N-LOREM FOUNDATION - Key Persons


Alan Beggs

Job Titles:
  • Professor of Pediatrics at Boston Children 's Hospital

Alfred Sandrock, Jr

Job Titles:
  • Executive Vice President, Research & Development and Chief Medical Officer at Biogen

Allison Bateman-House

Job Titles:
  • Assistant Professor of Population Health at NYU Grossman School of Medicine

Amy C. Williford

Job Titles:
  • Senior Director
  • Director of Communications and Donor Relations
  • Senior Director of Communications and Donor Relations at N - Lorem
Dr. Amy Williford is the senior director of communications and donor relations at n-Lorem Foundation. Amy is a seasoned communications professional who is well versed in translating complex scientific concepts and ideas into concise and easy-to-understand concepts. A scientist by training, Amy supported multiple high-profile communications programs as the associate director of corporate communications at Ionis Pharmaceuticals. She also managed and built relationships with pharmaceutical partners, institutional investors and private shareholders. Prior to joining Ionis, Amy worked at a public relations agency, where she managed and led communications for a range of companies across the biotechnology industry, and built long-standing relationships with key media, investors and analysts. Amy received her Ph.D. in chemistry from The Scripps Research Institute, where she studied the selective binding of transcription factors to DNA.

Amy McCooe

Job Titles:
  • Executive Director of the Wolverine Foundation
Amy McCooe is the executive director of The Wolverine Foundation. Amy provides operational leadership for key partners in the academic research and patient communities that are seeking and/or developing treatments for the MAPK8IP3-Related Neurodevelopmental Disorder. Previously, Amy successfully grew an online STEM ED tech company from theory to curriculum implementation in over 1,500 schools, reaching 60,000+ students and successfully landed as a business unit within a major player in the education market. She brings over 10 years of experience in building high-performance teams with the ability to motivate, influence and lead multiple groups to achieve exceptional results. Amy holds an M.B.A. from the University of Texas at Austin and a B.A. in history from Trinity College-Hartford.

C. Frank Bennett - CTO

Job Titles:
  • Chief Technical Officer
  • Member of the Board of Directors
  • Chief Technical Officer of N - Lorem Foundation
  • Co - Chairman - Chief Technology Officer at N - Lorem and Chief Scientific Officer at Ionis Pharmaceuticals
Dr. Frank Bennett is the chief technical officer of n-Lorem Foundation. He also is a founding member of Ionis Pharmaceuticals and serves as the company's chief scientific officer. He is responsible for preclinical antisense drug discovery and antisense technology research. Dr. Bennett is also the franchise leader for neurological programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry. Dr. Bennett is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen) and the 2018 Hereditary Disease Foundation's (HDF) Leslie Gehry Brenner Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for Huntington's disease (HD). Dr. Bennett has published more than 200 papers in the field of antisense research and development and he is an inventor on more than 175 issued patents. Prior to joining Ionis, Dr. Bennett was associate senior investigator in the Department of Molecular Pharmacology at SmithKline and French Laboratories, currently, GlaxoSmithKline. He received his Ph.D. in Pharmacology from Baylor College of Medicine, Houston, Texas and his B.S. degree in Pharmacy from the University of New Mexico, Albuquerque, New Mexico. He performed his postdoctoral research in the Department of Molecular Pharmacology at SmithKline and French Laboratories. Dr. Bennett serves on the Advisory Board for the Experimental Therapeutics Centre in Singapore and the Hereditary Disease Foundation.

