Novartis Halts Development of Ophthalmic AAV Gene Therapy GT005 for Geographic Atrophy

On September 11, Syncona Investment Management Limited, an investor in Gyroscope Therapeutics (Gyroscope), stated that Novartis has ceased the development of the ophthalmic AAV gene therapy GT005.

It is reported that GT005, a key investigational product of Gyroscope Therapeutics, is a gene therapy based on the AAV2 vector. GT005 aims to restore the balance of an overactive complement system (a part of the immune system) by increasing the production of complement factor I (CFI) protein. Overactivation of the complement system is closely related to the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production can reduce the overactivity of the system and decrease inflammation, with the goal of preserving a person’s vision.

It is reported that the decision to halt the development of the GT005 project was based on the recommendation of its Data Monitoring Committee, which is responsible for overseeing and reviewing the data from the project's clinical trials. After reviewing the existing data, including that from the Phase 2 clinical trial study, the committee concluded that the project should no longer proceed.

Last February, Gyroscope announced that Novartis had completed the acquisition of Gyroscope Therapeutics, paying an "upfront payment" of $800 million (£587 million), with an additional potential "milestone payment" of up to $700 million (£514 million) to be paid later. Novartis is particularly optimistic about Gyroscope's AAV gene therapy pipeline for ocular diseases such as age-related macular degeneration (AMD). This acquisition further strengthens Novartis’s leading position in the field of ophthalmic gene therapy.

Gyroscope Therapeutics, headquartered in London, UK, does not treat eye diseases by correcting genetic factors but instead focuses on increasing the production of complement factor I (CFI). Since CFI downregulates the complement system, an increase in serine protease production can counteract the driving forces behind age-related macular degeneration (AMD). Currently, Gyroscope Therapeutics has three clinical trials targeting age-related macular degeneration (AMD).

Patients with age-related macular degeneration (AMD) are mostly over 50 years old, with both eyes affected successively or simultaneously, and their vision is progressively impaired, seriously affecting the quality of life of the elderly. According to different clinical manifestations and pathological changes, AMD is divided into two types: dry and wet. Dry AMD is the main cause of permanent vision loss in people over 50. As dry AMD progresses, it leads to irreversible degenerative atrophy of retinal cells, resulting in gradual vision loss until permanent blindness.

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