About

Carman Alenson, CPA, MBA, is Chief Accounting Officer and Senior Vice President, Finance at Prime.

Ms. Alenson has over 20 years of accounting and finance experience, most recently serving as Vice President, Accounting, Treasury and Tax at Agios Pharmaceuticals, Inc. Prior to that, she was Executive Director of Accounting, Controller, and Assistant Treasurer at AMAG Pharmaceuticals, Inc., where she managed the corporate accounting group.

Ms. Alenson is a certified public accountant, and received her M.B.A. and B.A. in Economics and Business Administration from Boston University.

Niamh Alix joined Prime Medicine in June 2022 as Chief Human Resources Officer.

Niamh is a passionate human resources leader who brings her experience, of helping to grow teams from small, early-stage organizations to global enterprises, to Prime. She has vast experience across multiple biotech and pharmaceutical organizations, where she led HR and Talent Acquisition teams from a couple of people to over 120 talented individuals.

Niamh joins Prime from Orchard Therapeutics, where she was Global Vice President, Talent, and HR Business Partner. She partnered with the leaders across all departments such as Research, Regulatory Affairs, Technical Operations and Commercial. Niamh and her team developed and deployed important people practices like Talent Management, Diversity and Inclusion and Learning and Development to the employees. Prior to that, Ms. Alix held global and regional roles at Lonza, including HR Business Partner for Americas, Mexico, and Canada where she led a large HR team and people strategy for a 5,000+ employee population across corporate and manufacturing clients. Niamh also held the Global Head, HR Business Partner role for the Cell and Gene Technology’s group which spanned three continents and grew rapidly.
Prior to that, Niamh was the Ireland Country HR Head for Novartis Pharmaceuticals and Oncology. Before this, she held additional senior roles of increasing responsibility at Novartis and Bristol Myers Squibb across Manufacturing and Commercial organizations.

Niamh was born and raised in Ireland and moved to the New England region in 2013. Admittedly, a self-confessed life-long learner, she holds a Master’s degree in HR Strategies from Dublin City University and a B.Sc. in Management from the Technological University of Dublin. In addition, she is trained in many people focused practices like Management Coaching, Employment Law, Neuroscience in Leadership, Learning Agility and many talent development tools.

Andrew Anzalone joined Prime Medicine in 2020 as the head of the prime editing platform, where he currently leads efforts to advance technological developments in prime editing for human therapeutic applications.

Dr. Anzalone received his Sc.B. degree in chemistry from Brown University, where he performed undergraduate research in synthetic organic chemistry. He completed his M.D. and Ph.D. degrees at Columbia University as part of the Medical Scientist Training Program. Dr. Anzalone’s thesis research in Virginia W. Cornish’s laboratory focused on the design and synthesis of fluorescent probes for biological imaging applications, and on the investigation and engineering of RNA-dependent protein translation reprogramming mechanisms in eukaryotes. After completing his graduate studies, Dr. Anzalone pursued postdoctoral research as a Jane Coffin Childs Memorial Fund Postdoctoral Fellow in the laboratory of David R. Liu at the Broad Institute of Harvard and MIT. In the Liu laboratory, Dr. Anzalone pioneered the development of prime editing, a novel search-and-replace gene editing technology that has the potential to correct the majority of known human genetic variants associated with disease.

Richard Brudnick is our Chief Business Officer.

Prior to joining Prime Medicine, Mr. Brudnick was Chief Business Officer and Head of Strategy for Codiak BioSciences, a leader in the field of exosome therapeutics. Before Codiak, Mr. Brudnick was Executive Vice President of Business Development and Alliance Management at Bioverativ, Inc., a company he helped found. Until Bioverativ’s acquisition by Sanofi, Mr. Brudnick led business development efforts to build a significant pipeline in rare blood disorders. Mr. Brudnick joined Bioverativ at its spin-off from Biogen where, over the course of nearly 15 years, he initiated, led and completed transactions that led to several marketed products including Leqembi, Tecfidera, Spinraza and its biosimilars joint venture with Samsung. Mr. Brudnick also was CEO of a regional pharmaceutical distribution business, which he sold to a strategic buyer; co-founded two companies; and was a strategy consultant at Bain & Company.

Mohammed Asmal, MD, PhD, is Senior Vice President, Head of Clinical at Prime Medicine. He is a physician-scientist with an AB in biology and biomedical engineering from Harvard University, and an MD-PhD in molecular and cellular biology from Columbia University. He trained in internal medicine and infectious diseases at the Brigham and Women’s and Massachusetts General Hospitals, and has devoted much of his academic career to studying the virology and immunology of HIV, and to treating patients with HIV and other infectious diseases in the clinic. After leaving academic medicine, Dr. Asmal took on roles in clinical development at Vertex Pharmaceuticals, where he led trials in oncology and hepatitis C, bringing multiple small molecules into the clinic through phase II. He subsequently transitioned to bluebird bio, where he led the clinical development team, overseeing lentiviral-based gene therapy programs in rare disease and oncology, culminating in the first approval of a gene therapy for a hemoglobinopathy in the world. Immediately prior to joining Prime Medicine, Dr. Asmal was a venture partner at OrbiMed Advisors, LLC, where he performed diligence on potential investments in the cell and gene therapy, COVID-19 and oncology spaces, and oversaw a seed stage investment in cell therapy.

