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Gyroscope Therapeutics, UPenn to develop gene therapies for serious eye diseases


Two teams of researchers from Gyroscope Therapeutics and the University of Pennsylvania are joining forces to explore gene therapy targets for three specific serious eye diseases.

Gyroscope Therapeutics and the University of Pennsylvania announced last month a research agreement to develop gene therapies for serious eye diseases.

Investigators from the clinical-stage gene therapy company and the university’s Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) will collaborate to analyze specific gene therapy targets for glaucoma, optic neuritis, and retinitis pigmentosa, according to a news release.

Related: Collaboration formed to develop AAV-based gene therapies for eye disease

Per the sponsored agreement, Gyroscope has an exclusive option to the intellectual property associated with and as a result of any research conducted.

Leading the CAROT team is Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology, along with Ken Shindler, MD, MD, PhD, an associate professor of ophthalmology, and Ahmara Ross, MD, PhD, an assistant professor of ophthalmology, of the university’s Perelman School of Medicine.

Too many people in the world have limited vision or complete blindness because current treatment options for many serious eye diseases are so limited, said Khurem Farooq, Gyroscope CEO, in the news release.

Related: Ocular gene therapy offers hope for inherited retinal disease

“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,” he said.

Glaucoma is the leading cause of irreversible blindness across the globe, with an estimated 80 million people having it. Research findings have projected that total to increase to over 111 million by 2040.1

An estimated 300 million people worldwide have been diagnosed with retinitis pigmentosa — a disease primarily passed down genetically from one or both parents.2

Optic neuritis is often associated to the onset of multiple sclerosis (MS), appearing as the first sign in 20% of patients diagnosed as well as over the course of disease progression in 50% of patients.3

Read more gene therapy content here



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.

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