Recombinant adeno-associated virus (rAAV) stands as a crucial in vivo gene therapy platform, valued for its high transduction efficiency and established safety. However, challenges such as immunogenicity and the transient nature of transgene expression impede its...
Atsena Therapeutics, a clinical-stage gene therapy company, is pioneering a breakthrough approach to treat X-linked retinoschisis (XLRS), a genetic eye disorder primarily affecting males. The company has launched its Phase I/II LIGHTHOUSE study, targeting male...
