ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

Ocugen, Inc. gene therapy outlook: key 2026 catalysts, Phase 2/3 eye-disease data, BLA plans to 2028, and risks. Click for ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

Cell and gene therapy are revolutionary approaches to treating diseases at the molecular level. They involve the manipulation ...