Matt Hoffman, Associate Editorial Director for CGTLive™, has covered medical news for MJH Life Sciences, CGTLive’s parent company, since 2017. He also hosts the Medical World News show, Deep Dive. Follow him on Twitter @byMattHoffman or email him at [email protected]
AMP-101 Shows Pharmacologic and Safety Promise for Neuromuscular Disorders in Preclinical StudyMarch 23rd 2023
The Amplo Biotechnology gene therapy showed positive findings in mouse models, suggestive of the therapy’s development for use in a clinical setting. The AAV therapy delivers DOK7 gene, which has shown benefit in neuromuscular disorders.
Sarepta’s SRP-9001 Shows Functional Improvements in DMD Integrated AnalysisMarch 22nd 2023
Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.
FDA Revisits Need for Advisory Committee on Sarepta’s DMD Gene TherapyMarch 18th 2023
Ahead of the May 29, 2023, action date, the FDA is now seeking to hold an advisory committee meeting on Sarepta’s investigational agent SRP-9001 (also known as delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy.
Hemgenix Displays Superiority to Factor IX Prophylaxis for Hemophilia B in HOPE-B Trial DataMarch 14th 2023
The full dataset, published in the New England Journal of Medicine, reveals the newly approved hemophilia B gene therapy reduced annualized bleeding rates and improved the need for factor IX infusions.
FDA Accepts BLA Resubmission for Remestemcel-L in Steroid-Refractory aGVHDMarch 9th 2023
Mesoblast’s allogeneic off-the-shelf therapy showed long-term benefit in supporting data among children with steroid-refractory acute graft versus host disease. The FDA has set a PDUFA action date of August 2, 2023, for its review.
UX111 Shows Continued Dose-Dependent Efficacy as an MPS IIIA TreatmentFebruary 28th 2023
Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.
Parkinson Disease Gene Therapy Demonstrates Continued Improvement in 6-Month DataJune 14th 2019
Axovant reported positive safety data as well as improvements in a number of measurements of motor function and dyskinesias in patients with Parkinson disease who received treatment with their investigational gene therapy.
Can Striatal CaV1.3 Genetic Silencing Prevent Levodopa-Induced Dyskinesias in Parkinson?May 17th 2019
New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.