Developing a gene therapy-based solution for early intervention in age related macular degeneration (AMD) to prevent disease progression

The National Eye Institute awarded Oculogenex Inc. $1.092536 million under the Vision Research program (CFDA 93.867) on August 1, 2026, to develop a gene therapy-based solution for early intervention in intermediate age-related macular degeneration (IAMD).

Oculogenex will develop and advance commercialization of OCLX-001, a novel gene therapy that restores BMI1 expression to mitigate oxidative damage and maintain retinal cell viability. The project has two primary objectives: developing a current Good Manufacturing Practice (cGMP)-compliant process for master cell bank creation, plasmid production, and adeno-associated virus (AAV) manufacturing with rigorous process controls and success metrics including high plasmid integrity and viral vector potency; and conducting Good Laboratory Practice (GLP) toxicology and pharmacokinetic studies in minipigs to assess safety profiles and support an Investigational New Drug (IND) application. Preclinical studies have demonstrated significant photoreceptor protection and improved retinal function through various delivery mechanisms, including a specialized suprachoroidal injection technique (OCLX-SCDD).

Performance takes place in La Habra, California. The period of performance runs through May 31, 2028. The assistance type is a Project Grant.

Abstract

Oculogenex is addressing the critical unmet need for early intervention in intermediate age-related macular degeneration (iamd), a condition marked by mitochondrial dysfunction and oxidative stress contributing to retinal degeneration and severe vision loss. Through gene therapy development, our goal is to prevent the progression from iamd to advanced AMD, thereby enhancing patient quality of life and reducing associated health and economic burdens. Our approach focuses on the development and commercialization of a novel gene therapy (oclx-001), using patented technology to restore BMI1 expression, which is crucial for mitigating OXI- dative damage and maintaining retinal cell viability. Oclx-001 has demonstrated promising results in preclinical studies, showing significant protection against photoreceptor damage and improved retinal function through VAR- ious delivery mechanisms, including a specialized suprachoroidal injection technique (oclx-scdd). The project has two primary objectives: 1. Develop a cgmp-compliant process for master cell bank (MCB) creation, plasmid production, and subsequent adeno-associated virus (AAV) manufacturing. We aim to ensure the quality and consistency of the therapy with rigorous process controls and success metrics, including high plasmid integrity and viral vector potency. 2. Conduct good laboratory practice (GLP) toxicology and pharmacokinetic studies to assess safety profiles and support an investigational new drug (IND) application. This will involve detailed stud- IES in minipigs, measuring both efficacy endpoints and safety parameters to ensure therapeutic benefits without significant adverse effects. Given the increasing prevalence of AMD and the limited treatment options for its early stages, our project stands to significantly affect public health outcomes for aging populations at risk of severe vision loss. By targeting iamd before IT progresses to more debilitating stages, oclx-001 could substantially reduce emotional and financial strain on patients and healthcare systems alike. Moreover, successful completion of the proposed studies will enable US to move towards first-in-human clinical trials, marking a significant step forward in the treatment and management of AMD.