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 ...

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under ...

Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, BioMarin is ...

RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

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 ...

The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago ...

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 ...

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 ...

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD , from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first ...

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