CRD founder and CEO, Rich Horgan, recently visited our collaborators at Heidelberg University in Germany to share how the work done in the Grimm lab translates into the delivery of potential treatments CRD is developing for muscle diseases. We are grateful for the work done by Dirk Grimm and his team to develop advanced viral vector technologies and their commitment to bringing more efficacious treatments to patients.
Cure Rare Disease
Biotechnology
Boston, Massachusetts 8,214 followers
501(c)(3) nonprofit leading a nationwide collaboration of researchers and clinicians to develop customized therapeutics.
About us
Cure Rare Disease is a nonprofit biotechnology company whose mission is to enable and finance the development of life-saving genetic medicines for rare and ultra-rare patient populations previously deemed too rare to treat. To realize this, our unique ecosystem facilitates collaboration between world-renowned researchers and clinicians, policy experts and our generous donors. Together, we are fundamentally changing the rare and ultra-rare disease experience and burden for millions of people around the world with our discoveries and methods—while bringing hope to patients waiting for a cure.
- Website
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http://www.cureraredisease.org
External link for Cure Rare Disease
- Industry
- Biotechnology
- Company size
- 2-10 employees
- Headquarters
- Boston, Massachusetts
- Type
- Nonprofit
- Founded
- 2017
- Specialties
- drug development, research, advocacy, education, pharmaceuticals, precision therapy, gene editing , CRISPR, nonprofit, fundraising, accessibility, duchenne , and rare disease
Locations
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Primary
1575 Tremont St.
Unit 1107
Boston, Massachusetts 02120, US
Employees at Cure Rare Disease
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Marshall ("Mark") Smith, NACD.DC
Senior Executive Leader | Operations | Board Governance | Corporate Finance | Strategic Planning | M&A | IPO | Restructuring | Turnaround
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Mychal Tepker
3x Salesforce Certified Professional
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Steph Herzog
Owner, Designer @ Bespoke Home (SVM Design)
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Brent Warner
CEO and Board Member
Updates
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Spinocerebellar Ataxia Type 3 (SCA3) is a neuromuscular disease, affecting 1 to 5 in 100,000 people, and is the most common form of spinocerebellar ataxia. Mutations in the ATXN3 gene lead to toxic protein accumulation, impacting ataxin-3 enzyme function. Learn more about SCA3 and our efforts to develop a treatment for it using ASO technology in collaboration with Leiden University Medical Center: https://lnkd.in/gqFKkZp8 #SCA3 #RareDiseaseAwareness #CureRareDisease
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We are excited to announce significant additions to our leadership team and Board of Directors. Markus Ward has joined us as Chief Advancement Officer, bringing a wealth of experience and passion to our mission. Additionally, we are excited to welcome Jeremy Sternberg, J.D., Klaus Veitinger, M.D., Ph.D., and Mike Williams to our Board of Directors. Their diverse expertise and commitment will undoubtedly strengthen our efforts in advancing our mission. Read More: https://loom.ly/Kq__Low #CureRareDisease
Cure Rare Disease Expands Leadership Team and Board of Directors to Advance Mission | | Cure Rare Disease
cureraredisease.org
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Introducing the Angel Treatment Pathway. Developed in collaboration with global value, access and policy expert, Eric Faulkner, and funded by the Muscular Dystrophy Association, the Angel Treatment Pathway is the first comprehensive approach mapped to-date in the United States that offers a potential avenue for ultra-rare disease patients to access potentially life-saving therapies. In addition to drafting the initial proposal for the Angel Treatment Pathway, our team held meetings with leading rare disease and advanced therapy associations, industry, payers and advocacy groups to gather feedback, insight, and pressure test the details of the proposal from various perspectives in the rare disease ecosystem. This assessment and roadmap shows that most of the components of the pathway can actionably leverage the practices and infrastructure of our existing health system. The Angel treatment pathway report also helps make a case for the value of shifting our thinking about rare disease. By the numbers, the collective of up to 8,000 rare diseases rivals or exceeds many of our most devastating diseases, but these patients often have poor or no options. By moving from a historical view of ultra-rare disease as thousands of small populations to a collective from a solutions perspective, we have the potential to advance cross-cutting solutions that can rise to meet this need. We are grateful to each of the collaborators who contributed valuable feedback for this initial draft, which we hope will be the beginning of solving the most pressing challenges to accessing potential therapies for ultra-rare disease patients. Visit our website to learn more and access the full report: https://loom.ly/IaRagS0 #RareDisease #AngelTreatmentPathway #CureRareDisease
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In 2023, we raised a total of $3,759,840 from the generous support of our community. Over 92% of these funds have been allocated directly to research and development initiatives. This significant investment enables us to continue our work to advance potentially life-saving treatments for ultra-rare diseases. Thank you for your continued support in fueling our mission! Learn more in our 2023 annual report: https://loom.ly/mkXc4-U #CureRareDisease #Research #RareDisease
Cure Rare Disease Annual Report
cureraredisease.org
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Join us in paving the way for a future where rare disease patients are no longer left behind. Together, we're advancing science and technology to make hope for safe and effective treatments a reality. Learn more: https://loom.ly/mkXc4-U #CureRareDisease #RareDisease #GeneTherapy
Cure Rare Disease Annual Report
cureraredisease.org
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Register now to be part of the Disney Wine & Dine Half Marathon Weekend! Celebrating 15 years of excellence, this event promises four immersive days filled with iconic Disney Chefs, delectable food, and unforgettable running experiences. Join the CRD team today and prepare to embark on a journey of culinary delights and athletic achievement. Secure your spot now! https://loom.ly/FRdRXcY #DisneyWineAndDine
2024 Disney Wine & Dine Half Marathon Weekend
runsignup.com
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Establishing a cell line for Duchenne Muscular Dystrophy (DMD) is the first stage in our process to gauge efficacy of a potential therapeutic approach before moving into testing in mouse models. This crucial part of the research process involves several key steps and can take a considerable amount of time. Read our latest blog to learn more about this process and its importance in developing potential therapies for DMD. Read more: https://loom.ly/uV5ceE0 #DMD #RareDiseaseResearch #CRISPR #CureRareDisease
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Our 2023 Annual Report is out! In this report, you will find a comprehensive overview of all our team accomplished last year and milestones for the years ahead, including updates on each of our drug development programs, technology, policy advancements, and much more. The work detailed here would not be possible without the commitment and dedication of each of our research collaborators, industry partners, board members, supporters, and staff. Thank you for your continued support of our work to transform realities for ultra-rare disease patients! View here: https://loom.ly/mkXc4-U #AnnualReport #RareDisease #CureRareDisease #CRD
Cure Rare Disease Annual Report
cureraredisease.org
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Calling all Disney enthusiasts and avid runners! Join CRD's team at the Disney Wine & Dine Half Marathon Weekend, celebrating its crystal anniversary with four days at Walt Disney World® Resort. Learn more and register now! https://loom.ly/FRdRXcY #CRDTeam #DisneyWineAndDine #RunForACause
Disney Wine and Dine Half Marathon Weekend 2024
runsignup.com