Fierce Biotech

Bayer tosses 1 Pompe gene therapy from $2B AskBio buyout to focus on alternative option

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 ...
Technology Networks

Could tRNA Drugs Replace the One-Gene-One-Treatment Model?

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 ...
Rolling Out

Why gene therapy could revolutionize how we treat disease

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 ...
BioSpace

Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In

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.
BioSpace

UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question ...
KING5

Fred Hutch scientists advance gene therapy with simplified treatment method: HealthLink

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 ...
Fierce Pharma

BioMarin pulls hemophilia gene therapy Roctavian, taking $240M hit after divestiture efforts flounder

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

FDA details rationale for rejecting rare disease gene therapy from Regenxbio

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 ...
The American Journal of Managed Care

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

The Gene‑Therapy Breakthrough Story You'll Be Mad You Ignored at These Prices

Now, it’s worth noting Stock Advisor’s total average return is 892 % — a market-crushing outperformance compared to 194% for the S&P 500. Don't miss the latest top 10 list, available with Stock ...

FDA releases hold on second of Intellia’s ATTR-CM gene therapy trials

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 ...