Dr. Asmal has over 30 peer-reviewed scientific publications in immunology, HIV virology and in clinical trials of gene therapy for hemoglobinopathies. He is a native of Durban, South Africa, but has lived in the Boston vicinity for most of his life.

Karen Brown, PhD, JD is Senior Vice President, Intellectual Property & Legal Affairs of Prime Medicine, where she brings more than 20 years of experience in the life science industries. Before joining Prime, she served as senior vice president, IP & Legal Affairs at Obsidian Therapeutics, where she was a member of the leadership team, formulated the company’s IP strategy, and oversaw corporate legal matters. Prior to joining Obsidian, Dr. Brown was vice president and chief IP counsel at Ironwood Pharmaceuticals, where she built and led the IP group and developed the company’s global patent and trademark portfolio. While at Ironwood, Dr. Brown achieved successful outcomes in ANDA litigation for LINZESS® in collaboration with Ironwood’s co-development partner Allergan. Earlier in her career, Dr. Brown served in patent counsel roles of increasing responsibility at Vertex Pharmaceuticals, Schering-Plough, and in private practice.

Dr. Brown holds an AB in biology from Princeton University, a PhD in biochemistry from Boston University, and a JD from Fordham University School of Law.

Jeremy Duffield, MD, PhD, FRCP, is the Chief Scientific Officer of Prime Medicine. He has many years of drug discovery experience at Vertex Pharmaceuticals and Biogen Inc. preceded by a distinguished career in academic medicine.

Dr. Duffield has held several leadership roles, with focus in the fields of human genetics, innate immunity and regenerative medicine. He served as Global Head of Human Biology at Vertex Pharmaceuticals and as Vice President of Business Development where he and his team played important roles in discovering and advancing candidates to clinical studies in rare diseases including cystic fibrosis, a1-antitrypsin deficiency, sickle cell disease, FSGS and muscular dystrophies. Several candidates are now approved therapies. He was instrumental in building Vertex Cell and Genetic Therapies.

At Biogen, Dr. Duffield served as Senior Research Fellow and Vice President with responsibilities in early research programs, as joint Head of Innate Immunity and Regenerative Medicine therapeutic area, and as Head of the Biogen Post-Doctoral program. There he contributed to advancing integrin inhibitors, TNF superfamily inhibitors and IRAK inhibitors to clinical evaluation for pulmonary fibrosis and autoimmune diseases.

Prior to joining the leadership at Biogen, Dr. Duffield had a distinguished academic career on the faculty at University of Washington and Harvard Medical School as Head of the National Institutes of Health/National Center for Advancing Translational Sciences/American Heart Association-funded Laboratory for Innate Immunity and Regeneration. His laboratory used contemporary genetic methods to study cell and molecular function in innate immune and vascular cells in disease. Inventions from the laboratory contributed to the creation of several biotech companies, two of which advanced candidates now in late clinical trials. Additionally, Dr. Duffield practiced Internal Medicine and Nephrology at Massachusetts General Hospital until 2019.

Dr. Duffield served on NIH study sections, several company scientific advisory boards, is a member of the American Society of Clinical Investigation and received many scientific awards including the ASN-AHA Young Investigator Award and the NIH Early Career Investigator/Scholar Award.

Dr. Duffield received his B.A. and M.D. (B.M., B.Ch.) from Oxford University and a Ph.D. in Immunology from the University of Edinburgh in the laboratory of Sir John Savill.

Meredith Goldwasser joined Prime Medicine in September 2020 as Head of Strategy and Corporate Operations.

Dr. Goldwasser has spent the last 19 years developing new drugs in the biopharmaceutical industry working across all phases of clinical development, and in various roles in project leadership, business development and company building. She is a demonstrated strategic leader with broad experience in oncology, rare genetic diseases and data sciences. Most recently she was the development leader for the IDH Hematology program and oncology business development lead at Agios, where she also helped to build the clinical development and data sciences group. Prior to Agios, she held roles of increasing responsibility at Novartis and Genentech which contributed to the development and approval of multiple oncology products. Meredith holds a ScD in biostatistics from Harvard University, after which she worked as a research scientist at the Dana Farber Cancer Institute. She holds a BA from the University of Pennsylvania.

Keith Gottesdiener, MD is President and Chief Executive Officer of Prime Medicine and has served as a member of our Board of Directors since July 2020.

From October 2011 until March 2020, Dr. Gottesdiener served as the Chief Executive Officer and a director of Rhythm Pharmaceuticals, Inc., a biopharmaceutical company that develops therapeutics in rare genetic obesity. During that time, Rhythm submitted a New Drug Application for setmelanotide in two indications, for which setmelanotide was subsequently approved.

Dr. Gottesdiener joined Rhythm after 16 years at Merck Research Laboratories, where, starting in 1995, he held positions of increasing responsibility, including serving as a leader of Merck’s late clinical development organization from 2006 to 2011 and leading Merck’s early clinical development across all therapeutic areas from 2001 through early 2006. In such roles, Dr. Gottesdiener oversaw, led, or played a lead role in the development of most of Merck’s approved therapeutics and vaccines.

Dr. Gottesdiener received his B.A. from Harvard College and his M.D. from the University of Pennsylvania. He completed his residency and fellowship at the Brigham and Women’s Hospital-Beth Israel Medical Center-Dana Farber Cancer Institute Children’s Hospital programs. After his fellowship, Dr. Gottesdiener did postdoctoral research in the laboratory of Dr. Jack Strominger at the Dana Farber Cancer Institute. He then joined the faculty as an assistant professor at Columbia University, where he started an independent research laboratory with NIH RO-1 funding, ending his academic career as Associate Clinical Professor of Medicine at the time he left to join Merck in 1995.

