Unleash the full potential of genetic science for patients

Founded with the vision of unleashing the full potential of genetic science, Skyline Therapeutics brings together a world-class team of experts and leaders in science, technology, and business to tackle some of today’s toughest scientific challenges.

The Skyline team’s capabilities span capsid discovery, vector engineering and design, in-vivo pharmacology, clinical development, analytical and process development, and CMC.

2019 Established
10+ Pipeline Programs
3 Global Sites
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Amber Cai, Ph.D., MBA Chief Executive Officer

Dr. Amber Cai is the Founder and Chief Executive Officer of Skyline Therapeutics. She has decades of leadership experience in the biopharmaceutical industry, including as a founding member and head of operations for Novartis’ research and development center in Shanghai, the first fully integrated multinational R&D center in China. Dr. Cai played an instrumental role in building the company’s China team and operations, as well as in developing Novartis’ China R&D strategy and external alliances.

Dr. Cai holds a Ph.D. in Biochemistry from the Massachusetts Institute of Technology (MIT), an MBA from MIT’s Sloan School of Management and a B.S. in Microbiology from Fudan University.

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Jay Hou, Ph.D. Chief Scientific Officer

Dr. Jay Hou is the Chief Scientific Officer of Skyline Therapeutics. He brings nearly three decades of experience in protein engineering, molecular/cellular biology, and biochemistry to the role. A molecular and cellular biologist by training, Dr. Hou has a solid track record of accomplishments in the development of innovative medicines in both biologics (AAV gene therapy, protein, and antibody) and small molecules. His expertise covers a number of therapeutic areas including metabolic diseases, neurodegenerative diseases, and cancer. Dr. Hou joined Skyline from the Boston-based gene therapy company Voyager Therapeutics, where he successfully built original AAV gene therapy research and platform capabilities.

Dr. Hou holds a Ph.D. in Cellular/Molecular Biology from Clarkson University, as well as a B.S. degree in Chemical Engineering from Tsinghua University.

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Ling Su, Ph.D. Chief Development Officer

Dr. Ling Su is the Chief Development Officer of Skyline Therapeutics. He has more than 30 years of experience in pharmaceutical regulatory and clinical development, having previously served as a senior executive for multinational pharmaceutical companies including Merck & Co., Roche, Wyeth and Novartis. He has successfully built and led pharma clinical development organizations across China and overseen numerous global clinical trials and drug development programs in China and the Asia Pacific region. Earlier in his career, Dr. Su worked in China’s Bureau of Drugs of the Ministry of Health (the predecessor of the National Medical Products Administration) and in the U.S. FDA’s Center for Drug Evaluation and Research (CDER). He was elected the President of the Drug Information Association (DIA) Board of Directors for 2012-2013.

Dr. Su is a professor and Research Fellow at the Yeehong Business School at Shenyang Pharmaceutical University and is an inaugural member of the Biotech Expert Advisory Panel for the Hong Kong Stock Exchange.

Dr. Su holds a Bachelor’s degree in Pharmacology from Shanghai Medical University, a Ph.D. in Epidemiology and a Master’s degree in Clinical Drug Development from the University of North Carolina at Chapel Hill.

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Michael Sun, Ph.D. Chief Operations Officer

Dr. Sun is the Chief Operations Officer of Skyline Therapeutics. He brings decades of experience in business and science, across different types of organizations in the industry. His experience and leadership have been focused on developing corporate infrastructure and operational systems, delivering effective and efficient business support to help companies succeed.

Prior to joining Skyline, Dr. Sun spent a decade at ThermoFisher Scientific, where he led the company’s Service and Support function and the Strategic Account Group for the Genetic Systems Division in Greater China. Before joining ThermoFisher, Dr. Sun was Head of Research and Senior Vice President at Shanghai Genomics, where he led the company’s internal drug discovery program and external collaboration.

Dr. Sun received a B.S. in Genetics and Genetic Engineering from Fudan University in Shanghai. He received a Ph.D. in Biochemistry from Rutgers University and trained as a postdoc fellow at Genentech in South San Francisco.

