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CHDI Foundation, Inc. and Genzyme Corporation have entered into a collaboration to develop adeno-associated virus delivery of a novel gene silencing therapeutic approach for Huntington’s disease. The agreed research plan encompasses the development of a therapeutic clinical candidate that will aim to lower huntingtin protein expression, as well as the preparation and filing of an investigational new drug application with one or more regulatory authorities for an initial clinical appraisal.

“We are pleased to be working with CHDI to develop a gene therapeutic strategy that addresses the underlying molecular basis of Huntington’s disease. By leveraging the extensive body of research tools and knowledge that CHDI has assembled for this disease, it is our hope that this will accelerate drug development,” said Seng Cheng, Head of Research and Early Development, Rare Diseases Science at Genzyme.

“We know that the expanded mutant huntingtin protein plays a pivotal role in the pathogenesis of Huntington’s disease and consequently CHDI is committed to developing diverse and complementary therapeutic strategies that aim to lower this protein in patients,” said Jonathan Bard, Director, Molecular Pharmacology at CHDI. “Genzyme’s expertise in gene therapy, particularly with regard to delivery vehicles, will be invaluable to this particular therapeutic program and we’re very excited to be working with them.”

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Source: CHDI Foundation 



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