CSL Limited (OTC:CSLLY) and uniQure Inc (NASDAQ:QURE) announced the four-year results from the pivotal HOPE-B study ...

Data from the study showed that HEMGENIX continues to deliver sustained factor IX activity levels for up to four years post-treatment. The treatment also provides long-term bleed protection, ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

The adeno-associated virus (AAV) vector-based therapy is given as a one-shot intravenous infusion, delivering a gene for Factor IX, a clotting factor that is deficient in haemophilia B.

H.C. Wainwright analyst Patrick Trucchio reiterated a Buy rating on Sangamo Biosciences (SGMO – Research Report) today and set a price target ...

Given Sobi's cash flow generation from its hemophilia business and mature product Synagis, the firm ended 2024 with a net debt/EBITDA ratio of 1.6 times. We expect the firm can support this level of ...

BENGALURU: Bengaluru-based Narayana Nethralaya is developing gene therapy solutions that could drastically reduce the cost of ...

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...

Financial writer discusses MGX's gene editing technology, cash runway, and lack of clinical data for potential future ...

Gene therapy using modified blood stem cells helped five adults with severe hemophilia A reach therapeutic levels of clotting ...

University of California San Diego Health now offers a brand-new gene therapy that may change the playing field for sufferers of hemophilia B forever. The FDA-approved therapy is a one-time ...