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Gurpreet Ratra, Ph.D. Chief Business Officer

Dr. Ratra is the Chief Business Officer of Skyline Therapeutics. He has extensive gene therapy industry experience and ​a strong track record in business development and partnerships. He joined Skyline from Spark Therapeutics, where he served as the Vice President of Business Development.

Prior to Spark, Dr. Ratra worked in business development at Adverum Biotech, ​leading a number of in-licensing and out-licensing deals with academic and biotech partners, including a merger between Avalanche Biotech and Annapurna Therapeutics to create Adverum Biotechnologies. 

Dr. Ratra holds a Ph.D. ​in Biochemical Toxicology ​from St. Bartholomew’s Medical School in London and an MBA from the University of California Berkeley.

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Ping Li, Ph.D. Chief Manufacturing Officer

Dr. Ping Li is the Chief Manufacturing Officer of Skyline Therapeutics. She has more than two decades of industry experience in innovative product development and registration for global markets, as well as in overall CMC development including process R&D, manufacturing, and regulatory submissions.

Formerly Dr. Li held various roles at Novartis US and China. She was the Global Program Head for a metabolic portfolio program responsible for its CMC, preclinical, and clinical development, as well as for its global market access and authorization. She was also the Head of Technical R&D (China) at Novartis, leading the CMC function to develop innovative product designs, robust and scalable manufacturing processes and controls that supported the manufacturing and supply of drug materials for global clinical studies, as well as the successful technical transfer to product launch sites for commercialization.

Dr. Li holds a Ph.D. in Pharmaceutical Sciences from the University of Arizona.

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Wen Chang, Ph.D. Head of Regulatory Affairs and Development Strategy

Dr. Chang is the Head of Regulatory Affairs and Development Strategy at Skyline Therapeutics. She has decades of industry experiences in regulatory and development work across the globe, including leading regulatory and clinical pharmacology efforts at several multinational companies including Amgen, BMS, and Novartis.

In her most recent role as Head of China Regulatory and JAPAC (Japan, AP, Australia, and China) Strategy at Amgen, Dr. Chang spearheaded the regulatory team and achieved several landmark market authorization approvals in China. Prior to her time at Amgen, she held several senior executive positions on the global regulatory team and in global clinical pharmacology at BMS and Novartis, USA.

Dr. Chang holds a Ph.D. in Biopharmaceuticals from the School of Pharmacy at the State University of New York, and a B.S. in Pharmacy from Taipei Medical College.

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Thaddeus (Ted) P. Dryja, M.D.

Dr. Thaddeus (Ted) P. Dryja, M.D. is a Professor of Ophthalmology at Harvard Medical School and an Associate Director of Eye Pathology at the Massachusetts Eye and Ear Infirmary.

Dr. Dryja joined the faculty of the Department of Ophthalmology at Harvard Medical School in 1983 and led research in the molecular genetics of inherited retinal diseases (IRDs), as well as practicing general ophthalmology and ophthalmic pathology. In 1992, he became a full professor, in 1993, the David Cogan Professor at Harvard, and in 1996, a National Academy of Sciences member. Dr. Dryja’s research accomplishments include the discovery of the recessive nature of oncogenic mutations at tumor suppressor genes, the first cloning of the retinoblastoma gene, and the identification of 16 IRD genes.

He became the Head of Translational Medicine in Ophthalmology at the Novartis Institutes for Biomedical Research in 2006, and from 2009-2017 served as the Global Head of Ophthalmology Research at Novartis. During his time at Novartis, Dr. Dryja led teams that initiated drug discovery projects and brought many topical and systemic drugs to phase I trials, including low-molecular weight and biologic drugs and for indications including glaucoma, dry eye, uveitis, diabetic retinopathy, and both advanced forms of age-related macular degeneration (neovascularization and geographic atrophy). He also initiated and led a gene therapy program for an IRD (RLBP1 retinal degeneration) that reached phase I soon after his retirement from Novartis in 2017. One of the drugs for neovascular macular degeneration is now approved by the FDA (brolucizumab).

