Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

Ultragenyx Pharma (RARE) stock is in focus as gene therapy DTX301 met its main goal in a late-stage trial for the most common ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.

Patients identified as having a Janus kinase inhibitor responder profile were much more likely to respond positively to the class of atopic dermatitis therapies. Scientists have identified gene ...

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...