Gene Therapy | CHOP Research Institute

Gene Therapy

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.

Dr. Kadauke is the associate director of the Cell and Gene Therapy Laboratory at CHOP and the medical director of the Cell and Gene Therapy Informatics team. His research focuses on developing novel ways to manufacture cell therapies with the goal of accelerating and broadening patient access to these life-saving therapies.

kadaukes [at]

The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as UCART22 ("study drug").

Published on
Oct 14, 2022
Read the latest findings and new grants from Children’s Hospital of Philadelphia researchers.

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)