On May 9, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, reported that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting, which will be held both in person and virtually May 16-19, 2022 (Press release, Ultragenyx Pharmaceutical, MAY 9, 2022, View Source [SID1234613949]). The company will present new data supporting its gene therapy portfolio and discuss critical topics during a scientific symposium on Accelerated Approval and at the pre-meeting workshops.

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Clinical and pre-clinical presentations include:

Oral presentation: Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #463)
Date/Time: Tuesday, May 17, 4:15-4:30 PM ET
Presenter: Cary Harding, M.D., Oregon Health & Science University

Oral presentation: Sustained Efficacy and Safety at Week 52 and up to Three Years in Adults with Glycogen Storage Disease Type Ia (GSDIa): Results from a Phase 1/2 Clinical Trial of DTX401, an AAV8-mediated, Liver-directed Gene Therapy (Abstract #1212)
Date/Time: Thursday, May 19, 11:00-11:15 AM ET
Presenter: Rebecca Riba-Wolman, M.D., University of Connecticut

Poster presentation: Improving Neuronal Gene Transfer to the Brain After CSF Administration of AAV9 in Juvenile Non-Human Primates (Board No. Tu-143)
Date/Time: Tuesday, May 17, 5:30-6:30 PM ET
Presenter: Maggie Wright, Ph.D, Ultragenyx

Poster presentation: Understanding the Educational Needs of United States Physicians Related to Gene Therapy (Board No. Tu-67)
Date/Time: Tuesday, May 17, 5:30-6:30 PM ET
Presenter: Emily Belcher, CE Outcomes
Manufacturing presentations covering the company’s Pinnacle PCL (AAV vector Producer Cell Line) platform and technology include:

Oral presentation: Development and Characterization of Highly Optimized Monoclonal Producer Cell Lines (PCLs) for the Treatment of CDKL5 Deficiency Disorder (CDD) (Abstract #863)
Date/Time: Wednesday, May 18, 4:15-4:30 PM ET
Presenter: Laurie Tran, MSc, Ultragenyx

Poster presentation: Development of High Density & High Purity AAV Production Processes (Board No. M-293)
Date/Time: Monday, May 16, 5:30-6:30 PM ET
Presenter: Jan Panteli, Ph.D, Ultragenyx

Poster presentation: Development of an In Vitro Model for the Evaluation of Adeno-Associated Virus Delivered Microdystrophin Transgenes (Board No. W-56)
Date/Time: Wednesday, May 18, 5:30-6:30 PM ET
Presenters: Eric Himelman, Ph.D, and Hwan June Kang, Ph.D, both Ultragenyx
In addition to the data presentations, Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx will deliver a presentation at ASGCT (Free ASGCT Whitepaper)’s session on "Accelerated Approval for Gene Therapies," taking place on Monday, May 16 from 8:00-9:45 AM ET.

Separately, several members of the team will present during the following pre-meeting workshops on Sunday, May 15:

Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances
Time: 8:55-9:45 AM ET
Presenter: Erin Frey, Director, State Government Affairs, Ultragenyx
AAV Vector Integration
Time: 2:50-4:50 PM ET
Presenter: Sam Wadsworth, Ph.D., Chief Scientific Officer of Ultragenyx Gene Therapy
What to Expect as a Participant in a Gene Therapy Clinical Trial
Time: 1:00-3:00 PM ET
Presenter: Heather Lau, M.D., Executive Director, Global Clinical Development, Ultragenyx