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Updates on gene therapy for ‘bubble boy’ disease and cellular immunotherapy at ASGCT

Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.



Interim results of an ongoing clinical trial found lentiviral gene therapy is safe and effective for treatment of infants with X-linked severe combined immunodeficiency (X-SCID). The report will be presented during the 25th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). The conference will take place in person and virtually May 16 – 19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.

“All of our patients are doing very well,” said Ewelina Mamcarz, M.D., of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy. “At a median follow up of over two and a half years, the therapy appears to be safe and effective. The patients develop functional immune systems with no evidence of abnormal cell division.”

X-SCID, also known as bubble boy disease, is a rare, life-threatening disorder caused by a mutation in a single gene that prevents proper immune function. In 2019, St. Jude published an initial report in the New England Journal of Medicine documenting the success of the gene therapy developed and produced at St. Jude. Thursday, May 19, at 8:45 a.m. ET, Mamcarz will present interim results for the first 23 patients treated in the multi-center clinic trial.

The ASGCT annual meeting is a venue for professionals in gene and cell therapy to display their work with colleagues. The meeting serves a wide community, including academic researchers, clinicians, bio-industry developers, regulatory agencies, equipment manufacturers, patient advocates and others. The meeting will also feature other St. Jude scientists discussing research related to immunotherapies using chimeric antigen receptor (CAR) T cells from the Krenciute, Velasquez, Zhang and Gottschalk laboratories and immune response to vectors from the Davidoff laboratory.

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Posted on: 15 May 2022, source: ASGCT
St. Jude Children’s Research Hospital scientists will discuss research on cellular therapies for pediatric cancer and more at the annual gathering of the professional society for gene and cellular therapy researchers.
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