Title

Regen Med & Advanced Therapies State of the Industry Briefing
Monday
January
11
Missed ARM's 6th Annual Regenerative Medicine & Advanced Therapies State of the Industry Briefing? View the webcast and get caught up on the sector!
San Francisco, CA

Webcast Now Available!
ARM's Annual Regen Med & Advanced Therapies State of the Industry Briefing is a top-level gathering for key stakeholders in the sector - including business executives, investors, patient advocates and academic leaders - to learn more on the recent progress and outlook for the industry in the coming year. This event annualy attracts over 300 attendees and is the largest gathering specifically for regenerative medicine and advanced therapies taking place during JP Morgan week in San Francisco.

The webcast is broken down into three separate videos - one for each part of the program. To see all of the video options click the three lines in the upper left corner. To view additional content on the sector, please visit ARM's YouTube page at https://www.youtube.com/user/AllianceRegenMed.

Program Agenda
8:00am - 8:20am | Program Introduction & Industry Update
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine
Edward Lanphier, President & CEO, Sangamo BioSciences; Chairman, Alliance for Regenerative Medicine

8:20am - 9:05am | The 2016 Sector Forecast: Upcoming Clinical Data Events
This session features CEOs and senior executives from leading companies in the regenerative medicine and advanced therapies sector for a forward-looking discussion of expected clinical data events in 2016 and beyond. Panelists will address key issues related to continuing momentum in the sector as well as unique strategies and partnerships that will be required for success going forward.
Jason Kolbert, Head of Healthcare Research & Senior Managing Director, Maxim Group (co-moderator)
Jason McCarthy, Equity Research Analyst, Biotechnology, Maxim Group (co-moderator)
Silviu Itescu, Managing Director & CEO, Mesoblast
Sven Kili, VP & Head of Gene Therapy Development, GlaxoSmithKline
Gil Van Bokkelen, Chairman & CEO, Athersys

Jeffrey Walsh, COO, bluebird bio
Sue Washer, President & CEO, AGTC

9:05am - 9:50am | The Promise of Regenerative Medicine & Advanced Therapies in Oncology
This in-depth session will examine the progress of regenerative medicine and advanced therapies specifically as the field relates to the area of oncology. The panel's expert speakers will discuss a number of key hurdles on the path to commercialization including reimbursement and market access and what progress can be expected in the sector in the coming year.
Joshua Schimmer, Managing Director & Senior Research Analyst, Piper Jaffray (moderator)
Jeffrey Abbey, President & CEO, Argos Therapeutics
Usman Azam, Global Head, Cell & Gene Therapies Unit, Novartis

André Choulika, Chairman & CEO, Cellectis
Gregg Sando, Founder & CEO, Cell Medica

9:50am | Program End

Who Attends?
Over 300 of the advanced therapy and regenerative medicine field's top stakeholders including company founders, C-level business executives, investors, media, patient advocates and academic leaders interested in learning more on the sector's recent advances and outlook for the industry in the coming year.

Location
Parc 55 Hotel
55 Cyril Magnin St.
San Francisco, CA 94102

Questions?
Please contact Laura Parsons at lparsons@alliancerm.org.

Speaker Biographies
Jeffrey Abbey, President & CEO, Argos Therapeutics
Mr. Abbey has served as President and CEO of Argos Therapeutics and a member of the board of directors since February 2010. Abbey served in various other positions at Argos from September 2002 to February 2010, including as VP of Business Development from February 2004 to January 2009 and as Chief Business Officer from January 2009 to February 2010. Prior to joining Argos, Abbey served as VP of Business Development and Finance at Internet Appliance Network, an information technology company, from 1999 to 2001. Abbey was a partner at Eilenberg and Krause, a corporate law firm, from 1994 to 1999. Abbey received an A.B. in Mathematical Economics from Brown University and an M.B.A. and J.D. from the University of Virginia.

