Greg Aronin

Vice President, External Affairs at Paragon Biosciences

Greg Aronin serves as vice president of external affairs at Paragon Biosciences. He works with all external stakeholders, including government and industry, and has oversight responsibility for corporate communications strategy, marketing and thought leadership.

Greg Aronin has more than 25 years of experience in public affairs. Prior to Paragon, he co-founded Forte Group, a non-partisan public affairs and advocacy consulting firm. He previously served as vice president of public affairs at Marathon Pharmaceuticals. Earlier in his career, Mr. Aronin led government affairs for multiple Johnson & Johnson companies. He began his career directing government affairs activities for the Michigan Medical Society. Mr. Aronin is a graduate of the University of Michigan where he earned his Master of Public Health. He earned a bachelor’s degree in international relations at the James Madison College of Michigan State University.

Jamie Bauersmith

Vice President, Translational Science at UPMC Enterprises

Jamie is Vice President of Translational Sciences at UPMC Enterprises. Before joining Enterprises, Jamie was the Chief Strategic Officer and General Counsel of ByHeart, Inc., an infant formula company, where he led the company’s FDA strategy, managed all legal affairs and led the acquisition and set-up of the company’s manufacturing facility. Prior to his role at ByHeart, Jamie worked in the pharmaceutical sector for over 15 years where he held a variety of leadership positions at Fresenius Kabi, where he was the SVP overseeing the company’s portfolio and marketing strategy unit, and Amneal Pharmaceuticals where he oversaw the corporate development and procurement groups and was part of the executive management team.

Before his tenure at Fresenius and Amneal, James spent 10 years practicing law, first as an attorney in New York with Haug Partners and then as in-house counsel at Teva Pharmaceuticals where he managed the company’s Hatch Waxman Patent litigations. He earned a B.S. from the University of Notre Dame, a J.D. from the Pennsylvania State University School of Law, and an M.S. of Bioscience and Biotechnology from Drexel University. He is a registered U.S. Patent Attorney and maintains an active Pennsylvania law license.

At Enterprises, Jamie focuses his time on identifying, analyzing and structuring investment opportunities that address unmet therapeutic need and align with UPMC’s vision of bringing life changing medicine to patients.

Julie Bick, PhD

Chief Scientific Officer at FlowMetric a KCAS Company

Julie brings over 18 years of biochemistry and cell biology research experience acquired in both academic and industrial laboratories. Following her initial relocation to the US, Julie worked as a postdoctoral research associate at The Rutgers AgBiotech Center, where she remained for 4 years as Associate Professor. She then pursued private-sector opportunities while at the Tory Mesa Research Institute, San Diego, CA, and with Sanofi-Aventis, Bridgewater, NJ working in the areas of therapeutic protein engineering, recombinant protein production, and cell line development.
Prior to joining FlowMetric Diagnostics, Julie was appointed Chief Scientific Officer/Vice President of Cellexus Biosystems, Inc., where she was responsible for launching innovative single-use bioreactor systems for bio-therapeutic production in North America. More recently, Julie has worked in partnership with several life science companies in the greater Philadelphia area in the development of medical devices, assays and diagnostic tools, and co-authored numerous publications and patents in these areas.

Educated in the UK, Julie holds a PhD in Biochemistry from the University of Southampton and degrees with honors in Medicinal Biochemistry and Genetics from the University of Nottingham.

Joe Colletti

Executive Vice President at JLL

Joe is an Executive Vice President in JLL’s Philadelphia office, where he focuses on working with life sciences companies to assist in developing real estate solutions that drive business performance. Joe is an active member of JLL’s Life Sciences Advisory Council, a national practice comprised of JLL industry leading professionals with extensive knowledge and expertise working with life sciences organizations across the US. With over 12 years of industry experience, Joe has completed over 350 transactions on behalf of his clients, including R&D lab, GMP manufacturing, warehouse, and office space assignments. Working exclusively with tenants, Joe partners with entrepreneurs, emerging biotech and global pharma companies throughout the Greater Philadelphia region to assist with their real estate needs.

Jonathan Epstein, MD

William Wikoff Smith Professor, Executive Vice Dean and Chief Scientific Officer, Perelman School of Medicine at the University of Pennsylvania, Senior Vice President and Chief Scientific Officer, Pe at University of Pennsylvania

Dr. Epstein graduated from Harvard College in 1983, Harvard Medical School in 1988 and completed his Residency and Fellowship in Medicine and Cardiology at the Brigham and Women's Hospital, where he also completed an HHMI Postdoctoral Fellowship in Genetics. In 1996 he accepted a position as Assistant Professor of Medicine in the Division of Cardiology at the University of Pennsylvania. From 2006-2015, he served as Chairman of the Department of Cell and Developmental Biology and Scientific Director of the Penn Cardiovascular Institute. He is currently the William Wikoff Smith Professor, Executive Vice Dean and Chief Scientific Officer at the Perelman School of Medicine at the University of Pennsylvania.

