Emily's Entourage
Join the quest to cure CF
Programs and results
What we aim to solve
Emilys Entourage accelerates research and drug development for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations. By providing critical leadership and coordination, Emilys Entourage drives high-impact research, cultivates multi-stakeholder collaboration, and facilitates information exchange to speed breakthroughs. Our goal is singular: to expedite lifesaving treatments and a cure for the final 10% who are waiting with bated and fading breath.
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Strategic Approach
The funds raised by Emily’s Entourage drive high-impact research, information exchange, and collaboration to accelerate breakthroughs for the roughly 10% of the CF population without a targeted therapy available or in development, including those with nonsense mutations.
The work funded by Emily’s Entourage is particularly significant because although nonsense mutations are relatively uncommon in the CF population, they are the cause of approximately 12% of all genetic diseases, affecting roughly 30 million people worldwide. Progress on nonsense mutations of Cystic Fibrosis can have far-reaching implications for patients suffering from many different genetic diseases, including certain types of muscular dystrophy, inherited blood disorders, and cancers.
Research Grants
Emily’s Entourage’s (EE) Grant Program provides critical seed funding to innovative investigators with promising strategies that accelerate research and drug development for those in the final 10% of the CF community who do not benefit from CFTR modulators, including those with rare and nonsense mutations. There are multiple grant options:
Translational Grants
Collaborative Grants
Venture Philanthropy Investments
The objective is to fund promising therapies and drug-delivery mechanisms that have the potential to accelerate the development of gene-based therapies for people with CF. Potential areas of interest include but are not limited to the following list.
Gene Editing
Exogenous Delivery of Full-length CFTR Gene
RNA Editing
Non-viral vectors for DNA and RNA delivery
Scientific Symposia and Gatherings
Emily’s Entourage hosts scientific symposia that bring together the world’s top CF minds from academia, clinical care, and the biopharmaceutical industry. The goal of these symposia is to generate multi-disciplinary discussions, exchange ideas, share data and updates, and foster partnerships to expedite research and drug development for nonsense mutations using the W1282X mutation as a prototype.
Cell Models
Emily is one of only a few hundred people worldwide with two copies of the rare W1282X nonsense mutation, making her “pure” cells a highly valuable research asset. One of the most vital resources that Emily’s Entourage offers the scientific community is cells, which are developed into critically important cell models, including immortalized cell lines, induced pluripotent stem cells, airway epithelial cells, and organoids, and shared with investigators and companies around the world to spur research and drug development.
Where we work
External reviews
Our results
How does this organization measure their results? It's a hard question but an important one.
Number of research studies funded
This metric is no longer tracked.Totals By Year
Population(s) Served
People with diseases and illnesses
Related Program
Research Grants
Type of Metric
Output - describing our activities and reach
Direction of Success
Increasing
Context Notes
Emilys Entourage has awarded over $6.7 million in grants at the world's top academic medical centers targeting Cystic Fibrosis-related nonsense mutations.
Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
Emily's Entourage aims to accelerate life-saving treatments and cures for for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations.
What are the organization's key strategies for making this happen?
Emily's Entourage is upending traditional methods of disease research by applying a business-like approach to accelerate cutting-edge treatments that will reach the clinic within three years. Under the direction of its Scientific Advisory Board, Emily's Entourage has established strategic research priorities to guide its targeted financial investments in research projects with the greatest potential for fast impact. This results-oriented approach to expediting research is paradigm shifting and represents a sharp departure from the norm, wherein the scientific community drives research direction and progress is slow, fragmented, and inefficient.
What are the organization's capabilities for doing this?
Emily's Entourage has an international Scientific Advisory Board with pre-eminent researchers and drug developers in Cystic Fibrosis, Nonsense Mutations, and Immunology. The organization also has a Scientific Director with decades of experience in CF-related drug development.
What have they accomplished so far and what's next?
Emily's Entourage has made significant progress in advancing research and drug development for nonsense mutations of Cystic Fibrosis since its founding. Since 2011, Emily's Entourage has awarded 9 grants to top researchers at leading academic institutions around the world, leading to publications in prominent peer-reviewed journals. By leveraging its connections in industry and academia, the organization brokered an FDA-approved investigator-initiated clinical trial of a combination therapy. In addition, Emily's Entourage has provided seed funding to launch a novel mutation-agnostic CF Gene Therapy company called Talee Bio with researchers at the University of Iowa and Children's Hospital of Philadelphia. One area of focus is developing cell model systems to spur research and drug development in nonsense mutations of Cystic Fibrosis. To that end, Emily's Entourage has developed and disseminated a "toolbox" of homozygous nonsense mutation cells in various formats, including organoids, nasal epithelial cells, induced pluripotent stem cells, and more to researchers around the world to expedite drug discovery efforts. In 2015, Emily's Entourage was named a White House Champion of Change for Precision Medicine in recognition of its trailblazing efforts.
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
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Connect with nonprofit leaders
SubscribeBuild 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.
Emily's Entourage
Board of directorsas of 01/19/2024
Michael Golinkoff
Nickie Boston
Joan Denenberg
Nicholas Dinges
Nancy Astor Fox
Liza Kramer
Coby Kramer-Golinkoff
Emily Kramer-Golinkoff
Beth Shapiro Lavin
Jessica Rutstein Lazarus
Greg Reh
Danielle Selber
Julie Wertheimer
Doug Young
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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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 ? No -
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? No
Organizational demographics
Who works and leads organizations that serve our diverse communities? Candid partnered with CHANGE Philanthropy on this demographic section.
Leadership
The organization's leader identifies as:
Race & ethnicity
No data
Gender identity
No data
Transgender Identity
No data
Sexual orientation
No data
Disability
No data