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Gene Therapy Trial Report

Summary

Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial


NCTID NCT06623279 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term DOID:10871
Compound Name HG202
Sponsor HuidaGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 15 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant VEGFA mRNA
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type hfCas13Y
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1
Submit Date 2024-09-30
Completion Date 2031-02-01
Last Update 2024-10-02

Participation Criteria


Eligible Age 50 Years - 85 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Males or females ≥ 50 and ≤ 85 years at the time of signing the ICF; * Active macular choroidal neovascularization (MNV) secondary to nAMD in the study eye; * Sentinel (1st) subject for each dose cohort must have a BCVA ≤ 20/63 and ≥ 20/400 (≤63 and ≥ 19 ETDRS letters) in the study eye. Following the sentinel subject evaluation, the rest of the subjects in the dose cohort must have a BCVA between ≤ 20/40 and ≥ 20/400 (≤ 73 and ≥ 19 ETDRS letters) in the study eye. * Able to perform visual acuity and retinal function tests and able and willing to comply with study procedures for this clinical trial. Exclusion Criteria: * Retinal or subretinal hemorrhage, scarring, or fibrosis of greater than 50% of the total lesion in the study eye; * Other ocular diseases that may affect central vision in the study eye; * Any other cause of CNV than nAMD in the study eye * Uncontrolled glaucoma in the study eye; * History or presence of corneal transplant or corneal dystrophy in the study eye; * History of other intraocular surgery in the study eye within 3 months prior to baseline; * Prior gene therapy or oligonucleotide therapy; * Other conditions judged by the investigator as inappropriate for the study.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites
Locations

Regulatory Information


Has US IND True
FDA Designations
Recent Updates FDA cleared IND November 2024

Resources/Links