The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...
At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...
Studies of Hunter and Hurler syndromes on hold ...
US biotech firm Fractyl Health is developing Rejuva, an experimental gene therapy that may mimic GLP-1 drugs for years after ...
Sangamo Therapeutics recently reported detailed data from its completed registrational Phase 1/2 STAAR study of isaralgagene ...
Taysha Gene Therapies recently advanced its TSHA-102 Rett syndrome program, dosing the first patient in the REVEAL pivotal phase and receiving FDA authorization to use ASPIRE trial data in a planned ...
Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...