As Global Head of Johnson & Johnson Innovation, JLABS (JLABS), Melinda Richter fosters the Johnson & Johnson Family of Companies external R&D engine and supports the innovation community by creating capital-efficient commercialization models that give early stage companies a big company advantage.
By providing infrastructure, services, educational programs and networks in global hotspots, JLABS is the best place to start a company working in healthcare, with a specific emphasis on Johnson & Johnson’s sectors: consumer, medical device and pharmaceuticals.
What if we could simply switch off a mutated gene causing a debilitating neurodegenerative disease or sensory disorder? Recent milestones in gene therapy have ignited interest in the space and especially its application to neurological disorders. As more monogenic targets for diseases affecting the CNS are identified, their ability to be drugged with viral gene therapy is opening doors for innovations in treatments for diseases like Huntington’s, spinal muscular atrophy and ALS. As the science progresses, how are challenges like optimizing delivery to the brain, limitations with current testing models and more affecting the advancement of solutions?
To discuss these topics, we’re bringing together the region’s pioneering researchers and entrepreneurs that are developing gene therapy innovations addressing neurological diseases. Join us for a half day symposium for presentations on cutting-edge science and a discussion on the future of gene therapy solutions and the potential hurdles innovators currently face.
Professor, Department of Pharmaceutical Sciences, University of California, Irvine
Co-founder, Responsive Polymers Therapeutics, Inc.
Co-founder, Jupiter Therapeutics, Inc.
Dr. Young Jik Kwon is a professor at UC Irvine in the Pharmaceutical Sciences, Chemical Engineering & Materials Science, Biomedical Engineering, and Molecular Biology & Biochemistry departments. Following his undergraduate education in Biological Engineering at Inha University, Dr. Kwon received his Ph.D. in Chemical Engineering from the University of Southern California with a focus on retroviral gene delivery in 2003 and did post-doctoral training in department of chemistry at UC Berkeley on polymeric vaccine carriers. He started his academic career in Biomedical Engineering at Case Western Reserve University in 2005 and moved to UC Irvine in 2007. He currently oversees research at the BioTherapeutics Engineering Laboratory (BioTEL) at UC Irvine and his current projects mainly focus on gene therapy, drug delivery, cancer-targeted therapeutics, and cancer vaccines.
Dr. Kwon is a member of the NCI-designated Chao Family Comprehensive Cancer Center, the Cancer Research Institute, and the Center for Virus Research at UC Irvine. Dr. Kwon’s work was awarded the Medical Research Award from Gabrielle's Angel Foundation for Cancer Research in 2011, the Faculty Early Career Development Award (CAREER) from the National Science Foundation in 2010, and the Faculty Career Development Award from UC Irvine in 2008. He has received the best reviewer in the subject area of Pharmaceutical Sciences, Elsevier (2011), FEBS Journal Top-Cited Paper Award, Wiley (2013), and Top downloaded article award, Journal of Materials Chemistry, Royal Society of Chemistry Publishing (2014). He is also an invited scholar to Federal University of Minas Gerais (UFMG) in Belo Horizonte, Brazil (2013-2015) and a Brain Pool Scholar in Korea University in Seoul, Korea (2015-2016). In 2017, he co-founded startup companies, Responsive Polymers Therapeutics, Inc. and Jupiter Therapeutics, Inc, based on his research discoveries.
Gene Yeo PhD MBA is a Professor of Cellular and Molecular Medicine at the University of California, San Diego (UCSD). Dr. Yeo has a BSc in Chemical Engineering and a BA in Economics from the University of Illinois, Urbana-Champaign, a Ph.D. in Computational Neuroscience (Machine Learning) from Massachusetts Institute of Technology and an MBA from the UCSD Rady School of Management. Gene’s research interest is in understanding and manipulating RNA processing in development and disease using induced pluripotent stem cell and animal models. Gene’s lab develops and utilizes multidisciplinary technologies ranging from machine learning, biochemistry, molecular biology, genomics, chemistry and materials research. Gene is widely collaborative and emphasizes on reproducibility and robustness in his data and results, and transparency and honesty in communicating with trainees and colleagues. Dr. Yeo has authored more than 160 peer-reviewed publications including invited book chapters and review articles in the areas of neurodegeneration, RNA processing, computational biology and stem cell models; and served as Editor on two books on the biology of RNA binding proteins. Recently, his lab developed enhanced CLIP for the purposes of large-scale mapping of protein-RNA interactions. His lab also demonstrated in vivo RNA targeting with CRISPR/Cas proteins with proof of concept in repeat expansion disorders. Gene is on the Editorial Boards of the journals Cell Reports and Cell Research. Gene joined UCSD as an Assistant Professor in 2008, was promoted with tenure to Associate Professor in 2014 and to Professor in 2016. Gene was the first Crick-Jacobs Fellow at the Salk Institute (2005-2008) and is a recipient of the Alfred P Sloan Fellowship in recognition of his work in computational molecular biology (2011), Alpha Chi Sigma-Zeta Chapter Krug Lecturer (2016), Singapore National Research Foundation Visiting Investigatorship Award (2017), the inaugural Early Career Award from the International RNA Society (2017), the Blavatnik National Award Finalist (2018 & 2019) and San Diego Xconomy Awardee for ‘Big Idea’ (2019). Gene is a co-founder of biotech companies which includes Locana, Eclipse Bioinnovations, Enzerna and Proteona. Gene played a critical role in the successful raising of $55M for Locana at series A (2019).
