Gene Therapy | CHOP Research Institute
 

Gene Therapy

Published on
Mar 15, 2024
This week, CHOP announces two new Frontier Programs, and researchers define the origins of hematopoietic failure in Fanconi anemia.
Published on
Jan 23, 2024
This milestone represents an important step in treatment options for patients with hereditary hearing loss caused by genetic mutations.
Published on
Jan 3, 2024
After decades of limited progress in treating sickle cell disease, we have reached an historical moment with two new gene therapies.

The purpose of this study is to test and find a safe dose of this investigational gene therapy PBGM01 (also known as, the “study drug”) in children who have been diagnosed with the early infantile or late infantile form of GM1.

Published on
Oct 10, 2023
CHOP's NoT Bleeding Program will conduct clinical trials to develop the next generation of therapeutics for hemophilia A, B, and other bleeding disorders.
Published on
Aug 29, 2023
The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.
Published on
Jul 31, 2023
Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.
Published on
Jun 26, 2023
The FDA has granted accelerated approval of the first gene therapy for Duchenne muscular dystrophy — a major win for those affected by the debilitating disorder, CHOP experts say.
Published on
May 18, 2023
Scientists from Children’s Hospital of Philadelphia attend the American Society of Cell & Gene Therapy Annual Meeting to share their expertise through posters, panel discussions, and workshops.
Published on
Feb 6, 2023
Two scientists with unique skill sets are developing an alternative method for gene therapy delivery with support from a CGTC Seed Grant.