At Nightstar, our mission is to maintain and restore sight in patients with inherited retinal diseases.
We are a clinical-stage company focused on developing and commercializing a pipeline of novel and potentially curative, one-time retinal gene therapies for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and, for which, there are no currently approved treatments.
Our lead retinal gene therapy product candidate, NSR-REP1, is being developed for the treatment of choroideremia (CHM), a rare, degenerative, X-linked genetic retinal disorder primarily affecting males that is caused by a mutation in the CHM gene.
We have an ongoing Phase 3 registrational clinical trial, known as the STAR trial, of NSR-REP1 for CHM. We anticipate that STAR study will be fully enrolled by the first half of 2019 and expect the one-year follow-up results of the STAR trial to be available in 2020. We are also currently conducting a prospective, natural history study, known as the NIGHT study, across multiple clinical sites in the United States, Europe and Canada. NSR-REP1 has been granted orphan drug designation for the treatment of CHM from both the U.S. Food and Drug Administration and the European Medicines Agency.
We are developing NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP), an inherited X-linked recessive retinal disease characterized by mutations in the RPGR gene, leading to a lack of protein transport and a loss of photoreceptors, the specialized cells in the eye that convert light into visual signals. NSR-RPGR is currently being evaluated in a dose-ranging Phase 1/2 clinical trial for the treatment of XLRP, known as the XIRIUS trial. We expect the initial data on safety and tolerability from the dose escalation cohorts of this trial to be available towards the end of 2018; this data will determine the dose for study in an expansion cohort. We have also initiated a prospective, natural history observational study, which we refer to as the XOLARIS study, to better understand the progression of untreated XLRP. We have received orphan drug designation for NSR-RPGR for the treatment of XLRP from the European Medicines Agency.
We also have product candidates in preclinical development for a number of inherited retinal diseases for which there are no approved treatments such Stargardt disease and Best vitelliform macular dystrophy, or Best disease. We are evaluating other in-licensing opportunities to broaden our pipeline and drive future growth.
Our team includes a group of seasoned experts who bring a breadth of knowledge on retinal disease drug development, gene therapy, manufacturing and ophthalmic product commercialization.
Chief Executive Officer and Board Member
David has served as chief executive officer of Nightstar and as a member of Nightstar’s board of directors since January 2015 and previously served as a non-executive director of Nightstar from February 2014 to January 2015. Prior to Nightstar, he was vice president of Johnson & Johnson’s Vision Care Franchise where he led the global marketing, new product and licensing activities across the business. Prior to that he spent over 20 years at Allergan, Inc., where he served primarily in the sales and marketing areas in a number of capacities, including regional president, corporate vice president and senior vice president in locations in North America, Europe and Asia.
David received a B.A. from Butler University and is currently a board member of the Glaucoma Foundation.
Chief Development Officer
Tuyen has served as chief development officer of Nightstar since August 2017. Prior to Nightstar, Tuyen served as chief medical officer at PTC Therapeutics Inc. (“PTC”) where he was responsible for advancing the company’s pipeline of product candidates for the treatment of rare diseases. Prior to PTC, Tuyen served as vice president of global clinical development and operations at Bausch and Lomb Inc. (subsequently acquired by Valeant Pharmaceuticals International, Inc.) and played a key role in the company’s filing of new drug and device applications. Prior to that, Tuyen worked at Pfizer Inc. as its global clinical lead across multiple therapeutic disease areas of high-unmet need, most recently in retinal diseases.
Tuyen holds an M.D. from the University College London and an MBA from New York University Stern School of Business. He is a member of the Royal College of Ophthalmologists.
Gregory Robinson, Ph.D.
Chief Scientific Officer
Greg has served as our chief scientific officer since August 2016. Prior to Nightstar, Greg was chief scientific officer at Agilis Biotherapeutics LLC, a company focused on developing gene therapies for rare CNS disorders. Prior to Agilis, he was a member of the discovery research group and served as senior director of scientific licensing evaluating rare disease opportunities at Shire plc. Prior to that, Greg led the biology and drug discovery group at Eyetech Pharmaceuticals Inc. and was involved with the development of treatments for wet age-related macular degeneration (MACUGEN® and FOVISTA®). Earlier in his career, he held positions at Pharmacia Corporation and Hybridon, Inc.
