Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.

UNC Charlotte researchers are contributing to pioneering breakthroughs in cancer therapeutics, communicable disease ...

Childs Cure co-founders Jainu and Shruti Jogani founded the organization to address the genetic causes of rare diseases, ...

New genetic therapy has brought “life-changing improvements” in the vision of the infants born with one of the most severe forms of childhood blindness.According to Kings College London, an ...

The FDA has accepted for Priority Review the BLA for PRGN-2012 for the treatment of adults with recurrent respiratory papillomatosis.

Anova Enterprises, Inc. (Anova), a technology-enabled CRO dedicated to accelerating promising treatments, has announced enrollment of the first two pa ...

Ten of 11 children born with a rare form of congenital hearing loss experienced improvements after receiving the company’s ...

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...

A gene therapy from Regeneron Pharmaceuticals Inc. improved hearing in nearly all deaf children in a clinical trial, results ...

New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.

Jace, from Connecticut, is one of four children all born with severe childhood blindness who gained 'life-changing ...

Regeneron’s gene therapy, dubbed DB-OTO, is a cell-selective AAV gene therapy for children with hearing loss stemming from a ...