Dr. Epstein has been the recipient of numerous awards, including the Sir William Osler Young Investigator Award from the Interurban Clinical Club (2001) and the Outstanding Investigator Award from the American Federation for Medical Research (2006). He is a member of the American Academy of Arts and Sciences, American Association of Physicians, Past President of the Interurban Clinical Club, Past President of the American Society for Clinical Investigation and a member of the National Academy of Medicine (previously the Institute of Medicine). He serves on several editorial boards, and is a past Deputy Editor of the Journal of Clinical Investigation. He is a member of the NIH Council of Councils and the NIH Precision Medicine Initiative Cohort Program Advisory Committee. Dr. Epstein was a founding co-director of the Penn Institute for Regenerative Medicine in 2007.

Dr. Epstein’s research has focused on the molecular mechanisms of cardiovascular development and implications for understanding and treating human disease. His group has been at the forefront of utilizing animal models of congenital heart disease to determine genetic and molecular pathways required for cardiac morphogenesis, with implications for pediatric and adult cardiovascular disease. Stem cell, angiogenesis and epigenetic studies have had direct implications for the development of new therapeutic agents for heart failure and myocardial infarction.

Matt Fenske

Vice President, Lifecycle & Portfolio Strategy at Harmony Biosciences

Matt is currently the Vice President, Lifecycle & Portfolio Strategy at Harmony Biosciences. He responsibilities include advancing new products and lifecycle programs in the company portfolio as well as building the pipeline through business development. Prior to Harmony, Matt led the New Products Group at Shire for the Immunology and Hematology Franchises and also a similar role at Baxalta prior to that. Baxalta was a spin-off from Baxter where he held numerous roles in Global Marketing and US Marketing in the BioScience Business Unit. Matt also spent time with Boston Scientific (Guidant) in sales and marketing roles.

Sarah Fuller

Vice President, North America Business Development & Alliance Management at Kyowa Kirin
Princeton
USA

Sarah entered the pharmaceutical industry in 1998, working in Business Development at a global CRO where she led the North American proposal team for Clinical Development services. In 2005, she joined Bristol-Myers Squibb in R&D Finance, managing clinical development budgets and providing financial guidance to Senior Management. Most recently before joining BioWa/Kyowa Kirin, Sarah managed over 20 collaborations in BMS’s Business Development/Alliance Management group. Sarah joined BioWa/Kyowa Kirin in 2020, and was named President of BioWa (Kyowa Kirin’s technology licensing business) in 2021. In addition to managing the BioWa business, she and her team also lead business development and alliance management activities for Kyowa Kirin North America.

Nicholas Galakatos

Senior Managing Director at Blackstone

Nicholas Galakatos is the Global Head of Life Sciences and is the Chairman of its Investment Committee, having joined Blackstone as part of its acquisition of Clarus in December 2018.

Prior to joining Blackstone, Dr. Galakatos was a co-Founder and Managing Director of Clarus since the firm's inception in 2005. Dr. Galakatos has over 30 years of industry and investment experience in the healthcare sector and has led investments in biotechnology, pharmaceutical company partnerships, and diagnostics, from startups to commercial-stage companies. Before Clarus, Dr. Galakatos was a General Partner at MPM Capital, a healthcare venture capital firm. From 1997 to 2000 he was Vice President of New Business at Millennium Pharmaceuticals (presently Takeda) and a member of the Management Team. He is a founder of Millennium Predictive Medicine and TransForm Pharmaceuticals, where he was the Chairman and founding CEO. Dr. Galakatos is the Chairman of Anthos Therapeutics and a member of the Board of Directors of Talaris, Inc (NASDAQ: TALS) and BioMed Realty. He is a member of the Director's Council of the Koch Institute at MIT and a member of the Board of Trustees at Reed College.

Dr. Galakatos earned his PhD in Organic Chemistry from MIT and performed his post-doctoral studies at Harvard Medical School. He earned his undergraduate degree at Reed College.

Lori Gavrin

Chief Business & Strategy Officer at NeuExcell

Lori Gavrin, PhD has over 20 years of industry experience, holding a variety of leadership roles at several major pharma and biotech companies, including Wyeth, Pfizer, and GSK. Most recently, Lori served as VP of Corporate Development at Tmunity Therapeutics where she helped develop and execute the business development strategy, successfully managed complex strategic partnerships, and became an integral part of the Philadelphia cell and gene therapy community.

Dr. Gavrin graduated Summa Cum Laude with a B.A. in chemistry from the Colgate University and obtained her Ph.D. in organic chemistry from the University of Pennsylvania. In addition to her business development acumen, Lori is a recognized leader in the medicinal chemistry community, having worked in the field for a dozen years. Lori previously chaired the Medicinal Chemistry Gordon Research Conference and has 18 patents to her name.

Lori serves on the Executive Committee of the Medicinal Chemistry Section of the American Chemical Society, the Scientific Advisory Board of the Chemistry Department at the University of Pennsylvania, the Oversight Committee of the Coulter Translational Drug Discovery Program at Drexel University and the Board of Directors of the Philadelphia-Israel Chamber of Commerce.

John Grady

Northeast Region Executive and Senior Vice President, Development at Wexford Science & Technology

Brianna Gross

Genetics Counselor at Children's Hospital of Philadelphia

Abhi Gupta

Senior Director, Global Gene Therapy Business at Pfizer Biopharma Group

Abhi is a Senior Director of Global Gene Therapy at Pfizer. He is responsible for developing asset strategies and generating competitive insights to drive R&D investment decisions that maximize pipeline value.