Dr. Dryja received his medical degree from the Yale University School of Medicine. He received residency training in Ophthalmology at Harvard Medical School and completed doctoral fellowships in Pathology at Massachusetts Eye and Ear, and in Genetics at Boston Children's Hospital. He currently works as an attending eye pathology in the Cogan Eye Pathology Laboratory at the Massachusetts Eye and Ear.

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Mitchell H. Finer, Ph.D.

Dr. Mitchell H. Finer has spent three decades building companies in the areas of regenerative medicine, cell-based cancer immunotherapy, and in vivo viral and non-viral gene therapy, developing products from conception through phase III development and market launch. To date, he has developed three cell therapy products marketed in the U.S. and Europe.

Dr. Finer is currently serving as the President of Research and Development at Elevate Bio, as well as Chief Executive Officer of Life Edit Inc., a wholly owned subsidiary of Elevate Bio since 2020. He co-founded Elevate Bio in 2018 and served as the Chief Scientific Officer & President of Elevate Bio Basecamp from 2018 to 2020. He has also served as an Executive Partner at MPM Capital since 2015. Currently, Dr. Finer is a member of the board of directors at Abata Therapeutics, Tabby Therapeutics, Turmeric Acquisition Holdings and Coda Biotherapeutics. He also serves as Chairman of the Board for Oncorus (ONCR), where he previously served as founder & CEO and most recently as Executive Chairman.

Prior to Elevate Bio, Dr. Finer served as the CSO of Bluebird Bio, CEO of Intracel Corporation and Genteric Inc, and as Vice President of Research for Cell Genesys and the Gencell division of Aventis Pharma (now Sanofi). Mitch also successfully co-founded the retinal disease gene therapy company Avalanche Biotechnologies, now Adverum, Oncorus and Coda Biotherapeutics. He has previously served on the boards of Semma Therapeutics, TCR2 (TCRR), and Adverum (ADVM).

Dr. Finer received his Ph.D. in Biochemistry and Molecular Biology from Harvard University and a B.S. in Biochemistry and Microbiology from the University of California at Berkeley. Dr. Finer completed a post-doctoral fellowship at the Whitehead Institute for Biomedical Research at MIT.

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Steven K. Galson, MD, MPH

Dr. Steven K. Galson, M.D., M.P.H., is a consultant to Amgen and serves on the Board of Directors of Insilico Medicine and Biocryst Pharmaceuticals. Until June 2020, Dr. Galson was a Senior Vice President of Research and Development at Amgen. He joined the company in 2010 as Vice President of Global Regulatory Affairs. Prior to Amgen, Dr. Galson was Senior Vice President for Civilian Health Operations and Chief Health Scientist at Science Applications International Corporation.

Galson spent more than 20 years in government service, including two years as Acting Surgeon General of the United States. Previously, he served as director of the FDA’s Center for Drug Evaluation and Research (CDER), where he provided leadership for the center’s broad national and international programs in pharmaceutical regulation.

Dr. Galson began his public health service career as an epidemiological investigator at the Centers for Disease Control and Prevention (CDC) after completing a residency in internal medicine at the Hospitals of the Medical College of Pennsylvania. He also held senior-level positions at the Environmental Protection Agency; the Department of Energy, where he was chief medical officer; and the Department of Health and Human Services.

Dr. Galson holds a bachelor’s degree from Stony Brook University, an MD from Mt. Sinai School of Medicine, and an MPH from the Harvard School of Public Health. Dr. Galson is a Trustee of the Keck Graduate Institute and a founding Governor of the KGI School of Community Medicine. In 2008, he received an Honorary Doctor of Public Service degree from Drexel University School of Public Health, and in 2015, he received the Jacobi Medallion Award from Icahn Mount Sinai School of Medicine. In 2018, he was named Health Leader of the Year by the Commissioned Officers Association of the United States Public Health Service. Dr. Galson is on the Executive Committee of the Clinical Trial Transformation Initiative and is a Member of the Forum on Drug Discovery, Development, and Translation at the National Academy of Medicine.

