News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, ...

AviadoBio has opened the multi-centre Phase I/II ASPIRE-FTD trial in the UK, aimed at assessing the gene therapy candidate ...

A new clinical trial, delivered in Cardiff, will investigate the use of a one-time gene therapy to stop the disease ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

Oral presentation highlights data showing FLT201 results in durable enzyme expression and sustained clinical benefit in Gaucher disease Preclinical data for SPR301 support best-in-class potential for ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry ...

Mass photometry enables rapid, purification-free quantification of rAAV vectors, offering a faster solution for gene therapy ...

AGC Biologics has collaborated with Quell Therapeutics to advance the development of several T-regulatory (Treg) cell therapy ...

Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing ...

A Type D meeting was held with the FDA in March 2025 to discuss the potential regulatory path for OPGx-LCA5, including the ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...