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

The FDA has approved Capsida Biotherapeutics' investigational new drug (IND) application for CAP-003 to treat Parkinson’s ...

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received clearance from the U.S. Food and Drug Administration (FDA) to begin a ...

Fractyl Health, Inc. has announced the submission of the first module of its Clinical Trial Application (CTA) for RJVA-001, a gene therapy candidate aimed at treating inadequately controlled type ...

Supply chains for a new generation of therapies are complex, costly, and require meticulous management to ensure patient ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

Upfront fees and near-term development milestone payments totaling up to $11 millionSales milestones of $150 million or more ...

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

The US Food and Drug Administration (FDA) has approved Ocugen’s investigational new drug (IND) amendment to initiate a Phase ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

For gene therapy developers ... optimising research processes for large-scale and GMP applications presents additional difficulties. AI algorithms can help identify capsid variants that are ...