On May 8, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultrarare 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) 26th Annual Meeting, which will be held May 16-20, 2023 at the Los Angeles Convention Center in Los Angeles, California (Press release, Ultragenyx Pharmaceutical, MAY 8, 2023, View Source [SID1234631182]).

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The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.

Clinical and pre-clinical presentations:

Oral presentation: Efficacy and safety at week 52 and up to four years in adults with glycogen storage disease type Ia (GSDIa): Results from a Phase 1/2 clinical trial and long-term follow-up study of DTX401, an AAV-8-mediated, liver-directed gene therapy (Abstract #4)
Date/Time: Thursday, May 18, 8:00 – 8:15 AM PDT
Location: Concourse Hall 152 and 153
First author: Rebecca Riba-Wolman, M.D., University of Connecticut
Oral presentation: Long-term Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #128)
Date/Time: Thursday, May 18, 2:45 PM – 3:00 PM PDT
Location: Room 403 AB
Presenter: Cary Harding, M.D., Oregon Health & Science University
Poster presentation: Immunosuppression to Inhibit Capsid-Specific Humoral Immune Responses in High-dose AAV Gene Therapy in Cynomolgus Macaques (Board No. 1191)
Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Deniz Erturk-Hasdemir, Ph.D., Ultragenyx
Additional presentations including the company’s manufacturing capabilities and Pinnacle PCL (AAV vector producer cell line) platform and technology:

Oral presentation: In Vivo and In Vitro Assessment of Residual DNA Impurity-Derived Transcriptions (Abstract #109)
Date/Time: Thursday, May 18, 1:30 – 1:45 PM PDT
Location: Concourse Hall 150 and 151
Presenter: Hsin-I Jen, Ph.D., Ultragenyx
Poster presentation: Early Development of In Vitro Potency Assays for Rare and Ultrarare Disease Gene Therapy Products (Board No. 782)
Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Elena Balkanska-Sinclair, Ultragenyx

Poster presentation: Global Strategy to Improve the Downstream Manufacturing Process of the Adeno-associated Viral Vector for Glycogen Storage Disease Type Ia Treatment (Board No. 412)

Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Chao Huang, Ph.D., Ultragenyx

Poster presentation: Genome-wide analysis of triple-play plasmid integration in Pinnacle PCL producer cell lines across multiple rAAV programs (Board No. 1162)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Nicholas Richards, M.S., Ultragenyx

Poster presentation: Identification of novel mutations in PCCA and PCCB genes from Propionic Acidemia patient fibroblasts via long-read sequencing (Board No. 972)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Rea Guertler, M.Sc., Ultragenyx

Poster presentation: Development of innovative, scalable, high productivity manufacturing process to enable Ph1/2 clinical supply of UX810, an investigational AAV gene therapy to treat Duchenne’s Muscular Dystrophy (Board No. 1424)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Wei Xue, Ph.D., Ultragenyx

Poster presentation: Development of UX055 AAV Gene Therapy for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD), a Rare Neurological Disorder (Board No. 1340)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Julie Wei, Ph.D., Ultragenyx

Poster presentation: Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy (Board No. 1578)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Kristin Voorhees, Ultragenyx
In addition to the data presentations, Ultragenyx will present at one pre-meeting workshop on Tuesday, May 16:

Pre-meeting workshop: How to Become a Site for AAV Gene Therapy Trials
Time: 8:25-8:50 AM PDT
Location: Concourse Hall 152 and 153
Presenter: Sandra Nino-Siddens, Pharm.D., Ultragenyx