The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...

R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Deloitte urged pharma executives to “be bold” in a new report tracking the top 20 pharmaceutical companies’ R&D performance.

The biotechnology sector is gaining new momentum with better market conditions, cutting-edge innovations, and growing ...

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible ...

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

Base editing corrected a mutation that causes macular degeneration, highlighting the potential of gene therapy to treat ...

Manufacturing engineers are at the forefront of bringing new medicines derived from human sources such as cells, blood and genes to patients. They produce what are often first-of-their kind ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...