April 4 (Reuters) - Uniqure NV: * Uniqure NV -publication in gene therapy of data demonstrating widespread transduction in cns following direct injection of co's aav5 vector in large animal model Sign ...
The phase 3 GENEr8-1 study demonstrated that valoctocogene roxaparvovec, an AAV5-vectored gene therapy, effectively enabled endogenous FVIII production in adults with severe hemophilia A without ...
-- Data Suggest AAV5 Gene Therapies May be Viable Treatments for at Least 97% of Patients -- -- Data Presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Chicago -- ...
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