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

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

In June, the monoclonal antibody garadacimab (Andembry) was approved for prevention of HAE attacks at a dose of once monthly ...

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

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

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

Single-cell proteomics with RNA sequencing uncover hidden gene expression dynamics in stem cell differentiation.

DAWNZERA is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks ...

On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera ...

As aging progresses, the quality of DNA and proteins inside cells declines, which is known to be the cause of various ...

Using artificial intelligence, MIT researchers have come up with a new way to design nanoparticles that can more efficiently ...

Skyhawk Therapeutics, Inc., a leader in the discovery and development of small molecules that modulate RNA expression, today ...