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Nightstar Therapeutics

Founded Year



Acq - P2P | Acquired

Total Raised




About Nightstar Therapeutics

Nightstar Therapeutics (NASDAQ: NITE) is a clinical-stage gene therapy company focused on developing and commercializing one-time treatments for patients suffering from rare inherited retinal diseases including choroideremia, X-linked retinitis pigmentosa and Best vitelliform macular dystrophy.

Headquarters Location

215 Euston Road

London, England, NW1 2BE,

United Kingdom


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Expert Collections containing Nightstar Therapeutics

Expert Collections are analyst-curated lists that highlight the companies you need to know in the most important technology spaces.

Nightstar Therapeutics is included in 2 Expert Collections, including Biopharma Tech.


Biopharma Tech

5,241 items

Companies involved in the research, development, and commercialization of chemically- or biologically-derived therapeutic & theranostic drugs. Excludes vitamins/supplements, CROs/clinical trial services.


Regenerative Medicine

1,818 items

Regenerative medicine refers to the process of activating, replacing, engineering or regenerating human genetic material, cells, tissues or organs to restore normal function. It also includes bioengineered tissues used for in vitro testing (e.g. organ-on-a-chip, organoids).

Nightstar Therapeutics Patents

Nightstar Therapeutics has filed 1 patent.

patents chart

Application Date

Grant Date


Related Topics



Disorders of choroid and retina, Diseases of the eye and adnexa, Rare diseases, Visual system, Blindness


Application Date


Grant Date


Related Topics

Disorders of choroid and retina, Diseases of the eye and adnexa, Rare diseases, Visual system, Blindness



Latest Nightstar Therapeutics News

Rectify Pharmaceuticals Expands Clinical Drug Development Expertise with Board and SAB Appointments

Feb 14, 2023

Rectify Pharmaceuticals Expands Clinical Drug Development Expertise with Board and SAB Appointments Dr. Tuyen Ong, M.D., M.B.A. appointed to Board of Directors and Dr. Jason Campagna, M.D., Ph.D. to Scientific Advisory Board CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Rectify Pharmaceuticals, Inc. , (“Rectify”) a biotechnology company developing small molecule disease-modifying therapeutics that restore and enhance ABC transporter function, today announced the appointments of Tuyen Ong, M.D., M.B.A., to the Board and Directors, and Jason Campagna, M.D., Ph.D., to the Scientific Advisory Board. “We are excited to announce the additions of Dr. Ong and Dr. Campagna to our Board of Directors and Scientific Advisory Board, respectively, as they will further support Rectify’s mission to develop a pipeline of therapeutics for patients with serious genetic and large common ABC transporter mediated diseases,” said Rajesh Devraj, Ph.D., President and Chief Executive Officer of Rectify. “Dr. Ong has a proven track record of building world class companies and a wealth of experience in the development of novel therapies. We look forward to drawing on his expertise as we advance our initial pipeline candidates into and through the clinic. Dr. Campagna has deep expertise in biliary and liver disease drug development and extensive clinical development experience. Both Dr. Ong and Dr. Campagna will be important advisors for the company as we continue to advance our ABC transporter-targeted disease-modifying therapeutics for patients. We welcome them both to Rectify.” Dr. Tuyen Ong is currently the Chief Executive Officer at Ring Therapeutics and a CEO-Partner at Flagship Pioneering. Prior to Ring, Dr. Ong was Senior Vice President, Head of the Ophthalmology Franchise at Biogen and was Chief Development Officer at Nightstar Therapeutics until its acquisition by Biogen in 2019. Additionally, while at Nightstar, Dr. Ong was involved with the company’s NASDAQ IPO. Previously, Dr. Ong served as Chief Medical Officer at PTC Therapeutics and was responsible for progressing its pipeline of therapies for rare and genetic diseases. He also held leadership roles at Bausch and Lomb and at Pfizer, with more than 20 years’ experience in clinical and drug development. Dr. Ong was awarded his M.D. from University College London and his M.B.A. from New York University Stern School of Business. Dr. Jason Campagna currently serves as the Chief Medical Officer at Q32 Bio Inc. Prior to this, Dr. Campagna served as Chief Medical Officer at Intercept Pharmaceuticals where he had global responsibility for major functions supporting the Primary Biliary Cholangitis (PBC) business, Non-Alcoholic Steatohepatitis (NASH) development and pipeline efforts. Before becoming Chief Medical Officer, Dr. Campagna led the Global NASH Program at Intercept, with responsibility for implementation and execution of the NASH Program, culminating in the first submitted NDA for a therapeutic intended to treat patients with NASH. Dr. Campagna has also held a number of roles of increasing responsibility at The Medicines Company, was Chief Medical Quality Officer at Cottage Health System, and has held faculty appointments at the University of Pennsylvania and Massachusetts General Hospital. Dr. Campagna obtained his Ph.D. and M.D., as well as his bachelor’s degree in Cellular and Molecular Pharmacology/Biology, from the University of Miami Miller School of Medicine. About Rectify Pharmaceuticals, Inc. (“Rectify”) Rectify is developing Positive Functional Modulators (PFMs), disease-modifying therapies that restore and enhance ABC transporter function to address the underlying cause of serious ABC transporter mediated diseases. Rectify is the first company to directly address the untapped therapeutic potential of the ABC transporter target class. ATP-binding cassette (ABC) transporters are a 48-member superfamily of membrane-bound proteins that actively export a diverse range of substrates (e.g., lipids, ions, peptides, and salts) across lipid membranes. Rectify has built a breakthrough ABC transporter product platform that is enabling efficient and rapid discovery of first-in-class small molecule therapies that restore and enhance ABC transporter function for rare and common liver, biliary, eye and CNS diseases. Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds and were joined by Forbion and Longwood Fund. The company is headquartered in Cambridge, Massachusetts. For more information, please visit . Contacts

