Scientists have made a key breakthrough in treating respiratory diseases by developing a new drug delivery system that ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

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

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP). The OCU400 (AAV5-h NR2E3) gene therapy platform ...

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

Ocugen is currently advancing OCU400 through Phase 3 clinical development with a target Biologics License Application filing of mid-2026.

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

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

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

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

UniQure said it has aligned with the FDA on components of a statistical analysis plan for a registrational trial of AMT-130.