Dr. Gottesdiener is also a director at Intercept Pharmaceutics, and at Cardurion Pharmaceuticals.

Ann Lee, PhD, is the Chief Technical Officer of Prime Medicine. Ann is an accomplished biotech executive with extensive experience and accomplishments in vaccines, biologics, small molecules and cell therapy development and manufacturing. She brings a passion for helping patients, innovating new technologies, and developing employees. Over the course of her career, she has contributed to the licensure and commercialization of 25 new vaccines and medicines, with the most recent being two CAR T cell therapies.

Ann recently worked at BMS, which acquired Celgene and Juno Therapeutics. Ann joined Juno Therapeutics in 2017 as Executive VP of Technical Operations to work in the new field of cell and gene therapy. She built the global Cell Therapy Development and Operations organization responsible for developing new manufacturing processes and technologies, manufacturing, quality assurance, designing new facilities, and creating the global supply chain network and digital systems to deliver these autologous products.

Ann was at Genentech in 2005 as VP of Process R&D and became SVP and Head of Global Technical Development at Roche in 2009. She was responsible for delivering Roche’s global R&D pipeline, as well as tech transfer and technical support for all commercial products. She was a member of corporate strategic portfolio governance committees and worked extensively cross-functionally as well as with external partners across the globe.

Prior to Genentech, Ann joined Merck Research Laboratories, where she advanced through several leadership positions in vaccine development over 14 years. She then became Vice President of Chemical Technology and Engineering in the Manufacturing Division at Merck, overseeing process engineering and technical operations at 10 chemical sites around the world.

Ann is a member of the National Academy of Engineering, a Fellow of the American Academy of Arts and Sciences, and a Fellow of the American Institute of Medical and Biological Engineering. She is the recipient of many scientific and engineering awards and has served on several professional, academic and company boards. She holds a B.S. in chemical engineering from Cornell University, and a Ph.D. in biochemical engineering from Yale University.

Allan Reine, MD, joined Prime Medicine in January of 2024 as Chief Financial Officer. A seasoned financial executive with more than twenty years’ experience in the biotechnology industry, Dr. Reine is responsible for the company’s financing strategy and investor relations and oversees all financial operations. Prior to joining Prime Medicine, Dr. Reine was Chief Financial Officer at Foghorn Therapeutics and, before that, at Pieris Pharmaceuticals. Dr. Reine also serves as Chairman of the Board of ONK Therapeutics. Previously, Dr. Reine managed various healthcare portfolios primarily focused on biotechnology and pharmaceutical companies at Lombard Odier Asset Management, Citi Principal Strategies, SAC Capital, Trivium Capital and Alexandra Investment Management. He started his career at CIBC World Markets where he worked in both biotechnology investment banking and biotechnology equity research. Dr. Reine received his M.D. from the University of Toronto, and his Bachelor of Science in statistical sciences from the University of Western Ontario.

Fubao Wang, Ph.D., is the Head of Regulatory Affairs and Senior Vice President.

Prior to Prime, Dr. Wang served as Senior Vice President, Head of Regulatory Affairs at Asklepios BioPharmaceutical, Inc. (AskBio), as Vice President, Head of Regulatory CMC of Global Regulatory Affairs at Sarepta Therapeutics, as Associate Vice President, US site head of CMC Dossiers at Sanofi. Dr. Wang worked at Merck & Co., Inc., for 16 years, with increasing responsibilities in both technical and regulatory roles. His last position at Merck was as Director of Global and Emerging Markets Regulatory Affairs.

Dr. Wang received his postdoctoral training in Molecular Biology from Stanford University, his Ph.D. in Molecular Biology from University of Heidelberg, Germany, and his M.S. and B.S. in Microbiology from Nankai University, China.

Tom Cahill, MD, PhD is the Founder and Managing Partner of Newpath Partners, a Boston based life science venture fund focused on building therapeutic companies around transformational scientific discoveries. Newpath is a founding investor in most of their life science companies and focuses on uniquely aligning interests between academic scientists, investors, and management teams. Dr. Cahill currently serves as a Director at the following companies: Chroma Medicine, Exo Therapeutics, Kisbee Therapeutics, Kojin Therapeutics, Magnet Biomedicine, Myeloid Therapeutics, Nvelop Therapeutics, Prime Medicine, Resonance Medicine, and YourBio Health. In addition, Dr. Cahill is involved with non-profit and non-partisan groups such as Scientists to Stop COVID-19 (STSC-19) and the Personal Genetics Education Project (pgEd). Dr. Cahill received both his M.D. and Ph.D. from Duke University.

Wendy Chung, M.D., Ph.D. is a clinical and molecular geneticist and the Chief of the Department of Pediatrics at Boston Children’s Hospital and Harvard Medical School. Dr. Chung directs NIH funded research programs in human genetics of pulmonary hypertension, breast cancer, obesity, diabetes, autism, birth defects including congenital diaphragmatic hernia and congenital heart disease. She is a national leader in the ethical, legal, and social implications of genomics. She was the recipient of the Rare Impact Award from the National Organization of Rare Disorders, and is a member of the National Academy of Medicine and the American Academy of Physicians. Dr. Chung received her B.A. in biochemistry from Cornell University, her M.D. from Cornell University Medical College, and her Ph.D. from The Rockefeller University in genetics.

