Clinical Trial

Disease: Neovascular Age-related Macular Degeneration, nAMD, (NCT06031727)

Disease info:

Age-related macular degeneration (AMD) is an eye disorder caused by genetic and environmental factors. AMD is one of the leading causes of vision loss in older people. Mild symptoms may manifest in an individual's forties or fifties, with distorted vision or vision loss usually prevalent in a person’s sixties or seventies, which progresses over time. Vision loss in AMD is caused by a gradual degeneration of light-sensing cells in the macula, a small area at the centre of the retina that detects light and colour. The macula is responsible for central vision. AMD primarily affects central vision, impairing an individual's ability to read, write, drive and recognise faces. Peripheral and night vision are usually unaffected, but a slower adjustment of vision to darkness and dim lights is often present during the early stages of the disease.

AMD is categorised into two major types: the dry form and the wet form. Approximately 85% of individuals with AMD are classified as having dry-form AMD, which is characterised by a buildup of yellowish deposits called drusen beneath the retina and progressive vision loss. In advanced stages, geographic atrophy occurs whereby areas of the macula deteriorate completely, resulting in severe loss of vision. Vision loss often begins in one eye and progresses to include both eyes. In approximately 15% of individuals with dry-form AMD, the disease will progress to wet form. The wet form is associated with severe vision loss that exacerbates rapidly. 

Wet-form AMD is also defined as exudative AMD or choroidal neovascularisation (neovascular).

 

Frequency:
Approximately 8% of the global population shows signs of AMD. The disease currently affects about 170 million people worldwide, and its prevalence is expected to increase.
Official title:
A Trial to Evaluate the Safety, Tolerability, and Efficacy of CRISPR-Cas13 RNA-editing Therapy Targeting Knockdown of Vascular Endothelial Growth Factor A (HG202) in the Treatment of Neovascular Age-related Macular Degeneration (nAMD)
Who:

Contact

Name: Study Director

Phone: +86 021-25076143

Email: HG20201@huidagene.com 

Sponsor:

HuidaGene Therapeutics Co., Ltd.

Partners:

Tianjin Medical University Eye Hospital

Eye & ENT Hospital of Fudan University

Locations:

Shanghai
Eye & ENT Hospital of Fudan University, Shanghai, Shanghai, China

Tianjin
Tianjin Medical University Eye Hospital, Tianjing, Tianjing, China

Study start:
Sep. 4, 2023
Enrollment:
12 participants
Gene editing method:
CRISPR-Cas13
Type of edit:
Partial knock-down
Gene:
Vascular endothelial growth factor A (VEGF-A)
Delivery method:
Adeno-associated virus (AAV) vector - In-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting

Description

Age-related macular degeneration (AMD) is a progressive disease leading to severe and irreversible vision loss of which the neovascular AMD (nAMD) accounted for 90% blindness in AMD. nAMD is primarily driven by the perturbation of vascular endothelial growth factor (VEGF). VEGF overexpression leads to abnormal growth of choroidal neovascularization (CNV), which is a hallmark of AMD. Although anti-VEGF agents are effective in treating nAMD, long-term efficacy decreases over time due to the need for repeated injections impacting patient compliance with treatment regimen while patients still may lose vision during the 7th or 8th year of treatment. These frequent intravitreal injections can increase the risk of complications, including submacular hemorrhage, intraocular hypertension, inflammation, and retinal detachment. Furthermore, there are up to 46% of nAMD patients using anti-VEGF agents who have shown poor response or have developed tachyphylaxis with anti-VEGF therapies. HG202 is a CRISPR/Cas13 RNA-editing therapy packaging novel high-fidelity Cas13 technology using one single AAV vector to partially knock-down the expression of VEGFA and thus inhibit CNV formation in AMD patients who are either responsive or non-responsive to anti-VEGF agents. The long-term, stable delivery of HG202 following a one (1) time gene-editing therapy treatment for nAMD could potentially reduce the frequent injection treatment burden of currently available therapies AND treat nAMD patients who are non-responsive to anti-VEGF therapies and have no treatment.

Last updated: Dec. 19, 2023
close
Search CRISPR Medicine