Cell & Gene Therapy News
New Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment
New model and vector may hold the keys to transforming the lives of XLSA patients.
Two CHOP Patients with an Inherited Blindness Successfully Treated with Gene Editing
The findings from the clinical trial were published in the New England Journal of Medicine.
Researchers Publish Final Results of Key Clinical Trial for Gene Therapy for Sickle Cell Disease
In a landmark study, an international consortium led by researchers at CHOP published the final results of a key clinical trial of the gene therapy CASGEVY for the treatment of sickle cell disease.
CHOP, Stanford Researchers Identify Protein That Controls CAR T Cell Longevity
FOXO1 is required for memory in T cells and is associated with more durable clinical responses to CAR T cell therapy.
Children’s Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss
Children’s Hospital of Philadelphia is proud to announce the initial results of an experimental gene therapy treatment of a patient with hereditary hearing loss in the United States. Findings to date indicate that the treatment was successful.
FDA Approves Two Gene Therapies for Sickle Cell Disease
The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
Gene Splicing Reduces Effectiveness of CD20-Targeting Monoclonal Antibodies Designed to Treat Variety of Blood Cancers and Disorders
Researchers found CAR T-cell therapy may serve as effective alternative for patients with these disorders.
CHOP, Penn researchers develop gene editing approaches for PKU treatment
The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease
CHOP Researchers Improve Fitness of Cells Used in Cell Transplants
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
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.