Greg received a B.S. in biology from Macalester College and his Ph.D. in biochemistry from Boston University. He has authored 43 peer-reviewed publications and is an inventor or co-inventor on seven issued patents.
Chief Financial Officer
Senthil has served as chief financial officer of Nightstar since April 2017. Prior to Nightstar, Senthil was vice president and head of business development at Intercept Pharmaceuticals, Inc., where he was responsible for a wide range of activities including business development, strategy, financial analysis, investor relations and capital raising. Prior to Intercept, he spent 13 years in the healthcare investment banking groups at Lehman Brothers/Barclays, Citigroup and Lazard. His transaction experience includes mergers & acquisitions, initial public offerings and other equity and debt financings in the biopharmaceuticals sector.
Senthil received a B.S. in computer engineering and a B.A. in economics from Brown University.
General Counsel and Secretary
Bryan has served as our General Counsel since November 2017 and has acted as our Secretary since February 2018. From January 2013 to November 2017, Mr. Yoon headed up the global legal, compliance and intellectual property functions at Intercept Pharmaceuticals, Inc., where he most recently served as Senior Vice President—Legal Affairs and Secretary. Prior to joining Intercept, Mr. Yoon worked at Mintz, Levin, Cohn, Ferris, Glovsky and Popeo P.C. and Simpson Thacher & Bartlett LLP, where he advised clients, including a number of life sciences companies, on a variety of corporate and other legal matters.
Mr. Yoon holds a B.A. in Economics and an M.Eng. in Operations Research and Industrial Engineering from Cornell University. Mr. Yoon received his J.D. from the University of Michigan Law School.
SVP, Global Head of CMC
Julian has more than 25 years experience in biologics product development with specific expertise in viral and non-viral gene therapy, antibody and recombinant vaccine products. Prior to Nightstar, he served as commercial director at Cobra Biologics, where he managed the international business development and sales team, as well as the project management and commercial technical support teams. Prior to that, Julian was senior vice president of development at Renovo plc, where he was responsible for drug development and outsourcing. He previously held positions of increasing responsibility at Cobra Biologics’ Keele facility for 11 years progressing from senior scientist to director of production. He has also held positions at the National Institute for Medical Research, Bio Products Laboratory Ltd and AstraZeneca. Julian received an Honours Degree in biochemistry from University College London and an MSc at The University College of North Wales.
Mark De Rosch, Ph.D., FRAPS
SVP, Regulatory Affairs and Quality Assurance
Mark De Rosch, Ph.D., FRAPS joined Nightstar as Senior Vice President of Regulatory Affairs and Quality Assurance in April 2018 overseeing the global regulatory and quality organizations. Prior to Nightstar, he was Senior Vice President of Regulatory Affairs, Quality Assurance, and CMC at Akebia Therapeutics, Inc. He also served as Vice President of Regulatory Drugs/Biologics & Head of U.S. Operations for Voisin Consulting Life Sciences, and Vice President and Head of Global Regulatory Affairs for Inspiration Biopharmaceuticals, Inc. Mark also served in multiple key roles at Vertex Pharmaceuticals, Inc., including Senior Director, Global Clinical Regulatory Strategy, where he led the regulatory strategy and submissions process for Kalydeco® in cystic fibrosis leading to approvals in the U.S. and E.U.
Mark earned his B.S. in Chemistry/Biochemistry at the University of Wisconsin – Parkside and his Ph.D. in Inorganic Chemistry at the University of California, San Diego. Mark was named a Fellow of the Regulatory Affairs Professional Society in 2017.
BOARD OF DIRECTORS
Paula has served as a member of Nightstar’s board of directors since September 2018. Ms. Cobb currently serves as the executive vice president of corporate development at Decibel Therapeutics, Inc.
Prior to joining Decibel, she served as senior vice president of the rare disease group at Biogen, where she was responsible for the company’s marketed hemophilia assets and Phase 3 programs in spinal muscular atrophy and neuropathic pain. Prior to this role, she managed the multiple sclerosis franchise, led new product commercialization for early pipeline programs, served as chief of staff to the CEO and held various operational management roles with Biogen in Europe. Before Biogen, she worked for various consulting groups in the Boston area.
