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 ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

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

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

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

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung ...

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

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 ...