Arthur is currently CEO of DTx Pharma—a company he founded out of JLABS with John Grundy and Adam Pavlicek. He received his Honors B.Sc. in Biological Sciences from University of Delaware and his Ph.D. in Biomedical Sciences with a focus in Pharmacology from the University of California, San Diego. He was a Beckman and NSF Fellow during his undergraduate and graduate studies, respectively. During graduate school, he founded Synervate–a cell therapy company focused on a treatment for Alzheimer’s Disease. Following Synervate, he held positions of increasing responsibility at Janssen (J&J), MedImmune/AstraZeneca and Regulus Therapeutics.
Gerry Rodrigues is an Associate Vice-President in Research with Allergan and oversees the early stage research activities in retina. In addition to managing early stage retina projects, Gerry serves on retina Development and Clinical Core teams. He is also chair of the early stage portfolio team, which manages Discovery research activities and actively participates in helping drive the early stage pipeline through external diligence activities. Gerry currently leads the Allergan research team for the Allergan/Editas Medicines R&D Alliance to discover and develop CRISPR genome editing therapeutics for eye diseases. He received his PhD from McGill University and completed a post-doctoral fellowship at New York University Medical Center.
Kenneth P. Serbin’s mother Carol died of Huntington’s disease in 2006 after a 20-year battle against the disease. Serbin tested positive for the HD gene in 1999. His daughter Bianca tested negative for HD in the womb and is today a healthy 19-year-old college sophomore. Since 1998, Serbin has served as a volunteer advocate for the Huntington’s Disease Society of America (HDSA). Adopting the pseudonym “Gene Veritas,” in January 2005 he started the blog “At Risk for Huntington’s Disease” (www.curehd.blogspot.com), where he has posted more than 270 articles on numerous medical and social facets of the disorder and the quest for treatments. Starting in 2007, he initiated the effort in California to obtain funding for HD research from the state’s world-leading stem cell institute. In 2011, he was named the HDSA Person of the Year. In 2012, Serbin went public about his gene-positive status with his article “Racing Against the Genetic Clock,” in The Chronicle of Higher Education. On May 18, 2017, Serbin and his family joined Huntington’s advocates from around the globe at a special audience in Rome with Pope Francis, who declared Huntington’s “hidden no more.” A professor at the University of San Diego, he researches Brazilian social, cultural, political, and religious history and the history of science, technology, and medicine. He lives with his wife Regina in San Diego.
Alexis received her B. S. degree in chemistry from the University of California, Berkeley in December of 2008. She then joined the lab of Jacqueline K. Barton at the California Institute of Technology for her doctoral studies. While at Caltech, she worked as an NSF Graduate Research Fellow on the design, synthesis, and study of DNA mismatch-binding metal complexes and received her Ph.D. in 2014. She pursued postdoctoral work as a Ruth L. Kirschstein NIH Postdoctoral Fellow in the laboratory of David R. Liu, where she developed base editing, a new approach to genome editing that enables the direct, irreversible chemical conversion of one target DNA base into another in a programmable manner, without requiring double-stranded DNA backbone cleavage. Alexis joined the Department of Chemistry and Biochemistry at the University of California at San Diego in 2017, where her lab develops and applies new precision genome editing techniques to the functional genomics field.
As Head of JLABS @ San Diego Sanjay is responsible for setting the strategy and overseeing all operational activities. He joined after leading R&D finance operations for the Janssen Pharmaceuticals R&D CFO. Prior to this role he led early stage R&D divestments in partnership with JBD, JJI & JJDC, leading to a number of successful asset out-licenses and the formation of four new companies: Fusion Pharmaceuticals, Provention Bio (PRVB), Mebias Discovery and Aro Therapeutics. He has also led two internal discovery platform venture startups within Janssen R&D whilst managing overall venture operation.
Prior to Janssen Sanjay was with healthcare venture capital firm Quaker Partners, a fund with $700MM under management. Here he primarily invested in bio-pharmaceuticals and diagnostic/tools, including key deals Durata Therapeutics, Rapid Micro Biosystems and RainDance Technologies. Prior to Quaker Partners Sanjay was with Centocor Johnson & Johnson where he cofounded an internal venture to develop potential cell therapies for retinal diseases. Before joining Centocor he was as an entrepreneur at San Diego-based start-up Morphogen Pharmaceuticals, where he led pre-clinical operations to develop cell therapies for cardiovascular and neurological indications.
Leah Aluisio is an Associate Director working with Janssen, Neuroscience R & D, late stage Clinical Development. She believes passion and her experiences in Neuroscience Early Discovery brings a unique set of skill to the development of clinical compounds. Leah has contributed to over 25 new molecular entities across 15 years in Neuroscience discovery and more recently as a Study Responsible Scientist for Phase 1 and Phase 3 clinical development programs. Her work as a Clinical Scientist contributed to a successful FDA Advisory Committee and US launch of SPRAVATO™, the first new mechanism of action in decades to treat major depressive disorder.
The views expressed during this event by anyone other than an employee of Johnson & Johnson Innovation LLC, its parent company or any affiliate companies (herein after referred to as “JJI”) are those of the speakers or experts alone, and such speakers or experts are solely responsible for the information and opinions expressed by them. By hosting this event and the presentations, JJI does not endorse the views of the speakers, experts or the attendees, and JJI makes no warranties, express or implied, as to the content, the views, advice or the information presented.
