Patient-Partnered Collaborations for Rare Neurodegenerative Disease

Patient-Partnered Collaborations for Rare Neurodegenerative Disease is sponsored by Chan Zuckerberg Initiative. This RFA invites applications from collaborative teams bringing together patient-led rare disease organizations and research teams for 4-year research projects aimed at advancing the understanding of the fundamental science of rare diseases, particularly neurodegenerative and ne…

Patient-Partnered Collaborations for Rare Neurodegenerative Disease - Chan Zuckerberg Initiative This RFA closed on May 24, 2022 Patient-Partnered Collaborations for Rare Neurodegenerative Disease The Chan Zuckerberg Initiative invites applications from collaborative teams bringing together patient-led rare disease organizations and research teams for 4-year research projects aimed at advancing our understanding of the fundamental science of rare diseases.

The Patient-Partnered Collaborations for Rare Neurodegenerative Disease RFA aims to advance the understanding of the pathophysiology and mechanistic underpinnings of rare neurodegenerative and neurological disorders. Awards are $2,000,000 USD total costs for four years for each project (distributed amongst research institutions and a patient organization).

The award period for each RFA is an initial two years of funding, followed by an additional two years, pending evaluation. Applicants are welcome to apply to both this opportunity and the Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease RFA ; however, the application and scope of work must be distinct.

CZI is hosting a series of workshops to facilitate networking and the creation of new collaborative teams for this RFA. Learn about our grantees . For administrative and programmatic inquiries, or other questions pertaining to this RFA, please contact sciencegrants@chanzuckerberg.

com . Applications due by 5 p. m.

Pacific Time Earliest notification of decisions (subject to change) Expected start date of award period Award period and start date: The award period for each RFA is for an initial two years of funding, followed by an additional two years, pending evaluation. The expected start date is December 1, 2022.

Institutional Approval Form The CZI Neurodegeneration Challenge Network has three key goals: (1) To make fundamental advances toward understanding neurodegeneration; (2) To bring new ideas and talent to the field of neurodegeneration; and (3) To encourage and experiment with a new interdisciplinary, collaborative, and open science research model involving scientists, clinicians, and engineers.

CZI Neurodegeneration Challenge Network The Neurodegeneration Challenge Network model is built on the vision that progress in solving neurodegenerative diseases will come from bringing new people into the neurodegeneration field from diverse disciplines and expertise; building interdisciplinary collaborations; empowering the broader scientific community with robust tools and platforms, and creating a culture of open science.

Scientifically, we aspire to motivate the collective field to shift the approach to neurodegenerative diseases to a framework, where these diseases—currently addressed largely as distinct diseases and problems—are considered more holistically as a class of disorders with common features, mechanisms, and solutions.

The Patient-Partnered Collaborations for Rare Neurodegenerative Disease RFA extends on this vision by expanding the scope of diseases and biological problems covered within NDCN, and widens the circle of experts and stakeholders to include patient organizations as collaborators, building on learnings from CZI’s Rare as One Network and CZI’s efforts in patient-directed research.

In the three years since launching the Neurodegeneration Challenge Network (NDCN) in 2018, the Network has grown to a thriving collaborative network that includes 56 teams, 108 labs and over 500 affiliated students, postdocs, and staff scientists.

We are now pleased to invite applications to join the Neurodegeneration Challenge Network through a new Request for Applications (RFA) and grant program, the Patient-Partnered Collaborations for Rare Neurodegenerative Disease.

Building on the previous NDCN grant programs, including the NDCN Ben Barres Early Career Acceleration Award , the Collaborative Science Awards , and the Collaborative Pairs Pilot Projects , this RFA will also prioritize bringing new investigators and early-career scientists into the field.

There will be an additional emphasis on recruiting scientists from fields outside the core of neurodegeneration and rare disease, and will target work that addresses the biology of rare neurodegenerative diseases in a novel, innovative, interdisciplinary, and disease cross-cutting way.

To learn more, read about the Neurodegeneration Challenge Network , Rare As One Cycle One grantee announcement and Cycle Two announcement, and watch the NDCN video .

Patient-centered and foundational science-focused, this RFA aims to expand the understanding of the pathophysiology and mechanistic underpinnings of rare neurodegenerative disease and to channel the focus of research efforts towards issues and questions that are critically relevant to the patient experience. This RFA will not focus on therapeutic or clinical development.

However, in centering the work on foundational science and closing critical knowledge gaps, these projects will build a strong foundation for translational efforts. We also see tremendous opportunity for the study of these rare neurodegenerative conditions to inform our understanding of neurodegenerative diseases more broadly, including common diseases.

