2022 - Gene Therapies for Rare Diseases: Advancing Research with Humanized Models and AI

The discovery and development of orphan drugs and gene therapies for rare diseases face many challenges, including: insufficient knowledge regarding the underlying disease mechanisms, the lack of clinically relevant humanized animal disease models, insufficient AAV vector productivity and targeted delivery in gene therapy development and more.
In light of the challenges faced with orphan drug development, there has been growing interest in leveraging bioinformatics and artificial intelligence (AI) tools for accelerating drug discovery. It has become increasingly essential for researchers, biotechs and pharmaceutical companies developing rare disease therapies to implement AI tools that get results both quickly and cost-effectively — vastly reducing time to clinical trials.
The Rare Disease Data Center (RDDC) provides interfaces for the interactive visualization and exploration of biomedical data alongside several gene diagnosis tools based on the sophisticated AI and machine learning models. The RDDC integrates all reported rare disease data and information, including: epidemiological data, disease-related gene profile, variants, phenotypes, drug developmental outlines and disease-associated mouse models.
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Register for the webinar now and join Dr. Kugeng (KG) Huo, for a discussion on how to use the Rare Diseases Data Center (RDDC) resources and Cyagen’s one-stop CRO platform to accelerate the R&D of effective gene therapies for rare diseases.