Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

Spacecraft unveils 1st-ever images of sun's south pole: 'Beginning of a new era' ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

SpliceBio's lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by ...

Spain’s SpliceBio will use $135 million from a series B round backed by Sanofi and Roche to advance its lead gene editing ...

RNA gene scissors (CRISPR-Cas13) are gaining significant attention as a next-generation gene therapy with fewer side effects.