About Us

Mission & Values

Our mission is to transform the treatment paradigm for patients with inherited disorders by developing durable, broadly applicable, life-altering therapies grounded in innovative and rigorous science. By advancing a new category of genetic medicine, at SalioGen, we aspire to treat all inherited disorders, rare or common. At SalioGen, we bring together multidisciplinary group of scientists, drug developers, business leaders and key opinion leaders both from academia and the biotech industry with the breadth and depth of experience needed to bring new, innovative and transformative therapies to patients in need.

Leadership

Jason F. Cole

Jason F. Cole

CEO & Chairman of the Board

Jason F. Cole

CEO & Chairman of the Board

Jason F. Cole is a strategic C-Level biotechnology executive and Board Member with over 20 years of experience working with public and private biotech companies focused on strategy, capital raising, business development, operations and enabling teams.  At bluebird bio, he served as Chief Strategy and Financial Officer, Chief Business Officer and Chief Operating and Legal Officer and provided strategic, financial, operational and legal oversight during key growth and transition periods – scaling from 100 to 1,200 employees (U.S. & Europe), securing FDA approval of three first-of-their-kind gene and cell therapies (ZYNTEGLO, SKYSONA, ABECMA) and closing over $3.7 billion in financings and strategy-enabling business development transactions.  Prior to bluebird bio, Jason served on the executive leadership teams of Zalicus and CombinatoRx, with responsibility across corporate development, strategic alliances, legal, IT, and facilities.  Jason holds a J.D. from Columbia University and an A.B in Government for Dartmouth College.

Joseph J. Higgins, M.D.

Chief Innovation Officer
Board Member

Joseph J. Higgins, M.D.

Chief Innovation Officer Board Member

Joe is an executive leader, clinician and scientist dedicated to advancing gene therapies for children and adults with genetic disorders involving the cardiovascular, ophthalmological, neurological, and immunological systems. His background spans pediatrics, child neurology, medical genetics and molecular developmental neurobiology. Joe’s notable scientific achievements include, as part of his efforts on the Human Genome Project as the lead of the Human Genetic Initiative, identifying loci and genes for Parkinson’s disease and essential tremor as well as conducting multiple first-in-human Phase 1/2 clinical trials in enzyme replacement and gene therapies in lysosomal and other disorders. As the Vice President of Clinical Development at uniQure, Joe led Phase 1/2 first-in-human gene therapy trials in Huntington’s disease. His academic background includes positions as an instructor at Harvard Medical School, an Assistant Professor at George Washington University, an Associate Professor at SUNY Albany, and a Professor at Cornell University. Joe has published more than 100 medical journal publications and book chapters as well as numerous gene therapy patents.

Joe received his medical degree from New York Univeristy and graduated cum laude with a B.A. in Biology from Marist College. He is double-board certified in Pediatrics and Neurology with Special Qualification in Child Neurology and is licensed to practice medicine in New York and Massachusetts.

Joe Senn, Ph.D.

Joe Senn, Ph.D.

Senior Vice President,
Head of R&D

Joe Senn, Ph.D.

Senior Vice President, Head of R&D

Joe has experience with nearly all therapeutic modalities, including small molecules, biologics, antisense, gene editing and mRNA therapeutics. He most recently served as the Vice President of Nonclinical Development at Moderna Therapeutics for eight years, where he and his team were responsible for progressing over 40 candidates into the clinic. Prior to Moderna, Dr. Senn served as Site Head for Drug Safety at Takeda Pharmaceuticals, where he oversaw development of all immunology products across the portfolio.

Dr. Senn received his Ph.D. in Pharmacology and Physiology from the University of Rochester School of Medicine and Dentistry.

Jeff Kopacz

Jeff Kopacz, M.S., J.D.

Senior Vice President of
Intellectual Property & Contracts

Jeff Kopacz, M.S., J.D.

Senior Vice President of Intellectual Property & Contracts

Jeff has extensive intellectual property experience, including experience with building and defending intellectual property protection for platform-based technologies and FDA approved biotherapeutics. Prior to joining SalioGen Therapeutics, he served as Vice-President of Intellectual Property at Omega Therapeutics, where he led intellectual property and contract activities related to their genomic control technology.

Prior to joining Omega, Jeff was at Alnylam Pharmaceuticals, Inc., for 10 years and served most recently as Senior Director, Senior Intellectual Property counsel. His primary responsibilities included developing and executing the intellectual property strategy for Alnylam’s therapeutic candidates, and he helped build the intellectual property portfolio for the world’s first RNAi-approved medicines, Onpattro®, Givlaari®, Oxlumo®, and Leqvio®. He also helped defend the Alnylam platform intellectual property estate from patent challenges and worked closely with business development supporting various license agreements and collaborations.

