A Peninsula biotech company raised $68 million from investors — including a venture philanthropy fund started by Lululemon ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

CRISPR has made it cheap and easy. How CRISPR therapy could cure everything from cancer to infertility The imminent approval of the world's first CRISPR treatment for sickle cell disease is just ...

CRISPR technique can target single genes essential for cancer cell survival, toppling the whole structure, says nanomedicine ...

Stoke Therapeutics CEO Ed Kaye is stepping down, the company said. He will be replaced on an interim basis by Director Ian ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24 ... by using a novel in vivo CRISPR genome editing ... Researchers Discover Potential Mole Reversal Therapy in ...

SGT-003 gene therapy showed significant microdystrophin expression and muscle integrity improvements in DMD patients, with no serious adverse events reported. The novel AAV vector demonstrated ...

The company added that the therapy was designed using the company’s proprietary CRISPR/Cas gene-editing ... candidate for patients with Duchenne muscular dystrophy (DMD) has elicited a 110% ...

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the ...