Shahram Hejazi, PhD

Managing Director and CEO at BioAdvance

Dr. Shahram Hejazi is a life science investor and entrepreneur with general management experience in both early-stage ventures and large global companies. As Managing Director and CEO, Dr. Hejazi is responsible for the overall leadership and strategic direction of BioAdvance. His areas of investment focus are research tools, devices, diagnostics and digital health. He serves on the boards of Halo Labs Inc., WellTrackOne Inc., Talex Medical LLC, Bainbridge Health LLC, RightAir Inc., Oncora Medical Inc., Wellsheet Inc. and Keriton Inc. He is the Chairman of Philadelphia Pediatric Device Consortium at CHOP. Dr. Hejazi is also the 2014 James Wei Visiting Professor of Entrepreneurship at Princeton University, where he is a part-time faculty and teaches a well-regarded course on the topic of venture capital.

Previously, Dr. Hejazi was the President of Kodak’s life science division (later called Carestream Molecular Imaging), where he had global responsibility for R&D, manufacturing, operations, sales, marketing and service. While holding this position, Dr. Hejazi led the system business to successful double-digit annual growth and directed the launch of a new imaging agent business.

Before Kodak, he was the CEO of Zargis Medical Corporation (an early stage medical device company that spun-off from Siemens), where he led the company’s efforts in areas ranging from raising capital and technology development to completion of several clinical trials, FDA approval and product launch- the company was later sold to 3M. Prior to Zargis, Dr. Hejazi was the Head of the Strategic Business Development group at Siemens where he was instrumental in establishing Siemens first corporate venture fund. Earlier in his career, Dr. Hejazi held R&D management positions within the diagnostic imaging and nanotechnology fields at Kodak and IBM, respectively. In addition to his Board responsibilities at BioAdvance, Shahram is also the Chairman of the advisory board of Integral Molecular Inc. His past Board responsibilities includes FDA Industry Advisory Panel Member for Molecular and Clinical Genetic Devices, Fox Chase Cancer Center, Bioscan Inc., Alpha Innotech Corp. Shahram is the author of over 10 scientific publications and has a PhD in electrical engineering with emphasis in biophysics (SUNY at Buffalo) and an executive business education (Stanford University).

Gerri Henwood

President & CEO at Baudax Bio

Gerri Henwood has served as our President, Chief Executive Officer and a director of the Company since November of 2019. From 2008 to December 2020, Ms. Henwood was the President, Chief Executive Officer and a director of Recro Pharma, Inc. (REPH), a company she founded in 2008. From 2006 to 2013, Ms. Henwood served as the President of Malvern Consulting Group, Inc., or MCG, a pharmaceutical incubator and consulting firm. Before founding MCG, Ms. Henwood was the President and Chief Executive Officer of Auxilium Pharmaceuticals, Inc. (formerly AUXL), or Auxilium, a biopharmaceutical company she founded in late 1999. From 1985 to 1999, Ms. Henwood was the founder and Chief Executive Officer of IBAH, Inc., or IBAH, a contract research organization listed on NASDAQ. Ms. Henwood began her career with Smith Kline & French, now part of GlaxoSmithKline plc. She rose through the ranks to be a brand manager, then the head of Regulatory and Medical Affairs for the U.S. business and then to the position of Group Director—Marketing in the International Pharmaceutical Division.

Ms. Henwood previously served on the board of directors of Tetraphase Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, from 2015 until its acquisition by La Jolla Pharmaceutical Company in June 2020. Before that, she served on the board of directors of Alkermes, Inc. and its successor company, Alkermes, plc, a global biopharmaceutical company, from 2003 until March 2015, and on the board of directors of MAP Pharmaceuticals, Inc., a biopharmaceuticals company, from 2004 until its acquisition by Allergan, Inc. in March 2013. Ms. Henwood also served on the compensation committee of the board of directors of Tetraphase Pharmaceuticals, Inc.

Ms. Henwood holds a B.S. in Biology from Neumann University.

Jesse Hwang

Scientific Engagement Lead at Johnson & Johnson Innovation - JLABS US Northeast
New York City
USA

As the Scientific Engagement Lead for Johnson & Johnson Innovation - JLABS US Northeast, Jesse Hwang is responsible for external engagement with regional academic research institutions, start-ups and investment partners and JLABS portfolio management. He also collaborates with the Head of JLABS US Northeast and Johnson & Johnson Innovation colleagues in sourcing and evaluating innovative companies with the aim to foster a productive life science ecosystem and bring value to Johnson & Johnson's pipeline. Jesse received his BA from Cornell University and PhD from Princeton University and completed post-doctoral training at Yale University.

David Kellman

Managing Director, Head of Healthcare Investment Banking at JMP Securities

David Kellman is a Managing Director and is head of healthcare investment banking at JMP Securities. He serves on the firm’s investment banking management committee.

Prior to joining JMP, David was a Vice President at Paramount BioSciences, a life sciences investment firm. Previously, he was a hedge fund analyst at S.A.C. Capital Advisors focused on healthcare investing and a member of the healthcare investment banking group at Piper Jaffray. David began his career in the healthcare group of Investor Growth Capital, the venture capital arm of Investor AB.