Prior to joining Alnylam, Jeff was an attorney at Edwards Angell Palmer & Dodge LLP (now Locke Lord LLP), where he built the intellectual property estates for numerous commercial products and counseled clients on intellectual property matters in the biotechnology and pharmaceutical industries. Jeff began his biopharmaceutical career at Millennium Pharmaceuticals, now Takeda Pharmaceuticals.

Jeff received his undergraduate degree in microbiology for University of Massachusetts at Amherst, his graduate degree in molecular microbiology and immunology from Johns Hopkins University and a law degree from Suffolk University Law School.

Sandeep Nema

Sandeep Nema, Ph.D.

Senior Vice President of
Technical Operations

Sandeep Nema, Ph.D.

Senior Vice President of Technical Operations

Sandeep has exhaustive developmental experience in bringing candidates to clinic that span various modalities including gene therapy, ADCs, mAbs, vaccines, oligonucleotides, bi/trispecific, and CART. Prior to joining SalioGen, he worked for 24 years at Pfizer. Recently his team was successful in bringing Pfizer’s COVID-19 LNP-mRNA vaccine to the clinic and achieving emergency use authorization. In his last role as Executive Director, Biotherapeutics Pharmaceutical Sciences, Global Biologics he was responsible for all pre-proof of concept biologics projects, approximately 50 in total, from discovery up to Phase 3 start. Before that, Sandeep established the Protein Pharmaceutical R&D group at Pfizer that included Formulation, Microbiology and Stability functions. Sandeep is active in AAPS and PDA where he has served as Chairman, Arden House Conference on “Parenteral Products: Integrating Science, Innovation and Patient Needs,” Co-Chair, AAPS/FDA/USP workshop on “Future Direction in Aseptic Processing,” and past Chair of Sterile Product Focus group. He is also an adjunct Professor at University of Tennessee. In 2019 Sandeep co-edited ‘Pharmaceutical Dosage Forms: Parenteral Medications” (4th edition). In addition, he has served as Steering Committee Member for the Handbook of Pharmaceutical Excipients. Sandeep has been lead formulator for six launched products and has filed more than 100 INDs/IMPDs (CMC module).

Sandeep earned his Ph.D. in Pharmaceuticals from the University of Tennessee Health Science Center and his Bachelor of Pharmacy from Banaras Hindu University. He is also a Certified Regulatory Affairs Professional.

Jill Murray

Jill Murray

Vice President,
Human Resources

Jill Murray

Vice President, Human Resources

Jill is an accomplished, results oriented Senior Human Resources professional with 20+ years of progressive experience within the Biotech industry. She leverages her expertise working with executive leadership to develop & execute talent management initiatives that support the overall business strategy. She has expertise in organizational design, employee development, employee relations, leadership coaching, change management, compensation, benefits administration and legal compliance. Prior to joining Saliogen, Jill spent 14 years at ImmunoGen, Inc. covering all facets of Human Resources and Talent Management, which led her to a passion for HR Business Partnering. She joined Sarepta Therapeutics in 2015, where she was responsible for establishing and building the HR Business Partnering function. She joined Rubius Therapeutics in January 2018 as the lead HR Business Partner, and soon took on the additional responsibility as Head of Total Rewards, which ultimately led to her becoming Head of Human Resources for Rubius Therapeutics in 2022. Jill holds a Bachelors degree in Art and Psychology from Carnegie Mellon University. She is also a certified Executive Coach. 

Will McGown

Executive Director,
Finance & FP&A

Will McGown

Executive Director, Finance & FP&A

Will is a financial leader with over fifteen years of experience working in both public and private biotechnology companies at all stages of development, from discovery through commercialization. Most recently, Will was the Sr. Director of FP&A for Allena Pharmaceuticals. Prior to that role, Will spent five years as the Sr. Director of Finance and Controller for Navitor Pharmaceuticals, and nine years at AMAG Pharmaceuticals in various roles including Controller and Director of FP&A. Will holds an MBA from Quinnipiac University, and Bachelor of Science degrees in Finance and Accounting from the Darla Moore School of Business at the University of South Carolina. 

Extended Leadership

Nancy Craig

Nancy L. Craig, Ph.D.

