Cell & Gene Therapy News
Children’s Hospital of Philadelphia Researchers Announce Promising Results from First-of-its-Kind, Multicenter, Phase 1 Gene Therapy Trial for Danon Disease
Researchers at Children’s Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a single gene mutation. The data on the results of the RP-A501 Phase 1 trial, presented at a late breaking session today at the American Heart Association Scientific Sessions 2024 in Chicago, were also published in the New England Journal of Medicine.
Children’s Hospital of Philadelphia Launches Global Partnership to Establish a CAR-T Therapy Program in Brazil
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.
Researchers at Children’s Hospital of Philadelphia Reveal New Insights into Non-Inflammatory Causes of Rare Neurological Symptoms in Children, Adolescents and Young Adults Following CAR-T Therapy
Researchers at Children’s Hospital of Philadelphia (CHOP) revealed for the first time that children, adolescents and young adults may experience very rare neurological issues of paraparesis and quadriparesis following chimeric antigen receptor T-cell (CAR-T) therapy, a type of immunotherapy used to treat B-cell Acute Lymphoblastic Leukemia (B-ALL). Additionally, these complications occurred without the type of inflammation typically seen in adults. The findings were published today in the journal Blood.
Children’s Hospital of Philadelphia Researchers Report Encouraging First Evidence of Effective New Gene Therapy to Treat Multiple Sulfatase Deficiency
The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models
CHOP Researchers Develop Innovative Model Offering New Insights to Improve and Overcome Current Obstacles in Hemophilia A Gene Therapy
Researchers at CHOP announced the results of a new study offering insights into the development of next-generation gene therapies to treat hemophilia A.
Researchers at Children’s Hospital of Philadelphia Find Lentiviral Gene Transfer Improves Human Alpha Globin Production for the Treatment of Alpha Thalassemia
Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a new model that offers a potential platform for developing novel therapies to treat Alpha Thalassemia (AT), a severe blood disorder. The findings were published in the journal Blood.
CHOP Researchers Develop Safer, Alternative Conditioning Regimen for the Treatment of Beta-Thalassemia
Innovative model highlights bone marrow transplantation without toxic side effects.
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.