Expression Therapeutics announced promising Phase 1 clinical trial results for its novel gene therapy, ET3, treating hemophilia A.
The trial, detailed in the New England Journal of Medicine, showed significant increases in Factor VIII levels and a reduction in bleeding events in participants with severe hemophilia A.
The therapy uses a lentiviral vector targeting hematopoietic stem cells, offering a potential for a permanent cure and overcoming limitations of existing AAV-based therapies.
Positive safety and efficacy results pave the way for further development, potentially benefiting both adult and pediatric populations.
https://www.expressiontherapeutics.com
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