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Speaker Interview With David Schaffer

David Schaffer
Professor of Chemical & Biomolecular Engineering, UC Berkeley
Co-Founder& Acting Chief Scientific Officer, 4D Molecular Therapeutics 
Gene Therapy for Neurological Disorders Speaker Faculty


What do you think is exciting about the gene therapy for neurological disorders space and why?

There is serious unmet medical need in the CNS, from idiopathic diseases including Parkinson’s and Alzheimer’s, to monogenic disorders including lysosomal storage disease, Huntington’s, familial ALS, Rett, spinal muscular atrophy, and others. In addition, the retina is considered part of the CNS, and over 200 monogenic diseases as well as complex disorders such as macular degeneration rob people of their vision. Gene therapy has major promise to treat these monogenic disorders, and with additional advances in understanding disease biology, potentially also the more complex diseases.

Speaker Interview With Lamya Shihabuddin

Lamya Shihabuddin
Head of Genetic Neurologic Diseases
Gene Therapy for Neurological Disorders Speaker Faculty


Could you start by telling us a bit about how you came to be working on gene therapies targeting the CNS?

Initially, I joined Genzyme to develop cell therapies for lysosomal storage diseases with CNS involvement. Previously, I used AAV vectors, as well as lentiviral vectors, to transduce cells with marker genes both in vitro and in vivo to track their development and differentiation. At Genzyme, in thinking of developing a transformative holistic approach for treating lysosomal storage diseases with central nervous system involvement (CNS), targeting therapies to the CNS across the blood brain barrier represented a big challenge. Developing a gene therapy strategy that will circumvent the blood brain barrier (BBB), and deliver a therapeutic gene that best suits the disease directly to the area of...