Ms. Cobb graduated cum laude from Amherst College with a B.A. in political science and English. She also received an MBA from Harvard University Graduate School of Business Administration.
Chief Executive Officer and Board Member
David has served as chief executive officer of Nightstar and as a member of Nightstar’s board of directors since January 2015 and previously served as a non-executive director of Nightstar from February 2014 to January 2015.
Prior to Nightstar, he was vice president of Johnson & Johnson’s Vision Care Franchise where he led the global marketing, new product and licensing activities across the business. Prior to that he spent over 20 years at Allergan, Inc., where he served primarily in the sales and marketing areas in a number of capacities, including regional president, corporate vice president and senior vice president in locations in North America, Europe and Asia.
David received a B.A. from Butler University and is currently a board member of the Glaucoma Foundation.
Chris Hollowood, Ph.D.
Chairman and Board Member
Chris has served as a member of Nightstar’s board of directors and its chairman since November 2013. He has served as chief investment officer and managing partner of Syncona Partners LLP, Syncona Limited, a leading FTSE250 listed healthcare investor, since December 2016, and previously as a partner from 2012 to 2016.
Prior to Syncona, Chris was a partner of Apposite Capital LLP, a venture and growth capital company focused on the healthcare and life science sector, from 2007 to 2012. Prior to that, he held various roles with Bioscience Managers Ltd. and Neptune Investment Management Ltd.
Chris received a degree in natural sciences and a Ph.D. in organic chemistry, both from Cambridge University.
David has served as a member of Nightstar’s board of directors since July 2017. He is currently the executive vice president and chief financial officer of Ra Pharmaceuticals, Inc.
Prior to that, he served as a member of the senior management team of Tetraphase Pharmaceuticals, Inc., a biotechnology company, from its inception in 2006 through 2015. From 2010 to 2015, David also served as senior vice president and the chief financial officer of Tetraphase, where he led financial operations and was responsible for corporate finance activities. From 1999 to 2005, he served as the chief financial officer of PharMetrics Inc., a pharmacy and medical claims data informatics company. Prior to joining PharMetrics, David was vice president and chief financial officer of ProScript, Inc.
David is a member of the American Institute of CPAs and a Certified Public Accountant in the Commonwealth of Massachusetts. He holds a B.S. in business administration from Northeastern University and an M.S. in taxation from Bentley University.
James McArthur, Ph.D.
James has served as a member of Nightstar’s board of directors since November 2016. He is the founder and president of research and development of Cydan Development, Inc., a rare disease accelerator, and the founder and chief executive officer of Imara, Inc., which develops therapeutics for the treatment of sickle cell disease, since 2016.
James was previously head of research and principal scientist at Cell Genesys Inc., and began his career in gene therapy at Somatix Therapy Corp. He also serves as a member of the Board of Directors and the Scientific Advisory Board of the Friedreich’s Ataxia Research Alliance. James also founded Vtesse, Inc. and Synovex Corporation (renamed Adheron Therapeutics, Inc., and which was subsequently acquired by Roche Holding AG) and has published and patented extensively in the areas of gene therapy and rare diseases.
James was a postdoctoral fellow at the Massachusetts Institute of Technology and the University of California, Berkeley. He holds a Ph.D. in biochemistry from McGill University.
Dave has served as a member of Nightstar’s board of directors since August 2015. He has served as a general partner of New Enterprise Associates, an investment firm focused on venture capital and growth equity investments, since September 2008, where he leads the healthcare investing practice.
From 1992 until 2008, Dave worked at MedImmune Limited, a biotechnology company and subsidiary of AstraZeneca plc, and served in numerous roles during his tenure including from October 2000 to July 2008 as president and chief executive officer, and previously as chief financial officer, and as president and chief operating officer. During that time, he also served as executive vice president of AstraZeneca plc following its acquisition of MedImmune in June 2007. Prior to joining MedImmune, Dave was a vice president in the healthcare investment banking group at Smith Barney, Harris Upham & Co. Inc.
Dave received a B.A. from Dartmouth College. He serves as chairman of the board of directors of Adaptimmune Therapeutics, Ardelyx, Inc., Epizyme, Inc., TESARO, Inc., and Mersana Therapeutics, Inc.
Scott Whitcup, M.D.