Kaye Foster has served as a member of our board of directors since December 2021. Ms. Foster has been a Senior Advisor at the Boston Consulting Group since August 2014. Previously, she was Senior Vice President, Global Human Resources at Onyx Pharmaceuticals, Inc., an Amgen, Inc. subsidiary and a biopharmaceutical company, from October 2010 to January 2014. At Onyx, she led all aspects of human resources for U.S. and global operations. Prior to joining Onyx, Ms. Foster was Global Vice President of Human Resources and an Executive Committee member at Johnson and Johnson Corporation, from May 2003 to March 2010. Before Johnson and Johnson, Ms. Foster held several senior human resources executive positions with Pfizer Inc.. She currently serves on the board of directors of Agios Inc., where she chairs the compensation and Nominations and Governance Committees; and serves on the board of directors of Resilience Inc., on the compensation and community equity committees; on the board of directors and real estate and nominations committees of Stanford Health Care, a hospital and healthcare system; on the board of trustees and the human resources committee of Spelman College; and chairs the Glide Memorial Foundation Board of Trustees. She received her B.B.A. in Business Administration from Baruch College of the City University of New York and her M.B.A. from Columbia University, Graduate School of Business.

Keith Gottesdiener, MD is President and Chief Executive Officer of Prime Medicine and has served as a member of our Board of Directors since July 2020.

From October 2011 until March 2020, Dr. Gottesdiener served as the Chief Executive Officer and a director of Rhythm Pharmaceuticals, Inc., a biopharmaceutical company that develops therapeutics in rare genetic obesity. During that time, Rhythm submitted a New Drug Application for setmelanotide in two indications, for which setmelanotide was subsequently approved.

Dr. Gottesdiener joined Rhythm after 16 years at Merck Research Laboratories, where, starting in 1995, he held positions of increasing responsibility, including serving as a leader of Merck’s late clinical development organization from 2006 to 2011 and leading Merck’s early clinical development across all therapeutic areas from 2001 through early 2006. In such roles, Dr. Gottesdiener oversaw, led, or played a lead role in the development of most of Merck’s approved therapeutics and vaccines.

Dr. Gottesdiener received his B.A. from Harvard College and his M.D. from the University of Pennsylvania. He completed his residency and fellowship at the Brigham and Women’s Hospital-Beth Israel Medical Center-Dana Farber Cancer Institute Children’s Hospital programs. After his fellowship, Dr. Gottesdiener did postdoctoral research in the laboratory of Dr. Jack Strominger at the Dana Farber Cancer Institute. He then joined the faculty as an assistant professor at Columbia University, where he started an independent research laboratory with NIH RO-1 funding, ending his academic career as Associate Clinical Professor of Medicine at the time he left to join Merck in 1995.

Dr. Gottesdiener is also a director at Intercept Pharmaceutics, and at Cardurion Pharmaceuticals.

Michael A. Kelly has served as a member of our board of directors since November 2021. Mr. Kelly is currently acting as Founder & President of Sentry Hill Partners, LLC, a global life sciences transformation and management consulting business founded by Mr. Kelly in January 2018. From February 2003 to December 2017 he was a senior executive of Amgen, Inc., a biotechnology company, where he most recently served as Senior Vice President, Global Business Services and Vice President & Chief Financial Officer, International Commercial Operations. He also serves on the boards of directors of: Amicus Therapeutics, Inc., a biotechnology company, which he joined in December 2020; DMC Global Inc., a composite materials and oil field products company, which he joined in July 2020; NeoGenomics Laboratories, Inc., a genetics testing company, which he joined in July 2020; and Hookipa Pharma Inc., a biopharmaceutical company, which he joined in February 2019. Mr. Kelly has also held positions at Tanox, Inc., Biogen, Inc., and Nutrasweet Kelco Company, a division of Monsanto Life Sciences. He also serves on the Council of Advisors and was the former audit committee chairman for Direct Relief, a humanitarian aid organization focused on health outcomes and disaster relief. Mr. Kelly received his B.Sc. in Business Administration from Florida A&M University, concentrating in Finance and Industrial Relations.

Jeff Marrazzo founded and built Spark Therapeutics from an idea incubated within the Children’s Hospital of Philadelphia (CHOP) into the world’s first fully integrated, commercial gene therapy company with over 850 employees at the time of his departure. Under Jeff’s leadership, Spark developed and launched LUXTURNA^® for a rare blinding disorder, the first FDA-approved gene therapy for a genetic disease in the United States, spearheading the creation of novel reimbursement models to ensure patient access to genetic medicines. As CEO, Jeff shepherded multiple gene therapies into the clinic for patients with conditions ranging from inherited retinal diseases to rare bleeding and neuromuscular disorders.

While at Spark, Jeff raised $1 billion and established major partnerships with Pfizer and Novartis. Jeff’s successful orchestration of Spark’s $4.8 billion sale to Roche in 2019 marked a 100-fold increase in the company’s market value over six years and ensured Spark’s ongoing operations would remain in Philadelphia.

Jeff currently serves as a board member and strategic advisor to several biotechnology companies and entrepreneurs, primarily in the Philadelphia area.

