Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...

Biotechs in the charter city of Próspera aim to cure aging, but experts question the feasibility of their goals.

Progress of in vivo gene therapy for use in malignancies, monogenic disorders, and degeneration disease. AAV, adeno-associated virus; TK, thymidine kinase; ADA-SCID, adenosine deaminase-deficient ...

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 ...

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 ...

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, ...

In October 2025, Karolinska Institutet launched a brand-new two-week course giving PhD students a unique insight into how ...

The cell and gene therapy CRO market presents growth opportunities driven by FDA-approved therapies, over 90 global approvals, and 3,500 trials across 16 therapeutic areas, primarily in North America.