A collaboration between scientists at the Institute of Molecular and Clinical Ophthalmology Basel (IOB) and Beam Therapeutics has resulted in a new base editing therapy for Stargardt disease, the most ...

X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder characterized by high susceptibility to infections that results from genetic mutations in the cytochrome b-245 beta chain ...

Baby KJ was the first to receive a bespoke gene-editing treatment. Personalized drugs for others could be approved within the ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

Beam Therapeutics’ experimental gene-editing therapy for sickle cell disease now has its first cut of patient data showing signs of treating the rare blood disorder in a way that could differentiate ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...