David holds a BS in finance from the University of Pennsylvania’s Wharton School and a BSE in bioengineering from its School of Engineering and Applied Science.

Paige Lacatena

Director, Business Development and Licensing, HQ Neurology at Merck & Co.
Philadelphia
USA

Paige is currently the S&E Neuroscience Director in Merck’s HQ office and has been with Merck since October 2012. Prior to joining BD&L, she was the Global Director Scientific Affairs for neuroscience and anesthesia, responsible for the Merck Investigator Sponsored Studies Program. In this role she also led the Scientific Leader Input Engagement (SIE) meetings for Merck’s neuroscience and anesthesia programs, supporting the end-to-end pipeline from discovery through lifecycle management by engaging with external experts and key stakeholders. Prior to this role Paige was a member of Merck’s Global Management Acceleration Program where she spent a year as an Associate Director in MSD Finance in Kriens, Switzerland responsible for regional financial planning for corporate BD deals and another year as an Associate Director Global Marketing Neurology for verubecestat, a late stage Alzheimer’s asset. Earlier in her career Paige was an Associate Principal Scientist Biology in the Discovery Neuroscience Department at Merck where she focused on target identification and validation projects. In 2013 and in 2014 Paige was recognized by Forbes magazine as one of the “30 Under 30” in Healthcare and Science. Paige earned her Ph.D. in neuroscience from Case Western Reserve University and she completed a B.S. in Biology and Biomedical Engineering from Tufts University.

Colin Macphee

Leader, Discovery Partnerships with Academia (DPAc) at GSK

Claire Marrazzo Greenwood

Executive Director at CEO Council for Growth

As the Senior Vice President of Economic Competitiveness, Claire Greenwood is responsible for advancing the work of the Chamber designed to attract and expand companies, capital, and talent in the Greater Philadelphia region. She and her team engage C-Suite leaders from Chamber members and investors in these efforts to build on and amplify the region’s competitive advantages and accelerate equitable economic growth. They drive the work of two Action Teams (Health Care and Life Sciences and Energy), a multi-platform approach to the region’s talent and workforce challenges, and the organization’s partnerships and efforts to ensure companies Select Greater Philadelphia.

Ms. Greenwood also serves as the Executive Director of the Chamber’s regional CEO Council for Growth, a committed group of nearly 60 business, higher education and civic leaders working to make Greater Philadelphia one of the global first-tier 21st-century innovation economies. Ms. Greenwood graduated from the University of Delaware with a BA in Biological Sciences and a minor in psychology and holds a Master’s of Science in Organizational Dynamics from the University of Pennsylvania. Ms. Greenwood is a member of the Board of Directors for Campus Philly and the United Way of Greater Philadelphia and Southern New Jersey.

Michelle McMurry-Heath

President & CEO at Biotechnology Innovation Organization (BIO)

Michelle McMurry-Heath assumed the leadership of the Biotechnology Innovation Organization (BIO) as President and CEO on June 1, 2020. A medical doctor and molecular immunologist by training, Dr. McMurry-Heath becomes just the third chief executive to steward the world’s largest biotechnology advocacy group since BIO’s founding in 1993.

BIO represents 1,000 life sciences companies and organizations from 30 countries. The organization’s mission is to support companies that discover and deploy scientific breakthroughs that improve human health, environmental stewardship, and sustainable agriculture.

The common thread in McMurry-Heath’s work across academia, government and industry has been her focus on broadening access to scientific progress so more patients from diverse backgrounds can benefit from cutting-edge innovation. Driven by her own past family experiences navigating clinical trials and funding uncertainties within the rare disease community, McMurry-Heath calls “the distribution of scientific progress the social justice issue of our age.”

She comes to BIO from Johnson & Johnson where she served as Global Head of Evidence Generation for Medical Device Companies and then Vice President of Global External Innovation and Global Leader for Regulatory Sciences. She was also instrumental in bringing J&J’s incubator, JLabs, to Washington, DC. She led a global team of 900 with responsibilities in 150 countries around the globe.

Prior to her time at J&J, Dr. McMurry-Heath was also a key science policy leader in government. The Obama-Biden transition team tapped her to conduct a comprehensive analysis of the National Science Foundation’s policies, programs and personnel. President Obama then named her associate science director of the FDA’s Center for Devices and Radiological Health under Commissioner Peggy Hamburg. In that role, she championed clinical trial evolution, the use of real-world evidence in product evaluation, and an embrace of the patient’s voice in health research so new medical products deliver outcomes that matter to them.

McMurry-Heath was the founding director of the Aspen Institute’s Health, Biomedical Science, and Society Policy Program, where she promoted personalized medicine and bolstered international preparation for pandemic disease threats. She received her early training in science policy from the Robert Wood Johnson Foundation and later served as Senator Joe Lieberman’s top legislative aide for science and health. In that role, she drafted legislation to protect the country from biological attacks.

McMurry-Heath received her MD/PhD from Duke’s Medical Scientist Training Program, becoming the first African-American to graduate from the prestigious program. She spent 12 years working at the research bench before taking policy and leadership roles in government and industry.

