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Rocket Pharmaceuticals reports early success in gene therapy trial for heart condition PKP2-ACM, with 3 patients showing improved heart function. One had severe side effects.

Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene therapy to patients younger than 4 years old.

Initial clinical data from Genocury and EsoBiotec shows promise for in vivo CAR-T therapy, with complete remission in lymphoma and myeloma patients without lymphodepletion.

Merck's Keytruda shows 27% reduced risk of recurrence/death in head & neck cancer when used before & after surgery, with FDA decision expected by June 23

Gilead’s HIV drug business grew while its Covid drug sales tanked in the first quarter of 2025, as the company recorded the same amount of revenue compared to this time last year.

Gilead's antibody-drug conjugate Trodelvy combined with Merck's Keytruda reduced the risk of disease progression or death in a key study of an aggressive form of breast cancer.

Roche is stopping patients on the low dose of its experimental Huntington’s drug tominersen, and will be transitioning them to the high dose following recommendations by an independent data monitoring committee.

The FDA has permitted eGenesis to begin a clinical trial of a dialysis-like system that involves using a gene-edited pig liver outside of the body. It’s the second formal study ...

Verve Therapeutics is trying to swerve the gene editing industry downturn with new results on a potential one-time treatment to lower cholesterol. The experimental gene editing therapy cut bad cholesterol ...

Vertex halts VX-264 diabetes cell therapy due to poor efficacy in Phase 1/2 study, but continues Phase 3 trial of zimislecel (VX-880) with immunosuppression.

Phase 2 results on Eli Lilly’s experimental heart disease drug showed it could be dosed far less frequently than competing treatments in development from Novartis and Amgen. 

Heart gene therapy maker Tenaya Therapeutics is laying off 30% to 40% of its staff by the end of the year, the company’s CEO Faraz Ali told Endpoints News by email.

Decades ago, Amber Salzman remembers feeling like there was nothing she could do to help her cousin-in-law with a progressive muscle disease called facioscapulohumeral muscular dystrophy, or FSHD. She was ...

Regenxbio reports promising microdystrophin expression data in DMD patients, with highest levels in 3-year-old. Plans FDA filing mid-2025 for gene therapy RGX-202.

Cell therapy biotech RegCell is hoping to bridge science from Japan with the US biotech industry to develop new cell therapies for autoimmune conditions.

HHS Secretary Robert F. Kennedy Jr. and his staff last Wednesday hosted a regenerative medicine roundtable primarily to discuss how to lower regulatory barriers for stem cell treatments, three people familiar with the meeting confirmed to Endpoints News.

A pair of investors will acquire bluebird bio in a deal worth only $29 million upfront after the gene therapy maker ran out of time and options.

Solid Biosciences reports high microdystrophin levels in 3 children treated with SGT-003 gene therapy for Duchenne muscular dystrophy, shares rise 42% on promising data.

Radiopharmaceutical maker Lantheus Holdings plans to acquire Life Molecular Imaging for $350 million in a bid to build up its commercial Alzheimer’s diagnostics business. In addition to the $350 million ...

Dyne Therapeutics touted an early-stage study of its RNA treatment for myotonic dystrophy type 1 and has selected the dose it plans to move forward into pivotal studies. However, the company’s shares fell Friday morning,