Cure Rare Disease

Bridging the gap between researchers and a cure

Boston, MA   |  https://www.cureraredisease.org/

Mission

Cure Rare Disease forges collaborations with world-renowned researchers, clinicians and other stakeholders to develop a sustainable mechanism of customized therapeutics with unparalleled speed. We see the process being applied in a range of rare, genetic disorders that lack an effective treatment of cure.

Ruling year info

2018

Founder & President

Richard Horgan

Main address

1575 Tremont Street Unit 1107

Boston, MA 02120 USA

Show more contact info

EIN

82-2473513

NTEE code info

Alliance/Advocacy Organizations (G01)

IRS filing requirement

This organization is required to file an IRS Form 990 or 990-EZ.

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Communication

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Cure Rare Disease develops life-saving therapeutics for patients impacted by neuromuscular diseases. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors. Our therapeutics are designed specifically for the men and women who continue to fight for their right to live long, full, healthy lives despite having been diagnosed with a rare genetic disorder for which they’ve been told there is no treatment or cure.

Our programs

SOURCE: Self-reported by organization

What are the organization's current programs, how do they measure success, and who do the programs serve?

Educational and Referrals

Educating and referring impacted families and caregivers to centers of excellence.

Population(s) Served
Children and youth

Leading a collaboration with world-renown researchers to develop life-saving therapeutics for individuals suffering from neuromuscular rare diseases for which there is no treatment or cure.

Population(s) Served
Children and youth
Adults

Where we work

Awards

Researcher's Award 2022

Charles River Labs

Our results

SOURCE: Self-reported by organization

How does this organization measure their results? It's a hard question but an important one.

Number of patient neutralizing antibody analyses performed

This metric is no longer tracked.
Totals By Year
Population(s) Served

People with intellectual disabilities, People with learning disabilities, People with physical disabilities, Chronically ill people, Terminally ill people

Related Program

Gene Therapy Medicine Development

Type of Metric

Outcome - describing the effects on people or issues

Direction of Success

Increasing

Context Notes

Performed analysis on patient serum samples to understand if patients had elevated antibodies to therapeutic delivery modality to help inform and educate patients.

Number of Whole Genomes Sequenced

This metric is no longer tracked.
Totals By Year
Population(s) Served

People with learning disabilities, People with intellectual disabilities, People with physical disabilities, Chronically ill people, Terminally ill people

Related Program

Gene Therapy Medicine Development

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

Performed analysis on patient DNA to define and clarify the genetic mutation impacting the patient. Whole genome sequencing is critical to define the mutation to develop a drug to treat the patient.

Number of Pharmacology Studies Conducted

This metric is no longer tracked.
Totals By Year
Population(s) Served

People with intellectual disabilities, People with physical disabilities, People with learning disabilities, Chronically ill people, Terminally ill people

Related Program

Gene Therapy Medicine Development

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

Pharmacology studies are defined as in-vitro and in-vivo experiments performed with candidate therapeutics.

Number of patient education hours

This metric is no longer tracked.
Totals By Year
Population(s) Served

People with intellectual disabilities, People with learning disabilities, People with physical disabilities, Chronically ill people, Terminally ill people

Related Program

Educational and Referrals

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

Cure Rare Disease provides patient families and advocates the opportunity to tour labs, participate in educational webinars and learn about Cure Rare Disease’s efforts in the drug development space.

Number of cell lines established

This metric is no longer tracked.
Totals By Year
Population(s) Served

People with intellectual disabilities, People with learning disabilities, People with physical disabilities, Chronically ill people, Terminally ill people

Related Program

Gene Therapy Medicine Development

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

Cure Rare Disease’s offers patients the opportunity to directly contribute to research by providing a tissue biopsy which is then converted into a cell line for use in research.

Goals & Strategy

SOURCE: Self-reported by organization

Learn about the organization's key goals, strategies, capabilities, and progress.

Charting impact

Four powerful questions that require reflection about what really matters - results.

Cure Rare Disease envisions a future without rare diseases. To accomplish this goal, Cure Rare Disease collaborates with academic and clinical institutions across the Northern Hemisphere to engineer and test life-saving therapeutics for patients impacted by rare, neuromuscular diseases. Cure Rare Disease focuses on developing therapeutics for diseases for which there is no treatment or cure and for which there are too few patients to attract commercial drug development interests.

To realize our mission, Cure Rare Disease continues to develop research collaborations with institutions and industry members including Yale, Hospital for Sick Kids, Ohio State University, Leiden University Medical Center and other institutions around the globe. We have developed a novel framework by which therapeutics for rare and ultra-rare diseases are created. In partnership with our collaborators and patients, we are successfully advancing life-saving therapeutics.

