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Amicus Therapeutics, Penn joins forces.

Byline: Anthony Vecchione

Amicus Therapeutics entered into a joint venture with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency and one additional undisclosed rare metabolic disorder.Amicus Therapeutics entered into a joint venture with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency and one additional undisclosed rare metabolic disorder.

The alliance will combine Amicus' protein engineering and glycobiology expertise with Penn's adeno associated virus gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability.

Amicus Therapeutics Chairman and CEO John Crowley, told NJBIZ: "We look forward to continuing to build a portfolio of gene therapies that will one day bring a potential cure for these patients. Today's collaboration with Penn is just another proof point of our dedication to those living with rare diseases and their families, and our laser focus on making a meaningful difference for them."

In a statement, Amicus said Penn's AAV vector technology is designed to improve targeting, tropism, safety, immunogenicity and gene delivery, while Amicus' protein engineering capabilities may optimize protein expression, secretion, targeting and uptake of the target protein.

The pact between Amicus and Penn is a Research, Collaboration and License Agreement, with funding provided to Penn to advance the preclinical research programs in the Wilson Lab and license certain technologies invented under the collaboration.

Details and an overview of the collaboration will be provided during the Amicus Analyst Day on Thursday.View the full article from NJBIZ at http://www.njbiz.com/article/20181008/NJBIZ01/181009858/amicus-therapeutics-penn-joins-forces. Copyright 2018 BridgeTower Media. All Rights Reserved.

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Title Annotation:University of Pennsylvania. Perelman School of Medicine
Author:Vecchione , Anthony
Publication:NJ Biz
Date:Oct 8, 2018
Words:313
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