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 ...

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 ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

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

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

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

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

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

ROCKVILLE, MD / ACCESS Newswire / June 16, 2025 /uBriGene Biosciences, a leading cell and gene therapy CDMO, is proud to ...

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an ...

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 ...