Scott has served as a member of Nightstar’s board of directors since June 2017. He is the founder and chief executive officer of Akrivista LLC and has served as its chief executive officer since October 2015 and is the founder of Whitecap Biosciences, and has served as its Chief Executive Officer since November 2015.
Both companies are focused on developing new therapies in ophthalmology and dermatology. He previously served as executive vice president, R&D and chief scientific officer at Allergan, Inc. where his group focused on ophthalmology, CNS, urology, dermatology, medical aesthetics, anti-infectives and surgical obesity. Prior to Allergan, Scott was the clinical director at The National Eye Institute at the National Institutes of Health (NIH), where he led the intramural clinical research program. He is a Diplomate of both the American Board of Internal Medicine and the American Board of Ophthalmology, a licensed M.D. in California and serves on the Clinical Faculty of the Department of Ophthalmology at the Jules Stein Eye Institute, David Geffen School of Medicine.
Scott received a B.A. in neurobiology and behavior from Cornell University and an M.D from Cornell University Medical College. He currently serves on the board of directors of Semnur Pharmaceuticals and Menlo Therapeutics.
SCIENTIFIC ADVISORY BOARD
K. Reed Clark, Ph.D.
K. Reed Clark is an experienced molecular virologist/gene therapist with extensive experience in AAV vector engineering, pre-clinical and clinical gene transfer studies, AAV vector process development, and early phase clinical manufacturing. He received his Ph.D. from The Ohio State University and did his postdoctoral training at Nationwide Children’s Hospital.
His early research addressed shortcomings with rAAV vector production by identifying stabile mammalian cell lines for scalable rAAV production. Development of this vector system resulted in seminal discoveries for column affinity chromatography purification and qPCR titration of rAAV that are now standard practices in the field. Commercial friendly iterations of this mammalian manufacturing process have been used to produce multiple clinical grade rAAV vectors for several Phase 1/2 studies targeting a variety of inherited and acquired diseases. Dr. Clark’s interest in the clinical use of AAV vectors led him to explore the form and frequency of rAAV and wild-type viral genome persistence in vivo. Dr. Clark served as director of the Nationwide Children’s Hospital pre-clinical and clinical manufacturing core facilities as well as co-principle investigator on several NIH funded programs to develop biologics for early phase human proof of concept trials. He is currently senior vice president, head of pharmaceutical development at Dimension Therapeutics, a biotechnology company in Cambridge, Massachusetts that targets the liver for treatment of rare inherited metabolic diseases such as Ornithine transcarbamylase deficiency, Glycogen storage disease type I, Phenylketonuria and Wilson Disease. Dr. Clark leads 30 scientists in functional groups that include vector engineering, producer cell line isolation, upstream process development, downstream process development and pilot scale production up to 250L scale. Dr. Clark also has extensive CMC regulatory experience in AAV product development with deep experience in the construction and review of CMC regulatory filings and face-to-face interactions with both U.S. RAC and FDA officials and with global Health Authority agencies that include Health Canada, MHRA (UK), PEI (Germany), and ANSM (France). Dr. Clark has 57 publications in peer-reviewed journals and is an inventor or co-inventor on six issued patents.
Elias Reichel, M.D.
Dr. Reichel is professor and vice chair for research and director of the Vitreoretinal Diseases and Surgery Service at the New England Eye Center, Tufts University School of Medicine. Dr. Reichel earned his undergraduate degree from Harvard College and his M.D. from the Albert Einstein College of Medicine in New York.
He completed his residency at the Massachusetts Eye & Ear Infirmary and fellowships in vitreoretinal diseases and surgery at the New England Eye Center, electrophysiology and molecular genetics at the Massachusetts Eye and Ear Infirmary, and genetics at the Children’s Hospital-Boston. Dr. Reichel’s areas of expertise include both medical and surgical vitreoretinal disorders. His surgical interests include surgery for macular disorders and retinal detachment. He has a special interest in hereditary retinal degenerations, macular degeneration, and diabetic retinopathy and has pioneered the use of transpupillary thermotherapy for the treatment of exudative age-related macular degeneration (AMD). He has published more than 140 articles in peer-reviewed journals and written two books on retinal disorders. He is a founder of Hemera Biosciences, a clinical phase start-up developing a complement-based gene therapy for dry AMD.
SriniVas R. Sadda, M.D.