Senior Vice President of
Genetic Engineering and Mobile Elements, Chair, Scientific Advisory Board

Nancy L. Craig, Ph.D.

Senior Vice President of Genetic Engineering and Mobile Elements
Chair, Scientific Advisory Board

Nancy is widely regarded as a pioneer in the study of mobile genetic elements. Her research focuses on the molecular mechanisms of transposable elements, or mobile sequences of DNA found in the genomes of most known organisms. She has directed a research group focused on identifying mobile elements and studied their mechanisms of action since 1992. A member of the National Academy of Sciences since 2010, she currently serves as Professor Emerita of Molecular Biology & Genetics at the Johns Hopkins University School of Medicine. She was also an Investigator of the Howard Hughes Medical Institute from 1992-2015. Previously, Nancy was a Professor at the University of California, San Francisco in the Departments of Microbiology & Immunology and of Biochemistry & Biophysics. Nancy is the lead author of “Molecular Biology: Principles of Genome Function”, published by Oxford University Press and now in its third edition and also the editor of “Mobile DNA II” and “Mobile DNA III,” published by the American Society for Microbiology, and widely considered to be the definitive volumes on mobile genetic elements.

Nancy completed a postdoctoral fellowship at the National Institutes of Health with a focus on genomic integration, and she earned a Ph.D. in Biochemistry from Cornell University, during which her studies included mechanisms of DNA repair. Nancy graduated summa cum laude with an A.B. in Biology and Chemistry from Bryn Mawr College.

Jonathan Lu

Jonathan Lu

Sr. Director, Translational Medicine & Early Development

Jonathan Lu

Sr. Director, Translational Medicine & Early Development

Jonathan T. Lu, M.D., Ph.D., is a physician scientist with an extensive background in clinical medicine, scientific research and clinical development. His career spans various roles from academia to industry. including his current role as Senior Director in Translational Medicine at SalioGen Therapeutics. Notably, he served as a Global Clinical Lead for Group II Pulmonary Hypertension and made significant contributions to the development of Sotatercept at Acceleron Pharma, now a wholly owned subsidiary of Merck. As a Medical Director at Sarepta Therapeutics, Dr Lu held an important role in the development of three products that have received accelerated approval: Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen). Dr Lu also served as a head quarter Medical Director for AstraZeneca US Medical Affairs involved in medical strategies of Brillinta (tigacrelor).

Dr. Lu holds an M.D. and Ph.D. from Vanderbilt University School of Medicine, along with a ScB in Neuroscience from Brown University. Dr Lu trained as a cardiac electrophysiologist at UCSF and general cardiology at UCSD. Prior to joining industry, he was on faculty at Columbia University College of Physicians and Surgeons and took care of patients with heritable arrhythmia and ran a laboratory research program based on patient derived iPS cells.

Subhrangshu (Subhro) Guhathakurta Ph.D.

Sr. Director, Platform Development & Genetic Engineering

Subhrangshu (Subhro) Guhathakurta Ph.D.

Sr. Director, Platform Development & Genetic Engineering

Subhro is a trained molecular biologist and brings years of experience in genome and epigenome editing. He has a strong scientific background in regulation of disease-implicated key gene expressions by genome engineering. Dr. Guhathakurta has directed various cross functional research groups focusing on building cutting edge gene therapy tools and establishing novel genome editing based platform development. Subhro was one of the first few who developed an endogenous reporter system using CRISPR/Cas to monitor changes in expression of the key gene in the development of Parkinson’s Disease. He has also developed an epigenome editing platform to regulate precise histone posttranslational modification (PTM) status of any gene. Regulating gene expression in different disease conditions using viral and non-viral platforms by various modalities, such as small interfering RNAs, modulating local epigenetic environment, locus-specific integration or base pair editing, gene-specific knockdown/knockout, small molecules, etc. have been the key research areas of Subhro. He also worked on developing and optimizing expression cassettes for improved tissue specific delivery and enhanced expression of the cargo in various diseases in pre-clinical development.

Subhro earned his Ph.D. in Biotechnology from the University of Calcutta where he studied genetics of autism spectrum disorder and helped in establishing a peripheral biomarker for the disease. Later, he did his postdoctoral trainings at Cornell University and the University of Central Florida in understanding molecular biology of gene de-regulation, developing therapeutic tool and characterizing protein aggregation based neurodegenerative diseases. Subhro serves as a review editor for one of the Frontiers group of journals.

Michelle LeBlanc

Director, In-Vivo Biology

Michelle LeBlanc

Director, In-Vivo Biology

Michelle is a molecular and cellular biologist specializing in early-stage drug discovery, with a focus on ocular gene therapy. A retinal biologist by training, she has worked on a broad range of retinal diseases including diabetic macular edema, diabetic retinopathy, retinopathy of prematurity, age-related macular degeneration, and inherited retinal diseases such as Stargardt.

For her Ph.D., Michelle systematized and validated a high-thruput in vivo screening technology for discovery of novel drug targets. This work led to the identification of a previously unknown angiogenic factor and the development of a neutralizing monoclonal antibody. She is proficient in animal models of ocular pathology to understand and prevent disease. Following her Ph.D., Michelle completed her Postdoctoral Fellowship from Harvard University Department of Ophthalmology where she identified novel targets for pathogenic ocular neovascularization and leveraged biologics, mRNA, and gene editing tools for target inhibition in vivo. Prior to joining Saliogen, Michelle led the retina biology program at Generation Bio, focusing on non-viral delivery of nucleic acid cargos to the retina for inherited retinal diseases and conducted the company’s first nonhuman primate study for lipid nanoparticle delivery to the retina.

Michelle received her undergraduate degree in Biology from Syracuse University and her Ph.D. in Molecular and Cellular Pharmacology from the University of Miami Miller School of Medicine, Bascom Palmer Eye Institute.

Oleg Iartchouk

VP, Genomics & Biomarkers

Oleg Iartchouk

VP, Genomics & Biomarkers

Dr. Iartchouk is a technological leader specializing in genomics technologies applications and biomarkers profiling. With an extensive background in the field, he brings a wealth of experience and expertise to his current role at Saliogen.

Prior to his position at Saliogen, Dr. Iartchouk served as the Global Head of Genomics/Analytical Sciences at Novartis for nearly a decade. In this capacity, he led the Genomics Platform Group, which was dedicated to maintaining cutting-edge genomics expertise at Novartis/NIBR. Under his guidance, the group collaborated closely with biologists, striving to uncover new targets, biomarkers, and signatures in their pursuit of scientific discovery.

Before his tenure at Novartis, Dr. Iartchouk held the position of Director of Research Genomics and Genotyping at the Harvard Partners Center of Personalized Genomics Medicine.

Dr. Iartchouk's career also includes establishing and successfully leading several genomics platform groups at prominent organizations such as Sanofi/Oncology and Millennium/Cereon.

Educationally, Dr. Iartchouk earned his Ph.D. in Molecular Biology from Moscow State University, showcasing his strong foundation in the core principles of his field. Furthermore, he pursued postdoctoral training at renowned institutions, including Ecole Normal and Harvard University, refining his expertise and deepening his understanding of molecular biology.

Jungyeon Hwang

Jungyeon Hwang

Sr. Director, Drug Delivery

Jungyeon Hwang

Sr. Director, Drug Delivery

Jungyeon Hwang joined SalioGen Therapeutics as Sr. Director, Drug Delivery and brings significant experiences in formulation development and drug delivery technologies of both small molecule drugs and nucleic acids in large pharmaceutical and biotech sectors to the company. His most recent role was the Head of Formulations at PureTech Health, where he oversaw the development of oral delivery platforms for gene therapy. Prior to joining PureTech Health, he held the position of Associate Research Fellow at Pfizer, where he led the Novel Nanomaterial Group, focusing on nanomaterials and formulation development for immuno-oncology applications within the Oncology Research Unit. Earlier in his career, he occupied various scientific roles at drug delivery companies, progressively taking on more responsibilities and making significant contributions to advancing multiple cancer nanomedicine candidates from laboratory development to clinical stages. He received his undergraduate degree in Chemistry from the University of California, Davis. He obtained his Ph.D. from the University of California, Santa Barbara, and completed his Postdoctoral fellowship at University of California, Los Angeles.

Rob Lyng

Rob Lyng

Executive Director, Process Development & Analytical Science

Rob Lyng

Executive Director, Process Development & Analytical Science

Rob is a Process Development & Analytical Sciences team leader with 19 years of experience in CMC drug substance and drug product development. He comes with broad drug modality experience, including mRNA-based therapeutics, lipid nanoparticles, enzyme replacement, and antibodies. He was most recently at Omega Therapeutics, where he oversaw a team chartered to design and develop GMP purification processes and analytical methods in support of their novel therapeutic platform based on epigenetic regulation. Prior to Omega, Rob was an Associate Director of Analytical Development at Alexion Pharmaceuticals working on their FDA approved commercial products Soliris™, Ultomiris™, and Strenziq™. He got his start in CMC development at Synageva BioPharma where he was the lead process scientist responsible for the development of the GMP purification process for Kanuma™, an FDA approved enzyme replacement therapy.

