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

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...

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

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

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 ...

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, ...

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

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. The New York pharma is ending global development and commercialization of Beqvez ...

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.