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Mark A. Kay, M.D., Ph.D.

Dr. Mark Kay is the Dennis Farrey Family Professor in the Departments of Pediatrics and Genetics, as well as Head of the Division of Human Gene Therapy in Pediatrics at the Stanford University School of Medicine.

Before joining Stanford, Dr. Kay was an assistant/associate professor at the University of Washington in the Department of Medicine from 1993-1998. He has been a true bench-to-bedside physician-scientist, leading the first demonstration of therapeutic rAAV transduction of the liver in small and large animal models, working out the molecular process of AAV transduction in vivo, and playing a major role in developing a clinical trial representing the first systemic delivery of rAAV in humans.

Dr. Kay is one of the founders of the American Society of Gene Therapy and served as the President from 2005-2006. He has organized many national and international conferences, including the first Gordon Conference related to gene therapy. He was elected to the American Academy of Pediatrics in 2010. In 2020 Dr. Kay was elected to the National Academy of Inventors.

Dr. Kay received his medical degree and PhD from Case Western Reserve University and completed a residency in pediatrics and a fellowship in medical genetics and inborn errors of metabolism. He completed his postdoctoral research at Baylor College of Medicine.

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Adrian Thrasher, Ph.D.

Dr. Adrian Thrasher is a Professor of Pediatric Immunology at the UCL Great Ormond Street Institute of Child Health, London.

Trained initially in adult medicine, he completed clinical academic training scheme funded by the Wellcome Trust initially a Research Fellowship that led to a Ph.D. in molecular immunology and gene therapy. Dr. Thrasher then won a clinician scientist award, which allowed him to continue his research career while training in pediatrics, before receiving a highly prestigious principal research fellowship.

Dr. Thrasher quickly established himself as a leader of basic science gene therapy research, concentrating on the restoration of function in immunodeficiency diseases. He has played a leading role in defining the basic cytoskeletal defect in hematopoietic cells in Wiskott-Aldrich Syndrome and was the first to correct by gene transfer the underlying molecular defect in chronic granulomatous disease.

Dr. Thrasher has led the haematopoietic stem cell gene therapy program at Great Ormond Street Hospital for Children, which was the first institution in the UK to achieve the successful restoration of function and recovery of child patients with severe combined immunodeficiency. Dr. Thrasher has a respected global reputation and is a frequent keynote speaker at major gene therapy and immunology conferences. He is widely regarded as one of the leaders in gene therapy research worldwide.

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David A. Williams, M.D.

Dr. David A. Williams, M.D. is the Leland Fikes Professor of Pediatrics at Harvard Medical School, Chief of Hematology/Oncology at Boston Children's Hospital, and Associate Chair of Pediatric Oncology at the Dana-Farber Cancer Institute.

An internationally renowned researcher, Dr. Williams' work focuses on blood stem cell biology, leukemia, and gene therapy to correct genetic blood disorders.

Dr. Williams has won numerous prestigious awards for his research in the area of hematology. He is the recipient of the American Society of Gene and Cell Therapy’s Outstanding Achievement Award for his work in gene therapy in 2012. He is a member of the National Academy of Medicine and has served as President of both the American Society of Hematology and the International Society of Experimental Hematology. He co-founded the Transatlantic Gene Therapy Consortium and the North American Pediatric Aplastic Anemia Consortium and is a co-founder of Orchard Therapeutics and Alerion Biosciences. He has been continuously funded by the NIH since 1986 and was an investigator of the Howard Hughes Medical Institute for 16 years. He has multiple patents including those leading to Neumega™ and Retronecin™ drugs.

Dr. Williams was originally trained in pediatric hematology/oncology at Boston Children’s Hospital and Dana-Farber Cancer Institute. During his fellowship research at the MIT Cancer Center and the Whitehead Institute, he developed techniques that allowed for the introduction of genes into murine and human hematopoietic cells. Those techniques are still commonly utilized today.