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BioWorld - Monday, January 12, 2026
Home » Newsletters » BioWorld

BioWorld

Aug. 22, 2013

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Whatcha Say on Pompe, FDA? Biomarin Posts Endpoint Plan

Biomarin Pharmaceutical Inc. has made clearer its Phase III plans for the Pompe disease drug candidate BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (GAA), but the study has not yet begun to enroll patients and talks are still under way about an endpoint that could help better distinguish the compound from Sanofi SA's competitor Lumizyme. Read More

No 'Dry' Spell for Aridis in Anti-Infectives

Aridis Pharmaceuticals LLC is a textbook example of the motto that different roads can lead to the same destination. Read More

Patent Dustup Threatens Cozy Collaborations

When what was once an open and collegial research relationship erupted into a patent inventorship lawsuit between the University of Utah and the University of Massachusetts, researchers Brenda Bass and Thomas Tuschl, once colleagues in a closely related field, became parties in the suit. Read More

Studies Probe Reasons for Targeted Therapy Rashes

For reasons that are not yet understood, "many new targeted agents affect the skin," Stuart Yuspa told BioWorld Today. "It is an extremely important aspect of targeted therapies, and one that has to be addressed." Read More

Other News To Note

• International Stem Cell Corp., of Carlsbad, Calif., said it entered a master clinical research agreement with Duke University to conduct clinical trials research in Parkinson's disease using ISCO's neural stem cell product. Read More

Stock Movers

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Clinic Roundup

• Catabasis Pharmaceuticals Inc., of Cambridge, Mass., started Pathways I, a Phase II trial investigating the safety, tolerability and efficacy of CAT-2003, an oral SMART Linker conjugate constructed using the company's technology. Read More

U.S. Patent Disclosures

• Confometrx, of Palo Alto, Calif., was issued U.S. Patent No. 8,470,561, "GPCR comprising an IC2 insertion," related to G protein-coupled receptors (GPCRs) that have a stabilizing fusion in the second intracellular loop (IC2). Read More

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