Usman Azam, Global Head, Cell & Gene Therapies Unit, Novartis
Dr. Azam is a member of the Pharmaceutical Executive Committee of Novartis. His responsibilities in the Cell and Gene Therapy Unit (C&GTU) include commercial, business development licensing, new product commercialization, clinical development, regulatory affairs, technical research development, technical operations and quality. Previous roles at Novartis include Head of Global Medical Affairs and HEOR General Medicines, Chief Scientific Officer and Head of U.S. Clinical Development and Medical Affairs and Global Head, Regulatory Affairs for the neurosciences and ophthalmic franchise. Azam has an extensive U.S., global and international pharmaceutical industry background, with strong experience in science, regulatory and commercial. He joined Novartis in 2009 from Novaccel Therapeutics, where he was Chief Executive Officer, a company he founded focusing on accelerating assets in the translational medicine setting. Previously, he served as Chief Medical Officer at Aspreva Pharmaceutics, a company focused on orphan disease states and on the development of Mycophenolate Mofetil (Cellcept) in autoimmune diseases such as lupus nephritis, myasthenia gravis and pemphigus vulgaris. Prior to this, he worked at Johnson and Johnson in pharmaceutical research and development and also in medical devices at Johnson and Johnson Ethicon. Azam is a graduate of the University of Liverpool, where he completed undergraduate studies in Human Biology and Anatomy. He graduated from the University of Liverpool School of Medicine and attained board certification as an obstetrician and gynecologist in the UK.

André Choulika, Chairman & CEO, Cellectis
Dr. Choulika is one of the founders of Cellectis and has been Chairman of the Board and CEO since 2000. He has also been President of Calyxt (previously Cellectis plant sciences) since August 2010. From 1997 to 1999, Choulika worked as a post-doctoral fellow in the Division of Molecular Medicine at Boston Children’s Hospital, where he was a pioneer in the analysis and use of meganucleases to modify complex genomes. He has also served on the boards of directors of several biotechnology companies. After receiving his Ph.D. in Molecular Virology from the University of Paris VI (Pierre et Marie Curie), he completed a research fellowship in the Harvard Medical School Department of Genetics. Choulika also has management training from the HEC (Challenge +).

Silviu Itescu, Managing Director & CEO, Mesoblast
Dr. Itescu has served on Mesoblast's board of directors since the Company's founding in 2004, was Executive Director from 2007, and became CEO and Managing Director in 2011. Prior to founding Mesoblast in 2004, he established an international reputation as a physician scientist in the fields of stem cell biology, autoimmune diseases, organ transplantation and heart failure. Itescu has been a faculty member of Columbia University in New York and the University of Melbourne and Monash University in Australia. In 2013, Itescu received the inaugural Key Innovator Award from the Vatican’s Pontifical Council for Culture for his leadership in translational science and clinical medicine in relation to adult stem cell therapy. In 2011, he was named BioSpectrum Asia Person of the Year. He has consulted for various international pharmaceutical companies, has been an adviser to biotechnology and healthcare investor groups, and has served on the board of directors of several publicly listed life sciences companies.

Sven Kili, VP & Head of Gene Therapy Development, GlaxoSmithKline
Dr. Kili is the VP and Development Head for the Gene Therapy division of GlaxoSmithKline (GSK) where he leads the teams developing ex-vivo gene therapies for a variety of genetic disorders. They are currently developing solutions for a number of diseases including ADA-SCID; WAS; Metachromatic Leukodystrophy (MLD) and Beta-Thalassemia. Prior to this, Kili was Senior Director, Cell Therapy and Regenerative Medicine for Sanofi (Genzyme) Biosurgery where he led the clinical development and medical affairs activities culminating in the granting of the first combined ATMP approval in the EU for MACI®. His team also prepared and submitted regulatory filings for Australia and the U.S., including health technology assessments. Before joining Genzyme, Kili worked for Geistlich Pharma where, in addition to leading the cell therapy medical activities, he oversaw all UK regulatory functions and was the QPPV for the EU. Kili trained as an orthopaedic surgeon in the UK and South Africa and since leaving full-time clinical practise has developed expertise in cell and gene therapy clinical development, regulatory compliance, value creation, risk management and product safety, product launches and post-marketing activities. He sits on the board of a Swedish stem cell company and still maintains his clinical skills in the UK NHS and serves as an ATLS Instructor in his spare time.

Jason Kolbert, Head of Healthcare Research & Senior Managing Director, Maxim Group
Mr. Kolbert is Head of Healthcare Research and a Senior Biotechnology Analyst at Maxim Group. Kolbert has worked extensively in the healthcare sector as product manager for a leading pharmaceutical company, a fund manager and as an equity analyst. Prior to joining Maxim Group, he spent seven years at Susquehanna International Group where he managed a healthcare fund and later founded SIG’s sell-side biotechnology team. Previously, Kolbert served as the healthcare strategist for Salomon Smith Barney. He is frequently quoted in Barron’s and is regularly featured on CNBC. Prior to beginning his Wall Street career, Kolbert served as a product manager for Schering-Plough in Osaka, Japan. He received a B.S. in Chemistry from SUNY New Paltz, and an M.B.A. from the University of New Haven.

