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Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2020 Virtual Annual Meeting
NOVATO, Calif., April 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif., April 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting, which will be held virtually May 12-15, 2020. Information will be available at www.asgct.org.\n Nine Ultragenyx abstracts have been accepted for virtual presentation, including: An oral presentation of available data from the confirmatory cohort of the Phase 1/2 study of DTX401, an AAV-based gene therapy for the treatment of glycogen storage disease Type Ia (GSDIa) (Abstract #1306)An oral presentation of new data from the first three cohorts of the Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency (Abstract #505)Multiple presentations, including two orals, highlighting the HeLa producer cell line platform and improvements in the HEK293 triple transfection system (Abstracts #539 and #543) “We have made meaningful progress across all of our gene therapy programs, including continued improvements to both the HeLa PCL and HEK293 transient transfection manufacturing technology platforms which we believe will enable highly reproducible, more consistent and scalable gene therapy manufacturing,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “In addition to the presentations that support our gene therapy manufacturing approach, we look forward to sharing first-time results from the confirmatory cohort of our GSDIa study and updated clinical data from the OTC study.” Details of the nine presentations are as follows: Tuesday, May 12Oral PresentationAbstract #98: AAV9/hCDKL5 delivery to cerebrospinal fluid of juvenile CDKL5-deficient mice improves learning and memory and motor function in adult mice Time: 5:15-5:30 PM ET Poster PresentationAbstract #172: Characterization and correction of an in vitro model of Wilson Disease by recombinant adeno-associated virus (rAAV) delivered ATP7B transgene Time: 5:30-6:30 PM ET Poster PresentationAbstract #451: Rapid CMC development and...