Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLDSymposium(TM) 2015
- Optimized for Efficient Targeting to Multiple Key Muscle Tissues of Disease
- Significantly Better Than Approved ERT in Reducing Disease Substrate (Glycogen)
- Addition of Chaperone Further Improves Glycogen Reduction
ORLANDO, Fla. and CRANBURY, N.J., Feb. 10, 2015 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today highlighted results from preclinical studies of its next-generation Pompe ERT at WORLDSymposium™ 2015 in Orlando, Florida.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "Our goal is to develop a fundamentally better therapy for patients living with Pompe disease. The results presented at WORLDSymposium suggest that we have made significant progress with our Pompe program as we look toward addressing some of the major challenges with the current approved therapy including enzyme activity and stability; targeting and uptake; and tolerability and immunogenicity. These results also validate our internal biologics capabilities to develop and manufacture our own cell line at clinical scale. We look forward to moving our next-generation ERT into the clinic later this year." …
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