Oculogenex, a La Habra, California-based gene therapy company, has received a USD 1.09 million R44 SBIR grant from the National Eye Institute (NEI) to advance its AAV-based candidate OCLX-001 toward an Investigational New Drug (IND) application for intermediate age-related macular degeneration (iAMD). Principal investigator Lakshmi Ramkumar leads the program.
The award funds two IND-enabling workstreams: establishing a current Good Manufacturing Practice (cGMP)-compliant manufacturing process for plasmid production and adeno-associated virus (AAV) vector manufacturing, and conducting Good Laboratory Practice (GLP) toxicology and pharmacokinetic studies in minipigs. Successful completion would position Oculogenex to file an IND and enter first-in-human trials.
OCLX-001 uses an AAV vector to restore expression of BMI1, a polycomb repressor complex protein implicated in mitochondrial homeostasis and oxidative stress resistance in retinal cells. The company asserts that BMI1 downregulation in aging retinal pigment epithelium contributes to photoreceptor loss characteristic of dry AMD. Preclinical studies, according to the company, demonstrated protection against photoreceptor damage and improved retinal function. Delivery is via a proprietary suprachoroidal injection device designated OCLX-SCDD.