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All 23 treated patients are alive at 2.6-year median follow-up without evidence of malignant transformation Data representing largest cohort of infants with XSCID treated with gene therapy presented...
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AviadoBio to Present Pre-clinical Data on its Gene Therapy Candidate for Frontotemporal Dementia at ASGCT 2022 Intrathalamic delivery of AVB-101 shows promising efficacy in a pre-clinical disease...
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Data represent largest cohort of infants with XSCID, also known as bubble boy disease, who received lentiviral gene therapy with the longest follow-up to date Seventeen of 18 patients with follow-up...
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LEXINGTON, Mass. and AMSTERDAM, May 02, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical...