Gyroscope Therapeutics and the University of Pennsylvania Announce Research Agreement to Develop Gene Therapies for Serious Eye Diseases

LONDON – Friday 18 December 2020 – Gyroscope Therapeutics Limited, a clinical-stage gene therapy company focused on diseases of the eye, today announced the company has entered a sponsored research agreement with the University of Pennsylvania and the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) to develop gene therapies for serious eye diseases that can lead to permanent vision loss. Gyroscope has an exclusive option to the intellectual property associated with, and arising from, the research conducted under the agreement. 

A team of researchers from CAROT and Gyroscope will work together to explore specific gene therapy targets for glaucoma, optic neuritis and retinitis pigmentosa. The CAROT team is led by Jean Bennett, M.D., Ph.D., the F.M. Kirby Professor of Ophthalmology, along with Ken Shindler, M.D., Ph.D., an Associate Professor of Ophthalmology, and Ahmara Ross, M.D., Ph.D., an Assistant Professor of Ophthalmology, of the Perelman School of Medicine.

“Too many people around the globe face a life with limited vision or complete blindness because current treatment options for many serious eye diseases are so limited,” said Khurem Farooq, Chief Executive Officer, Gyroscope. “Gene therapy has the potential to be a completely new way of approaching these diseases, and we are very excited to work with Jean and the team of world leaders in ophthalmic gene therapy research at the University of Pennsylvania to evaluate new targets for these conditions.”

“Our team is passionate about the potential of gene therapies for people with serious eye diseases,” said Dr. Bennett. “We are looking forward to furthering our research in glaucoma, optic neuritis and retinitis pigmentosa, which combined currently cause a devastating loss of vision for millions of people around the world.”

Glaucoma is a leading cause of irreversible blindness globally. An estimated 80 million people have glaucoma worldwide, and this number is expected to increase to more than 111 million by 2040.¹ There is no cure for glaucoma. If it is caught early, people with glaucoma can be treated with surgery or medication to help control the disease. Because glaucoma typically does not cause pain, it often progresses silently and is not diagnosed until the optic nerve is irreparably damaged.

Retinitis pigmentosa (RP) refers to a group of rare genetic retinal diseases that cause progressive loss of night and peripheral vision. The condition is often diagnosed in childhood or adolescence and can lead to legal, and sometimes complete, blindness. An estimated 300,000 people worldwide have RP, mainly caused by a genetic variant inherited from one or both parents.²

Optic neuritis occurs when the optic nerve is damaged as a result of inflammation. Symptoms of optic neuritis include temporary vision loss in one eye and pain with eye movement. Optic neuritis is closely associated with multiple sclerosis (MS): It is the first sign of MS in 20% of patients, and occurs during the course of the disease in 50% of MS patients.³


Gyroscope Therapeutics is a clinical-stage gene therapy company developing gene therapy beyond rare disease to treat diseases of the eye that cause vision loss and blindness. Our lead investigational gene therapy, GT005, is currently being evaluated in Phase II clinical trials for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), a leading cause of blindness. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of people with GA.

Syncona Ltd, our lead investor, helped us create a leading gene therapy company combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

Charlotte Arnold
VP, Corporate Affairs
Gyroscope Therapeutics


[1] Tham YC, Li X, Wong TY, Quigley HA, Aung T, Cheng CY. Global prevalence of glaucoma and projections of glaucoma burden through 2040: a systematic review and meta-analysis. Ophthalmology. 2014 Nov;121(11):2081-90.
[2] Cowen Equity Research Therapeutic Categories Outlook: Comprehensive Study. 2020 Feb;P.2334.
[3] Kale N. Optic neuritis as an early sign of multiple sclerosis. Eye Brain. 2016;8:195-202.