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NIH Awards Grant for Engineered Mechanosensitive Channel Glaucoma Gene Therapy

The grant goes toward developing an innovative approach to gene-agnostically and autonomously regulate pressure.

This content originally appeared on our sister site, Ophthalmology Times.

The NIH has awarded a $1.5 million SBIR Direct-to-Phase 2 grant to Nanoscope Therapeutics to support advancement of its Engineered Mechanosensitive Channel (EMC) glaucoma therapy.

Nanoscope's EMCs are designed to sensitize cells toward abnormal mechanical stress related to pressure elevation caused by Glaucoma. SBIR Direct-to-Phase II grant award is based on demonstration of feasibility of the technology equivalent to meeting phase 1 objectives and providing a solid foundation for the proposed phase 2 activity.

The company hopes to address vision loss due to glaucoma, which is the second leading cause of blindness in America with no cure. Glaucoma is recalcitrant to treatment and the resulting optic nerve damage is irreversible. Primary Open Angle Glaucoma (POAG), characterized by poor drainage of aqueous humor through the conventional outflow pathway, is common. Currently available pharmacological and surgical treatments come with significant limitations and side-effects including systemic reactions to medications, patient non-compliance, eye infections, surgical device failure, and eye damage.

EMC acts as an ideal drainage valve for trabecular meshwork (TM), because it is a relatively small homo-oligomeric channel and does not need any associated proteins or energy sources to assemble and function. Using patented gene-agnostic EMC therapy, Nanoscope researchers have demonstrated that lowering of the intraocular pressure (IOP) in animal models of glaucoma.

"EMC is an engineered version of a stretch-activated channel, which directly senses tension in the membrane lipid bilayer of cells and in response, transiently opens its pore for fluid exchange. Use of EMC in the impaired TM of glaucomatous eyes as an autonomous pressure modulator is novel and has significant promise for Glaucoma", Samarendra Mohanty, President and chief scientific officer, said in the news release.

POAG is heterogenous, and known to be caused by different genetic mutations, however, most contributing mutations are still unknown. Therefore, Nanoscope’s approach of correcting the pathological phenotype rather than correction of genetic mutation in a gene-independent manner would be transformative in clinical ophthalmology. The development of a safe, effective, long-lasting single dose therapeutic for the treatment of POAG would improve the lives of millions of people suffering from this devastating disease.