Rob earned his Ph.D. in Biochemistry, Cell, and Molecular Biology from Emory University and his Bachelor of Arts in Biochemistry from Harvard University.

Pat Sacco

Pat Sacco

Senior Vice President of Manufacturing, Quality, and Operations

Pat Sacco

Senior Vice President of Manufacturing, Quality, and Operations

Pat is highly experienced as a biotechnology and life sciences executive in technical and general operations, including manufacturing, supply chain, CMC, quality, process sciences and engineering. Most recently, as an independent consultant, he has worked with a number of advanced therapy medicinal product (ATMP) biotech companies, CDMOs and specialized program management clients. Previously, he was the Senior Vice President of Technical Operations at both Translate Bio and Shire (now Takeda), and prior to that held roles of increasing responsibility at Wyeth Biopharma (formerly Genetics Institute) and Genzyme. He has contributed to the development, manufacturing and commercialization of the enzyme replacement therapies, REPLAGAL®, ELAPRASE®, and VPRIV®.

Pat holds a Bachelor of Science degree in Chemical Engineering and Biotechnology from Worcester Polytechnic Institute (WPI). He also serves as a board member of NewAge Industries Inc., QbDVision Inc., and as an advisory board member of Vernal Biosciences Inc.

Board of Directors

Jason Cole

CEO & Chairman, Board of Directors

Joseph J. Higgins, M.D.

President & Chief Innovation Officer | Board member

Sukumar Nagendran, M.D.

Board Member

Sukumar Nagendran, M.D.

Board Member

Suku has more than 30 years of experience in key functional areas, including gene therapy development, clinical development strategy, medical affairs, diagnostics, payer strategy and commercialization of therapeutic products. He currently serves as the President of R&D at Jaguar Gene Therapy. Previously, he was Chief Medical Officer & Senior Vice President of AveXis Inc., prior to the company’s acquisition by Novartis. There, he was responsible for overseeing and driving all clinical development and medical affairs strategy, notably for the company’s late-stage AAV-mediated gene therapy program for Spinal Muscular Atrophy (SMA). Prior to AveXis, Suku held key leadership positions at Pfizer, Novartis, Daiichi Sankyo, Reata Pharmaceuticals and Quest Diagnostics. During his tenures at these companies, Dr. Nagendran played significant roles in several high-profile product launches and across multiple functions, including clinical trials/operations, field medical, medical product teams, pricing and reimbursement, publications and advocacy. He was also instrumental in building the medical affairs department at Quest Diagnostics, one of the largest lab/diagnostics providers in the world. Suku is a board member of Solid Bio and Taysha Gene Therapies, as well as an advisor for Neurogene, Medocity, Encoded Therapeutics, ANZU Medical and Prognos. Previously, he was a board member and advisor to two SPAC’s, HSACU and Chardan.

Prior to moving to industry, Suku practiced internal medicine, with a focus on diabetes and cardiovascular disease. He is a Mayo Alumni Laureate and founding member of the Robert Wood Johnson Legacy Society. He is also the sponsor for the Fonseca-Nagendran Scholar award at the American Diabetes Association (ADA) to enhance research in minority populations and the sponsor of the Jerry Mendell translational medicine award at ASGCT. Suku received his undergraduate degree in Biochemistry at Rutgers University and his medical degree at Rutgers Medical School. He trained in Internal Medicine at Mayo Clinic, Rochester. He has was awarded the Distinguished Alumni Award at Rutgers Medical School in 2020.

Sean Stalfort

Board Member

Sean Stalfort

Board Member

Sean is the President of PBM Capital where he sources and structures new investment opportunities, oversees portfolio companies and manages the daily operations of the company. He has been an investor and, on the Board/Board observer of AveXis (sold to Novartis), ArcherDx (sold to Invitae), Dova (sold to SOBI), Verrica (NASDAQ: VRCA), Taysha (NASDAQ: TSHA), and Acumen Pharmaceuticals (NASDAQ: ABOS) as well as current portfolio companies including Xalud Therapeutics, Candel Therapeutics and Lexeo Therapeutics. Prior to PBM Capital, Sean was the Executive Vice President for New Business Development and M&A for PBM Products where he sourced and managed new growth opportunities internationally as well as various capital market activities through the sale of the company to Perrigo for over $800 million. Earlier in his career, Sean worked at several investment firms including Greenwich Capital Markets and CS First Boston.