McMurry-Heath lives in Washington, D.C. with her husband Sebastian Heath, a veterinarian, and their daughter, Isabella. To relax, she enjoys yoga, snorkeling and her daughter’s sporting events.

Vered Nohi

Executive Director at US-Israel Binational Industrial R&D Foundation (BIRD)
Philadelphia
USA

Vered Nohi has been the Executive Director of the Philadelphia-Israel Chamber of Commerce (PICC) and Representative of the US-Israel Binational Industrial Research and Development (BIRD) Foundation since 2013. Her expertise is in growing bilateral trade relations in North America, Asia and Israel. The PICC is an independent, membership based non-profit, which unique mission is to connect Israeli and Greater Philadelphia, southern New Jersey and Delaware-based businesses, academic institutions, governmental agencies, and economic development organizations for innovative joint ventures. Under her leadership the BIRD Foundation awarded 17 grants of up to $ 1million each to Pennsylvania, Delaware, and southern New Jersey companies. Vered‘s career includes leadership roles at the Diamond State Port Corporation / Port of Wilmington, Delaware expanding its handling of international cargoes, and prior at the State of Israel’s Economic and Cultural Office/Mission in Taipei, Taiwan as Tourism Representative. Vered is a graduate of Hebrew University in Jerusalem in East Asian Studies and Sociology and a veteran of the Israeli Navy.

Heather Petty

Associate Director, Program Development at Xontogeny

Heather currently serves as Associate Director of Program Development where she collaborates cross-functionally to construct strategic initiatives and priorities across the portfolio. These activities span design and optimization of early preclinical through clinical development, identifying and building upon existing KOL and vendor relationships, and maintaining competitive landscapes. Heather has been with Xontogeny since it was founded in 2016, starting as an Associate with the investment team to support the build out of the company’s due diligence process before moving to portfolio-related activities in 2021.

Heather obtained her MBS degree from Keck Graduate Institute, a specialized program that focuses on translating ideas into commercial products in the biopharmaceutical industry across all modalities and therapeutic areas. She has an entrepreneurial background stemming from several years of breast cancer cell research and the development of a novel diagnostic for early detection of sepsis while acquiring her undergraduate degree at Southwestern University. She is particularly passionate about rare diseases and serves as an advocate through the Young Professional Executive Committee for The Genesis Foundation and through her athletic endeavors on American Ninja Warrior.

Ron Philip

Chief Executive Officer at Spark Therapeutics

As Spark Therapeutics Chief Executive Officer, Ron Philip leads a team of talented individuals working together to develop new therapies for people living with genetic diseases. Since joining Spark in 2017, he has played a key role in Spark’s rapid growth and achievements — most notably, directing the commercial launch of Spark’s first approved gene therapy, LUXTURNA® (voretigene neparvovec-rzyl).

Ron previously served as Chief Operating Officer, where he was responsible for leading product strategy development and execution for Spark’s early and late-stage pipeline assets, including the governance and proactive management necessary to bring more gene therapies to patients living with genetic diseases. He also was responsible for the other functions, including commercial development, marketing, pricing and reimbursement, government affairs, program and alliance management, commercial operations, diagnostics, market access and patient services functions.

Before becoming COO, Ron served as Spark’s Chief Commercial Officer and Senior Vice President, Head of Global Commercial.

With more than 30 years of experience across biopharma companies and management consulting, Ron has a proven track record of improving operating performance in multiple therapeutic categories and global markets. He held several senior leadership roles at Pfizer, including Regional President for Africa/Middle East, Head of Global Business Unit Strategy, Head of Business Development Search and Evaluation and Commercial Development Lead within Pfizer’s Innovative Products Business. He also worked at Wyeth Pharmaceuticals in U.S. commercial operations, with Deloitte Consulting in their strategy and operations practice, and at Merck and Co. in the manufacturing and marketing divisions.

Ron currently serves as the Trustee and Chair of the Science Committee at the Academy of Natural Sciences of Drexel University in Philadelphia and previously was an active board member of Cure Duchenne. Ron is a graduate of Drexel University.

Barbara Ryan

Senior Advisor, Life Sciences at Ernst & Young

Barbara is a senior advisor who works on internal projects and thought leadership. She speaks at events on life sciences, capital markets and the future outlook. She also works on the quarterly life sciences trends report.

Barbara collaborates with EY Public Relations to communicate thought leadership and provide insights on life sciences trends and key drivers and challenges.

