Hematopoietic stem cell (HSC) gene therapy for hemophilia
Roctavian and Hemgenix are in-vivo gene therapies where an AAV vector delivers a functional copy of the defective gene to hepatocytes, the phy…
For years, digital biomarkers have seemed like a promising way to transform drug clinical trials, yet their adoption journey has been uneven. We’ve watched as the excitement for Proteus's ingestible …
In February 2025, the FDA gave IND approval to United Therapeutics for the first xenotransplantation clinical trial, which followed a series of high-profile transplants via the compassionate use pathw…
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