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 ...

Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in autoimmune diseases like lupus — but only if patients can get access to them.

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

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 ...

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