Sean graduated from Brown University with a B.A. in Business Economics and Political Science.

Scientific Advisory Board

Nancy L. Craig, Ph.D. | Johns Hopkins University School of Medicine

Chair, Scientific Advisory Board

Beverly Davidson, Ph.D. | Children's Hospital of Philadelphia; Perelman School of Medicine at the University of Pennsylvania

Beverly Davidson, Ph.D.

Dr. Davidson is the Chief Scientific Strategy Officer, the Arthur V. Meigs Chair in Pediatrics, and the Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia (CHOP). She is Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine of the University of Pennsylvania (UPENN). Her research group at CHOP is focused on inherited genetic diseases that cause central nervous system dysfunction. In 2013, she co-founded the gene therapy company Spark Therapeutics, and in 2016 she was a co-founder of Talee Bio (now Spirovant), a biotech company applying gene therapy to treat cystic fibrosis and other genetic lung diseases.

Among other awards, Dr. Davidson received the Hereditary Disease Foundations’ Leslie Gehry Prize for Innovation in Science in 2015, and she was elected to the American Academy of Arts and Sciences in 2017 and the National Academy of Medicine in 2019. She is president-elect of the American Society of Gene and Cell Therapy, the largest international association of gene and cell therapy research.

Ellis J. Neufeld, M.D., Ph.D. | St. Jude Children’s Research Hospital

Ellis J. Neufeld, M.D., Ph.D.

Dr. Neufeld, an esteemed hematologist, is the Clinical Director and Physician-in-Chief at St. Jude Children’s Research Hospital, where he holds the John and Lorine Thrasher Chair in Pediatric Medicine. He previously, he served on the faculty of Harvard Medical School for 26 years. Before coming to St. Jude in 2017, he was Associate Chief of the Division of Hematology/Oncology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Medical Director at the Boston Hemophilia Center, and Professor of Pediatrics at Harvard Medical School.

Dr. Neufeld has worked as a physician-scientist in the laboratory, studying the genetic basis of inherited blood disorders, and as a clinical researcher in hematology with numerous publications in the areas of hemophilia, thalassemia, and immune thrombocytopenia.

He received his medical degree and Ph.D. from the Washington University School of Medicine in St. Louis, Missouri.

Jesse B. Owens, Ph.D. | University of Hawaii John A. Burns School of Medicine

Jesse B. Owens, Ph.D.

Dr. Owens is an Assistant Professor at the University of Hawaii at the Institute for Biogenesis Research and John A. Burns School of Medicine. Dr. Owens’ lab is currently investigating new tools for improved genome editing, specifically developing vectors that use transposons and recombinases to enable safer and more efficient gene delivery to diverse tissues.

Matthew H. Wilson, M.D., Ph.D. | Vanderbilt University Medical Center

Thomas Quertermous, M.D.

Dr. Quertermous is a noted cardiovascular geneticist currently serving as the William G. Irwin Professor of Medicine in the Division of Cardiovascular Medicine at Stanford University. His research group employs large-scale human genetics studies to characterize the genetic basis of atherosclerosis, and related risk factors such as hypertension and insulin resistance. He is also the founder and director of Cardiovascular Metabolic Consulting.

Dr. Quertermous established an independent laboratory in the Cardiac Unit at Massachusetts General Hospital in 1987. He was recruited to Vanderbilt University in 1991 as Chief of Cardiology and Professor of Medicine and Molecular Physiology and Biophysics. Dr. Quertermous moved to Stanford University in 1997, where he assumed leadership of the Division of Cardiovascular Medicine. He completed clinical training in cardiology at the Massachusetts General Hospital and research training in molecular genetics in the Department of Genetics at the Harvard Medical School.

D. Holmes Morton, M.D. | Recipient of the Albert Schweitzer Prize for Humanitarianism and Founding Pediatrician, Clinic for Special Children and Central Pennsylvania Clinic

Thomas Quertermous, M.D.

Dr. Quertermous is a noted cardiovascular geneticist currently serving as the William G. Irwin Professor of Medicine in the Division of Cardiovascular Medicine at Stanford University. His research group employs large-scale human genetics studies to characterize the genetic basis of atherosclerosis, and related risk factors such as hypertension and insulin resistance. He is also the founder and director of Cardiovascular Metabolic Consulting.

