Advances in viral vector technology: Anc-AAV & evolutionary guided vector design

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Presenter: Dr Luk H. Vandenberghe

Assistant Professor, Harvard Medical School & Director of the Grousbeck Gene Therapy Center, Schepens Eye and Ear Institute, Massachusetts Eye and Ear Infirmary, Boston MA, USA
Dr Luk Vandenberghe, Mass Eye and Ear

Luk H. Vandenberghe, PhD is Assistant Professor at Harvard Medical School and Director of the Grousbeck Gene Therapy Center at the Massahusetts Eye and Ear Infirmary in Boston, Massachusetts in the United States. Dr. Vandenberghe was trained as a biochemical engineer at the University of Leuven in Belgium and pursued a PhD in Biomedical Sciences with a focus on virology and viral gene therapy. His laboratory is interested in questions on the host-vector biology of human gene transfer, the development of enabling technologies to broaden gene therapy applications, and translation of preclinical programs for indications with unmet need. Dr. Vandenberghe is a co-author on over 50 peer reviewed publication including in Nature Medicine, Science and other high impact journals. He holds over a dozen patents related to gene therapy technology which are licensed to multiple biopharmaceutical entities including Biogen, Pfizer, Lonza, Avexis and Voyager Therapeutics.

Question and Answer Session moderator: Dr Dirk Grimm

Group Leader, BioQuant, Heidelberg University Hospital, Germany
Dr Dirk Grimm Heidelberg University Hospital
Dirk Grimm, PhD is Full Professor for Viral Vector Technologies at Heidelberg University Hospital. He was trained as a biologist at the Universities of Kaiserslautern and Heidelberg (Germany) and pursued a PhD in Biology with a focus on Adeno-associated viral (AAV) vector technology. His laboratory is interested in engineering novel capsid and genome variants of AAV that are specifically optimized for tissue-specific expression of elements for therapeutic gene regulation and genome editing. Dr. Grimm is a co-author on over 70 peer-reviewed publications including in Nature, Nature Structural and Molecular Biology, Journal of Clinical Investigation, Blood and other high-impact journals.


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Gene therapy is a potent modality with demonstrated ability to intervene in diseases of unmet medical need. Its safety and efficacy is often governed by the vector system used. Adeno-associated viral vectors (AAV) have shown great promise for sustained expression of a therapeutic gene in vivo. Obstacles remain however for gene therapy to broaden its impact beyond niche indications; currently clinically considered AAVs are repurposed naturally occurring viral entities that bring along limitations imposed by its viral biology. For example, AAVs is endemic in humans which results in a large proportion of patients to harbor memory responses to AAV antigens, preventing them to benefit from a future AAV gene therapy. Other constraints include AAV’s limited genetic cargo capacity, poor targeting of tissue targets (e.g. kidney, cochlea), and a desire to increase transduction specificity of an inherently promiscuous virus.

This webinar provides insight into:

  • The novel discovery efforts aimed at building synthetic AAV-based gene transfer vehicles that diverge sufficiently from their natural peers
  • The challenges in retaining the superior features that have justified AAV's current clinical use
  • Their work in deconstructing the putative viral evolutionary lineage of most of the commonly used AAVs using Ancestral Sequence Reconstruction
  • Subsequent efforts that have led on the one hand to a set of uniquely distinct AAVs with potent transduction of therapeutically relevant tissues (including liver, retina, muscle, and cochlea). On the other hand, they actively exploit these novel reagents to deepen our structure-function understanding toward the development of improved clinical gene transfer reagents