Education Background: BA/MA in Biochemistry & Molecular Biology and Biotechnology

Research Projects: AAV treatment for Familial Dysautonomia in mice

Research Interest: Genetic therapies

Career Ambition: Researching and developing viral therapies for disease

klewis38@meei.harvard.edu

Anil Chekuri, PhD, has been focused on understanding the molecular bases of Inherited Retinal Degenerations (IRDs) and Age-related Macular Degeneration (AMD). He aims to investigate key molecular pathways underlying several forms of retinal dystrophies and development of therapeutic targets to rescue retinal function. His research also focuses on the development of novel Adeno Associated Viral (AAV) vectors with improved retinal tropism and efficacy for therapeutic delivery suitable for translational studies. Dr. Chekuri is currently working to develop gene therapy for various forms of retinal disorders.

achekuri@mgh.harvard.edu

Luk H. Vandenberghe, PhD, is an Associate Professor at Harvard Medical School and Associate Member of the Broad Institute of Harvard and MIT in Boston, MA, USA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, a part of the the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye.

He received a degree in cellular and genetic engineering from the University of Leuven, Belgium. His previous work led to the discovery of novel AAV serotypes such as AAV9, novel insights into AAV structure-function, and vector immunobiology. His laboratory aims to gain a deeper understanding of mechanisms of gene transfer, to develop technologies to overcome hurdles to gene therapy clinical applications, and to translate specific gene therapy programs in vision, hearing, and other fields. Recent studies leverage structural and evolutionary information on AAV as a starting point for the rational design of synthetic viral vector systems, a first generation of which is referred to as AncAAVs which are now progressing to the clinic for a number of indications. Dr. Vandenberghe previously co-founded GenSight Biologics and Akouos. He also is a founder, board member, and advisor to Odylia Therapeutics, a non-profit catalyzing translation for gene therapies within the challenging field of ultra-rare disorders. Dr. Vandenberghe has over 50 peer reviewed publication and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.

luk.vandenberghe@gmail.com