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News & Trends - Pharmaceuticals

First investigational gene therapy trial for macular degeneration

Health Industry Hub | May 3, 2021 |

Pharma News: Australia’s first clinical trials of an investigational gene therapy to treat dry age-related macular degeneration (AMD) has begun.

Centre for Eye Research Australia (CERA) Principal Investigator of Retinal Gene Therapy Research and vitreoretinal surgeon Dr Tom Edwards performed the first surgeries to administer this investigational gene therapy to patients at the Royal Victorian Eye and Ear Hospital in Melbourne.

This investigational gene therapy, being developed by Gyroscope Therapeutics Limited, is being studied in two studies evaluating its safety and effectiveness for the treatment of geographic atrophy (GA) secondary to AMD.

AMD causes a gradual and permanent loss of central vision that worsens over time.

“Having the dry form of AMD is a devastating diagnosis, which robs people of their ability to read, drive or even see the faces of loved ones,’’ says Dr Edwards.

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“There is currently no approved treatment for dry AMD. As dry AMD advances, it leads to an irreversible degeneration of retinal cells, causing a gradual and permanent loss of central vision.

“We look forward to advancing important research for people facing devastating vision loss.”

Gyroscope Therapeutics Limited recently announced it has raised USD $148.0 million (£107.8 million) in a Series C financing to advance the clinical development of GT005, the company’s lead investigational gene therapy being evaluated for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

These funds will also enable Gyroscope to further advance its early stage pipeline and innovative delivery technology, including its proprietary OrbitTM subretinal delivery system, which is designed for precise delivery to the back of the eye.

Around 20 Australians with dry AMD are expected to take part in the Phase II HORIZON and EXPLORE trials evaluating the safety and effectiveness of this investigational gene therapy.

Khurem Farooq, Chief Executive Officer, Gyroscope said “We recently announced encouraging Phase I/II clinical trial data with our lead investigational gene therapy, GT005, that give us confidence in its potential as a treatment for geographic atrophy and are continuing to advance our Phase II clinical programme.”


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