Cullen Chair

Job Titles:
  • Professor

Dr. Cedrik Ngongang

Job Titles:
  • Senior Director
  • Director, Medical Geneticist
  • Senior Director, Medical Geneticist at N - Lorem
Dr. Cedrik Ngongang is the senior director medical geneticist at n-Lorem Foundation. Prior to joining n-Lorem, Dr. Ngongang was a scientist at the National Human Genome Research Institute, National Institutes of Health. Dr. Ngongang's clinical and research experience in genetic medicine spans various aspects of the diagnostics and management of rare disorders, including clinical assessment of genomic variants, elucidation of the genetic basis of Mendelian phenotypes in individuals from ancestrally diverse backgrounds, and application of machine learning approaches to the clinical and genomic diagnosis. He received his M.D. from the University of Yaoundé 1 Faculty of Medicine, and his Master of Medicine in medical genetics from the University of Cape Town. He completed his postdoctoral training in molecular genetics at the National Institutes of Health, following his board-certification in clinical genetics in South Africa.

Dr. Jeff Carroll

Job Titles:
  • Scientific Advisor
  • Scientific Advisor for N - Lorem
Dr. Jeff Carroll is a scientific advisor for n-Lorem. Jeff began assisting n-Lorem's access to treatment committee at a very early stage, given his passion for helping families with rare genetic diseases. This passion emerges from a personal connection - Jeff's mother died after suffering from Huntington's Disease, placing her 6 children at 50% risk of developing the disease. In 2003, Jeff learned that he inherited this mutation from his mother, sparking a passion to help understand the disease, and to help hasten the development of novel treatments for all HD patients. Jeff is now an associate professor in the Department of Neurology at the University of Washington. Jeff's primary scientific focus is on conducting translational studies in support of "Huntingtin Lowering" approaches to Huntington's Disease, primarily through the use of antisense oligonucleotides. Jeff's Ph.D. and postdoctoral studies all involved using mouse models to understand basic mechanisms of HD and preclinical testing of therapeutic interventions in these models. During his Ph.D., he trained with Michael Hayden (UBC) and his postdoctoral studies were conducted under the supervision of Marcy MacDonald (MGH, Harvard Medical School).

Dr. Joseph Loscalzo

Job Titles:
  • Member of the Executive Committee
  • Professor of the Theory
  • Senior Physician & Head of the Department of Medicine
Dr. Joseph Loscalzo is Hersey Professor of the Theory and Practice of Medicine at Harvard Medical School, chairman of the Department of Medicine, and physician-in-chief at Brigham and Women's Hospital. Dr. Loscalzo received his A.B. degree, summa cum laude, his Ph.D. in biochemistry, and his M.D. from the University of Pennsylvania. His clinical training was completed at Brigham and Women's Hospital and Harvard Medical School, where he served as Resident and Chief Resident in medicine and Fellow in cardiovascular medicine. Post-training, Dr. Loscalzo joined the Harvard faculty and staff at Brigham and Women's Hospital in 1984. He rose to the rank of associate professor of Medicine, Chief of Cardiology at the West Roxbury Veterans Administration Medical Center, and director of the Center for Research in Thrombolysis at Brigham and Women's Hospital. He joined the faculty of Boston University in 1994, first as Chief of Cardiology and, in 1997, Wade Professor and Chair of Medicine, Professor of Biochemistry, and Director of the Whitaker Cardiovascular Institute. He returned to Harvard and Brigham and Women's Hospital in 2005. He currently serves on the board of directors of Leap Therapeutics, Inc., a publicly held biopharmaceutical company, and is an executive committee member of the UDN.

Dr. Julie Douville

Job Titles:
  • Executive Director of ASO Discovery and Development at N - Lorem Foundation
  • Executive Director, ASO Discovery and Development
  • Executive Director, ASO Discovery and Development at N - Lorem
Dr. Julie Douville is the executive director of ASO discovery and development at n-Lorem Foundation. She previously served as senior director of preclinical development. Prior to n-Lorem, Julie was at Charles River Laboratories from 2007 to 2022 as the director of infusion, parenteral administration and neurotoxicology, where she specialized in cerebrospinal administration, and in the development of antisense oligonucleotides, gene therapies and other therapeutic entities. In this role, Julie oversaw several IND-supporting programs for indications including ALS, Alzheimer's Disease, Parkinson's, Angelman's Syndrome, Duchenne's Muscular Dystrophy, and many others. Working with academic investigators, she personally oversaw the toxicology program for milasen, the first personalized ASO treatment that was customized for a mutation in a single patient, which was approved for clinical use by the FDA in 2018. Julie led several additional n=1 preclinical programs, in addition to being involved on many other rare disease programs for which she developed strategies for acceleration of the preclinical phase. She has taken part in key discussions with regulatory authorities in the FDA and EMA, helping to establish precedents for the U.S. guidelines around individualized antisense oligonucleotide treatment. Julie carries a strong background in GLP preclinical development, and in-depth knowledge of ASO-associated toxicities, especially in the central nervous system. Julie received her Ph.D. in Microbiology and Immunology from the University of Montreal.

Dr. Konstantina Skourti-Stathaki

Job Titles:
  • Director of ASO Discovery and Design at N - Lorem Foundation
Dr. Konstantina Skourti-Stathaki is the Director of ASO Discovery and Design at n-Lorem Foundation. Konstantina has a strong passion for RNA research and her drive and mission is to utilize it to save people's lives. Prior to n-Lorem, Konstantina led the RNA Biology and Technology team at MiNA Therapeutics. In this role, she specialized and oversaw pre-clinical RNA Therapeutics drug discovery, specifically target identification and validation, oligonucleotide design and targeting, and mechanism of action studies. She led the strategic planning of new targets and shaped key early decisions on the drug discovery process of rare disease targets. Konstantina also co-led programs from drug discovery to IND filing. Prior to her industry position, Konstantina spent 15 years in academia and her expertise is on RNA biology and biochemistry, transcription and epigenetics. Konstantina did her Ph.D. at the University of Oxford where she made the pioneering discovery that naturally formed RNA/DNA hybrids control transcription and chromatin in mammalian cells. She was then awarded a prestigious Sir Henry Wellcome independent post-doctoral fellowship which she spent at the University of Edinburgh, UK and the Max Delbruck Centre for Molecular Medicine in Berlin, Germany, where she pioneered how RNA structures affect stem cell homeostasis and DNA damage repair. Konstantina has a strong track record of innovative research including numerous high impact factor peer-reviewed publications and patent filings.

Dr. Laurence Mignon

Job Titles:
  • Director of Clinical Development
  • Senior Director of Clinical Development at N - Lorem Foundation
Dr. Laurence Mignon is the senior director of clinical development at n-Lorem Foundation. Laurence has more than 20 years of experience as a seasoned academic researcher and clinical developer with a passion for rare diseases. Most recently, Laurence worked at Ionis Pharmaceuticals both in the Translational Medicine group and the Neurology Franchise Clinical Development group. In those roles, she implemented a novel genomic study to better understand genotype-phenotype associations and disease pathways, initiated the first clinical study in myotonic dystrophy type 1 and was a key member of the clinical team that brought SPINRAZA® (nusinersen), a disease-modifying therapy for patients with spinal muscular atrophy, to the market. Prior to Ionis, Laurence worked at Orexigen Therapeutics on the development of an obesity medication and at the Neuroscience Education Institute developing Continuing Medical Education material aimed at deconstructing the mechanism of action of psychiatric medications for psychiatrists, primary care physicians, and registered nurses. Laurence's broad and diverse experience allows her to be strategically creative in her approach to deal with uncharted challenges. Laurence received her PhD in Pharmacology and Experimental Therapeutics from Loyola University Chicago and was a researcher for 6 years in the Department of Neurology at UCLA in the laboratory of Marie-Françoise Chesselet, studying properties of muscle-derived stem cells and the role of the serotonin pathway in Parkinson's disease.

