The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

CRISPR stocks are high risk but hold potential for aggressive investors. CRISPR Therapeutics and its partner Vertex Pharmaceuticals have the only approved CRISPR therapy. Several other biotech ...

Using a new gene-editing technique, a team of scientists from UT Southwestern (UTSW) Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. University of Texas (UT) ...

Gene therapy treatments have been making major headlines as advances in gene editing have gone mainstream thanks to the advent of CRISPR ... therapy for Duchenne Muscular Dystrophy (DMD) costs ...

28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR ... New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy ...

Three patients are in “safe, deep remission” after receiving a CRISPR-Cas9-modified cell therapy for autoimmune disease treatment, according to a new published study in Cell. Unlike traditional ...

A therapy based on CRISPR gene-editing technology that the ... enlarged organs and delayed puberty. The median age of death is 37 years. Stem cell transplants are another treatment option, but ...

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 ...

the latest shift for a tarantella company that has repeatedly switched direction and leadership since first emerging as one of the first CRISPR biotechs a decade ago. Editas will now focus ...

Now, Editas is turning its focus to “in vivo” gene editing, where a CRISPR therapy does its work modifying genes after it’s infused into the body. Reni-cel and Casgevy are both built from a ...