iBio Inc., a biotechnology company that develops vaccines and therapeutic proteins based on its plant-based iBiolaunch Platform Technology, recently announced the engagement of CBR International Corporation (CBR) for crucial regulatory and clinical services in order to advance iBio’s Orphan Drug design designates, plant-produced human alpha galactosidase A (“?-Gal A”) enzyme component for development as a therapeutic option for its patients with Fabry disease.
CBR International is a global, full service product, clinical, and regulatory company that strives to provide comprehensive development services to the biotechnology, pharmaceutical, and device industries worldwide.
“We expect this engagement to significantly accelerate our path to the clinic for this important product application of our platform technology,” said Robert Erwin, President of iBio. “CBR managed the successful application for Orphan Drug designation of plant-made ?-Gal A, and has substantial experience in this field. They will join the team comprising our regular research and development collaborator, Fraunhofer USA Center for Molecular Biotechnology and our manufacturing partner, Kentucky Bioprocessing, LLC.”
The Fabry disease therapeutic program is a part of iBio’s program to bring forth approximately ten more candidates proteins for commercialization as “biobetter” or “biosimilar” therapeutic commodities. iBio expects to advance these candidates to at least a point sufficient to showcase the advantages of using the iBioLaunch™ platform technology and then enter into a collaborative plan with its partners in the industry. iBio has already shown the applicability of its platform technology to various therapeutic protein classes, extending from cytokines and growth factors to enzymes and antibodies.
Dr. Jeanne Novak, President and CEO of CBR commented, “Enzyme replacement therapy has been constrained by production limitations, interruptions and treatment cost. Patients with Fabry disease who do not receive enzyme replacement therapy are at increased risk of strokes, heart attack, heart disease, and renal failure. iBio’s innovative platform technology promises to add greater capacity for the supply of therapeutic protein biologics so that ?-Gal A and other critical enzymes can be made available to more patients who need them in the future. We are confident that CBR’s clinical and regulatory expertise and extensive prior experience with ?-Gal A will help expedite the development of iBio’s Fabry disease product program.”
Current estimates indicate approximately 8,000 to 10,000 people are affected by Fabry Disease. Present therapies being utilized are estimated to cost over $200,000 per patient a year.
Fabry disease is caused by the deficiency of an enzyme, alpha galactosidase A, whose function is to break down a fatty substance, globotriaosylceramide. The defective gene is carried on the X chromosome, and males with the defect have a tendency to experience worse symptoms than do females who carry only one copy of the detective gene. However, many females who are heterozygous for the defect on the chromosome are symptomatic and are often under-treated. Symptoms typically begin during childhood and include pain in the feet and hands, agiokeratomas, and changes in the cornea. The disease is progressive and symptoms of the heart, kidney, and neurological damage can occur in young adulthood. Enzyme replace therapy with recombinant human alpha-galactosidase has proven to be clinically beneficial in the decline of symptoms.
For more information on iBio and its products, visit the company’s website: www.ibioinc.com
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