gene therapy

News

Ultragenyx wins FDA approval for first treatment targeting r...

Ultragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla.

News

Skylark trials gene therapy for common form of child deafnes...

Skylark Bio has dosed the first patient in a phase 1/2 trial of SKY-GJB2, a gene therapy for the most common form of genetic deafness in children.

R&D

pharmaphorum podcast Episode 267, Haseeb Ahmad - Purespring Therapeutics

Purespring on nephrology & gene therapy in kidney disease

Purespring CEO Haseeb Ahmad discusses transforming the treatment of kidney disease, an area of enormous unmet need, and the company's gene therapy.

News

Regenxbio's Duchenne gene therapy clears phase 3 hurdle

Regenxbio is pitching at FDA approval in 2027 for its Duchenne muscular dystrophy gene therapy RGX-202, a potential rival to Sarepta/Roche's Elevidys.

R&D

Podcast interview with Boyang Wang, the founder of Immortal Dragons

Why everyone should care about longevity, with Boyang Wang

In today’s episode of the pharmaphorum podcast, host Jonah Comstock speaks with Boyang Wang, the founder of Immortal Dragons.

News

FDA clears first genetic hearing loss gene therapy

Regeneron's Otarmeni is the first gene therapy for congenital hearing loss to be cleared by the FDA, and will be made available for free in the US.

Ultragenyx wins FDA approval for first treatment targeting r...

Skylark trials gene therapy for common form of child deafnes...

Purespring on nephrology & gene therapy in kidney disease

Regenxbio's Duchenne gene therapy clears phase 3 hurdle

Why everyone should care about longevity, with Boyang Wang

FDA clears first genetic hearing loss gene therapy

ESC26: Isomab safety evidence on blood vessel growth for CAD

Discover a new era of healthcare panel engagement: Grounded ...

gene therapy

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