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|April 11, 2025
World Parkinson's Day 2025: Looking Back at Progress for Cell and Gene Therapy
Author(s)Noah Stansfield
In observance of World Parkinson's Day, held annually on April 11, we took a look back at the past year's news in cell and gene therapy for PD.
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According to the Michael J. Fox Foundation, almost 1 million people in United States live with Parkinson disease (PD) and more than 6 million people in the world live with PD. Notably, the symptoms of PD and their severity vary widely, and can be different for every person. Although some treatment options for the disease exist, there is currently no cure and great unmet need remains for the patient community.
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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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