Amanda, William and William Jr. Shirk

Patient Spotlight Family

Our journey with SMA began in Fall 2018, when my son William was about 9 months old. Up until that point, he was the most perfect happy baby who met all of his milestones right on time, a great eater, a great sleeper, rolled over and all across the room, sat up… as he got closer to his first birthday he still had no interest in crawling and would not bear any weight on his legs. Even his pediatrician chalked it up to him being a super chunky, calm, content baby who just needed a little more time to support his weight. So, we tried not to worry and hoped for progress. He began early intervention physical therapy 1x per week and we soon were incorporating PT into almost every part of his daily routine—playtime, bath time, meal time; he was constantly working those muscles, yet he wasn't really improving. In fact, we began to notice that he could not reach his arms over his head, he would fall over more frequently when in a sitting position and seemed floppy when we held him. After another visit to the pediatrician, we took him to see a pediatric neurologist and neuromuscular specialist at Children's Hospital of Philadelphia who, after a few rounds of testing, sorrowfully told us that our sweet 15-month-old little boy has a rare, serious genetic disease called Spinal Muscular Atrophy, or SMA…. But today on this diagnosis day, March 12, 2019 while our hearts are broken into a billion tiny pieces, we also have SO MUCH HOPE because NOW there is treatment… a life-saving treatment, with more just on the horizon! But time is of the essence...children with type 2 SMA, like William, may live full lives depending on the severity of symptoms. Earlier treatment means better outcomes!

Less than 4 weeks later William was lucky enough to receive the first ever treatment for SMA, Spinraza, which was administered through a series of injections into his spine. By the time he received treatment he had lost the ability to roll over or reach his arms high enough to his mouth to feed himself, but, while it sounds crazy, literally in just a few days after his first treatment he started rolling over again and over the next several weeks, slowly started gaining more and more strength. He's started to reach higher, kick harder and sit without falling over! We soon started exploring the single-dose gene therapy, Zolgensma, approved by the FDA just 2.5 months after his initial diagnosis! And in July 2019 at 19 months old he received it. Less than a month later, he was bearing weight on his legs with support and those hands that could hardly reach his mouth a few months ago he can now get up over his head. Since, he’s only gotten stronger every day. He works hard with about 2 physical therapy and 2 occupational therapy appointments every week, as well as 8-week sessions of aquatic therapy on and off, we’ve gotten the slew of equipment from new braces every few months to wheelchairs to walkers, respiratory treatment, PT benches and balls and harnesses… but this kid is determined, he always has a smile on his face. About 4 months after receiving gene therapy he was able to cruise along furniture and take small steps with a walker, about 4 months after that he started crawling and about 2 months after that on May 12, 2020, at 2.5 years old, he took his very first step on his own and has only continued to get stronger since. Today, 4 year old William rarely uses his wheelchair anymore, he is in a regular preschool classroom, navigating the room, opening his own markers, using the bathroom himself, playing on the playground and making friends! And while we still have a journey ahead of us, as many daily tasks are still very difficult for him, he is determined to be independent. Our little boy always has a smile on his face, he is so brave and his fighting spirit will let nothing stop him.

Much of this is owed to incredible families before us and amazing research organizations like CureSMA who have made strides in finding an end to the monster that SMA is without early detection and early treatment. We have an amazing team of doctors and therapists at CHOP, as well as therapists through the Montgomery County Intermediate Unit who follow him closely and work with him to help him continually make immense improvements. Because of them, my family never had to hear the words, "take him home and love him" or "there's nothing we can do." Instead, we had hope from the day we received William’s diagnosis, my son quickly received treatment that halted his progressive muscle weakness, we heard the words “he will walk one day”, and today my son walked all the way downstairs from his bedroom to our kitchen to grab a snack on his own.

Sri Sriadibhatla, PhD

Investment Director, Healthcare Investment Group (HIG) at Ben Franklin Technology Partners of Southeastern PA

Sri Sriadibhatla is Investment Director, Healthcare Investment Group (HIG) at Ben Franklin Technology Partners of SEP. Sri works closely with the Managing Director, and is responsible for sourcing, diligence, negotiating, and managing investments in early-stage companies in the healthcare sector. He has extensive experience in the drug discovery & development, formulations, regulatory, and commercialization process.

Prior to joining Ben Franklin, Sri worked as Technology Scout at FMC Corporation and led initiatives in developing, adapting, streamlining, and executing an Open Innovation model. He has over 14 years of experience in technology evaluation, licensing, investment and commercialization. Sri is a registered pharmacist and holds a PhD in Pharmaceutical Sciences (Center for Drug Delivery and Nanomedicine) from the University of Nebraska Medical Center.

Erika Swift

President, BioStrategy Partners at Pennsylvania State University

Dr. Ron Thiboutot

Executive Vice President at Life Sciences Greenhouse of Central Pennsylvania

Ronald P. Thiboutot, Ph.D., is Executive Vice President of the Life Sciences Greenhouse of Central Pennsylvania. His responsibilities include identifying promising new areas of technology, reviewing science and business opportunities, and investing in early stage opportunities. Dr. Thiboutot oversees technical due diligence of the funded companies and provides technical and business mentoring to funded applicants. Dr. Thiboutot sits on four corporate and one university board and provides interim CEO services for selected start-ups as required.

Prior to joining the Life Sciences Greenhouse, Dr. Thiboutot was president of RT Consultants, Inc. which provided technical, equipment procurement, and facility design consulting services to the US and EU pharmaceutical industry. Among his other employers were Wyeth Pharmaceuticals, where he was the plant director of the vaccine manufacturing facility located in Marietta, PA; Bristol-Myers Squibb, where he worked in the international technology transfer division; and Baxter Travenol. He currently serves as CEO of Pharmateck, Inc.

