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 The leading web portal for pharmacy resources, news, education and careers December 15, 2017
Pharmacy Choice - Pharmaceutical News - BioStrategies LC Awarded NTSAD Grant to Develop an Enzyme Replacement Therapy for GM1 Gangliosidis - December 15, 2017

Pharmacy News Article

 11/10/14 - BioStrategies LC Awarded NTSAD Grant to Develop an Enzyme Replacement Therapy for GM1 Gangliosidis
November 10, 2014 BioStrategies LC, developers of plant-derived therapeutics and enzyme replacement therapeutics (ERT) for rare lysosomal diseases, announced today that the company has been awarded a two-year research grant from the National Tay-Sachs and Allied Diseases Association (NTSAD) to develop a central nervous system-targeted enzyme replacement therapy for GM1 gangliosidosis.

"This grant from NTSAD will allow BioStrategies to test our novel protein carrier technology as a potentially breakthrough treatment for GM1 gangliosidosis, " said BioStrategies CEO David N. Radin, Ph.D. "We are optimistic about the ability of this protein fusion technology to deliver enzyme therapy to the brain based our previous success with nasal delivery of vaccines."

GM1 gangliosidosis is a lysosomal storage disease caused by an inherited gene defect that prevents the breakdown of certain complex lipids, which then accumulate to pathological levels, particularly in cells of the central nervous system. The disease is marked by progressive neurodegeneration

The research will also determine the feasibility of BioStrategies' plant-based bioproduction platform for this complex protein drug. Plants bring key advantages in safety and the speed and cost of large-scale production of complex human proteins.

The principal investigators on the project are Drs. David N. Radin (lead) and Carole L. Cramer from BioStrategies, and GM1 gangliosidosis expert, Dr. Alessandra d'Azzo, of St. Jude Children's Research Hospital.


National Tay-Sachs & Allied Diseases Association (NTSAD) is one of the oldest patient advocacy groups in the country. NTSAD focuses on supporting research and the needs of over 500 families and individuals worldwide in several ways, including raising awareness to prevent disease. Their programs and services includes providing comprehensive support services to affected families through their Peer Support Group. The NTSAD research support program aims to direct, fund and promote promising research to develop treatments and cures. NTSAD also offers educational and awareness programs directly and through collaborations with other rare disease programs and community partners. On a broader front, the association advocates for families and persons of all ages with disabilities on an individual, state and national level.

About BioStrategies

BioStrategies LC is a biotech company focused on the development of innovative protein-based therapeutics for human health and veterinary applications. The company has received multiple grant awards from NIH and other federal and state agencies and received the prestigious SBA Tibbett's Award in 2012 for innovative research excellence in the national Small Business Innovative Research Program (SBIR). This award recognized the company's pioneering research to develop novel enzyme replacement therapeutic (ERT) technology, which employs biotechnology approaches for production of the human enzyme in a safe, low cost plant-based production system for the rare genetic condition, Gaucher's Disease. This innovative technology was the foundation for FDA approval in 2012 of a new plant-produced ERT drug for Gaucher's, Elelyso, that was taken through clinical trials and is marketed worldwide by a partnership between Pfizer and Protalix. Building on this success, the first plant-produced biologic drug to receive FDA approval. BioStrategies' new lectin-based ERT fusion protein delivery system is designed to increase the effectiveness of ERT drugs by more efficiently delivering them to cells and tissues of the body that are not treated adequately by existing drugs. The grant award to BioStrategies from NTSAD will be complemented by another recently approved grant for GM1 research by NIH/NINDS. These studies are being conducted at the preclinical stage in animal models of this disease.


David Radin, Ph.D.

Managing Director

BioStrategies LC


Susan Kahn

Executive Director

National Tay Sachs & Allied Diseases Association


 c. NewMediaWire, LLC

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