Dr. Quertermous established an independent laboratory in the Cardiac Unit at Massachusetts General Hospital in 1987. He was recruited to Vanderbilt University in 1991 as Chief of Cardiology and Professor of Medicine and Molecular Physiology and Biophysics. Dr. Quertermous moved to Stanford University in 1997, where he assumed leadership of the Division of Cardiovascular Medicine. He completed clinical training in cardiology at the Massachusetts General Hospital and research training in molecular genetics in the Department of Genetics at the Harvard Medical School.

Executive Advisory Board

Mathai Mammen, M.D., Ph.D. | Founder, Theravance; formerly EVP of Pharmaceuticals, R&D at Johnson & Johnson; formerly SVP, R&D at Merck

John Maraganore, Ph.D. | Founding Chief Executive Officer (CEO) and director of Alnylam Pharmaceuticals

Mark McClellan, M.D., Ph.D. | The Robert J. Margolis Professor of Business, Medicine, and Policy, and founding Director of the Duke-Margolis Center for Health Policy at Duke University, former commissioner of the U.S. Food and Drug Administration (FDA) and former administrator of the Centers for Medicare & Medicaid Services (CMS)

Advisors

Brian Hew, Ph.D. | University of Hawaii

Brian Hew, Ph.D.

Dr. Hew is a researcher specializing in molecular biology at the University of Hawaii. Previously, he was a lecturer at Kapiolani Community College, a research scientist at Black Ivory Biotech, and a research technician and graduate assistant at the Cancer Research Center of Hawaii. He received his undergraduate degree at the University of Washington, and his Ph.D. in Cellular and Molecular Biology at the University of Hawaii at Manoa.

Andrew Knudten, M.S., MBA | Jaguar Gene Therapy

Andrew Knudten, M.S., MBA

Mr. Knudten serves as chief operating officer at Jaguar Gene Therapy. He previously occupied the positions of chief technical officer at Cirius Therapeutics, Inc., operations director at Amgen, Inc., global head of contract manufacturing at Novartis AG and chief technical officer at AveXis, Inc. (now Novartis Gene Therapy), where he led development and commercialization of Zolgensma® from a manufacturing, process development and supply chain perspective.

Mark E. Pennesi, M.D., Ph.D. | Casey Eye Institute, Oregon Health & Science University

Mark E. Pennesi, M.D., Ph.D.

Dr. Pennesi is an inherited retinal degeneration specialist at the Casey Eye Institute, Oregon Health & Science University, where he leads the institute’s gene therapy programs. He serves as the Kenneth C. Swan associate professor in ophthalmic genetics and as the chief of the Paul H. Casey Ophthalmic Genetics Division, where his research focuses on developing novel treatments for inherited retinal diseases.

Both Research to Prevent Blindness and the Foundation Fighting Blindness have recognized Dr. Pennesi with career development awards to support his ambition to conduct transitional research that will bring treatments for diseases such as retinitis pigmentosa from the laboratory to the clinic.
Dr. Pennesi completed a residency at University of California, San Francisco and a combined M.D./Ph.D. at Baylor College of Medicine in Houston, Texas.

Renee Ryals, Ph.D. | Casey Eye Institute, Oregon Health & Science University

Renee Ryals, Ph.D.

Dr. Ryals is a Research Assistant Professor in the department of Ophthalmology at the Casey Eye Institute, Oregon Health & Science University. She has a joint appointment in the Molecular and Medical Genetics department at OHSU. Dr. Ryals has been studying inherited retinal degenerations for over ten years and has authored 19 peer-reviewed publications to date.

Gaurav Sahay, Ph.D. | Oregon State University

Gaurav Sahay, Ph.D.

Gaurav Sahay is an associate professor in the Department of Pharmaceutical Sciences, College of Pharmacy at Oregon State University. Dr. Sahay’s lab is developing novel nanotechnology-based platforms including lipid-based nanoparticles for effective delivery of messenger RNA therapeutics for treatment of cystic fibrosis, retinal degeneration and against SARS-CoV-2. He has done pioneering work to dissect the intracellular transport essential for nucleic acid delivery to the cytosol and developed methods to overcome endosomal barriers. Dr. Sahay has 48-peer-reviewed publications in top tier journals including Science Advances, Nature, Nature Communications, Nature Biotechnology, Nature Nanotechnology, Journal of Controlled Release and Nano Letters. Dr. Sahay is the winner of a 2013 American Association of Pharmaceutical Scientists (AAPS) Postdoctoral Fellow Award, the 2015 Controlled Release Society (CRS) T. Nagai Award, a 2016 American Association of Colleges of Pharmacy (AACP) New Investigator Award, a 2019 Oregon Health & Sciences University (OHSU) Distinguished Faculty Senate Award for Collaboration, 2020 Phi Kappa Phi OSU Emerging Scholar Award and 2020 CMBE Young Innovator Award. Sahay lab is funded through the National Institutes of Health, Cystic Fibrosis Foundation, and biotech companies. He serves as a consultant and scientific advisory board member to several biotech and venture capital firms and Gaurav was the Chair of the 2018 NanoMedicine and Drug Delivery Symposium (NanoDDS, Portland, OR) and is current Chair of CRS GDE Focus Group. Dr. Sahay completed his postdoctoral research with Prof. Robert Langer and Prof. Daniel Anderson at the Koch Institute for Integrative Cancer Research at MIT and received his Ph.D. from the University of Nebraska Medical Center under the mentorship of Prof. Alexander Kabanov.

