HG202 / HuidaGene Therap - LARVOL DELTA

In-vitro and in-vivo studies to support SIGHT-I clinical trial, the world’s first CRISPR/RNA-targeting therapy of HG202, for patients with neovascular age-related macular degeneration (ESGCT 2024) - P1 | "Four subjects with nAMD had an acceptable local and systemic adverse-event profile after the delivery of HG202 in the SIGHT-I study. Therefore, HG202, as a novel therapy, has the potential to provide safe and effective RNA-targeting therapy to treat nAMD patients who are non-responsive or develop tachyphylaxis to anti-VEGF agents with a one-time treatment."

Preclinical • Age-related Macular Degeneration • Diabetic Retinopathy • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

CRISPR-Cas13 Gene Editing Therapy Targeting VEGFA Demonstrated Early Efficacy in Neovascular Age-Related Macular Degeneration (ASGCT 2025) - P1 | "Subretinal injection of HG202, a CRISPR-Cas13 RNA-targeting gene therapy, demonstrates a favorable safety profile with no retinal detachment, drug-related inflammation, or retinal toxicity. Early efficacy data reveal significant reductions in CRT and the frequency of anti-VEGF injections. These results suggest that HG202 has the potential to provide a durable, one-time therapeutic option for nAMD, addressing critical unmet needs in the management of this chronic disease."

Clinical • Age-related Macular Degeneration • Cataract • Gene Therapies • Hematological Disorders • Inflammation • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

CRISPR/cas13-medIated RNA TarGeting THerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: HuidaGene Therapeutics Co., Ltd. | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Dec 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

HuidaGene Therapeutics Receives the First-Ever FDA Clearance of CRISPR/Cas13 RNA-Editing HG202 for Macular Degeneration (PRNewswire) - "HuidaGene Therapeutics...announced that the U.S. FDA has cleared its investigational new drug (IND) application for HG202, the world's first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD), which affects millions worldwide....'The BRIGHT trial will evaluate HG202's safety and efficacy to address this gap. We look forward to enrolling patients soon.'"

IND • Trial status • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

BRIGHT: Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: HuidaGene Therapeutics Co., Ltd.

New P1 trial • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Exploring the potential of a novel Cas13‐based RNA‐targeting therapy for age‐related macular degeneration (ESGCT 2023) - "This innovative approach allows for precise and efficient gene knockdown with the potential for the patients to reduce frequent IVT injections and minimize the risk of complications, providing a promising therapeutic strategy to ameliorate the social and economic burdens as well as to improve safety profile associated with chronic anti-VEGF therapy by providing reliable and safer option for nAMD. More importantly, HG202 offers a new approach for nAMD patients who are non-responsive to IVT anti-VEGF therapies."

Age-related Macular Degeneration • Cardiovascular • Glaucoma • Hematological Disorders • Macular Degeneration • Ocular Infections • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Retinal Disorders

CRISPR/Cas13 RNA-Targeting Therapy to Treat Patients with Neovascular Age-Related Macular Degeneration (ASGCT 2024) - P1 | "Three subjects with nAMD had an acceptable local and systemic adverse-event profile after the delivery of HG202. Subject #202 had significant improvement in BCVA, CRT, and elimination of persistent fluid within 8 weeks after the injection of HG202. Although only a low-dose cohort completed the enrollment, this study provides the basis for CRISPR/Cas13 RNA therapy is relatively safe in humans."

Clinical • Age-related Macular Degeneration • Diabetic Retinopathy • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

In-Vitro and In-Vivo Studies of HG202 as a CRISPR/Cas13 RNA Targeting Therapy for Macular Degeneration (ASGCT 2024) - "By successfully blocking VEGFA signaling, HG202 demonstrates long-term transgene expression and RNA editing after a single injection, thus eliminating the need for monthly injections and providing enduring therapeutic benefits. Therefore, HG202 has the potential to provide an effective and long-lasting treatment option for patients with nAMD who do not respond well or are resistant to anti-VEGF therapy. HG202 as a CRISPR RNA-targeting strategy is currently being investigated in humans."

Preclinical • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Efficient In-vitro and In-vivo CRISPR RNA Targeting Therapy of HG202 for Macular Degeneration (ARVO 2024) - "HG202 successfully blocks VEGFA signaling and achieves long-term transgene expression and RNA editing after a single injection, thus eliminating the need for monthly injections and providing enduring therapeutic benefits. HG202 can potentially offer an effective and long-lasting nAMD treatment for patients who are non-responsive or resistant to anti-VEGF therapy. HG202 as a CRISPR RNA-targeting strategy is currently being investigated in humans."

Preclinical • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

World's First CRISPR/RNA-Targeting Therapy (HG202) for Patients with Neovascular Age-Related Macular Degeneration (ARVO 2024) - P1 | "Current anti-VEGF therapies have significantly changed the landscape to treat nAMD but require life-long repeated invasive injections. HG202 as a novel therapy has the potential to provide safe and effective RNA-targeting therapy to treat nAMD patients who are responsive and non-responsive or develop tachyphylaxis to anti-VEGF agents with a one-time treatment. Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand."

Clinical • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

CRISPR/Cas13-mediated RNA Targeting Therapy for the Treatment of nAMD Investigator-Initiated Trial (SIGHT-I) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: HuidaGene Therapeutics Co., Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

CRISPR/Cas13-mediated RNA Targeting Therapy for the Treatment of nAMD Investigator-Initiated Trial (SIGHT-I) (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: HuidaGene Therapeutics Co., Ltd.

New P1 trial • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration