Cure Rare Disease Enters Homestretch to Clinical Trial

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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Key Takeaways:

  • 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


Nina Pfister

Source: Cure Rare Disease

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