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  4. Webinar: Gene Therapy and Rare Disease

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, bioinformatics and artificial intelligence (AI) tools have become essential for researchers, biotechs, and pharmaceutical companies developing rare disease therapies to accelerate drug discovery and vastly reduce 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 is consistently developing new tools to meet the rapidly evolving demands of rare disease scientific research. For example, the RDDC RNA Splicing Tool is based on deep neural network (DNN) strategy and bioinformatics that predict splicing by analyzing the splicing abnormalities that have a greater effect on protein coding translation, and eventually finds out the causes of genetic diseases at the base mutation level. By combining conventional bioinformatics analysis and AI models, we can screen the loci of interest before heading onto validation experiments.

For AAV-mediated gene therapy approaches, Cyagen has produced substantial experimental data for AI model training and developed proprietary machine learning algorithms to accelerate the AAV capsid identification and optimization processes compared to traditional directed evolution methods. Utilizing AI and single-cell RNA-sequencing technologies, Cyagen’s high-throughput AAV vector discovery platform helps overcome the present limitations of gene therapy R&D by quickly identifying next-generation AAV capsids that have enhanced tissue targeting capability, tissue specificity, and productivity.

Even with the available data resources and AI tools, researchers often face difficulties in acquiring clinically relevant models of a rare disease. The Humanized Model Project has developed genetic humanization models of rare disease for preclinical research, with a focus on ophthalmology and neurology applications. These clinically relevant humanized models are readily available to researchers worldwide via Cyagen’s repository, which can also perform downstream services as a part of their comprehensive gene therapy research solution platform.

Join our expert, Dr. Kugeng (KG) Huo, for a discussion on how you can use the Rare Disease Data Center (RDDC) resources and Cyagen’s one-stop CRO platform to accelerate your research and development of effective gene therapies for rare diseases.

Key Topics

Learn how you can accelerate research and development of effective gene therapies for rare diseases with a range of innovative AI-powered resources:

  • Introduction to the Rare Disease Data Center (RDDC)
  • Examples - How the RDDC’s analytic tools can help provide new insights on rare diseases
  • How Cyagen’s humanized model project provides clinically relevant genetic humanization models of rare disease for preclinical research, including ophthalmology and neurology applications, alongside downstream services
  • AI-powered AAV design & virus packaging platform improves delivery and transduction performance for significantly accelerated gene therapy R&D
Who Should Attend?

This session is packed with relevant insights for clinical research professionals and pharmaceutical sponsors in the rare disease space. The webinar will be of interest to members of the following fields:

  • Clinical Research Hospitals
  • Biotechnology
  • Pharmaceutical & Biopharma
  • Preclinical and Academic Research
  • Rare Disease Research Organizations & Non-Profits

Relevant areas:

  • Clinical operations
  • Innovation
  • Medical affairs
  • Scientific Affairs
  • Project Management
  • Research and Development

Titles may include:

  • Drug Discovery Professionals
  • VP and Head of Data Science
  • VP/Director of Biology
  • VP/Director of Research
  • Scientists
  • Biologists
  • Bioinformaticians
  • Medicinal Chemists
  • Pharmacologists
  • Data Scientists
  • Drug Discovery Professionals
  • Research & Development Professionals
  • Artificial Intelligence/Machine Learning Professionals
  • Therapeutic Lead/Head
  • CIO
  • CTO
Kugeng (KG) Huo, PhD
Senior Scientist
Cyagen Biosciences
Kugeng (KG) Huo is a Senior Scientist at Cyagen Biosciences. He holds a BSc in Biology from Concordia University and a PhD in Experimental Medicine from McGill University, followed by a post-doctoral training at the UHN of University of Toronto, and has almost 10 years of research experience in vascular disease, oncology and bioinformatics.
About Cyagen

Cyagen Biosciences is a 900-employee company headquartered in Santa Clara, California, with additional locations in Germany, Japan, and China. Cyagen offers a “one-stop shop” tailored to biomedical scientists’ gene research needs. Cyagen is the world's largest provider of custom-engineered mouse and rat models. Our custom murine model generation services range from DNA vector construction to embryonic stem cell manipulation, microinjection, breeding, and more. All projects are fully customizable and flexible. Cyagen provides comprehensive Contract Research Organization (CRO) services for cell and gene therapy (CGT) research, including viral vector development, CAR design and construction, disease models, phenotype analysis, and pharmacodynamic evaluations.

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About the Rare Disease Data Center

The Rare Disease Data Center (RDDC) is committed to providing unlimited access to comprehensive rare disease research data visualization & AI-powered pathogenic analysis tools. It 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. The RDDC provides a rapid analysis and visual understanding for a rare disease of interest, offering the maximum convenience for people who are dedicated to the treatments of rare diseases, such as clinical doctors, scientific researchers from hospitals and universities, and pharmaceutical companies.