Troy Short | Jaguar Gene Therapy

Troy Short

Mr. Short is currently the vice president of manufacturing and supply chain at Jaguar Gene Therapy. Previously, at AveXis, Inc. (now a subsidiary of Novartis AG), he served as the vice president of supply chain, building the global supply chain for the gene therapy product for spinal muscular atrophy now marketed as Zolgensma®. He was also the director of supply chain at Pfizer, where he managed all activities related to supply chain for the company’s McPherson, Kansas site. Mr. Short received his Bachelor of Science degree in mechanical engineering at Kansas State University.

Wayne Tschetter, Ph.D. | Casey Eye Institute, Oregon Health & Science University

Wayne Tschetter, Ph.D.

Dr. Tschetter is a senior research associate at the Casey Eye Institute, Oregon Health & Science University. Prior to joining the Casey Eye Institute, he was an Associate Professor of Biology at Concordia University in Portland. He received his undergraduate degree in Neuroscience from the University of Lethbridge in 2006, and his Ph.D. in Physiology, Biophysics and Systems Biology from Cornell University, Weill Medical College.

Matthew Wilson, M.D., Ph.D. | Vanderbilt University

Matthew Wilson, M.D., Ph.D.

Dr. Wilson is an associate professor of medicine at Vanderbilt University Medical Center. His clinical expertise is in treating patients with chronic kidney disease, and his research group is focused on introducing new therapeutics approaches harnessing the biology of transposons for gene transfer for preventing kidney disease progression and complications.

Dr. Wilson earned his medical degree and his Ph.D. in pharmacology from Vanderbilt University.

Jonathan Yen, Ph.D. | St. Jude Children’s Research Hospital

Jonathan Yen, Ph.D.

Jonathan Yen is a director in the Department of Hematology at St. Jude Children’s Research Hospital in Memphis, Tennessee. He received his Bachelor of Science degree in biomedical engineering from Johns Hopkins University and his Ph.D. in bioengineering at the University of Illinois at Urbana Champaign. His postdoctoral training took place in the Engineering Therapeutics group in the Chemical Biology & Therapeutics at the Novartis Institutes for BioMedical Research. He worked on the application and development of non-viral delivery modalities and CRISPR-Cas9 for the treatment of sickle cell disease. After his postdoctoral training, he joined Beam Therapeutics as a scientist to develop the therapeutic application of base editors for the treatment of hemoglobinopathies. He is currently at St. Jude Children’s Research Hospital to continue to develop and apply novel genome editing tools and new delivery modalities for the treatment of sickle cell disease.

Collaborators

Frederick Dyda, Ph.D.

Jaguar Gene Therapy

Frederick Dyda, Ph.D.

Dr. Dyda is a senior investigator at the Laboratory of Molecular Biology of the National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health in Bethesda, Maryland. His scientific areas of focus include molecular biology, biochemistry and structural biology. Currently, his research at the NIH is focused on molecular and structural biology of mobile genetic elements. Dr. Dyda received his undergraduate education at Eötvös Lóránd University and his Ph.D. at the University of Pittsburgh in 1992.

Alison B. Hickman, Ph.D.

National Institutes of Health

Alison B. Hickman, Ph.D.

Dr. Hickman is a staff scientist in Dr. Frederick Dyda’s research group at the Laboratory of Molecular Biology of the National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health in Bethesda, Maryland. Dr. Hickman received her Bachelor of Science degreefrom McGill University, and her Ph.D. at the Massachusetts Institute of Technology in 1990.

Investors

General Inception
Superstring Capital
Yonjin Venture
SSI Accelerator