ULTRAGENYX PHARMACEUTICAL INC.
NOVATO -
GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).
Abstracts are available to view online at www.aan.com/events/annual-meeting-abstracts. The GTX-102 emerging science presentation will include data beyond what is currently available online.
AAN 2024 Presentation Details
Title: Clinical Activity and Safety of GTX-102, an Investigational Antisense Oligonucleotide for the Treatment of Patients With Angelman Syndrome
Format: Oral (abstract #192, presentation #008)
Session: Emerging Science 2
Presenter:
Date/ Time:
Title: Proteomic Profiling of Angelman Syndrome for Disease-Associated Biomarker Discovery
Format: Poster (#8.001)
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter:
Date/ Time:
Title: Clinical Characteristics of Creatine Transporter Deficiency (CTD): Final Results of the Vigilan Observational Study
Format: Poster (#8.002)
Session: Child Neurology and Developmental Neurology: Neurogenetics 2
Presenter:
Date/ Time:
About
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, business plans and objectives for GTX-102, expectations regarding the tolerability and safety of GTX-102, and future clinical and regulatory developments for GTX-102 are forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company to successfully develop GTX-102, the company's ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company's behalf, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
In addition to its
Contact:
Tel: +1-415-475-6370
Email: ir@ultragenyx.com
Tel: +1-415-225-5050
Email: media@ultragenyx.com
(C) 2024 Electronic News Publishing, source