Dr. Michael Hayden

Job Titles:
  • Killam Professor of Medical Genetics at University of British Columbia
Dr. Michael Hayden is a Killam Professor at the University of British Columbia and the director of the Translational Laboratory in Genetic Medicine at the National University of Singapore and the Agency for Science, Technology and Research (A*STAR). From 2012 to 2017, he served as president of Global R&D and chief scientific officer at Teva Pharmaceutical Industries Ltd. Dr. Hayden built and transformed research and development at Teva to become a highly productive research engine and was instrumental in the approval of approximately 30 new products. Dr. Hayden has founded three biotechnology companies and has been the recipient of numerous prestigious honors and awards including being inducted into the Canadian Medical Hall of Fame, receiving the July 2012 Diamond Jubilee Medal, on behalf of HRH Queen Elisabeth II and the Margolese National Brain Disorder Prize, awarded to Canadians who have made outstanding contributions to the treatment, amelioration, or cure of brain diseases. He's also received the Canada Gairdner Wightman award for his outstanding leadership in medicine and medical science as a physician-scientist. Dr. Hayden was awarded the Order of Canada, the Order of British Columbia, named Canada's Health Researcher of the Year by Canadian Institutes of Health Research, and has received the Prix Galien for his contribution to Canadian pharmaceutical research. Most recently, Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world.

Dr. Neil Shneider

Job Titles:
  • Associate Professor of Motor Neuron Disorders
  • Associate Professor of Neurology in the Neuromuscular Division at Columbia University, Vagelos College of Physicians and Surgeons
  • Claire Tow Associate Professor of Motor Neuron Disorders and Director of the Eleanor and Lou Gehrig ALS Center at Columbia University
Dr. Neil Shneider serves as the Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University. He is an investigator in the Center for Motor Neuron Biology and Disease where his lab focuses on the study of models and mechanisms of ALS and the discovery and development of novel therapeutics for ALS and related disorders. Dr. Shneider worked with Ionis Pharmaceuticals to develop ION363 (Jacifusen), an anti-sense oligonucleotide (ASO) for ALS patients with rare mutations in the FUsed in Sarcoma (FUS) gene. Dr. Shneider is a graduate of Harvard College and earned his M.D. and Ph.D. degrees at the Columbia University College of Physicians and Surgeons. In partnership with n-Lorem and Columbia University, Dr. Shneider founded Silence ALS, an initiative to develop ASOs for ALS patients with nano-rare, pathogenic mutations in ALS genes. Dr. Shneider was co-chair of the Translating Fundamental Research into Potential ALS Therapies Working Group for the NIH ALS Strategic Planning Workshop.

Dr. Rosanne M. Crooke

Job Titles:
  • Co - Founder of N - Lorem
  • Founder of N - Lorem
Dr. Rosanne M. Crooke is co-founder of n-Lorem and serves on the Board of Directors. Dr. Crooke received a B.A. degree in Biology at Williams College and Ph.D. in Pharmacology at the University of Pennsylvania. She was the vice president of Cardiovascular Research at Ionis Pharmaceuticals and one of the founding members of the company. At Ionis, she was responsible for leading the cardiovascular, metabolic, and renal research programs, where she successfully helped identify and advance multiple first-in-class drugs, two of which have already reached the market. She contributed significantly to a broad understanding of ASOs through her extensive work characterizing the in vitro and in vivo toxicological and pharmacokinetic parameters of antisense agents. She authored more than 110 primary research manuscripts, reviews and book chapters, and is an inventor on more than 67 issued patents. She also served on the board of the Deuel Lipid Conference.

