SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...

Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...

Wilson celebrates the first U.S. patient to complete gene therapy for sickle cell, but says advocacy must continue.

Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...

In an update posted today, Neurogene said that the "emerging treatment-related serious adverse event (SAE)" was a case of ...

Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

Neurogene is proceeding with a scaled-back study of its proposed NGN-401 gene therapy for the rare genetic neurological disorder Rett syndrome after a serious adverse event left a study participant in ...

In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...

Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that ...

It is driven by developments in personalised medicine, gene therapy, and CRISPR technology. The global regenerative medicine ...

We recently compiled a list of the ARK Invest Stock Portfolio: Top 10 Stock Picks for 2024. In this article, we are going to ...