Browsing Tag
gene therapy
66 posts
Sarepta Therapeutics awards 25 Route 79 scholarships in 2025–2026, but what makes this Duchenne program unique?
Sarepta Therapeutics awarded 25 Route 79 scholarships for 2025–2026 to support students impacted by Duchenne. See how this initiative strengthens community ties.
Why Rocket Pharmaceuticals shareholders are filing suit over adverse event risks in RP-A501
A class action lawsuit claims Rocket Pharmaceuticals misled investors about the safety and effectiveness of its gene therapy. Find out what’s at stake.
Rocket Pharmaceuticals receives FDA clearance to begin human trials for RP-A701 in treating BAG3-associated dilated cardiomyopathy
Rocket Pharmaceuticals earns FDA IND clearance for RP-A701, advancing gene therapy for rare BAG3-associated dilated cardiomyopathy. Trial begins in U.S.
Klotho Neurosciences begins manufacturing KLTO-202 gene therapy to advance clinical trials in ALS
Klotho Neurosciences begins KLTO-202 manufacturing to prepare for ALS clinical trials, with a gene therapy targeting neuroprotection in spinal neurons.
GenSight Biologics completes LUMEVOQ tech transfer to Catalent to enable global clinical expansion
GenSight Biologics advances gene therapy LUMEVOQ® through strategic manufacturing transfer to Catalent, bolstering regulatory readiness and clinical scale-up.
Genethon and Atamyo expand gene therapy pipeline with key trial milestones and global collaboration momentum
Genethon and Atamyo Therapeutics progress gene therapy trials for Duchenne and LGMD, while France’s GenoTher Biocluster strengthens global biotech alliances.
AstraZeneca moves deeper into cell therapy with $1bn EsoBiotec acquisition
AstraZeneca’s $1 billion acquisition of EsoBiotec signals a major shift in cell therapy innovation. Find out how this deal could transform cancer treatment.
4D Molecular Therapeutics strengthens cash position, eyes Phase 3 success for 4D-150
4D Molecular Therapeutics (4DMT) has announced its full-year 2024 financial results, highlighting key advancements in its late-stage clinical…
Pacira BioSciences strengthens gene therapy pipeline with GQ Bio takeover
Pacira BioSciences, Inc. has taken a significant step toward expanding its presence in genetic medicine by acquiring the…
MeiraGTx’s gene therapy trial shows transformative results for children with LCA4 retinal dystrophy
MeiraGTx Holdings plc has announced groundbreaking results from its gene therapy trial targeting Leber congenital amaurosis 4 (LCA4),…