Charlesson gets $2.35M to develop ocular nanoparticle drug delivery

October 23, 2007 — Charlesson LLC says it has received several Small Business Innovative Research (SBIR) awards from the National Institutes of Health (NIH), and an award from the Oklahoma Center for the Advancement of Science and Technology (OCAST). The four grants total approximately $2.35 million and will support development of several pharmaceutical drug candidates, as well as nanoparticle-based gene therapies for eye disease.

“These funds will greatly assist the company in finalizing preclinical studies for an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA),” Mike Moradi, Charlesson’s CEO, said in a news release. “We expect to file our first IND in 2008 and are hopeful that Charlesson’s products will improve the quality of life for over 13.3 million Americans suffering from these blinding diseases.”

Moradi added that Charlesson is seeking commercial partners.

Charlesson was awarded $840,000 in a Phase II SBIR award from the NIH to develop CLT-003 for Diabetic Macular Edema (DME). These funds will be used to finalize IND-enabling efficacy and safety data. CLT-003 is a small molecule therapeutic with potent effects on reducing vascular leakage and subsequent neovascularization and inflammation that can occur in the eyes of diabetic patients. Charlesson is also developing efficacy profiles of CLT-003 for treating the “wet” and “dry” forms of Age-Related Macular Degeneration (AMD).

“Our preclinical studies have demonstrated that CLT-003 is a safe and potent therapeutic for treating various forms of blinding retinal disease, and we look forward to working with the FDA to initiate human clinical trials,” said Moradi said.

Charlesson also received about $1.1 million in a special request for proposals from the NIH to develop therapies to treat complications of type 1 diabetes. These funds will be used to develop Charlesson’s gene therapy program to deliver DNA encoding CLT-001 to the retina. CLT-001 is a naturally occurring peptide found in the eye, and Charlesson has demonstrated that supplementation of this peptide in animal models of diabetes and AMD has profound effects on reducing neovascular and inflammatory events in the eye.

In contrast to typical gene therapy where viral vectors are used to deliver the gene of interest, Charlesson’s program employs biodegradable nanoparticles for gene delivery. This non-viral approach eliminates many of the safety concerns in eliciting an immune response following gene therapy. Additionally, Charlesson has demonstrated that topical delivery of nanoparticle-encapsulated CLT-001 DNA can result in gene transfer to retinal cells in the back of the eye.

“Our scientists have demonstrated the safety and efficacy of CLT-001 and we are excited to advance our nanoparticle gene therapy efforts,” said Moradi. “This delivery system is expected to allow topical delivery of therapeutics that produce a sustained effect in the eye, without the need for frequent dosing.”

Direct, back-of-the-eye delivery methods for macular degeneration medication has long been sought by pharmaceutical companies. Current techniques involve frequent dosing, with much of the medication wasted in indirect delivery, and elderly patients often forgetting or unwilling to stick with a strict schedule of frequent dosing.

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