Clinical In Vivo Gene Therapy

Clinical In Vivo Gene Therapy
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The Clinical In Vivo Gene Therapy (CIGT) group at Children's Hospital of Philadelphia (CHOP) is dedicated to advancing clinical in vivo gene therapy. In vivo gene therapy involves directly infusing a new, corrected gene into a child’s body, with the goal of stopping disease or making it less severe. Through industry partnerships and clinical trials, CIGT seeks to make transformative discoveries and build upon CHOP’s legacy as a leader in this field.

Led by experts with extensive experience in gene therapy clinical trials, our team aims to offer more breakthrough therapies to our patients sooner, by streamlining complex gene therapy trials and ensuring licensed gene therapies are available in our clinics.

How we serve you

We partner with programs throughout CHOP to make game-changing advances.

Conditions we treat

We constantly strive to create more in vivo gene therapy options for children with a variety of conditions.

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Meet your team

Our experts are at the forefront of clinical in vivo gene therapy breakthroughs.

Patient stories
Our Stories
Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.
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Our contributions

View this slideshow to see the breakthroughs we've made over the years.
March 2000

Hemophilia breakthrough
First in vivo gene therapy for hemophilia B and early use of AAV vector developed at CHOP
2005
Founding of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics
Molecular Genetics Laboratory
March 2006

First systemic administration of an AAV vector
CHOP has more experience than any other institution using systemic AAV vectors.
White doctor analyzing sample in a research laboratory
2007

Clinical Vector Core established at CHOP
This core manufactures clinical and pre-clinical adeno-associated virus (AAV) and Lentivirus (LV) vectors.
March 2007

First outline of human cellular immune response to AAV
Researchers at CHOP outlined the initial human immune response to AAV vectors.
April 2008

Congenital blindness breakthrough
Researchers from CHOP and Penn used gene therapy to safely improve vision in patients with Leber's congenital amaurosis (LCA).
October 2009

Defining FIX-Padua
A CHOP investigator defined FIX-Padua, now in use for all current hemophilia B gene therapy efforts.
December 2017

Factor IX Padua breakthrough
First successful use of gene therapy with Factor IX Padua for near curative levels in hemophilia B.
July 2018

Inherited retinal dystrophy breakthrough
First CHOP patient treated with approved in vivo gene therapy for inherited retinal dystrophy
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July 2019

SMA breakthrough
First CHOP patient treated with approved in vivo gene therapy for spinal muscular atrophy (SMA).
2021
Founding of Clinical In Vivo Gene Therapy group, dedicated to advancing these therapies at CHOP.
November 2021

Developments in hemophilia A
CHOP led the clinical trial that demonstrated stable multi-year Factor VIII expression after AAV gene therapy for hemophilia A
Lab technician with sample
2022
In vivo gene therapy trials at CHOP span across 7 clinical divisions: ENT, endocrinology, neurology, genetics, cardiology, opthalmology and hematology.
April 2022
First-ever in vivo delivery of an experimental CRISPR gene editing medicine to a pediatric patient enrolled in CHOP clinical trial.
CRISPR-edited lung cells

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A gift of any size helps us make life-changing breakthroughs and advances our work for children everywhere.

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