One-year KYSA-8 and KYSA-6 data showed durable miv-cel benefit in SPS and gMG, supporting Kyverna's rolling BLA submission targeted for Q4 2026.
News|Articles|May 29, 2025
ALS Awareness Month 2025: A Review of the Cell and Gene Therapy Pipeline
Author(s)Noah Stansfield
In observance of ALS Awareness Month, held annually in May, we took a look at the clinical pipeline of advanced therapeutics currently in development for the neurodegenerative disease.
Advertisement
Advertisement
According to the ALS Association, a person is diagnosed with amyotrophic lateral sclerosis (ALS), progressive neurodegenerative disease affecting brain and spinal cord nerve cells, every 90 minutes. Diagnosis with ALS can take a substantial period of time—months or years—and although about 10% of patients can have their condition linked to an inherited mutated gene, 90% of cases are not known to be related to any genetic cause or family history. There is currently no cure for the condition.
Related to this article
FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A, based on cognitive data from an open-label trial in children 2 to 5 years old.
Novartis suspended several autoimmune and neurology trials of rap-cel after 3 patient deaths linked to immune effector cell-associated hemophagocytic syndrome, and BMS separately paused zola-cel studies after observing inflammatory safety events.
The FDA placed a new clinical hold on REGENXBIO's RGX-121 for Hunter syndrome after asymptomatic spine MRI findings were identified in 5 trial participants, the gene therapy's second regulatory setback this year.
The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data as a major BLA amendment.
PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.
FDA grants Kyverna's miv-cel RMAT designation in non-active secondary progressive MS, based on Phase 1 data showing CAR T expansion and B-cell reconstitution.
FDA advisory committee votes 9-3 against deramiocel's effectiveness for Duchenne cardiomyopathy; PDUFA decision due August 22, 2026.
Advertisement
Advertisement