Sebastien Beauzile, 21, became the first person in New York state to be made symptom-free of sickle cell disease after ...

But they are not the same. Gene therapy, according to the FDA, modifies a person’s genes to treat or cure disease, and can ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Mía had dystrophic epidermolysis bullosa, better known as butterfly skin due to the extreme fragility of this tissue in ...

Development strategies designed to reduce the unknowns so that cell and gene therapy innovators can develop robust processes efficiently.

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

That’s important because thousands of mutations across more than 50 genes have been associated with RP, so a gene therapy that targets just one mutation would only work in a subset of patients.

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses how key regulatory designations, such as ...

Haseeb Ahmad brings over 25 years of leadership experience in life sciences, most recently serving as President of Novartis ...

Two years after Neil Armstrong walked on the Moon, US president Richard Nixon declared a new frontier in American scientific ...

“The KickStart Grant Awards help de-risk these technologies by establishing a collaboration agreement between the startup ...