USHER 1F COLLABORATIVE INC

Vision for Our Future

Newtonville, MA   |  http://usher1f.org

Mission

Usher 1F Collaborative is a 501c3 nonprofit foundation whose mission is to fund medical research to find an effective treatment to save or restore the vision of those with Usher Syndrome type 1F.

Ruling year info

2014

President and Director

Melissa Kershman Chaikof

Vice President and Director

Dr. Elliot L. Chaikof

Main address

321 Walnut St., No 228

Newtonville, MA 02460 USA

Show more contact info

Formerly known as

Vision for a Cure

EIN

46-4309387

NTEE code info

Fund Raising and/or Fund Distribution (H12)

Eye (H41)

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

Blog

Programs and results

What we aim to solve

SOURCE: Self-reported by organization

Usher Syndrome is the leading cause of inherited deaf-blindness. Type 1 is the most severe, with those affected born profoundly deaf with impaired balance. During childhood to adolescence, they begin to lose their vision to retinitis pigmentosa, first with night blindness, followed by increasingly narrowing tunnel vision, until total blindness results sometime during adulthood. Usher 1F Collaborative seeks to find a cure for Usher Syndrome type 1F, which is caused by mutations on the PCDH15 gene.

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?

Usher 1F Project, Westerfield Lab, University of Oregon Institute of Neuroscience

Monte Westerfield, Ph.D., is at the Institute of Neuroscience and Department of Biology at the University of Oregon. His research is focused on Usher Syndrome with particular expertise in creating zebrafish models of Usher Syndrome mutations. Usher 1F Collaborative has funded the creation of a zebrafish model of the Usher Syndrome Type 1F R245X mutation that is carried by ~2% of those of Ashkenazi Jewish descent. Dr. Westerfield and his team, including Jennifer Phillips, Ph.D., are now beginning testing of drugs and gene therapies on our zebrafish that have the potential to halt or slow the progression of vision loss.

Population(s) Served
People with vision impairments
People with hearing impairments

Edwin Stone, M.D., Ph.D., who is working to develop a cure for Usher 1F along with other types of retinitis pigmentosa at his lab at the University of Iowa, is working to halt further retinal degeneration from Usher 1F through gene replacement and to repair damage that has already occurred through replacement of damaged retinal photoreceptor cells with new cells made from the patient’s own skin cells, with a goal of a clinical trial once safety and efficacy are determined.

Population(s) Served
People with vision impairments
People with hearing impairments

Zubair Ahmed, Ph.D., at the University of Maryland, has developed a mouse model of the most prevalent Usher 1F mutation that he is now using to test potential gene therapies and, in collaboration with Dr. Westerfield, potential drug therapies. He is also working to develop a mini gene for gene replacment for Usher 1F.

Population(s) Served
People with vision impairments
People with hearing impairments

Livia Carvalho, Ph.D., at the University of Western Australia, is collaborating with Dr. Ahmed to develop gene replacement therapy for Usher 1F. She has developed a dual vector gene replacement therapy, which Dr. Ahmed is testing for efficacy on our mouse.

Population(s) Served
People with vision impairments
People with hearing impairments

Leah Byrne, Ph.D. at the University of Pittsburgh is developing a split gene dual vector system for gene replacement. Her unique approach increases the efficiency of viral vector co-infection, increasing efficacy of large gene expression. Once complete, Dr. Ahmed will test her vectors for efficacy on our mouse.

Population(s) Served
People with vision impairments

David Corey, Ph.D., is the Bertarelli Professor of Translational Medical Science, Blavatnik Institute of Neurobiology, Harvard Medical School. After years of studying protocadherin 15, the Usher 1F gene, in relation to hearing, he decided to pursue research on treating the vision loss of Usher 1F after attending Usher 1F Collaborative's May 2017 researchers symposium. We are very fortunate that he has obtained a substantial amount of funding from outside sources, most notably from the Bertarelli Foundation. Dr. Corey is working on three approaches to gene therapy, mini genes, split genes, and gene editing. He has begun testing of mini genes for efficacy on our zebrafish.

Population(s) Served
People with vision impairments
People with hearing impairments

Where we work

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 research studies funded

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

People with hearing impairments, People with vision impairments

Related Program

Usher 1F Project, Westerfield Lab, University of Oregon Institute of Neuroscience

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

We fund research labs to conduct translational research for a cure for the vision loss of Usher Syndrome type 1F.

Number of research studies conducted

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

People with hearing impairments, People with vision impairments

Related Program

Usher 1F Research Project at the Corey Lab

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

Context Notes

Testing begun of multiple gene therapy treatments for the vision loss of Usher 1F.