Nightstar Therapeutics Frequently Asked Questions (FAQ)

  • When was Nightstar Therapeutics founded?

    Nightstar Therapeutics was founded in 2013.

  • Where is Nightstar Therapeutics's headquarters?

    Nightstar Therapeutics's headquarters is located at 215 Euston Road, London.

  • What is Nightstar Therapeutics's latest funding round?

    Nightstar Therapeutics's latest funding round is Acq - P2P.

  • How much did Nightstar Therapeutics raise?

    Nightstar Therapeutics raised a total of $100M.

  • Who are the investors of Nightstar Therapeutics?

    Investors of Nightstar Therapeutics include Biogen IDEC, Syncona Partners, New Enterprise Associates, Redmile Group, Wellington Management and 3 more.

  • Who are Nightstar Therapeutics's competitors?

    Competitors of Nightstar Therapeutics include Audentes Therapeutics.

Compare Nightstar Therapeutics to Competitors


AAVLife, registered in Paris, is a privately held company dedicated to advancing gene therapy for rare diseases. AAVLife's lead indication is for Friedreich's Ataxia Cardiomyopathy. Its program is based on the successful use of a gene-therapy approach in a mouse model developed to mimic the gene defect and cardiac symptoms of Friedreich's Ataxia. The approach made use of an adeno-associated virus (AAV) to introduce a normal gene into cardiac tissue. The therapy restored cardiac function and reversed pathological heart enlargement in mice that had already progressed to heart failure.

Renova Therapeutics Logo
Renova Therapeutics

Renova Therapeutics is a San Diego-based biopharmaceutical company and an industry leader in gene therapy, pursuing the development of treatments for congestive heart failure (CHF) and other chronic diseases. CHF is the most rapidly growing cardiovascular disorder in the United States, with an estimated six million people afflicted and 550,000 new cases diagnosed every year. Approximately 26 million patients have CHF worldwide. Renova Therapeutics, in a Public-Private Partnership with the National Institutes of Health, is currently supporting a Phase I/II trial of its novel lead therapy for patients with severe CHF (Class II, III and IV). This initial product is a single-dose gene therapy that, in animal studies, has shown substantially improved, sustained outcomes in heart failure models by safely and significantly improving myocardial function. The product is being evaluated for its safety and initial efficacy in a 3:1 randomized, double-blinded, placebo-controlled dose-ranging study involving 56 patients across seven US medical centers.


StrideBio is a gene therapy company focused on creating and developing novel adeno-associated viral (AAV) vector technologies. The company is building a proprietary therapeutic pipeline focused on rare diseases.

Evive Biotech

Evive Biotech is an integrated biotech company developing therapeutic biologics. Products under development are mainly aimed at unsolved medical problems, such as oncology, inflammation, and autoimmune diseases. The company was formerly known as Generon Biomed, Inc. and changed its name to Evive Biotech in June 2020. The company was founded in 2004 and is based in Singapore, Singapore.

Applied Genetic Technologies

Applied Genetic Technologies (NASDAQ: AGTC) is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. The company was founded in 1999 and is based in Alachua, Florida.


Calimmune is a clinical-stage biotechnology company focused on developing novel gene therapies that have the potential to improve and protect the lives of patients by enhancing their immune systems. Calimmune's lead product candidate for HIV, now in Phase I/II studies, is being evaluated as a one-time treatment to prevent HIV progression to AIDS. Calimmune is developing a full product pipeline of therapeutic candidates to address the unique needs of individuals at different stages of HIV infection and with various treatment histories.

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