A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits, the new approach developed at UC San Diego ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, ...

Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on ...

Beam Therapeutics rises after the FDA grants orphan drug status to its gene-editing therapy BEAM-101 for sickle cell disease.

VG801 is a dual AAV gene therapy that leverages mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.