Two CHOP Patients with an Inherited Blindness Successfully Treated with Gene Editing
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The findings from the clinical trial were published in the New England Journal of Medicine.
Cell and Gene Therapy Innovations News and Updates
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CHOP Researchers Improve Fitness of Cells Used in Cell Transplants
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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.
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
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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.
NIH Awards Penn Medicine and Children’s Hospital of Philadelphia $26 Million Grant to Develop Therapies for Rare Newborn Genetic Diseases
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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.
Researchers Develop “In Vivo” Gene Editing Model for Blood Disorders
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CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.
CHOP Treats 500th CAR T Patient
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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.
CHOP Expert Breaks Down Latest Hemophilia A Gene Therapy Data
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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
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CHOP will be a provider in Independence Blue Cross’s new Advanced Network for Gene-Based Therapeutics, effective March 1, 2023.
CHOP Researchers Join Global Team to Take on Solid Tumors in Children
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Four CHOP researchers are part of a world-class research team that will receive $25 million to study solid tumors in children, funded by Cancer Grand Challenges.
First Approved CAR T-Cell Therapy Shows Continued Benefits Five Years Later
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New data on show CAR T-cell therapy leads to durable remission and long-term survival in children and young adults with relapsed or refractory B-ALL.