Jeff holds a B.A. in systems science and engineering and B.A. in economics from the University of Pennsylvania as well as a M.B.A/M.P.A from Wharton and Harvard University.

Robert Nelsen is a co-founder and a Managing Director of ARCH Venture Partners. He joined ARCH at its founding and played a significant role in the early sourcing, financing, and development of more than 100 companies, including twenty-seven which have reached valuations exceeding $1 billion. His seed and early-stage investments include Illumina (ILMN); Alnylam Pharmaceuticals (ALNY); Juno Therapeutics (JUNO); Sana Biotechnology (SANA), Unity Biotechnology (UBX); Sienna Biopharmaceuticals (SNNA); Vir Biotechnology(VIR); Agios Pharmaceuticals (AGIO); Sage Therapeutics (SAGE); GRAIL; Ikaria; Kythera Biopharmaceuticals (KYTH); Receptos (RCPT); Aviron (AVIR); Denali Therapeutics (DNLI); Prime Medicine; Beam Therapeutics (BEAM); NetBot; Bluebird Bio (BLUE); R2 Technology; XenoPort (XNPT); Caliper Life Sciences (CALP); Trubion Pharmaceuticals (TRBN); Adolor (ADLR); deCODE Genetics; Array BioPharma (ARRY); Editas (EDIT); IDUN Pharmaceuticals; Classmates.com; Hua Medicine; Fate Therapeutics (FATE); Rubius Therapeutics (RUBY); KSQ Therapeutics; WuxiNextCODE; and Everyday Learning Corporation.

Mr. Nelsen is a director of Vir Bio, GRAIL, Sana Biotechnology, Beam Therapeutics, Prime Medicine, Resilience, Lyell, RBNC, Revolution Healthcare Acquisition Corp., Denali Therapeutics, Karuna Therapeutics, insitro, Maze Therapeutics, Inc., and serves as Chairman of Hua Medicine, among others. He previously served as a Trustee of the Fred Hutchinson Cancer Research Institute, the Institute for Systems Biology, and was a director of the National Venture Capital Association. Mr. Nelsen holds an M.B.A. from the University of Chicago and a B.S. from the University of Puget Sound with majors in Economics and Biology.

Dr. David Schenkein is a general partner at GV, where he co-leads the life science investment team. Previously, Dr. Schenkein spent ten years as chief executive officer and a member of the board of directors at Agios Pharmaceuticals, where he remains chairman.

Dr. Schenkein has been a hematologist and medical oncologist for 30 years. He currently serves as an adjunct attending physician in hematology at Tufts Medical Center. Prior to joining Agios, Dr. Schenkein was the senior vice president, clinical hematology/oncology at Genentech, where he was responsible for numerous successful oncology drug approvals and leading the medical and scientific strategies for their BioOncology portfolio. While at Genentech, he served as an adjunct clinical professor of medical oncology at Stanford University School of Medicine.

Prior to joining Genentech, he served as the senior vice president of clinical research at Millennium Pharmaceuticals, overseeing the clinical development and worldwide approval of Velcade, a first-in-class cancer therapy now approved to treat multiple myeloma and non-Hodgkins lymphoma. He serves on the board of directors of Denali Therapeutics.

Dr. Schenkein holds a B.A. in chemistry from Wesleyan University and an M.D. from the State University of New York Upstate Medical School.

Andrew Anzalone joined Prime Medicine in 2020 as the head of the prime editing platform, where he currently leads efforts to advance technological developments in prime editing for human therapeutic applications.

Dr. Anzalone received his Sc.B. degree in chemistry from Brown University, where he performed undergraduate research in synthetic organic chemistry. He completed his M.D. and Ph.D. degrees at Columbia University as part of the Medical Scientist Training Program. Dr. Anzalone’s thesis research in Virginia W. Cornish’s laboratory focused on the design and synthesis of fluorescent probes for biological imaging applications, and on the investigation and engineering of RNA-dependent protein translation reprogramming mechanisms in eukaryotes. After completing his graduate studies, Dr. Anzalone pursued postdoctoral research as a Jane Coffin Childs Memorial Fund Postdoctoral Fellow in the laboratory of David R. Liu at the Broad Institute of Harvard and MIT. In the Liu laboratory, Dr. Anzalone pioneered the development of prime editing, a novel search-and-replace gene editing technology that has the potential to correct the majority of known human genetic variants associated with disease.

David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice chair of the faculty at the Broad Institute of Harvard and MIT, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries. Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases.

Liu graduated first in his class at Harvard College in 1994. During his doctoral research at UC Berkeley, Liu initiated the first general effort to expand the genetic code in living cells. He earned his PhD in 1999 and became assistant professor of chemistry and chemical biology at Harvard University in the same year. He was promoted to associate professor in 2003 and to full professor in 2005. Liu became a Howard Hughes Medical Institute investigator in 2005 and joined the JASONs, academic science advisors to the U.S. government, in 2009. In 2016 he became a Core Institute Member and Vice-Chair of the Faculty at the Broad Institute of MIT and Harvard, and Director of the Chemical Biology and Therapeutics Science Program.

