On April 28, 2020 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) 23RD Annual Meeting, which will be held virtually May 12-15, 2020 (Press release, Ultragenyx Pharmaceutical, APR 28, 2020, View Source [SID1234556694]). Information will be available at www.asgct.org.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 12
Oral Presentation
Abstract #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 Presentation
Abstract #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 Presentation
Abstract #451: Rapid CMC development and pre-commercial considerations for rAAV gene therapy products for rare diseases

Time: 5:30-6:30 PM ET
Wednesday, May 13
Oral Presentation
Abstract #539: HeLa 3.0: CRISPR knockout of genes modulating titer in established rAAV-producing cell lines

Time: 4:00-4:15 PM ET
Oral Presentation
Abstract #505: AAV8 gene therapy as a potential treatment in adults with late-onset OTC deficiency: results from a Phase 1/2 clinical trial

Time: 4:15-4:30 PM ET
Oral Presentation
Abstract #543: Characterization of phenotypic and genotypic stability of rAAV producing HeLa cell lines

Time: 4:45-5:00 PM ET
Poster Presentation
Abstract #586: Gene therapy for Wilson Disease using rAAV to restore ATP7B gene function

Time: 5:30-6:30 PM ET
Thursday, May 14
Poster Presentation
Abstract #1008: Elongation of the Rep-Cap cassette with a cellular intron reduces reverse-packaged Rep-Cap trans plasmid sequences and increases therapeutic vector genome packaging in a HEK293 triple transfection rAAV vector production system

Time: 5:30-6:30 PM ET
Friday, May 15
Oral Presentation
Abstract #1306: AAV8-mediated liver-directed gene therapy as a potential therapeutic option in adults with glycogen storage disease type Ia (GSDIa): results from a Phase 1/2 clinical trial

Time: 10:30-10:45 AM ET