Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

Genetic carrier screening is conducted to determine if an asymptomatic person is a carrier for a potentially inheritable pathogenic gene.

Already offering the only non-invasive treatment for SMA in liquid form, Roche’s Evrysdi now comes as a tablet.

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

Dr. Tejal Aslesh Tailor is an accomplished neuroscientist and molecular biologist with years of expertise in preclinical ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation ...