A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin ...

How cell and gene therapy developers can lessen the chances of receiving FDA complete response letters through the use of ...

In a preclinical study, UC Davis Comprehensive Cancer Center scientists have developed a highly targeted gene therapy that ...

The FDA has granted Fast Track designation to SAR446268 for the treatment of non-congenital myotonic dystrophy type 1.

Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the ...

By Daniella Parra NanoCell Therapeutics, Inc. has appointed three new members to its Scientific Advisory Board: Prof. Zoltán ...

M&A headlined for a second straight week as Genmab acquired Merus for $8 billion; Pfizer strikes most-favored-nation deal ...

After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s ...

I cried with every single patient,” Sung says. “It just was this crazy feeling that, for the patients and families, almost can’t feel real.” The treatment, known as AMT-130, is delivered deep into the ...

With several disease-modifying therapies available for transthyretin amyloid cardiomyopathy (ATTR-CM) -- and more waiting in ...

We caught up with the team behind the first ever mobile leukapheresis center to see what community-based care could look like ...

A student who had sworn off having children in case she gave them the deadly Huntington's disease says a 'life-changing' ...