A Revolutionary Approach to Gene Therapy

Raj is a medical technology pioneer and entrepreneur. In addition to degrees in pharmaceutical science from the University of British Columbia, he has extensive experience in the pharmaceutical industry, medicinal chemistry, and pharmacology, with a particular focus on nanobiotechnology and survey research as well as data analysis on molecular biomarkers and related compounds. Nano-biosensors for rapid detection of molecular targets at the point of care have been a major part of his industry research. During his work on sensor development, he developed the world's first point of care molecular biomarker test for Alzheimer's disease. As CSO, he laid the groundwork for over ten patents in nano-sensor technology. Furthermore, Raj has extensive experience in global strategic management, specialty biologicals, and market access. Aside from that, he has managed risk management programs for multiple myeloma and inflammatory diseases for some of the world's largest pharmaceutical companies. The key strength of Raj is his ability to inspire high-performing teams that change the world.

Anna is an expert in global health, advocacy, and business development with more than 20 years of experience in the development of drugs and medical devices, development of partnerships, and expansion of footprints in developing markets. In the last five years, Anna helped take two companies public. She was VP for Public Affairs at Medicines for Malaria and a senior advisor to the Bill & Melinda Gates Foundation. She also advises family offices, philanthropic organizations and impact investment funds.

Guangping Gao, PhD is the Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor in Biomedical Research, UMass Chan Medical School. Dr. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, hugely impacting many currently untreatable human diseases. Dr. Gao has published 250+ research papers and serves as Editor of Human Gene Therapy.

Dr. Kazak is an Assistant Professor in the Department of biochemistry and Goodman Cancer Research Centre at McGill University. His lab explores molecules and metabolic pathways that control mitochondrial energetics in adipocytes and cancer cells.

Short Biography
Claire Henchcliffe, M.D., D.Phil. is the Chair and Stanley van den Noort Professor of Neurology, at the University of California, Irvine. She read for her BA in Biochemistry and completed her graduate thesis at the University of Oxford in the UK. She then pursued post-doctoral training in genetics and neuroscience at the University of Cambridge, UK, and the University of California at Berkeley, California. Dr. Henchcliffe received her medical degree from the College of Physicians and Surgeons of Columbia University in New York City. She completed neurology residency and fellowship at the College of Physicians and Surgeons of Columbia University in New York City, prior to joining Weill Cornell Medical College, New York City in 2003. At Weill Cornell Medical College she developed a clinical and research program in movement disorders with a focus on clinical trials including stem cell-based therapeutics, eventually serving as Professor of Neurology and Neuroscience, and Vice Chair for Clinical Research. She served as the Weill Cornell site Principal Investigator for the NIH-funded NeuroNEXT clinical trial network and as Medical Director of the Trial Innovation Unit, for the Weill Cornell Clinical and Translational Science Center to the national NCATS-supported Trial Innovation Network. She left in 2020 to take up her position at UCI. Her current research focuses on developing new treatments for Parkinson’s disease, and she has a specific interest in the potential for data-driven personalized approaches to care, including gene- and cell-based therapies. She has been an investigator and on the steering committees for multiple clinical trials in Parkinson’s disease.

Christine is the Medical Director of Clinical Research and Clinical Development, Gene Therapy Program, Boston Children's Hospital and has been a Pediatric Hematology/Oncology Fellow at the Dana Fabor Cancer Institute for over 20 years. She is also the Director of Clinical Research and Development at, Dana-Farber/Boston Children's Cancer and Blood Disorders Center. Her research and clinical focus is on cellular therapies for rare pediatric diseases- especially neuro-metabolic disease and brain tumors. Widely published, Christine was also the clinical leader in partnership with a biotech company and MGH in the research that lead to the first FDA approved therapy for children with cerebral adrenoleukodystrophy (ALD) in the world. She is also a leader in multiple international pediatric stem cell transplant groups.

Troy Lund, MD, PhD, is a pediatric blood and marrow transplant physician who cares for children with adrenoleukodystrophy, leukemia and lymphoma. He sees patients at the University of Minnesota Masonic Children’s Hospital Pediatric Specialty Care Journey Clinic. Dr. Lund's research focuses on improving the outcomes for all patients undergoing blood and marrow transplantation by increasing the speed at which hematopoietic stem cells reconstitute the immune system after transplant. He is also an associate professor in the Department of Pediatrics and a faculty member in the Division of Blood and Marrow Transplantation at the University of Minnesota Medical School.

Doctor Sanjay Gandhi is a board certified interventional cardiologist with more than 15 years experience in interventional cardiology, clinical trials, service line operations, and hospital strategy in academic institutions. A member of the faculty of Case Western Reserve University in Cleveland, he also serves as the president of the Ohio Chapter of American College of Cardiology. He holds a Master of Business Administration degree with high honors from the University of Michigan. He is passionate about helping healthcare technology startups achieve full potential and clinical validation.