Phase 1 data indicate Intellia’s medicine could be a powerful treatment for a cardiac form of ATTR amyloidosis. But rival drugs are further ahead.
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.
Analysts appear optimistic for Intellia’s gene editor nex-z, which showed a greater serum TTR reduction than Alnylam’s ...
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 ...
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