Abeona gene therapy candidate selected for FDA RDEA Pilot Program

UniQure’s gene therapy slowed Huntington’s by 75% in a Ph1/2 trial. With an FDA filing planned for early 2026, it’s eyeing a potential launch next year. https://lnkd.in/e3Rhhv2Y

A UK-led study of uniQure’s AMT-130 gene therapy reported about 75% slowing of Huntington’s disease over 36 months in high-dose patients compared with Enroll-HD controls. Published in Nature and Science, this is the first convincing disease-modifying signal, though results are early and based on a small trial.

Powering Global Access Through an Integrated Supply Chain Infrastructure The cell and gene therapy revolution is global. With clinical trials being conducted in countries around the world and commercial therapies reaching new patient populations every year, the need for integrated, compliant, and scalable supply chain support has never been more urgent. Read our feature: https://hubs.li/Q03KD-Pw0 Sponsored by Cryoport Systems

Rare Therapeutics launched today! It is an affiliate company of GEMMA Biotherapeutics, and it focuses on gene therapy for ultra-rare diseases. Its clinical-stage portfolio includes GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy. Read more in today's press release: https://lnkd.in/ekqJeq3h Visit us at https://www.raretx.us/. #UltraRare #GeneTherapy #GlobalAccess

Exciting news✨ Huntington's disease successfully treated for the first time with a new gene therapy. The treatment has shown spectacular results, slowing the disease's progression by an average of 75% in patients. This could mean decades of extended, good quality life. Details: https://lnkd.in/dRVpSpdv #HuntingtonsDisease #GeneTherapy #Biotechnology

📢 Exciting news in the fight against spinal muscular atrophy (SMA)! An expanded use of Zolgensma may soon be available for patients aged 2 to 17 years. This new formulation could make a significant impact on a larger portion of the SMA community. Explore our latest pipeline report to learn more about innovative developments in gene therapy: https://cvs.co/3IwK1Zt

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first genetic treatment for the condition. The Amsterdam-based company announced that patients who received a high dose of its AMT-130 therapy for Huntington’s disease saw disease progression slow by 75% after 36 months. The treatment was “generally well-tolerated,” with a “manageable” safety profile, the company said. The full study results have not yet been published in a peer-reviewed journal. Read more: https://cnn.it/4gIXuu0

📢 Exciting news in the fight against spinal muscular atrophy (SMA)! An expanded use of Zolgensma may soon be available for patients aged 2 to 17 years. This new formulation could make a significant impact on a larger portion of the SMA community. Explore CVS Caremark's latest pipeline report to learn more about innovative developments in gene therapy: https://cvs.co/3IwK1Zt #CVSCaremark #SMA #GeneTherapy #Zolgensma #FDAApproval #TeamCVS

📢 Exciting news in the fight against spinal muscular atrophy (SMA)! An expanded use of Zolgensma may soon be available for patients aged 2 to 17 years. This new formulation could make a significant impact on a larger portion of the SMA community. Explore CVS Caremark's latest pipeline report to learn more about innovative developments in gene therapy: https://cvs.co/3IwK1Zt #CVSCaremark #SMA #GeneTherapy #Zolgensma #FDAApproval #TeamCVS

Exclusive: Cirrus Therapeutics launched with $11M to develop a gene therapy for dry age-related macular degeneration, aiming to slow vision loss by boosting an immune protein in the eye. https://lnkd.in/g8BzRhmT