The goal of the agreement is to advance cell and gene therapy accessibility by enabling scalable and efficient cell therapy manufacturing
Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector
The company is rolling out ARTEMIS, a next-generation AI platform for capsid design
CTx001 targets Geographic Atrophy (GA) secondary to Age-related Macular Degeneration
Heart failure affects an estimated 64.3 million people worldwide
Applications include detailed scientific rationale and supporting clinical evidence
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
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