Edward Lanphier, President & CEO, Sangamo BioSciences; Chairman, Alliance for Regenerative Medicine
Mr. Lanphier, the Founder of Sangamo BioSciences, has served as President, CEO and as a member of the board of directors since Sangamo's inception in 1995. Lanphier has over 30 years of experience in the pharmaceutical and biotechnology industry. From June 1992 to May 1997, he held various positions at Somatix Therapy Corporation, a gene therapy company, including EVP, Commercial Development and Chief Financial Officer. Prior to Somatix, he was President and CEO of BioGrowth, a biotechnology company that merged with Celtrix Laboratories to form Celtrix Pharmaceuticals in 1991. From 1986 to 1987, he served as VP of Corporate Development at Biotherapeutics, Inc. From 1984 to 1986, Lanphier served as VP of Corporate Development at Synergen. Prior to Synergen, he was employed by Eli Lilly and Company in the strategic business planning biotechnology group. He currently serves on the board of directors of the Biotechnology Institute, is Chairman of the Alliance for Regenerative Medicine, serves on the board of trustees for The Buck Institute for Research on Aging, and the Dean's Advisory Board for the University of Michigan School of Public Health. Lanphier holds a B.A. in Biochemistry from Knox College.

Jason McCarthy, Equity Research Analyst, Biotechnology, Maxim Group
Dr. McCarthy is an equity research analyst covering the biotechnology industry at Maxim Group. McCarthy joined Maxim Group in July 2014. Prior to this, he received his Ph.D. in Biomedical Sciences from the Albert Einstein College of Medicine where his focus was on the development of monoclonal antibody-based therapeutics for the prevention and/or treatment of invasive fungal disease associated with immune compromise. He also holds master's degrees in both biomedical research (Albert Einstein College of Medicine) and molecular biology (Adelphi University), as well as a bachelor's degree in biochemistry from Stony Brook University. The biotechnology research group at Maxim covers over 50 names with strong focus on cell-based therapeutics, immune-based therapeutics and gene therapy, and how these platforms are causing a paradigm shift in not only medicine, but also in how we think about disease.

Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine
Mr. Ruffin has more than 20 years of experience in the biotech and healthcare industries. He is a Founder and Managing Director of the Alliance for Regenerative Medicine (ARM), the global organization representing the interests of the regenerative medicine community. Ruffin is also the Managing Partner of Adjuvant Partners, a boutique regenerative medicine and advanced therapies business consulting firm. Prior to joining Adjuvant Partners, he was CEO of LifeTech Innovations, a business development consulting firm. Prior to his position at LTI, Ruffin was EVP of Capital Formation and Business Development at the Biotechnology Industry Organization (BIO), the largest trade organization representing the biotech and drug development industries. Joining BIO in 1994 as one of its original employees, Ruffin was responsible for building the organization's global business development and investor outreach programs focused on helping companies raise capital and identify strategic partnering and licensing opportunities. This BIO business development franchise is now the largest in the world, with operations in the U.S., Europe and Japan. In addition to his business development work at BIO, Ruffin was responsible for leading the industry's capital formation advocacy efforts with a focus on economic incentives to promote investment in early stage biotech and med-tech businesses. He was also a founder and board member of the Interoperable Informatics Infrastructure Consortium (I3C), an international standards setting body for the bioinformatics industry. Prior to joining BIO, Ruffin worked for U.S. Senator Arlen Specter for five years as his Senior Legislative Assistant. Prior to that, he spent five years working in varying capacities, including Policy Analyst at Systems Planning Corporation International and the Center for Strategic and International Studies. Ruffin received his M.A. in International Studies and Economics from the Johns Hopkins School for Advanced International Studies and his B.A. from the University of Virginia.

Gregg Sando, Founder & CEO, Cell Medica
After a career in investment banking based both in London and New York, Mr. Sando completed his Immunology MSc degree at the medical school of Imperial College. Recognizing the significant progress taking place in the field of cellular immunotherapy, Sando formed Cell Medica in cooperation with a small group of the leading clinicians and immunologists who were pioneering this new mode of medical treatment.

