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Webinar

In Silico Trial Design in Development of Rare Disease Cell and Gene Therapies

Available on-demand
60 Minutes

There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease. The majority of CGT approvals have been based on small, open-label, non-randomized, single-arm studies which either evaluate treatment effectiveness without a comparator or utilize a historical control comparator to evaluate treatment efficacy. Among the several unique study design challenges common to CGT trials is the inability in many rare diseases to secure suitable historical controls that enrich the single-arm trial by providing an opportunity for evaluating efficacy. In this presentation, we highlight key study design challenges and considerations for rare disease CGT trials and discuss the potential for leveraging in silico approaches for generating synthetic control arms that enable a control-based comparative assessment of efficacy.

Speakers

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Abie Ekangaki, Ph.D.

Dr. Abie Ekangaki is responsible for bringing expertise in biostatistics to shape clinical trial design strategies and methodologies for regulatory submissions and otherwise, and provides broad biostatistical consulting for both internal and external clients. He advises on efficient trial designs for clinical trial programs and applies his statistical expertise, drug development, and operational experience across different therapeutic areas.

With 28 years of experience as a biostatistician, Dr. Ekangaki has worked as a research scientist with the World Health Organization in Switzerland, as a statistics lecturer at Macquarie University in Australia, and has 23 years of experience in the pharma industry in both large pharma and CROs, where he has held several lead technical and senior leadership positions.

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Kenneth Ndugga-Kabuye, MD, FACMG

Dr. Ken Ndugga-Kabuye is Vice President, Cell & Gene Therapy at Premier Research. A clinical geneticist by training, Dr. Ndugga-Kabuye is an expert in the management and clinical development of rare genetic diseases. He has extensive experience executing clinical studies that leverage cutting edge approaches in the treatment of hereditary disease inclusive of Synthetic Biotics, RNA Therapies, Gene Therapies and Enzyme Replacement Therapies.

In previous roles, Dr. Ndugga-Kabuye has supported CRO and biotechnology clients in the hereditary and rare disease space. Dr. Ndugga-Kabuye received a Bachelor of Science in Microbiology, Immunology & Molecular Genetics (MIMG) from the University of California, Los Angeles (UCLA) and a medical degree from Ross University School of Medicine. Dr. Ndugga-Kabuye performed his residency and fellowship training in Medical Genetics & Genomics at the University of Washington in Seattle, WA. He is board certified in Clinical Genetics and Genomics, by the American Board of Medical Genetics and Genomics (ABMGG).

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Duration:
60 Minutes