Dr. Sarah Glass - COO

Job Titles:
  • Chief Operating Officer
  • Chief Operating Officer at N - Lorem
  • Chief Operating Officer of N - Lorem Foundation
Dr. Sarah Glass is the chief operating officer of n-Lorem Foundation. Sarah received her Ph.D. in Molecular Genetics at Ohio State University where she trained in rare inherited cancer syndromes. She has over 20 years of experience in clinical development and research across academia, pharmaceutical companies, and CROs. Sarah brings significant strengths and experience as an accomplished research geneticist, rare disease drug developer, and clinical trialist. She is acclaimed for forging key strategic partnerships across rare disease sectors and has driven efficiencies to decrease patient/caregiver burden in clinical research. Most notably, Sarah combines her professional expertise and training with the perspective of a parent of a child with a nano-rare disease. This allows Sarah to not only personally understand the challenges faced by n-Lorem patients and their families, but also to translate this understanding into n-Lorem's paradigm shifting platform solution for the nano-rare community. Outside of n-Lorem, Sarah volunteers as chair of the DYRK1A Medical and Scientific advisory board and is engaged in many rare disease organizations to ensure collaboration in reaching common goals.

Francis Sessions Cole, III

Job Titles:
  • Professor of Pediatrics at Washington University School of Medicine in St. Louis

Frank J. Sasinowski

Job Titles:
  • Director Hyman, Phelps & McNamara, P.C
  • Is Director of Hyman
Frank J. Sasinowski is director of Hyman, Phelps & McNamara, P.C., and an Adjunct Professor of Neurology at the University of Rochester School of Medicine. He brings more than 20 years of board experience at prestigious institutions, including the Alliance for Regenerative Medicine (ARM) Foundation for Cell and Gene Medicine, National Organization for Rare Disorders (NORD), EveryLife Foundation for Rare Diseases, the IndoUSrare patient organization, the United States Pharmacopeia (USP) and various notable biotechnology companies. After joining the U.S. Food and Drug Administration (FDA) in 1983 as regulatory counsel in the Center for Drugs and Biologics, Sasinowski became a key player in the implementation of foundational legislation such as the 1983 Orphan Drug Act and 1984 Hatch-Waxman Act. He worked with the FDA for nearly half a decade before leaving his position as the Deputy Director of health policy in the Commissioner's office and joining Hyman, Phelps & McNamara, P.C. Sasinowski used his experience working in the drug regulatory process to help secure FDA approval for hundreds of new drugs, including more than 100 new molecular entities - often for serious and rare diseases. Sasinowski has been widely recognized for his expertise by the life sciences sector, patient advocacy organizations and notable world leaders, including former President Barack Obama, who recognized his contributions to the President's Council of Advisors on Science and Technology (PCAST) report in 2012. He is the recipient of numerous awards, including NORD's first-ever Lifetime Achievement Award, as well as the FDA's Award of Merit and NORD's Humanitarian Award, where he was honored for his work benefiting the rare disease community. Most recently, Sasinowski received Pope Francis' 2021 Pontifical Hero Award for Inspiration as recognition for his leadership, advocacy and unwavering dedication to patients living with rare disease.

Gene Yeo

Job Titles:
  • Professor of Cellular and Molecular Medicine at the University of California
Gene Yeo is a Professor of Cellular and Molecular Medicine at the University of California, San Diego. Dr. Yeo has a B.S. in Chemical Engineering and a B.A. in Economics from the University of Illinois, Urbana-Champaign, a Ph.D. in Computational Neuroscience from Massachusetts Institute of Technology and an M.B.A. from the UC San Diego Rady School of Management. His primary research interest is in understanding the importance of RNA processing and the roles that RNA binding proteins (RBPs) play in development and disease. Dr. Yeo has authored more than 200 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. Gene is on the Editorial Boards of the journals Cell Reports, Cell Research and eLife, and on the Advisory Board of Review commons. Gene is also a Paul Allen Distinguished Investigator (2020) and received the 2021 Elisa Izaurralde Award for Innovation in Research, Teaching and Service from the RNA Society. Gene is a co-founder of biotech companies which includes Locanabio, Eclipse Bioinnovations, Proteona, CircBio and Trotana Therapeutics. Gene serves or had served on the scientific advisory boards of the Allen Institute of Immunology, Locanabio, Eclipse Bioinnovations, Proteona, Aquinnah, Cell Applications, Tecan, LGC, Nooma, Insitro and Ribometrix. Gene is a senior advisor to Accelerator Life Sciences Partners.

