GlobalData on MSN

Huntington’s gene therapy research has reached a turning point, expert says

An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and challenges with the FDA.

1st Michigan patient undergoes FDA-authorized sickle cell gene therapy

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...
Fierce Pharma

CSL warns of supply shortfall, treatment delays for hemophilia gene therapy Hemgenix

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working with regulators to ensure stable ongoing supply for the one-time gene therapy ...
Hoodline

Detroit Man Becomes First Michigander To Get Breakthrough Lyfgenia Gene Therapy

At just 24, Chantez "CJ" Sanford is quietly making Michigan medical history. He is the first person in the state to receive ...

SK pharmteco Reaffirms Three-Business-Unit Strategy, Strengthens Global Gene Therapy Business Unit with Funding Commitment

SK pharmteco today announced a significant funding commitment to support strategic initiatives within its viral vector ...

Ultragenyx’s gene therapy meets co-primary endpoint in Phase III trial

Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.
Technology Networks

Milk-Derived Nanoparticles Deliver Targeted Gene Therapy to Bile Duct Cancer

Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...

An AI-guided gene-editing tool for more precise and safer DNA correction

Researchers at the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) have developed a revolutionary new method to improve compact gene-editing tools known as base ...

Belief BioMed announced BBM-H901 (Dalnacogene Ponparvovec Injection), China's first hemophilia B gene therapy, was officially approved in Macao, China

Belief BioMed (BBM) today announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially ...
The University of Alabama at Birmingham

Investigational gene therapy trial for rare kidney disease begins clinical trial at UAB

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Man receives gene therapy to cure sickle cell disease, hopes to become licensed pilot

Daniel Cressy is the first person in the area to receive the groundbreaking treatment.

Apertura Gene Therapy and Viralgen Announce Strategic Collaboration to Manufacture TfR1 CapX, a Next-Generation Capsid for Gene Therapies Designed to Treat Central Nervous ...

NEW YORK and SAN SEBASTIÁN, Spain, March 17, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and ...