Clinical Trials | CHOP Research Institute
 

Clinical Trials

Published on
Jun 20, 2024
Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.
Published on
Jun 3, 2024
The CHOP-Penn Autism Research Symposium explored opportunities for breakthroughs in the understanding and treatment of autism spectrum disorder.
Published on
May 29, 2024
Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.
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.
Published on
Dec 22, 2023
Our research news roundup highlights newly approved gene therapies, insights into common genetic epilepsy disorders, and head injury prevention for young athletes.

The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF).

This is a study to measure the effectiveness of the medication voclosporin compared to placebo in adolescent patients with active lupus nephritis.

The purpose of this study is to test an investigational medication in children and adolescents aged 5-17 years old with irritability associated with autism spectrum disorder (ASD).

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.

E-mail:
kadaukes [at] chop.edu

Combining our proven clinical expertise in hemophilia gene therapy and care of bleeding disorders with our research expertise to solidify Children’s Hospital of Philadelphia as the premier center for the therapeutic development and advanced clinical care of inherited bleeding disorders.