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Genisphere's 3DNA drug delivery platform achieves success in preclinical treatment of Posterior Capsular Opacification (PCO)

HATFIELD, Pa -
March 26, 2015 -

Genisphere LLC, provider of the 3DNA® nanotechnology platform, reported today it has achieved specific immunodepletion of problematic Myo/Nog cells implicated in posterior capsular opacification (PCO), a clouding of the eye lens which can be a complication of cataract surgery. The company envisions a wide application of this immunodepletion strategy in other fibrotic diseases and cancer, and has recently completed in- licensing intellectual property and additional assets for targeting Myo/Nog cells for therapeutic purposes from LIMR Development Inc. (LDI), a for-profit subsidiary of the Lankenau Institute for Medical Research where an early stage of the technology was developed.

Myo/Nog cells were discovered in the laboratory of Dr. Mindy George-Weinstein, and named for their ability to form muscle (Myo) and their production of Noggin, an inhibitor of bone morphogenetic proteins involved in cell communication. A unique monoclonal antibody (mAb) specifically recognizes these cells and has been used to identify, track, deplete and isolate this population from embryonic and adult tissues. Genisphere has collaborated with Dr. George- Weinstein to study the ability of the mAb to target cells implicated in PCO, a disease that typically occurs in approximately 30% of adults and greater than 70% of children after cataract surgery. They reported specific immunodepletion of Myo/Nog cells, when the mAb was used as a targeting device on Genisphere’s 3DNA® nanocarrier loaded with doxorubicin. The targeted 3DNA® approach is similar to that of an antibody drug conjugate (ADC), but delivers 100 times more of the drug to the targeted cells than an ADC and has no observed toxicity. Additional studies testing this formulation in rabbits undergoing cataract surgery are ongoing with Drs. Liliana Werner and Nick Mamalis, Co-Directors of the Intermountain Ocular Research Center at the University of Utah. Dr. Werner commented, "This is a preventative treatment of PCO, and the preliminary efficacy and safety results look very promising."

Dr. Mindy George-Weinstein, Professor of Biomedical Sciences at the Cooper Medical School of Rowan University, stated "Myo/Nog cells have also been found in a variety of tumors, where we predict they contribute to tumor growth. This targeted 3DNA immunodepletion strategy may be useful as an adjuvant therapy to reduce tumor expansion and recurrence."

Dr. Robert Getts, Chief Science Officer of Genisphere, said "Since the antibody has broad utility and 3DNA nanocarriers can deliver a variety of drug cargoes, we can easily generate targeted drugs for many of these indications." He added, "Genisphere's partnership model for development of nanotherapeutics has set the path forward for clinical testing and future commercialization of these and other candidates."

About Genisphere

Genisphere LLC is the provider of the 3DNA® platform for targeted drug delivery. 3DNA® is a nanoscale, multivalent scaffold made from proprietary, synthetic DNA formed in a flexible, branched structure. 3DNA® nanocarriers are engineered and cross-linked to form a stable architecture while maintaining the biocompatibility of the nucleic-acid building blocks, and demonstrate efficacy and safety with a variety of drug cargos across multiple indications. Genisphere's technology is IP-protected and fully customizable to deliver small molecules, biologics, and nucleic acids with precise specificity enabled by multivalent targeting via antibodies, peptides and other molecular entities. Genisphere leverages a collaborative model to advance its 3DNA® drug delivery platform, and seeks additional partnerships with biotechnology and pharmaceutical companies to improve efficacy and reduce toxicity. Genisphere is also advancing its own lead compounds based on 3DNA® nanotechnology. For more information, please visit http://genisphere.com.

For more information contact:

Tom Bliss
Genisphere
215-996-3027
Tom_Bliss@genisphere.com