Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...

The Philadelphia Citizen is recognizing CHOP doctors Alexis Thompson and Dr. Stephan Grupp with the 'Disruptors of the Year' ...

Pfizer said on Thursday it will end global development of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors. Beqvez was approved in the U.S. last year, for the ...

Pfizer will end global development of its bleeding disorder gene therapy, Beqvez, the Nikkei reported on Thursday.

A Phase III trial evaluating nex-z in transthyretin amyloidosis with cardiomyopathy is underway, with plans for another ...

Recent innovations in DNA-modifying will revolutionize the treatment of many diseases and prompt crucial discussions.

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters ...

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.

The trial was tailored to evaluate the therapy at multiple ascending dose levels, with three dose administrations per level.

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...

Cardiac arrhythmias affect millions across the world and are responsible for a fifth of all deaths in the Netherlands.