Science First - Gene Therapy: Back to the Future

Jun 16 2016https://www.eventbrite.com/e/science-first-gene-therapy-back-to-the-future-ticke...

Will gene therapy become a reality in your lifetime? We have heard about gene therapy for decades, often with the promise of success. Modulating gene expression in vivo instead of undergoing surgery or taking drugs has been touted and doubted as the future of fighting diseases, but the tides have ebbed and flowed with many successes and failures.

Recently, we are seeing progress toward a sea change. Today there is much greater optimism around gene therapy and its applications. Johnson & Johnson Innovation, JLABS is bringing together three of the companies on the front lines of gene therapy. Join the experts as they present their latest research in this area, and continue the conversation about the future of gene therapy.

This event will tackle the following questions:

  • What are the barriers; what are the difficulties that need to be addressed before we see successful gene therapies?
  • How will potential technology overcome preexisting blocks to success? How close are we?
  • What is the future of gene therapy, and how can we get there?



Agenda:
2:00 PM | Registration opens and Networking
2:30 PM | Presentations
3:15 PM | Panel discussion
4:00 PM | Networking Reception
4:30 PM | Close

Speakers:
Philip Gregory | CSO, Bluebird Bio read bio»
Rob Kotin | VP, Virology & Gene Therapy, Voyager Therapeutics, Inc., Adjunct Professor, Gene Therapy Center, University of Massachusetts Medical School read bio»
Laura Sepp-Lorenzino | VP, Entrepreneur in Residence, Alnylam Pharmaceuticals read bio»
[moderator] Charles Bridges | VP, Medical Devices, Cardiovascular Therapeutic Area Expert, Johnson and Johnson MD&D read bio»

Fees:
$25 | General Public
$15 | Student/Academic
$35 | Onsite

Location:
JLABS @ LabCentral
700 Main Street
Cambridge, MA

About the Science First series:
The Science First series, sponsored by Johnson & Johnson Innovation, JLABS, brings together top experts to explore pressing topics in their scientific field. It is a chance for local scientists, researchers, and entrepreneurs to present and dialogue on new discoveries, advancing techniques, and other cutting-edge science themes.

Speakers' Biographies:

Philip Gregory | Chief Scientific Officer, Bluebird Bio
Philip D. Gregory, D. Phil., has served as Chief Scientific Officer of bluebird bio since June 2015. Dr. Gregory was formerly with Sangamo BioSciences, Inc., where he held multiple leadership positions, most recently serving as Chief Scientific Officer and Senior Vice President, Research. While at Sangamo Dr. Gregory led pioneering research in genome editing and was named by Thompson Reuters as one of "The World's Most Influential Scientific Minds 2014". Prior to Sangamo, Dr. Gregory was at the University of Munich, Germany, where he studied the role of chromatin structure in gene regulation and published extensively in this field. He served as a member of the Scientific Advisory Board of Keystone Symposia from 2009 to 2015. Dr. Gregory earned a D. Phil. in biochemistry from the University of Oxford and holds a B.Sc. in microbiology from the University of Sheffield.
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Robert Kotin | Vice President, Virology & Gene Therapy, Voyager Therapeutics, Inc., Adjunct Professor, Gene Therapy Center, University of Massachusetts Medical School
Robert Kotin, Vice President of Virology and Gene Therapy, established an advanced adeno-associated virus vector platform at Voyager Therapeutics. Robert has made several landmark discoveries during a 30-year career in AAV research. He discovered that AAV DNA integrates preferentially into the AAVS1 locus on human chromosome 19 and characterized novel activities of the AAV non-structural protein necessary for integration. As the head of the Molecular Virology and Gene Therapy Laboratory in the National Heart, Lung, and Blood Institute of the NIH, Robert's research on parvoviruses led to the discovery and development of methodologies to produce rAAV using the baculovirus / Sf9 cell system. This scalable, economical process has been licensed by uniQure to produce alipogene tiparvovec (Glybera™), the only regulatory agency approved rAAV product. Robert continues his basic science projects at the University of Massachusetts Medical School where he is an Adjunct professor in the Gene Therapy Center.
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Laura Sepp-Lorenzino, Ph.D. | Vice President, Entrepreneur in Residence, Alnylam Pharmaceuticals
Dr. Sepp-Lorenzino joined Alnylam in 2014 as VP, Entrepreneur-in-Residence, and responsible for the Hepatic Infectious Disease Strategic Therapeutic Area. Before joining Alnylam, Dr. Sepp-Lorenzino spent 14 years at Merck & Co., having most recently served as Executive Director and Department Head, RNA Therapeutics Discovery Biology. In this role, she was responsible for identification and optimization of siRNAs and delivery vehicles, advancement of pre-clinical candidates, and development of an siRNA-conjugate platform to expand the repertoire of tissues accessible to in vivo siRNA delivery. Prior to RNAi, Laura worked in oncology drug discovery and development, having led the Cancer Research Department at Merck West Point, and having been an Assistant Lab Member and Assistant Attending Molecular Biologist at Memorial Sloan-Kettering Cancer Center. Laura received her Professional Degree in Biochemistry from the University of Buenos Aires, and her M.S. and Ph.D. in Biochemistry from New York University.
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Charles R. Bridges, M.D., Sc.D. | Vice President, Medical Devices, Cardiovascular Therapeutic Area Expert, Johnson and Johnson MD&D
Received an A.B. in Applied Physics, magna cum laude from Harvard College; an M.D. from Harvard Medical School; a Master of Science in Electrical Engineering and Doctor of Science in Chemical Engineering from M.I.T. as a Whittaker Foundation Fellow. He completed residency training at the Brigham and Women's Hospital and the Hospital of the University of Pennsylvania. Dr. Bridges is formerly Professor of Surgery, Division of Cardiovascular Surgery at Penn, Chief of Cardiac Surgery at Pennsylvania Hospital and Professor and Chairman, Department of Thoracic and Cardiovascular Surgery at Carolinas Healthcare System, the second largest public healthcare system in the US. He is currently Cardiovascular Therapeutic Area Expert and Vice President of Johnson and Johnson Medical Devices and Professor of Cardiovascular Surgery at the Icahn School of Medicine at Mount Sinai. His basic and translational research interests include studies continuously funded for over a decade by the National Heart Lung and Blood Institute (NHLBI) for the development of novel methods and devices for highly efficient vector-mediated cardiac gene delivery using minimally invasive cardiac surgical techniques. Dr. Bridges invented "molecular cardiac surgery with recirculating delivery" (MCARD), a patented technology and arguably, the most efficient and cardiac-specific gene delivery platform in the world. Dr. Bridges has over 150 peer-reviewed publications and fourteen issued and pending US patents for inventions in the fields of cardiovascular devices and cardiac and systemic gene and cell delivery. He has served as a consultant to the Circulatory System Devices Panel of the US Food and Drug Administration, a regular member of the Bioengineering, Technology and Surgical Sciences Study Section of the National Institutes of Health and on the Steering Committee of the Gene Therapy Resource Program of the National Heart Lung and Blood Institute. His current interests are focused on leveraging his extensive background in biotechnology; biomedical engineering and clinical cardiovascular surgery to both source and drive disruptive innovation in the cardiovascular device space.
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