Jason Wood

Job Titles:
  • Founder and CEO of J. Wood Capital Advisors
Jason Wood is founder and CEO of J. Wood Capital Advisors, an advisory firm and registered broker dealer focused on providing capital structure advice and solutions to corporate clients. Previously, Jason worked for nearly 20 years at J. P. Morgan, where he was Head of Equity-Linked Capital Markets for the Americas and a member of the Equity Commitments Committee. Jason graduated from Princeton University cum laude with a B.A. in Economics.

Joseph G. Gleeson - Chairman

Job Titles:
  • Chairman
  • Chief Medical Officer
  • Chief Medical Officer at N - Lorem and Rady Professor, Neurosciences and Pediatrics, University of California, San Diego
  • Chief Medical Officer of N - Lorem Foundation
Dr. Joe Gleeson is the Chief Medical Officer of n-Lorem Foundation. Joe graduated from the University of Chicago Pritzker School of Medicine, then trained in pediatrics, neurology and neurogenetics at Children's Hospital, Boston and Harvard Medical School. He has been a faculty member at University of California San Diego School of Medicine and an attending physician at Rady Children's Hospital San Diego since 1999. From 2008-2019, he was an investigator with the Howard Hughes Medical Institute. From 2014-2015, he was the Hess Professor and head of the Laboratory for Pediatric Brain Disease at Rockefeller University, and director of mendelian sequencing at the New York Genome Center. Since 2015, he has held the Rady Children's Hospital auxiliary endowed professorship of neuroscience and served as director of neuroscience research at the Rady Children's Institute for Genomic Medicine. He is an investigator with the Simons Foundation for Autism Research Initiative, and an elected member of the National Academy of Medicine.

Kim Butler - COO

Job Titles:
  • Administrative Assistant
  • Director of Operations
  • Senior Director of Operations at N - Lorem Foundation
Kim Butler is the senior director of operations at n-Lorem Foundation. Kim is a broadly experienced administrative assistant who has been employed by Ionis Pharmaceuticals since 2014 . During her tenure with Ionis she has been promoted several times and has assumed substantial managerial responsibilities. Additionally, she is accountable for leading the training of all administrative personnel and leads the management of administrative activities for Ionis. Currently, Kim is the senior administrative assistant to the Chairman and CEO of n-Lorem and has played an instrumental role in the formation of the foundation. Prior to joining Ionis, Ms. Butler served in senior administrative positions in several organizations and has help found and participated in a number of charitable efforts. She is active in a wide range of civic service groups such as the American Legion and a frequent volunteer committed to serving the needy empathetically.

Lauren Black

Job Titles:
  • Distinguished Scientist at Charles River Lab

Liz Berry Kravis - Chairman

Job Titles:
  • Chairman
  • External Physician and Patient Subject Matter Experts

Lynne Parshall

Job Titles:
  • Board Member of N - Lorem Foundation
  • Board of Directors
Ms. Parshall has been a board member of n-Lorem Foundation since its inception. She is Chair of the Audit Committee. Ms. Parshall is a seasoned biotechnology executive with a focus on finance and strategy. She received her B.A. from Harvard University, and her J.D. from Stanford University. Ms. Parshall has served as a Director of lonis Pharmaceuticals since September 2000 and previously was an executive officer and then Senior Strategic Advisor to lonis Pharmaceuticals from 1991 to 2022. Prior to joining lonis, Ms. Parshall practiced law at Cooley LLP, where she was a partner from 1986 to 1991. Ms. Parshall serves on the board of directors of Cytokinetcs Inc. and Foghorn Therapeutics, both public biopharmaceutical companies and numerous private biotechnology companies.

