In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...

Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study. This gene therapy is being evaluated for profound genetic hearing loss due ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

The MCO-010 vector uses surviving cells in the retina to produce a photosensitive protein that may help patients regain some ...

Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; ...

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

The gene therapy for neurological diseases market is poised for growth due to rising neurological disorder prevalence, demand ...

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

MIT engineers develop a new system for precise control of synthetic gene expression, potentially advancing gene therapy.