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|June 18, 2025
Alzheimer's & Brain Awareness Month 2025: Looking Back at Recent Progress in Cell and Gene Therapy
Author(s)Noah Stansfield
In honor of Alzheimer's & Brain Awareness Month, observed annually in June, we took a look back at recent news and insights in cell and gene therapy for Alzheimer disease and other dementias.
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According to the Alzheimer's Association, more than 55 million people across the globe currently live with Alzheimer disease (AD) or another dementia. Furthermore, 2 in 3 Americans possess 1 or more major potential dementia risk factor.
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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.
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