The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

In a challenging market environment, Adverum Biotechnologies Inc. (NASDAQ:ADVM) stock has reached a 52-week low, dipping to $4.01. The biotechnology firm, which specializes in gene therapy targeting ...

Researchers at the Johns Hopkins Kimmel Cancer Center and Bloomberg~Kimmel Institute for Cancer Immunotherapy have developed a computer model to help scientists identify tumor-fighting immune cells in ...

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval On track to dose first patient in Phase 3 trial in second half of 2025 LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) ...

Scientists have progressed from merely provoking an anti-cancer immune response to designing it ...

NHS approves £1.65m gene therapy for sickle cell disease, offering a potential cure. A milestone for patients, especially in ...

Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...

Researchers at Cellectis leveraged gene editing to create a CAR T cell that acts in an IF/THEN system. Traditional methods to develop CAR T cells that use the patient’s cells introduce challenges ...

Researchers at the Johns Hopkins Kimmel Cancer Center and Bloomberg-Kimmel Institute for Cancer Immunotherapy have developed ...

One sickle cell patient told ITV News the new gene therapy being rolled out for use on the NHS means they "finally have something to look towards". | ITV National News ...

Previous research has shown that voretigene neparvovec administered subretinally early in childhood for RPE65-mediated ...

In 2019, the first CRISPR clinical trials were conducted to treat sickle cell diseases and in 2020, this treatment was ...