gene therapy

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.

R&D

pharmaphorum podcast Episode 253, Lance Baldo, Beacon Therapeutics

Looking to gene therapy for ocular diseases, with Lance Bald...

Lance Baldo, CEO of Beacon Therapeutics, discusses the transformative power of gene therapy to deliver meaningful outcomes for severe ocular diseases.

R&D