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

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

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

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.

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

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Advancements in genome editing, particularly CRISPR, are reshaping precision biology, driving innovations in medicine, agriculture, and synthetic biology.

Genetic testing, via germline and somatic testing, is crucial in breast cancer to understand inherited risks and tumor ...

In an earlier study, patients with biomarker-positive high-grade glioma lived longer after treatment with DB107 than biomarker-negative patients.

However, they can also have unwelcome side effects, including adverse immune system reactions, gene errors and tissue damage. What excites me most about cell therapy is its potential to revolutionize ...