GT005

Unassigned

New Medicines

Geographic atrophy in patients with age-related macular degeneration

Information

New molecular entity
Gyroscope Therapeutics
Gyroscope Therapeutics

Development and Regulatory status

Phase II Clinical Trials
None
None
Nov 20FDA has granted two fast track designations to GT 005 for the treatment of GA secondary to dry AMD. one is for patients with variants in their complement genes; the other is for those who have specific mutations in their Complement Factor I (CFI) gene and low levels of the CFI protein in their blood [5].
Nov 20GT005 is a one-time therapy delivered under the retina. The US has designated the treatment Fast Track status [4].
Feb 20Gyroscope announces the successful completion of a £50.4 million Series B financing. With this new round of financing, Gyroscope will continue to advance the clinical development of GT005, the manufacturing platform to meet patient need worldwide, and a second-generation Orbit Subretinal Delivery System (SDS) [3].

Category

A retinal gene therapy consisting of a recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor
AMD is characterised by the appearance of drusen in the macula, accompanied by choroidal neovascularisation (wet AMD) or geographic atrophy (dry AMD). The estimated prevalence of AMD in the UK is 4.8% of those over 65 years of age and 12.2% of those aged 80 years or more. Dry AMD is the most common form of AMD, occurring in 90% of cases. AMD is the most common cause of severe visual impairment in older adults in the developed world [1].
Geographic atrophy in patients with age-related macular degeneration
Intraocular

Trial or other data

Dec 20EXPLORE is studying the safety and effectiveness of GT005 in people with GA secondary to dry AMD who have rare variants in their CFI gene and who have low levels of the CFI protein in their blood. HORIZON is evaluating GT005 in a broader group of people with dry AMD than EXPLORE [7].
Nov 20PII HORIZON trial to evaluate the safety and efficacy of two doses of GT 005 administered as a single subretinal injection in patients with GA secondary to AMD starts (GT005-03; NCT04566445). 180 adults will be recruited in the US, Germany and UK (at Sunderland Eye Infirmary). Primary outcome is change from baseline to Week 48 in GA area as measured by fundus fluorescence; collection of these data is due to complete Feb 22 [6].
Jul 20PII EXPLORE trial to assess the safety and efficacy of two doses of subretinally injected GT 005, in patients with GA secondary to AMD starts (NCT04437368; GT005-02). 75 adults will be recruited in the US and Germany (at present, no UK sites).apeutics announced that the first was dosed in the US. Primary outcome is change from baseline to Week 48 in GA area as measured by fundus fluorescence; collection of these data is due to complete Feb 23 [6].
Mar 20PI/II FOCUS trial is recruiting [2].
Dec 18PI/II FOCUS trial to assess the safety and biological activity of a single dose of GT 005 subretinal injection in patients with geographic atrophy due to dry AMD starts (GT005-01; NCT03846193). The non-randomised, open-label, dose-escalation trial is enrolling 35 adults in the UK; sites are Bristol Eye Hospital, Moorfields Eye Hospital, Manchester Eye Hospital, Oxford University Hospital and Sunderland Eye Infirmary. Primary outcome is safety; secondary outcomes include change from baseline in GA size measured in mm2 and change from baseline in retinal drusen volume measured in mm3. The study is due to complete Jun 21 [2].