Liu has been elected to the U.S. National Academy of Sciences, the U.S. National Academy of Medicine, and the American Association for the Advancement of Science. He has earned several University-wide distinctions for teaching at Harvard, including the Joseph R. Levenson Memorial Teaching Prize, the Roslyn Abramson Award, and a Harvard College Professorship. Liu has published more than 200 papers and is the inventor on more than 75 issued U.S. patents. His research accomplishments have earned distinctions including the Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society David Perlman Award, ACS Chemical Biology Award, the American Chemical Society Pure Chemistry Award, the Arthur Cope Young Scholar Award, the NIH Marshall Nirenberg Lecturer, and awards from the Sloan Foundation, Beckman Foundation, NSF CAREER Program, and Searle Scholars Program. In 2016 and 2020 he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology, and was named one of Nature’s 10 researchers in world and to the Foreign Policy Leading Global Thinkers in 2017. He is the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics, and Chroma Medicine.

Dr. Yvonne Chen is an Associate Professor of Microbiology, Immunology, and Molecular Genetics at UCLA. She is the co-director of the Tumor Immunology program in the Jonsson Comprehensive Cancer Center at UCLA, and a member researcher of the Parker Institute for Cancer Immunotherapy. The Chen Laboratory applies biomolecular engineering techniques to the development of novel mammalian-cell systems for clinical use, and led the first investigator-sponsored clinical trial on CAR-T cell therapy at UCLA. The Chen Lab’s work has been recognized by the NIH Director’s Early Independence Award, the NSF CAREER Award, the Mark Foundation Emerging Leader Award, and the Cancer Research Institute Lloyd J. Old STAR Award, among others. Prior to joining UCLA in 2013, Yvonne was a Junior Fellow in the Harvard Society of Fellows. Yvonne received her B.S. in Chemical Engineering from Stanford University and her Ph.D. in Chemical Engineering from the California Institute of Technology.

Prof. Czechowicz is a translationally oriented-physician scientist at Stanford University in the Department of Pediatrics Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine. She is also a member of the Stanford Institute of Stem Cell Biology and Regenerative Medicine. Dr. Czechowicz previously spent a decade on the Farm as a Stanford trainee completing her undergraduate, medical school and PhD degrees while conducting research with Prof. Irving Weissman. She subsequently did further clinical training in Boston, completing her residency in Pediatrics at Boston Children’s Hospital and pursued subspecialty training in Pediatric Hematology/Oncology at the Dana Farber Cancer Institute while simultaneously conducting postdoctoral research with Prof. Derrick Rossi in collaboration with Prof. David Scadden. Dr. Czechowicz’s research has focused around improving the utilization of hematopoietic stem cells for the treatment of diverse blood and immune diseases, and she has done pioneering work showing that hematopoietic stem cell depletion is a critical component to donor hematopoietic stem cell engraftment. Multiple pre-clinical and clinical therapies are in development based upon her studies aimed at the elimination of chemotherapy and irradiation from transplant protocols. Furthermore, Dr. Czechowicz’s clinical interests are in bone marrow failure syndromes with a particular interest in Fanconi Anemia and she has led several innovative cell and gene therapy trials attempting to improving outcomes for patients with this disease. Dr. Czechowicz is also quite passionate about mentoring and training future generations of physicians and scientists, and is very supportive of helping diverse trainees on various traditional and non-traditional career paths.

Guangping Gao, PhD, is the Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems, Penelope Booth Rockwell Professor in Biomedical Research, University of Massachusetts Chan Medical School; Elected fellows, both the US National Academy of Inventors (NAI) and American Academy of Microbiology; Past president, American Society of Gene and Cell Therapy.

Dr. Gao is an internationally well recognized gene therapy researcher who has played a key role in the discovery and characterization of new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, hugely impacting many currently untreatable human diseases. For 30+ years of his scientific research career, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy of rare genetic diseases, encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, viral vector manufacturing for preclinical and clinical gene therapy applications as well as technology platforms development as novel approaches for human gene therapy.

Dr. Gao has published 330+ research papers, 8 book chapters, and 5 edited books. Dr. Gao holds 212 patents with 429 more patent applications pending. He serves as Executive Editor-In-Chief of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and on Editorial Boards of several other gene therapy and virology journals. Dr. Gao has been ranked as the World Top 20 Translational Researchers for several years in a row by Nature Biotechnology. Dr. Gao co-founded Voyager Therapeutics, Adrenas Therapeutics, and Aspa Therapeutics, focusing on developing rAAV gene therapeutics for treating a variety of devastating rare diseases.

Jim Haber received his AB from Harvard College and his PhD in Biochemistry at the University of California, Berkeley. After postdoctoral work at the University of Wisconsin, Madison, he joined the Department of Biology at Brandeis in 1972. He has been a leading researcher in the fields of DNA repair and DNA damage checkpoints for over forty years. By using inducible site-specific DNA cleavage, his lab has been able to monitor in real time the kinetics of DNA repair processes. They have identified many of the components and molecular intermediates in both nonhomologous end-joining and in several pathways of homologous recombination. In 2011 he received the Genetics Society of America’s Thomas Hunt Morgan Medal for Lifetime Achievement in Genetics. He is a member of the National Academy of Sciences, a Fellow of the American Academy of Microbiology, a Fellow of the American Association for the Advancement of Science and of the American Academy of Arts and Sciences.

Anastasia Khvorova, PhD, has more than 20 years of experience developing oligonucleotide technology and therapeutics. She is a professor in the RNA Therapeutics Institute and Program in Molecular Medicine at the University of Massachusetts (UMass) Chan Medical School, where her lab brings together hardcore organic and oligonucleotide chemists with RNA biologists and pharmacologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. Dr Khvorova founded the UMass Nucleic Acid Chemistry Center, the only nonprofit facility in North America capable of gram-scale synthesis of modified oligonucleotides.

