This makes the point that for each even genotype it’s a whole separate project. It’s a big population of patients, so that, as you know, is going on. ADA-SCID was the lowest hanging fruit because it’s expressed everywhere in the body. Yes, there’s been at least 20 different genes that have been described as causing SCID. The Office of Intellectual Property is talking to potential industry partners who may take this on and try and get it approved.Īre researchers exploring gene therapy for other genetic forms of SCID beside those that are currently in clinical trials? I don’t control the intellectual property which belongs to the University of California Regents. In the technical terms of the university, I’m just the inventor. The longer game is to get this back into the hands of someone who can get FDA approval and make it available on an ongoing basis. I don’t know how much that will be, and that will determine how many patients we can treat right away – right away meaning 3 to 6 months from now. There is one batch of the vector from the same source that we used for the trial that Orchard has that we will get some of. Can you share any information or an estimated timeline for when your compassionate use program might begin? Gene therapy for ADA SCID has been in the news a lot lately. ![]() ![]() The following is a condensed version of the June 2021 Q&A which touched on subjects such as the future of SCID treatment, criteria dictating treatment choices, and the types of SCID being treated in gene therapy clinical trials. In addition, they also fielded questions from members of the SCID Community. Jennifer Puck from the University of California San Francisco discussed topics including SCID definition, clinical features, diagnosis, inheritance, and treatment options. Donald Kohn from the University of California Los Angeles and Dr. During the 2021 IDF National Conference in June, two Severe Combined Immunodeficiency (SCID) experts teamed up to present a detailed update on the landscape of SCID.ĭr.
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