Joshua Schimmer, Managing Director & Senior Research Analyst, Piper Jaffray
Dr. Schimmer is a managing director and senior research analyst who joined Piper Jaffray in 2013 to cover biotechnology companies. Schimmer has been on the Street for more than 10 years. Prior to joining Piper Jaffray, he worked in equity research at Lazard Capital Markets, Leerink Swann and Cowen and Company. In addition, he spent time on the buy-side at Davidson Kempner as part of the healthcare investing team. Schimmer was awarded runner-up in the small/midcap biotechnology category in Institutional Investor magazine’s annual poll for 2015. He holds an M.D. from the University of Toronto, where he also specialized in Internal Medicine and subspecialized in Rheumatology. Schimmer also holds an M.B.A. from Harvard Business School.

Gil Van Bokkelen, Chairman & CEO, Athersys
Dr. Van Bokkelen has served as Athersys' CEO and Chairman since August 2000. Van Bokkelen co-founded Athersys in October 1995 and served as CEO and Director since Athersys' founding. Prior to May 2006, he also served as Athersys' President. Van Bokkelen is past Chairman of the Alliance for Regenerative Medicine, a Washington D.C. based consortium of companies, patient advocacy groups, disease foundations, and clinical and research institutions that are committed to the advancement of the field of regenerative medicine. He is also the Chairman of the Board of Governors for the National Center for Regenerative Medicine, and serves on a number of other boards, including the Biotechnology Industry Organization's ECS board of directors, the McGowan Institute for Regenerative Medicine and the Regenerative Medicine Foundation. He received his Ph.D. in Genetics from Stanford University School of Medicine, his B.A. in Economics and B.A. in Molecular Biology from UC Berkeley.

Jeffrey Walsh, COO, bluebird bio
Mr. Walsh joined bluebird bio as Chief Operating Officer and acting Chief Financial Officer in May of 2011 and has been a key contributor to bluebird’s growth from a private 20 person company in early clinical development to a public 250 person company with a late stage clinical pipeline. Walsh has more than 25 years of experience in the biotechnology field with both public and private life sciences companies with responsibility for financial management, corporate development, program management, commercial development, strategy and legal functions. Before joining bluebird bio, Walsh was the Chief Business Officer of Taligen Therapeutics where he built and led the financial management, corporate development and program management functions of the company. He played a key role in the strategy and overall growth of the company as well as the sale of Taligen to Alexion Pharmaceuticals in January 2011 for up to $478 million. Walsh started his career at SmithKline Beecham in finance and worldwide business development roles. He subsequently held senior-level business development, finance and operations roles at Allscripts (MDRX), PathoGenesis (PGNS sold to Chiron for $700 million), EXACT Sciences (EXAS) and Inotek Pharmaceuticals (ITEK). Walsh received his M.B.A. from the Kellogg Graduate School of Management at Northwestern University and his B.A. from Yale University. Walsh resides in Wellesley, MA with his wife and three daughters. He is currently on the board of directors for the Alliance for Regenerative Medicine (a non-profit advocacy group supporting innovative regenerative medicine and cell and gene therapy research and development) and the board of advisors for the Convergence Conference. Walsh has been a business school guest lecturer on topics of entrepreneurialism and biotechnology organizational behavior. Walsh played lacrosse at Yale and has coached soccer and basketball for over 10 years in the Wellesley community and holds his Massachusetts E soccer coaching license. He also enjoys competing in triathlons.

Sue Washer, President & CEO, AGTC
An experienced entrepreneur with a strong scientific and operational background, Ms. Washer brings a decade of experience in pharmaceutical management and research with Abbott Labs and Eli Lilly & Company and more than 16 years of senior management experience with entrepreneurial firms in Florida including three start-ups. At AGTC, Washer has successfully secured investments of more than $91 million from nationally recognized venture capitalists and granting agencies; negotiated and closed on a major collaboration with a top five biotech company; led the company to complete critical milestones; and recruited an experienced management team. Her experience as a group leader with Abbott’s Diagnostic Division helped hone her skills in bringing new products and technologies from the lab bench into the marketplace. Washer is the Chair of Southeast BIO, is a member of the Executive Committee of BioFlorida and sits on the board of the Florida High Tech Corridor Council. She has a degree in biochemistry from Michigan State University and an M.B.A. from the University of Florida where she was one of the first graduates from the Warrington College of Business Entrepreneurship program.

Biotech Showcase

This meeting is held in conjunction with Biotech Showcase 2016 hosted by EBD Group and Demy Colton.