Prior to UMass Chan Medical School, Dr Khvorova spent 12 years in industry in different executive positions. She has co-founded several biotechnology companies.

Dr Khvorova is named as inventor on more than 150 patents and 300 patent applications and has authored more than 100 peer-reviewed publications, including seminal articles in Cell, Nature, and Nature Biotechnology, defining the field of RNAi drug design and development. Dr Khvorova is principal investigator on three major National Institutes of Health grants. She has served as a director of the Oligonucleotide Therapeutics Society and currently is Director-at-Large and Scientific & Research Council Chair of the American Society of Gene and Cell Therapy.

Ben Kleinstiver is a biochemist and genome editor whose research lab works at the intersection of protein engineering and the development of genetic engineering technologies. He received his Ph.D in Biochemistry from the University of Western Ontario, where he studied homing endonucleases and their application as monomeric nuclease technologies. He then completed his postdoctoral studies at Massachusetts General Hospital (MGH) and Harvard Medical School, focused on developing improved CRISPR-Cas enzymes. The Kleinstiver laboratory is situated within the Center for Genomic Medicine at MGH and is focused on accelerating the development of CRISPR technologies as genetic medicines. The major research goals in Dr. Kleinstiver’s laboratory are to overcome the limitations of naturally occurring enzymes to design more optimal technologies, to develop new capabilities that solve outstanding needs in the genome editing field, with the hope of transforming these technologies into safe and effective genetic therapies for patients.

David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice chair of the faculty at the Broad Institute of Harvard and MIT, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries. Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases.

Liu graduated first in his class at Harvard College in 1994. During his doctoral research at UC Berkeley, Liu initiated the first general effort to expand the genetic code in living cells. He earned his PhD in 1999 and became assistant professor of chemistry and chemical biology at Harvard University in the same year. He was promoted to associate professor in 2003 and to full professor in 2005. Liu became a Howard Hughes Medical Institute investigator in 2005 and joined the JASONs, academic science advisors to the U.S. government, in 2009. In 2016 he became a Core Institute Member and Vice-Chair of the Faculty at the Broad Institute of MIT and Harvard, and Director of the Chemical Biology and Therapeutics Science Program.

Liu has been elected to the U.S. National Academy of Sciences, the U.S. National Academy of Medicine, and the American Association for the Advancement of Science. He has earned several University-wide distinctions for teaching at Harvard, including the Joseph R. Levenson Memorial Teaching Prize, the Roslyn Abramson Award, and a Harvard College Professorship. Liu has published more than 200 papers and is the inventor on more than 75 issued U.S. patents. His research accomplishments have earned distinctions including the Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society David Perlman Award, ACS Chemical Biology Award, the American Chemical Society Pure Chemistry Award, the Arthur Cope Young Scholar Award, the NIH Marshall Nirenberg Lecturer, and awards from the Sloan Foundation, Beckman Foundation, NSF CAREER Program, and Searle Scholars Program. In 2016 and 2020 he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology, and was named one of Nature’s 10 researchers in world and to the Foreign Policy Leading Global Thinkers in 2017. He is the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics, and Chroma Medicine.

Dr. Muthiah (Mano) Manoharan serves as the Senior Vice President of Drug Innovation, a Scientific Advisory Board Member, and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts. In 2003, he was the founding chemist hired at Alnylam as the Head of the Drug Discovery. He and his team pioneered the discovery and development of numerous chemical modifications, GalNAc conjugation chemistry, and LNP delivery platform that make RNA interference-based human therapeutics possible. This work led to the approval of five RNAi therapeutics: ONPATTRO^® (patisiran, 2018), GIVLAARI^® (givosiran, 2019), OXLUMO^® (lumasiran, 2020), LEQVIO^® (inclisiran, 2020, 2021) and AMVUTTRA^® (vutrisiran, 2022).

Dr. Manoharan has had a distinguished career as a world-leading chemist in the field of oligonucleotides. He is an author of more than 230 publications (nearly 56,300 Google Scholar citations with an h-index of 110 and an i10-index of 416) and over 500 abstracts, as well as an inventor of over 250 issued U.S. patents. Prior to Alnylam, Dr. Manoharan worked in the field of antisense oligonucleotide therapeutics at Ionis (formerly Isis) Pharmaceuticals and at LifeCodes Corporation. He also served as a member of the chemistry faculty at the American College, Madurai, Tamil Nādu, India.

Dr. Manoharan received his B.Sc. and M.Sc. degrees in chemistry at the American College, Madurai, Tamil Nādu, India. He earned his Ph.D. in chemistry (with Professor Ernest L. Eliel) at the University of North Carolina, Chapel Hill and carried out post-doctoral research (with Professor John A. Gerlt) in the field of oligonucleotide chemistry and DNA repair enzymes at Yale University and at the University of Maryland. Dr. Manoharan is the winner of the Lifetime Achievement Award of the Oligonucleotide Therapeutics Society (2019), the M. L. Wolfrom Award (2007), D. Horton Industrial Carbohydrate Chemistry Award (2021) from the Carbohydrate Chemistry Division of the American Chemical Society and the Chemical Research Society of India (CRSI) 2022 Medal by the Council of the Chemical Research Society of India.

