Sarepta is facing a number of issues currently, including platform-level safety concerns, management credibility issues, etc.

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not ...

The approval makes donidalorsen the only treatment in the US for hereditary angioedema that is an RNA-targeted prophylactic.

In 1957, just four years after Francis Crick and other scientists solved the riddle of DNA’s structure—the now famous double ...

Since 2008, the FDA has approved 11 treatments for a rare genetic condition called hereditary angioedema (HAE), three of ...

Dawnzera, a prekallikrein-directed antisense oligonucleotide, is supplied as a 80mg/0.8mL solution in a single-dose autoinjector.

Researchers at San Diego State University and Michigan State University are shedding new light on how viruses meticulously ...

Similar to the way the caps on the ends of a shoelace prevent it from fraying, telomeres—regions of repetitive DNA sequences ...

Genomics, Inc. (Nasdaq: TXG), a leader in single cell and spatial biology, today announced the launch of Xenium Protein, a ...

Merck KGaA has swooped into a neurological-focused pact with RNA biotech Skyhawk Therapeutics that could top out at $2 ...

The collaboration will explore RNA-based small molecule drug candidates aimed at neurological conditions where existing ...

The quest for a vaccine against HIV has been going on for about 40 years — almost as long as the virus that causes AIDS has ...