Cell and Gene Therapy Innovations News

Building on our proven track record in cell and gene therapy, CHOP is committed to discovering the next life-changing treatments for the most serious diseases.

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Children’s Hospital of Philadelphia Launches Global Partnership to Establish a CAR-T Therapy Program in Brazil

Nov 7, 2024

Made possible with a $4 million grant from the Brazilian Ministry of Health, CHOP cell therapy specialists will offer their expertise and resources to oversee this initiative.

CHOP Cell and Gene Therapy Researcher Receives NIH New Innovator Award

Oct 8, 2024

Evan Weber will use this “high risk, high reward” grant to fund research into improving CAR-T therapies.

Two CHOP Patients with an Inherited Blindness Successfully Treated with Gene Editing

May 6, 2024

The findings from the clinical trial were published in the New England Journal of Medicine.

CHOP Researchers Improve Fitness of Cells Used in Cell Transplants

Sep 26, 2023

CHOP researchers find exposing cells to a small molecule drug improves cell fitness during hematopoietic stem cell transplants, which could improve ex vivo gene therapy.

NIH Awards Penn Medicine and Children’s Hospital of Philadelphia $26 Million Grant to Develop Therapies for Rare Newborn Genetic Diseases

Aug 1, 2023

A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.

CHOP Treats 500th CAR T Patient

Jul 17, 2023

CHOP has treated its 500th patient with CAR T-cell immunotherapy, a “living drug” that harnesses the power of a patient’s own immune system to fight cancer.

Researchers Develop “In Vivo” Gene Editing Model for Blood Disorders

Jul 27, 2023

CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

Aug 22, 2023

For decades, doctors and researchers have been working diligently to discover new treatments and provide more options for patients with Duchenne muscular dystrophy (DMD), a debilitating genetic disease that ravages the muscles in the body and leads to a loss of movement and coordination.

CHOP Expert Breaks Down Latest Hemophilia A Gene Therapy Data

Feb 23, 2023

CHOP gene therapy expert Dr. Lindsey George reacts to a phase 3 hemophilia gene therapy study in an editorial in the New England Journal of Medicine.

CHOP Will Be Provider in Independence Blue Cross’s Gene Therapy Network

Jan 26, 2023

CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.

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