Eric Olson is the founding Chair of the Department of Molecular Biology at UT Southwestern Medical Center. He also directs the Hamon Center for Regenerative Science and Medicine and the Wellstone Center for Muscular Dystrophy Research at UT Southwestern. He holds the Robert A. Welch Distinguished Chair, the Pogue Chair Distinguished Chair in Cardiac Birth Defects and the Annie and Willie Nelson Professorship in Stem Cell Research.

Eric Olson and his trainees discovered many of the key genes and mechanisms responsible for development and disease of the heart and other muscles. His most recent work has provided new strategies for correction of genetic disorders of muscle and the heart using CRISPR gene editing.

Dr. Olson is a member of the U.S. National Academy of Sciences, the Institute of Medicine, and the American Academy of Arts and Sciences and has received numerous additional honors for his work. He has co-founded multiple biotechnology companies to design new therapies for heart and muscle disease.

Gerald Schwank is an Associate Professor at the Medical Faculty of the University of Zurich, specialized in translational genome editing. He completed his PhD in Molecular Biology at the University of Zurich, after which he moved to the Netherlands for his postdoctoral training at the Hubrecht Institute for Developmental Biology and Stem Cell Research. During this time he developed genome editing strategies to correct pathogenic mutations in patient-derived stem cell organoids. In 2015, he returned to Switzerland to establish his laboratory at ETH Zurich, where his team developed CRISPR-based screening methods to investigate key molecular mechanisms involved in cancer progression. Furthermore, his lab utilized DNA double-strand break-independent genome editing techniques, such as base- and prime editing, to develop novel therapies for genetic disorders. Following his appointment as an Associate Professor at the University of Zurich in 2019, his lab started integrating protein engineering and machine learning approaches to further refine genome editing tools for therapeutic applications. His ongoing research initiatives receive support from various organizations dedicated to fighting rare diseases, the Swiss National Science Foundation (SNSF), and the European Research Council (ERC).

Jay Shendure is an Investigator of the Howard Hughes Medical Institute, Professor of Genome Sciences at the University of Washington, Director of the Allen Discovery Center for Cell Lineage Tracing, and Scientific Director of the Brotman Baty Institute for Precision Medicine. His 2005 doctoral thesis with George Church included one of the first successful reductions to practice of next generation DNA sequencing. Dr. Shendure’s research group in Seattle pioneered exome sequencing and its earliest applications to gene discovery for Mendelian disorders and autism; cell-free DNA diagnostics for cancer and reproductive medicine; massively parallel reporter assays, saturation genome editing; whole organism lineage tracing, and massively parallel molecular profiling of single cells. Dr. Shendure is the recipient of the 2012 Curt Stern Award from the American Society of Human Genetics, the 2013 FEDERAprijs, a 2013 NIH Director’s Pioneer Award, the 2014 HudsonAlpha Life Sciences Prize, the 2018 Richard and Carol Hertzberg Prize for Technology Innovation, the 2019 Richard Lounsbery Award from the National Academy of Sciences, and the 2022 Mendel Lectureship from the European Society of Human Genetics. He currently or previously has served as an advisor to the NIH Director, the US Precision Medicine Initiative, the National Human Genome Research Institute, the Chan-Zuckerberg Initiative and the Allen Institutes for Cell Science and Immunology. He received his MD and PhD degrees from Harvard Medical School in 2007.

Dr. Samuel Sternberg, PhD, runs a research laboratory at Columbia University, where he is an assistant professor in the Department of Biochemistry and Molecular Biophysics. He received his B.A. in Biochemistry from Columbia University in 2007, graduating summa cum laude, and his Ph.D. in Chemistry from the University of California, Berkeley in 2014. He earned graduate student fellowships from the National Science Foundation and the Department of Defense, and was awarded the Scaringe Award from the RNA Society and the Harold Weintraub Graduate Student Award from the Fred Hutchinson Cancer Research Center. After a brief book-writing stint, Sam worked as a Scientist and Group Leader at Caribou Biosciences before beginning his independent position at Columbia in 2018. He is the recipient of the NIH Director’s New Innovator Award, and is a Sloan Fellow, Pew Biomedical Scholar, and Schaefer Research Scholar.

Sam’s lab explores the biology of CRISPR-Cas systems and transposable elements, and develops these systems for genome engineering. In addition to publishing his research in leading journals and speaking internationally, Sam remains actively involved in public outreach and ongoing discussions on the ethical issues surrounding genome editing. Together with Jennifer Doudna, he co-authored a popular science book about the discovery, development, and applications of CRISPR technology. Titled A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution, their book was a finalist for the Los Angeles Times Book Prize, and The New York Review of Books called it “required reading for every concerned citizen.”

Dr. Tsai is an Assistant Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T-cells for cancer immunotherapy. His group has recently developed CHANGE-seq, a state-of-the-art, sensitive, unbiased, high-throughput method for defining the genome-wide activity of genome editors. Previously, he has developed methods for high-throughput genome editing with TALENs, and for defining and improving the genome-wide specificity of CRISPR-Cas nucleases such as GUIDE-seq and CIRCLE-seq. Dr. Tsai completed a postdoctoral fellowship at Massachusetts General Hospital & Harvard Medical School, Ph.D. in Functional Genomics and M.S. in Bioinformatics from North Carolina State University, and B.S. from the University of Michigan.