Luk Vandenberghe, a gene therapy researcher at Massachusetts Eye and Ear and at Harvard Medical School, is spearheading an effort to develop a COVID-19 vaccine that uses an adeno-associated viral (AAV) vector to deliver genetic instructions for the SARS-CoV-2 spike protein into human cells. AAV vectors are used in the two commercial gene therapies in…

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1st Patient Dosed in Trial of Gene Therapy, OTL-201, for Sanfilippo Type A Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene therapy OTL-201 for Sanfilippo syndrome type A. OTL-201’s safety, tolerability, and efficacy is being investigated in the open-label, Phase 1/2 study (NCT04201405) taking place at Manchester University NHS Foundation Trust in the…

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Nationwide Children’s Hospital chooses Mayo Pujols, most recently of Novartis Pharmaceuticals, for its new biotech subsidiary Andelyn. As the new CEO for Andelyn Biosciences, a Nationwide Children’s Hospital-owned gene-therapy startup, Mayo Pujols will begin to assemble a diverse team of up to 200 scientists, biochemists, engineers and technicians. The portfolio of gene-therapy products to be…

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