Cure Rare Disease’s organizational capabilities include a staff members to support and engage with patients and patient families. Moreover, our research capabilities include 7+ research collaborations and 5+ partnerships with industry to advance our therapeutics. Moreover, we are supported by regulatory, clinical and manufacturing advisors and consultants to help guide the programs.

We have 19 active therapeutic development programs broken down into 5 phases. Our first therapeutic has entered the clinic while 3 other therapeutics are in the 4th stage of development. An additional 5 therapeutics are in the 3rd phase of development and the remaining therapeutics are split evenly between the first and second stages of development. In aggregate, over 200,000 people would be impacted if successful. Beyond drug development, we have conducted neutralizing antibody analyses on over 30 patients samples and have also conducted whole genome sequencing on over 15 patient samples.

How we listen

SOURCE: Self-reported by organization

Seeking feedback from people served makes programs more responsive and effective. Here’s how this organization is listening.

done We demonstrated a willingness to learn more by reviewing resources about feedback practice.
done We shared information about our current feedback practices.
  • Who are the people you serve with your mission?

    Cure Rare Disease serves the rare disease community including researchers, clinicians, patients impacted, their families and their communities.

  • How is your organization collecting feedback from the people you serve?

    Focus groups or interviews (by phone or in person), Community meetings/Town halls, Constituent (client or resident, etc.) advisory committees, Social media,

  • How is your organization using feedback from the people you serve?

    To identify bright spots and enhance positive service experiences, To make fundamental changes to our programs and/or operations, To inform the development of new programs/projects, To strengthen relationships with the people we serve,

  • What significant change resulted from feedback?

    We launched an antibody screening program in Fall 2020 when we learned that the community broadly wanted this program but was unable to have their needs fulfilled through other mechanisms.

  • With whom is the organization sharing feedback?

    The people we serve, Our staff, Our board,

  • Which of the following feedback practices does your organization routinely carry out?

    We collect feedback from the people we serve at least annually, We take steps to get feedback from marginalized or under-represented people, We aim to collect feedback from as many people we serve as possible, We take steps to ensure people feel comfortable being honest with us, We act on the feedback we receive,

  • What challenges does the organization face when collecting feedback?

Financials

Cure Rare Disease
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Operations

The people, governance practices, and partners that make the organization tick.

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lock

Connect with nonprofit leaders

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Build relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.

  • Analyze a variety of pre-calculated financial metrics
  • Access beautifully interactive analysis and comparison tools
  • Compare nonprofit financials to similar organizations

Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.

Cure Rare Disease

Board of directors
as of 06/23/2022
SOURCE: Self-reported by organization
Board chair

Richard Horgan

Melissa Hunter-Ensor

Greenberg-Traurig, LLP

Kwesi Acquay

JP Morgan

Stephanie Herzog

Mark Smith

California Resources Corp.

Sara Cole

IQVIA

Karen Morales

Magnet Marketing

Keven Lin

Cody Tranbarger

Prime Medicine

Ali Urman

Ark Invest

Jessica Curran

Kindness Over Muscular Dystrophy

Greg Klassen

Liz Duffy

Greenwich Country Day School

Board leadership practices

SOURCE: Self-reported by organization

GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.

  • Board orientation and education
    Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes
  • CEO oversight
    Has the board conducted a formal, written assessment of the chief executive within the past year ? Yes
  • Ethics and transparency
    Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes
  • Board composition
    Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes
  • Board performance
    Has the board conducted a formal, written self-assessment of its performance within the past three years? Yes

Organizational demographics

SOURCE: Self-reported; last updated 6/23/2022

Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.

Leadership

The organization's leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Male, Not transgender (cisgender)
Sexual orientation
Heterosexual or Straight
Disability status
Person with a disability

Race & ethnicity

Gender identity

 

Sexual orientation

No data

Disability

We do not display disability information for organizations with fewer than 15 staff.

Equity strategies

Last updated: 06/23/2022

GuideStar partnered with Equity in the Center - an organization that works to shift mindsets, practices, and systems to increase racial equity - to create this section. Learn more

Data
  • We review compensation data across the organization (and by staff levels) to identify disparities by race.
  • We ask team members to identify racial disparities in their programs and / or portfolios.
  • We employ non-traditional ways of gathering feedback on programs and trainings, which may include interviews, roundtables, and external reviews with/by community stakeholders.
  • We disaggregate data by demographics, including race, in every policy and program measured.
  • We have long-term strategic plans and measurable goals for creating a culture such that one’s race identity has no influence on how they fare within the organization.
Policies and processes
  • We seek individuals from various race backgrounds for board and executive director/CEO positions within our organization.
  • We have community representation at the board level, either on the board itself or through a community advisory board.
  • We help senior leadership understand how to be inclusive leaders with learning approaches that emphasize reflection, iteration, and adaptability.
  • We engage everyone, from the board to staff levels of the organization, in race equity work and ensure that individuals understand their roles in creating culture such that one’s race identity has no influence on how they fare within the organization.