Nonprofit Biotech Announces Major Milestones Achieved in 2020 to Date, Despite Pandemic Headwind
Boston, Massachusetts--(Newsfile Corp. - September 30, 2020) - Cure Rare Disease (CRD), the leading nonprofit biotech developing customized therapeutics for rare diseases, is pleased to announce its recent accomplishments that have advanced preclinical development of their first-in-human CRISPR therapeutic for Duchenne muscular dystrophy this year amidst headwinds of the global pandemic.
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Cure Rare Disease announces recent accomplishments that have accelerated preclinical development of their CRISPR-based drug for DMD despite global pandemic.
CRD has shown robust dystrophin upregulation from recent animal dosing studies, completed their preIND meeting with FDA, and are approaching their final study before dosing projected for early 2021.
The company has also established subsequent patient cohorts that are next in line for treatment, inspiring hope for all.
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Cure Rare Disease
Cure Rare Disease (CRD) is a Boston-based non-profit biotechnology company founded by Rich Horgan, Blavatnik Life Science Entrepreneur-in-Residence at Harvard Business School who has witnessed three generations of his family affected by Duchenne muscular dystrophy, a fatal, neuromuscular disease. As anyone who is touched by a fatal, rare disease knows, time is of the essence. To overcome the obstacles inherent in the existing model of drug development, CRD has taken a new approach, forming an unprecedented collaboration with leading researchers and institutions nationwide in genetics, genomics, bioinformatics and biotechnology. Horgan and team are pioneering the development of a new platform for customized therapeutic treatments that can be applied to a range of rare, genetic diseases, and aim to inspire hope for a cure. Learn more at www.cureraredisease.org.
Source: Cure Rare Disease
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