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Haematopoietic Stem Cell Gene Therapy For Severe Monogenic Disorders and Potential Beyond Rare Disease

The talk will demonstrate how gene-corrected HSCs can address a variety of disease states across multiple organ systems, and present emerging clinical data in severe immune deficiency disorders, MLD and other neurometabolic diseases which demonstrate proof of concept for the HSC gene therapy approach. The talk will also outline some of the exciting new research programs the Discovery Team are conducting within the CSCRM, aimed at harnessing the potential for HSC gene therapy to address unmet medical needs for more prevalent larger disease indications.

Speaker: Dr Pervinder Sagoo 

Piv Sagoo is Director of Discovery Research at Orchard Therapeutics, a commercial Gene & Cell therapy biotech company that develops haematopoietic stem cell-based gene therapies to treat patients with rare genetic disorders. She leads a team focused on basic and preclinical research into the application of stem cell gene therapy for new disease indications, and innovations in process development and technologies for Orchards early stage clinical drug products. Piv has an academic research background spanning basic, preclinical and clinical immunology, with expertise in leading projects with a translational context in the fields of transplantation, cell therapies, infection and immunity.


14:00-15:00 – Seminar

15:00-17:00 – Questions and chat with audience and PIs

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