Elevidys came under scrutiny in June when the FDA disclosed two fatal cases in non-ambulatory boys who developed dangerously high liver enzyme levels
The expanded mbDNA portfolio delivers enhanced stability, reduced immunogenicity, and broad compatibility with gene insertion and expression technologies
Results demonstrate favorable safety profile and clinically meaningful improvements in patients with non-ischemic heart failure
Adverum's lead program, Ixo-vec, is a Phase 3 gene therapy designed to treat vision loss associated with wet age-related macular degeneration with a single intravitreal dose
Several technologies for hemoglobinopathies are being transferred to commercial partners
New offering to debut at the 32nd ESGCT Congress, October 7–10, 2025, in Seville, Spain
Lonza boosts TheraPEAK line with new Cytokines and AAV Medium for next-gen therapies
NouvNeu001 is the world’s first allogeneic iPSC-derived cell therapy for Parkinson’s disease to receive this designation
Collaboration reinforces Boehringer Ingelheim’s expertise in microbial contract development and manufacturing
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