Researchers uncover key mechanisms in gene regulation that may lead to better design of RNA-based medicines.

The lab of The Wistar Institute's Jessie Villanueva, Ph.D., has identified a new strategy for attacking treatment-resistant ...

Scientists have progressed from merely provoking an anti-cancer immune response to designing it ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

In the literary world, this year is shaping up to be a good one for fans of cultural criticism: There are new books about Better Call Saul and biographies of James Gandolfini and Lorne Michaels on ...

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?

It may be, she added, that weekly therapy sessions are simply not scalable to a broad population, and the field should explore light-touch alternatives, like single-session interventions and ...

Courtesy RegenxBio The arrangement allows RegenxBio “to leverage our expertise in gene therapy manufacturing while also capturing milestones and a meaningful share of future product revenues ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

However, I see gene therapy companies having a rougher road ... I would set a few sell orders at key levels to book some profit and move my NTLA position to a “House Money” status to create ...