To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Adverse genetic mutations can cause harm and are due to various circumstances. 'Jumping genes' are one cause of mutations, but cells try and combat them with a specialized RNA called piRNA.

Adverse genetic mutations can cause harm and are due to various circumstances. "Jumping genes" are one cause of mutations, but cells try and combat them with a specialized RNA called piRNA.

An international team of clinical collaborators, led by physician scientists from Dana-Farber Cancer Institute, performed a ...

The rate of cGVHD-free survival was 78% at 1 year in patients who received Orca-T compared with 38% among patients who ...

Toon Van Gorp, MD, PhD, a MIRASOL trial investigator, emphasizes that the final analysis reinforces the efficacy of mirvetuximab soravtansine-gynx (Elahere; AbbVie) in patients with folate receptor ...

Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G) Fabhalta is the only oral alternative complement pathway ...

An intravenous form of the gene therapy is already approved and sold as Zolgensma in the US for patients less than 2 years of age.

Story Feb. 13 - Encoded Therapeutics: The gene therapy biotech is laying off 29% of staffers as it focuses resources toward its Dravet syndrome program. Most of the affected roles will be in ...