Protalix boasts of "revolutionary" advance on oral Gaucher's drug

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Israel's Protalix BioTherapeutics says it is blazing a scientific path that will lead to a new recombinant protein therapeutic for Gaucher's disease that can be delivered orally, eliminating the need for an IV solution.

Best known for its work on Gaucher's, Protalix has been tinkering with enzymes extracted from carrot cells and then delivering it to the patient intravenously. But its researchers have found in animal studies that the enzyme can be delivered orally and remain protected until it breaks down in the blood. And that could provide a significant advance over oral delivery work now being done at Genzyme, which has made a reputation for itself in the rare disease arena.

"Using the plant-cell expression system for oral delivery of GCD is revolutionary because it targets the disease-specific organs without the need for lifetime dependence on repeated intravenous infusions. Moreover, it is unlike substrate reduction therapy which is oral, but may have unpredictable long term untoward effects due to the inhibition of other non-disease-specific compounds. Finally, oral administration of the enzyme for patients with Gaucher disease will increase compliance and facilitate management," said Professor Ari Zimran, director of the Gaucher Clinic in Shaare Zedek Medical Center.

Protalix also reported progress with its preclinical stage program for Fabry disease: PRX-102. The therapy is made from a plant cell expressed modified version of the recombinant human alpha-GAL-A protein.

"We believe our proprietary Fabry product has the potential to be a 'best-in-class' enzyme resulting from the unique modifications made to the protein," said Dr. Yoseph Shaaltiel, Protalix's executive vice president, research and development. "Following the anticipated IND approval, we intend to conduct a phase I/II clinical trial in Fabry disease patients."

- see the Protalix release
- here's the report from Haaretz

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