Number of grants and research funding awarded to the institution

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

People with hearing impairments, People with vision impairments

Related Program

Usher 1F Research Project at the Corey Lab

Type of Metric

Output - describing our activities and reach

Direction of Success

Increasing

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.

Usher 1F Collaborative is working to fund medical research to find an effective treatment or cure for the vision loss of Usher Syndrome type 1F. Our goal is to both halt further vision loss and reverse vision loss in those already affected and to prevent vision loss in babies born with this disorder.

We have identified 3 potential paths to a cure, gene, drug, and stem cell therapies. Our initial goal was to develop an animal model of the Usher 1F mutation on which to test potential therapies. We now have both a zebrafish and mouse model, both of which display all 3 traits of Usher 1F, profound deafness from birth, an impaired vestibular system, and progressive vision loss. The challenge to developing gene therapy for Usher 1F is that our gene is large and will not fit on the current viral vectors used to deliver replacement genes. Thus, our researchers are working on 4 different strategies, a split gene dual vector approach, a mini-gene, base editing, and development of an AAV viral vector that will hold our entire large gene.
We have already begun testing on our mouse and zebrafish of four gene therapies and three drug therapies.
Finally, one researcher is developing stem cell therapies using patients' own skin cells

We have a board comprised of people with the necessary and complementary skills, 3 PhD research scientists, including one MD, PhD, one working in pharma venture capital, and one working in drug development, along with other board members with connections to wealthy individuals to help raise funds for research, and, last but not least, a group of affected, dedicated, and determined families. In 2020, we also formed our Scientific Advisory Council, which includes esteemed scientists to help guide us in our path to a cure. We have engaged six research teams who are specifically researching Usher 1F, including at Harvard, the University of Oregon Institute of Neuroscience, the University of Iowa Institute for Vision Research, the University of Maryland, the University of Pittsburgh, and the University of Western Australia. In addition, we are partnering with a team at the Center for Eye Research Australia. Finally, our two animal models have opened up paths for testing potential treatments.

We have:
Developed 2 animal models that share all 3 human Usher 1F traits
Engaged 6 research labs dedicated to Usher 1F specific research
Partnered with 2 research labs obtaining their own funding for Usher 1F specific research
Directly funded over $2 million in research and helped our partners raise another $2.6 million
Begun testing of gene and drug therapies for Usher 1F
Have signed an agreement and will soon begin a natural history study

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 shared information about our current feedback practices.
  • How is your organization collecting feedback from the people you serve?

    Community meetings/Town halls, Suggestion box/email,

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

    To inform the development of new programs/projects, To strengthen relationships with the people we serve,

  • What significant change resulted from feedback?

    Change in the frequency of emails to patients with Usher 1F with simpler language after one of our patients, who is part of the deaf culture, let us know that she and others could not understand the language used in our emails. Addition of online patient meetings to share progress.

  • With whom is the organization sharing feedback?

    Our staff, Our board,

  • What challenges does the organization face when collecting feedback?

    It is difficult to get the people we serve to respond to requests for feedback,

Financials

USHER 1F COLLABORATIVE INC
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Operations

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

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Connect with nonprofit leaders

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lock

Connect with nonprofit leaders

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  • 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.

USHER 1F COLLABORATIVE INC

Board of directors
as of 2/4/2021
SOURCE: Self-reported by organization
Board co-chair

Melissa Chaikof

Usher 1f Collaborative

Term: 2016 - 2018


Board co-chair

Dr. Elliot Chaikof

Beth Israel Deaconess Medical Center

Term: 2016 - 2018

Frank Gentile

Third Rock Ventures

Lijun Sun

Beth Israel Deaconess Medical Center

Rachel Root

Laurie Shapiro

Optical Expressions

Jared Root

Ideal Fastener

Matthew Shulman

Ardent Companies

Joshua Herz

Associated Agencies, Inc.

Julian Seewald

Julian Seewald, DDS

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 ? 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

SOURCE: Self-reported; last updated 02/04/2021

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
Female, Not transgender (cisgender)
Sexual orientation
Heterosexual or Straight
Disability status
Person without a disability

The organization's co-leader identifies as:

Race & ethnicity
White/Caucasian/European
Gender identity
Male, Not transgender (cisgender)
Sexual orientation
Heterosexual or Straight
Disability status
Person without a disability

Race & ethnicity

Gender identity

 

Sexual orientation

No data

Disability

Equity strategies

Last updated: 02/03/2021

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 disaggregate data to adjust programming goals to keep pace with changing needs of the communities we support.
  • 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.