Dr. Thiboutot holds a Bachelor of Science, Master of Science, and Ph.D. from the Massachusetts College of Pharmacy and has been active in numerous U.S. and international pharmaceutical trade organizations during his 25 years in the pharmaceutical industry.

Mark Tornetta

Vice President of Biologics Discovery at Tavotek Biotherapeutics
Lower Gwynedd
USA

Mr. Mark Tornetta is Vice President of Biologics Discovery at Tavotek Biotherapeutics. He has over 29 years of discovery biopharmaceutical experience, 23 years in antibody engineering and 6 years in target discovery working on orphan GPCRs. His biopharma activities involved targets in cardiovascular, pulmonary, metabolic, immune, oncological, and infectious diseases. His antibody engineering career started at SmithKline Beecham/GSK. After implementing the pCombIII system from Scripps Research Institute, he made his first contribution to phage display by developing a new selection technique called “epitope blocked panning”. This was essential for the discovery of a neutralizing anti-RSV antibody. He moved on to the exploration of de-orphanizing GPCRs where he played a key role in discovering the ligand and future MOAs for the C3aR. During this time, he gained experience as a scientist and project management skills by driving small molecule candidate evaluation from high throughput in vitro cellular assays through to ex vivo modeling assays. He then returned to antibody engineering at Centocor/Janssen Pharmaceuticals of Johnson & Johnson (J&J). He was a key contributor in implementing two phage display platforms. The first was the Morphosys HuCAL system where he performed therapeutic based experiments and developed new selection strategies that routinely generated candidates with sub-nM binding affinities. He also managed Morphosys AG to generate candidates for Centocor research teams and delivered on every target. The second technology was the Fab-pIX display system. He proved that a Fab protein can be displayed on phage by pIX fusion. This enabled the construction of a large Fab library. Over 6 years, he and a team of 5 scientists produced 8000 antibodies to more than 200 targets. His creativity in panning techniques led to the discovery of an antibody currently in Phase II development at GSK. He has expertise in cell surface panning which produced many antibodies to such targets like GPCRs. He also developed complimentary methods such as high throughput panning, micro-scale bacterial expression-purification, and high throughput ranking kinetics. Mr. Tornetta received his Bachelor of Science in biology from Widener University and a Master of Science degree in Biotechnology at Drexel University. He is an inventor on 19 patents, an author on 16 manuscripts and chaired as well as presented at multiple scientific conferences. He has received numerous awards and most notably 5 Leadership Innovation awards while at J&J.

Steve Tullman

Executive Chairman at Nexstone

Steve’s career in life sciences spans more than 30 years and includes numerous Chairman, CEO, and founding credits, with the creation of ten biotechnology companies. He currently serves as the Managing Member of Nexstone Innovation and Chairman of NexEos Bio. Steve co-founded NeXeption, Inc. in 2011 and NeXeption, LLC in 2012 to focus on building biotech organizations designed to identify and advance programs with significant unmet medical needs. His previous chairmanships include serving Onspira Therapeutics, Inc. (acquired by Altavant Sciences / Sumitomo Dainippon Pharma in 2019), Aclaris Therapeutics, Inc. (NASDAQ: ACRS), Ralexar Therapeutics, Inc., and Vicept Therapeutics, Inc. until its acquisition by Allergan, Inc. He also served as Chairman, President and CEO of Ceptaris Therapeutics, Inc. until its acquisition by Actelion in 2013. He created Ception Therapeutics, Inc. in 2005 and served as its President and CEO until its acquisition by Cephalon, Inc. in 2010.

Dominic Warrino, PhD

Senior Scientific Adviser at KCAS

Dominic Warrino, PhD, is a Senior Scientific Advisor. His role is to serve as scientific and technical advisor for both clients and internal teams for development, validation, and application of bioanalytical, immunogenicity, and biomarker methods for large molecule therapeutics. Dominic joined the company in 2013 and brought with him expertise in a full range of bioanalytical techniques including ECL, ELISA, RIA, Flow Cytometry, ELISpot, cell-based assays and Luminex. He has 20 years of experience developing and validating immunological assays for biotechnology and pharmaceutical companies. He most recently spent several years in the Biopharma Services department at Viracor-IBT (formally IBT), and previously also worked for Cytogen (formally Cellcor), Eligix, and Streck Laboratories developing novel compounds for treatment of various cancers. Dominic has worked on over 100 large molecule compounds developing and validating PK, PD, ADA, and Biomarker assays in support of pre-clinical to phase IV testing.

Michael Weingarten

Director, SBIR Development Center at National Cancer Institute

Michael Weingarten is the Director for the Small Business Innovation Research (SBIR) Development Center at the National Cancer Institute in Bethesda, MD. In this role, Mr. Weingarten leads a team of nine Program Directors who manage all aspects of the NCI SBIR & STTR Programs including a portfolio of $182M in grants and contracts annually. The SBIR & STTR programs are NCI's engine of innovation for developing and commercializing novel technologies and products to prevent, diagnose, and treat cancer. Mr. Weingarten has implemented a set of key initiatives for optimizing the performance of the NCI SBIR Program at the NIH. These include the establishment of a new model at the NCI for managing the program - the SBIR Development Center.

