researchers will use a $15 million federal grant to design individualized gene therapies for nine people with ultra-rare genetic forms of amyotrophic lateral sclerosis (ALS). The three-year grant comes from the National Institute of Neurological Disorders and Stroke’s (NINDS) Ultra-rare Gene-based Therapy (URGenT) Network, a program…

Coave Therapeutics has received a grant from the ALS Association to support the development of its gene therapy candidate CTx-TFEB for amyotrophic lateral sclerosis (ALS). The grant, made through the Lawrence and Isabel Barnett Drug Development Program, is part of the $2.9 million the ALS Association…

VectorY Therapeutics has raised about $138 million in funding aiming to bring into clinical testing VTx-002, its lead treatment candidate for amyotrophic lateral sclerosis (ALS). The therapy equips nerve cells to produce antibodies against the damaging TDP-43 protein clumps that are an ALS hallmark. “The investment…

SOL-257, an experimental one-time gene therapy, was well tolerated and significantly improved disease outcomes in two distinct mouse models of amyotrophic lateral sclerosis (ALS). Developed by Sola Biosciences, SOL-257 is designed to clear the toxic TDP-43 protein that accumulates and forms clumps in ALS nerve cells, contributing to…

Anew Medical‘s targeted gene therapy candidate, ANEW-202, eased multiple mechanisms associated with amyotrophic lateral sclerosis (ALS), leading to improved muscle function and survival in a mouse model of the disease. The gene therapy, licensed on an exclusive worldwide basis from the Autonomous University of Barcelona, in Spain, is…