Matthew Might

Job Titles:
  • Director at Hugh Kaul Precision Medicine Institute at University of Alabama at Birmingham

Megan Knutsen

Job Titles:
  • Director, Foundation and Program Management

Paul Compton

Job Titles:
  • Member of the Board of Directors
  • Global Head of Corporate & Investment Bank, and President of Barclays Bank PLC
  • Global Head of the Corporate
Paul Compton serves as the Global Head of the Corporate and Investment Bank, and President of Barclays Bank PLC (BBPLC), and is a member of the Group Executive Committee of Barclays. Paul leads Barclay's provision of funding, financing, strategic and risk management for money managers, corporate clients, financial institutions, governments and supranational organizations. Before joining Barclays in 2016, Paul served for nearly three decades in a variety of senior roles at JPMorgan Chase and Ernst & Young. Mr. Paul Compton joins n-Lorem with a wealth of finance and investment expertise, with more than three decades of experience helping companies raise capital. Paul holds a Bachelor of Commerce and a Bachelor of Economics from the University of Queensland, Australia. He received post-graduate degrees from the Institute of Chartered Accountants in Australia and the Securities Institute of Australia.

Richard Finkel

Job Titles:
  • Director, Experimental Neuroscience Program, and Endowed Chair in Neurotherapeutics at St. Jude Children 's Research Hospital

Sidney Carter

Job Titles:
  • Professor
Sidney Carter Professor of Neurology, Professor of Pediatrics, and Director Emeritus (1979-2000) of the Child Neurology Service at Columbia University Irving Medical Center, New York City

Stanley T. Crooke - CEO, Chairman, Founder

Job Titles:
  • Chairman of the Board
  • Chief Executive Officer
  • Founder
  • Founder, Chairman and Chief Executive Officer of N - Lorem
Dr. Stanley Crooke is founder, chairman and chief executive officer of n-Lorem, a nonprofit foundation focused on providing treatments for patients with nano-rare disease patients (1 to 30 patients worldwide), which he initiated in January 2020. Prior to n- Lorem, Dr. Crooke founded and was Chairman and Chief Executive Officer and Lead Scientist of Ionis Pharmaceuticals. During his tenure at Ionis, he led the scientific development of a new platform for drug discovery, antisense technology and the creation of one of the largest and more advanced development pipelines in the biotechnology industry, and commercialized several antisense drugs including, SPINRAZA® (nusinersen), TEGSEDI™ (inotersen) and others. Early in Dr. Crooke's career, he led the creation of the first broad anticancer program in the industry at Bristol-Myers, bringing numerous anticancer drugs to the market in the first five years of his career. He then assumed responsibility for worldwide R&D (president) at SmithKline Beckman (now GSK). During his tenure at SKB, Dr. Crooke led the restructuring of R&D and the development of several drugs that were commercialized. Dr. Crooke has also contemporaneously led a successful academic career becoming a full professor at Baylor College of Medicine and the University of Pennsylvania Medical School where he trained a number of Ph.D. students and won several teaching awards. Dr. Crooke has been an active scientist throughout his career as well. Dr. Crooke has received a number of awards, most recently, the Indiana University School of Medicine Steven C. Beering Award, the Prix Galien Roy Vagelos Pro Bono Humanum Award, the American Chemical Society's E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Lifetime Achievement Award presented by the Oligonucleotide Therapeutics Society, the Scrip Lifetime Achievement Award and the 2019 Massry Prize. Dr. Crooke received his M.D. and Ph.D. degrees and house staff training at Baylor College of Medicine, where he currently serves on the Board of Advisors. In 2021, Dr. Crooke has been named Distinguished Alumnus of both Baylor College of Medicine's Graduate and Medical schools and named one of the 20 of the most influential biopharma R&D executives by Endpoints News. He has published nearly 600 scientific publications, edited more than 20 books, has numerous patents, and led the development of more than 23 drugs that have been commercialized.

Toby Ferguson

Job Titles:
  • Vice President, Head of the Neuromuscular Development Unit at Biogen

Wei Zhang

Job Titles:
  • Assistant Director of Research

Wendy Chung

Job Titles:
  • Co - Chair