Under Mr. Weingarten's leadership, the NCI SBIR Development Center has launched a range of new initiatives to facilitate the success of small businesses developing cancer-related technologies. Recent initiatives include the launch of the NIH I-Corps™ pilot program in which teams of budding entrepreneurs engage in a hypothesis-driven approach to validate their proposed business models by conducting over 100 interviews with potential customers. Companies adjust their strategies based on direct customer feedback and analyze the information they collect to determine if there is a product/market fit. Other NCI SBIR initiatives introduced under Mr. Weingarten's leadership include the NCI SBIR Investor Forums, the NCI SBIR Phase II Bridge Award, and the workshop titled Federal Resources to Accelerate Commercialization (FRAC). Thus far, NCI SBIR has held three investor forums that in total have facilitated the closing of investment deals with NCI-funded SBIR companies valued at over $300M. The NCI SBIR Phase II Bridge Award, which was launched in 2009, incentivizes partnerships between NIH's SBIR Phase II awardees and third-party investors and/or strategic partners to help small businesses bridge the funding gap between the end of their SBIR Phase II awards and the next round of financing needed to advance a promising cancer therapy or imaging technology.

Travis Whitfill

Partner at Bios Partners

Travis Whitfill is a Partner at Bios Partners and serves as the Chief Science Officer of Azitra Inc. His background began in molecular biology and biochemistry after receiving scientific training at the MD Anderson Cancer Center and Duke University. He is the co-founder of several startup companies, including Azitra Inc. and several biotech and healthcare startups. He brings strong background in entrepreneurship and business and was recently acknowledged as Forbes’ 30 Under 30 in 2018. He’s also the Senior Analyst at Bios Research, which brings experience in public markets, and drug development to support its venture capital investments. He also is an Associate Research Scientist in the Departments of Pediatrics and Emergency Medicine at Yale University. Mr. Whitfill has led numerous grant-funded projects, holds several patents, and has co-authored over three dozen publications. Mr. Whitfill received degrees from Yale University (MPH) and Dallas Baptist University (BS) and is working on a PhD from University College London.

USA

David Kellman

Managing Director, Head of Healthcare Investment Banking

JMP Securities

Paige Lacatena

Director, Business Development and Licensing, HQ Neurology

Merck & Co.

USA

Person Name	Job Title	Organization
Greg Aronin	Vice President, External Affairs	Paragon Biosciences
Jamie Bauersmith	Vice President, Translational Science	UPMC Enterprises
Julie Bick, PhD	Chief Scientific Officer	FlowMetric a KCAS Company
Joe Colletti	Executive Vice President	JLL
Jonathan Epstein, MD	William Wikoff Smith Professor, Executive Vice Dean and Chief Scientific Officer, Perelman School of Medicine at the University of Pennsylvania, Senior Vice President and Chief Scientific Officer, Pe	University of Pennsylvania
Matt Fenske	Vice President, Lifecycle & Portfolio Strategy	Harmony Biosciences
Sarah Fuller	Vice President, North America Business Development & Alliance Management	Kyowa Kirin
Nicholas Galakatos	Senior Managing Director	Blackstone
Lori Gavrin	Chief Business & Strategy Officer	NeuExcell
John Grady	Northeast Region Executive and Senior Vice President, Development	Wexford Science & Technology
Brianna Gross	Genetics Counselor	Children's Hospital of Philadelphia
Abhi Gupta	Senior Director, Global Gene Therapy Business	Pfizer Biopharma Group
Shahram Hejazi, PhD	Managing Director and CEO	BioAdvance
Gerri Henwood	President & CEO	Baudax Bio
Jesse Hwang	Scientific Engagement Lead	Johnson & Johnson Innovation - JLABS US Northeast
David Kellman	Managing Director, Head of Healthcare Investment Banking	JMP Securities
Paige Lacatena	Director, Business Development and Licensing, HQ Neurology	Merck & Co.
Colin Macphee	Leader, Discovery Partnerships with Academia (DPAc)	GSK
Claire Marrazzo Greenwood	Executive Director	CEO Council for Growth
Michelle McMurry-Heath	President & CEO	Biotechnology Innovation Organization (BIO)
Vered Nohi	Executive Director	US-Israel Binational Industrial R&D Foundation (BIRD)
Heather Petty	Associate Director, Program Development	Xontogeny
Ron Philip	Chief Executive Officer	Spark Therapeutics
Barbara Ryan	Senior Advisor, Life Sciences	Ernst & Young
Amanda, William and William Jr. Shirk		Patient Spotlight Family
Sri Sriadibhatla, PhD	Investment Director, Healthcare Investment Group (HIG)	Ben Franklin Technology Partners of Southeastern PA
Erika Swift	President, BioStrategy Partners	Pennsylvania State University
Dr. Ron Thiboutot	Executive Vice President	Life Sciences Greenhouse of Central Pennsylvania
Mark Tornetta	Vice President of Biologics Discovery	Tavotek Biotherapeutics
Steve Tullman	Executive Chairman	Nexstone
Dominic Warrino, PhD	Senior Scientific Adviser	KCAS
Michael Weingarten	Director, SBIR Development Center	National Cancer Institute
Travis Whitfill	Partner	Bios Partners