SriniVas R. Sadda, M.D., is the president and chief scientific officer of the Doheny Eye Institute, the Stephen J. Ryan – Arnold and Mabel Beckman Endowed chair and professor of ophthalmology at the University of California – Los Angeles (UCLA), David Geffen School of Medicine. He received his M.D. from The Johns Hopkins University in Baltimore, Maryland.
After an internship at the William Beaumont Hospital in Royal Oak, Michigan, he returned to The Johns Hopkins University and the Wilmer Eye Institute in Baltimore for an ophthalmology residency as well as neuro-ophthalmology and medical retina fellowships. Dr Sadda’s major research interests include automated retinal image analysis, retinal substructure assessment, advanced retinal imaging technologies, and vision restoration approaches. He has organized multiple consensus efforts for the classification of various retinal disorders. Dr. Sadda has served as principal investigator for over 30 major clinical trials, and has led several international collaborative research programs. He is the founder and Emeritus director of the Doheny Image Reading Center, one of the largest centralized reading centers in the world. He has more than 340 publications in peer-reviewed journals and over 300 published abstracts. He authored the first edition of the textbook Emerging Technologies in Retinal Disease, as well as 14 other book chapters. As an invited lecturer, he has given more than 350 presentations around the country and the world, including multiple named lectures. Dr. Sadda also serves as an editorial board member of Ophthalmic Surgery, Lasers & Imaging, Retina, Graefe’s Archive for Clinical and Experimental Ophthalmology, Ophthalmology Retina, and Ophthalmology. He is also an editor of the 5th edition of the Ryan’s Retina textbook. Dr. Sadda has received multiple awards and honors that include Research to Prevent Blindness Physician-Scientist Award, a Senior Honor Award from the American Society of Retina Specialists, a Senior Achievement Award and Secretariat Award from the American Academy of Ophthalmology, John H. Zumberge Research and Innovation Award, the Silver Fellow designation from the Association for Research in Vision and Ophthalmology, and the Macula Society Young Investigator Award. He has been named to the Best Doctors of America list for several consecutive years.
Luk H. Vandenberghe, Ph.D.
Luk H. Vandenberghe, Ph.D., assistant professor at Harvard Medical School, directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, Massachusetts. He received a degree in cellular and genetic engineering from the University of Leuven, Belgium.
His doctoral work led to the discovery of novel adeno-associated virus (AAV) serotypes such as AAV9, novel insights into AAV structure-function and vector immunobiology. His current laboratory addresses mechanistic questions on AAV virology, develops technologies aiming to overcome hurdles to gene therapy clinical applications, and actively translates gene therapy programs in hearing and vision. His research focuses on AAV for therapeutic gene delivery, and more recently on using AAV as a starting point for the design of synthetic viral vector systems, a first generation which is referred to as AncAAVs and are now progressing to the clinic for a number of indications. Dr. Vandenberghe previously co-founded GenSight Biologics and works with several companies as a consultant or member of the scientific advisory board. He also is a founder, board member and advisor to Odylia Therapeutics, a non-profit catalyzing translation for gene therapies within the challenging field of ultra-rare disorders. Dr. Vandenberghe has over 50 peer reviewed publications and more than a dozen licensed patents, mostly related to gene therapy methods, technologies and applications.
We collaborate with global researchers and clinical experts in the United Kingdom, the United States, Canada and Germany. We have partnered with leading academic research institutions, such as the University of Oxford, to assist us in conducting clinical trials with our retinal gene therapy candidates, and to collaborate with the institutions’ researchers and clinical experts to provide key insights and vital resources to us as we advance our clinical programs and earlier-stage development pipeline.
In an effort to refine the delivery of gene therapy to the subretinal space and improve outcomes for patients with inherited retinal diseases, we collaborate with leaders in retinal imaging technology, robotic surgery and precision delivery, including Carl Zeiss Meditec AG, Oxular Limited and the Dutch Ophthalmic Research Center. To further enhance our surgical process, in 2017, we executed a collaboration with Preceyes B.V. (NL), a medical robotics company focused on ocular surgery, for the development of a high-precision drug delivery technology in the eye. We will use the Preceyes robotic device to further refine the delivery of gene therapy to the subretinal space for a range of inherited retinal diseases.