Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...

The new bid surpasses an offer SK Capital and Carlyle Group made last month for the gene therapy developer, which Bluebird’s board still supports.

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

For gene therapy developers, every key milestone significantly influences long-term success and the timeline for commercialisation. Yet, amidst this pivotal moment, a crucial question looms: ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.

The American Cancer Society estimates that 83,000 new cases of bladder cancer will be diagnosed this year, with almost 17,000 fatalities. But now, a new gene therapy is giving patients more hope of ...

Get Instant Summarized Text (Gist) A novel gene therapy has been developed that alleviates symptoms of Dravet syndrome in mice without side effects. This therapy uses a two-step strategy ...

The goal of the new therapy was “to be very precise” and “just deliver the gene that’s missing.” The result is a treatment that is safer and more effective with significantly fewer side ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment. The company behind the drug, Sarepta ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...