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CURE RARE DISEASE: A YEAR IN REVIEW

Significant Milestones Fuel Development of Customized Therapeutics for Duchenne muscular dystrophy

BOSTON,MA (January 8, 2020)—Cure Rare Disease (CRD), the emerging, Boston-based nonprofit on a mission to develop precision medicine for rare diseases, is pleased to provide an annual review of its activities and accomplishments in 2019. Successful fundraising efforts have allowed CRD to accelerate their drug development plan, achieving significant milestones that are leading to the first in-man, customized therapeutic for Duchenne muscular dystrophy (DMD). 

Using revolutionary CRISPR gene-editing technology implemented by a team of world-class geneticists and clinicians, CRD’s therapeutic has been designed specifically for the brother of Founder & President Rich Horgan, Terry Horgan (age 24), who has been battling DMD for much of his life. The close of the year marks the next phase of growth, as CRD moves into mouse testing, dosing, and ultimately application for an FDA-approved, human clinical administration expected in late 2020. The company has also established a subsequent patient cohort that is next in line for treatment, inspiring hope for all those impacted by rare diseases.

Groundbreaking Drug Development

CRD achieved groundbreaking milestones on the scientific front, including: showing in-vitro proof of concept for the custom CRISPR therapy, which restored the patient’s missing protein, dystrophin; Established the humanized DMD mouse colony for preclinical testing in early 2020; Preclinical development plan and securement of clinical site for the therapeutic administration; Establishment of their first patient cohort for their unique custom medicine development process; Formation of their research collaboration spanning 7 institutions and over 15 researchers; Commencement of FDA engagement process; Engagement with leading payors to begin discussion of a customized therapeutics reimbursement mechanism.

Fundraising Milestones

When time is of the essence, every dollar counts, and CRD is grateful to share that over the past 12 months, a combination of individual and corporate donations have pushed them across the $1M+ threshold of total capital raised that is fueling the nonprofit’s research and development initiatives. Namely, the Inaugural Golf to Cure Rare Disease that took place in Branford, CT attracted over 360 attendees and raised $370,000+; The Third Annual Cure Rare Disease gala in Boston included 130 attendees with $160,000+ raised. Household brands like East River Energy, Global Partners LP, Mystic Oil, and Murphy Beverage Company have all gotten on board in various capacities via corporate giving programs that have further moved the needle. Corporate partners continue to join in support of this unprecedented mission.

Key Appointments

In 2019, Cure Rare Disease added seven new board members including: Karen Morales to drive marketing plan, currently serving as the Founder of Marketing Magnet and is personally impacted by rare disease; Jon Bircher, Chief Commercial Officer & Board Director of Cello Heath who is advising on drug development for CRD; Stephanie Herzog, whose 4-year-old son is impacted by DMD and is Founder of a boutique interior design firm now leading fundraising efforts alongside newly appointed board members, Renata Maybruck and Mark Smith; Naomi Hoyle who is driving FDA pathway modification, and Carolina Alarco, the gala chair and Founder of Biotech Advisors LLC with more than 25 years of relevant industry experience.

Going Forward

Cure Rare Disease has a vision for 2020 that includes projected medical advancement and growth via successful completion of dose-finding for efficacy and GLP toxicology studies; FDA approval to dose their first patient, Terry Horgan, with first-in-man administration of their customized therapeutic; and subsequently advancing their first cohort of patients to the preclinical stage. 

“In reflection, we are beyond grateful for the contributions that all donors have generously offered over the course of the past year that have enabled our collaboration to advance our unique drug development process—inspiring hope for my brother and others who are impacted by rare disease,” stated CRD Founder & President, Rich Horgan. “We look forward to rolling out additional campaigns with our existing and new partners across the U.S. to support the larger-scale development of customized therapeutics, and are working diligently to pave the way for FDA approval later this year.”

ABOUT CURE RARE DISEASE

Cure Rare Disease (CRD) is a Boston-based nonprofit 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


Key Takeaways
  • Successful fundraising efforts have allowed CRD to accelerate their drug development plan.
  • CRD has achieved significant milestones in developing the first in-man, customized therapeutic for Duchenne muscular dystrophy.
  • Cure Rare Disease has a vision for 2020 that includes projected medical advancement and growth.
Quotes
“In reflection, we are beyond grateful for the contributions that all donors have generously offered over the course of the past year that have ena...
Rich HorganFounder & President, CRD
“We look forward to rolling out additional campaigns with our existing and new partners across the U.S. to support the larger-scale development of ...
Rich HorganFounder & President, CRD
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