The focus of the RFA is on rare neurodegenerative diseases and neurological diseases with neurodegenerative features.

Examples of diseases that would fall within scope include, but are not limited to, lysosomal storage disorders, neuropathies, spastic paraplegias, ataxias, leukodystrophies, rare metabolic and autoimmune disorders leading to neurodegenerative disease, myopathies and muscular dystrophies, encephalophathies, and rare epilepsies in cases where there are degenerative features.

Neurodevelopmental and neuropsychiatric disorders that are not considered neurodegenerative by consensus clinical criteria (autism, for instance) are not within scope for this RFA.

A key aim of this RFA is to encourage research in rare neurodegenerative diseases where research has been limited; rare disease areas where there are currently robust, mature research efforts and active research communities and/or significant funding by federal and philanthropic initiatives will be of lower priority.

Examples of rare neurodegenerative disease areas which we consider to be mature fields of research include, but are not limited to, ALS, Huntington’s Disease, Frontotemporal Dementia, Spinal Muscular Atrophy. We are open to consideration of proposals that address rare sub-populations of patients that are understudied and of unique scientific value.

Applicants will be required to provide an overview of the clinical and biological features of the disease that is the subject of the application and a statement of the evidence in support of the disease being neurodegenerative and a case for why the disease area is understudied.

We particularly encourage project proposals that address health disparities or focus on rare diseases that predominantly affect underrepresented populations, particularly ancestries that are underrepresented in biomedical research.

Consistent with NDCN’s focus on discovery science, this RFA is open to proposals for discovery science projects that are exploring foundational understanding of causes, progression, or mechanisms of rare neurodegenerative and neurological disorders. Proposals should center on a research question and clear research strategy.

Examples of the types of projects that would meet these criteria could include, but are not limited to: Projects that aim to address critical knowledge gaps about disease causes, clinical progression or mechanisms of action Projects that bridge natural history studies and mechanistic investigations Development of new models to support better understanding of disease mechanism Preclinical mechanistic work in support of therapeutic development; for instance, perturbation studies on a candidate mechanism in a disease model, proof of concept for reversal, disease mitigation in a disease model.

The emphasis of this RFA will be on mechanistic work that provides insight into disease biology rather than therapeutic drug screens.

Collaborative cross-disease research (for instance, exploring common mechanisms that affect more than one disease) Data-centered approaches that generate and/or analyze biological and clinical data sets, for the purpose of developing biomarkers, insights into disease mechanisms Longitudinal history studies that track disease development and provide insight into critical periods of disease progression and potential critical therapeutic windows Projects that provide critical insights into comorbidities, for instance, neuroimmune or metabolic influences on neurodegeneration.

Areas that are out of scope of this RFA: Clinical trials or pre-clinical research aimed primarily at clinical trial readiness (e.g., toxicology studies) Tool or resource development that is not tied to a specific research plan or agenda. This could include the development of research technologies, models (cell lines, animal models) or other types of infrastructure development (e.g., registries).

We view the development of hiqh-quality, robust research tools as critical for accelerating science; however, for this RFA, such tool and resource development efforts should be clearly connected to specific research questions. Collaboration is a central feature of this RFA and grant program.

Applications will each have two lead Principal Investigators (PIs): one representing the research team and the other representing the patient organization. Teams should be made up of a minimum of two investigators with a maximum of five investigators, including the patient organization leader. Applications should be co-written by the researchers and the patient-organization representative.

Applications will be accepted from newly formed collaborative teams who have not worked together previously or established researcher-patient organization collaborative teams who are proposing new research directions. We will prioritize teams that are taking a fresh, innovative approach to tackling these challenging diseases.

The criteria below for team composition are intended to allow for flexibility in team composition, while ensuring that there is strong co-ownership of the collaboration between the research and patient-advocate partners. If applicants have a question about their team’s composition and eligibility, please contact sciencegrants@chanzuckerberg. com .

To allow tight collaboration and coordination of research efforts, this RFA is focused on small group collaborations. Teams should be made up of a minimum of two Principal Investigators (PIs) to a maximum of five PIs. All PIs are expected to actively contribute to the project and engage in program and network level activities, such as investigator meetings and workshops where relevant.

Below we describe the roles of the PIs. Coordinating research PI / lead-research PI: Teams should designate a coordinating PI, the scientific lead of the research project, who coordinates the research team. The lead-research PI will also be the Coordinating PI of the grant to submit the application on behalf of the collaborating team.

The Coordinating PI will act as the administrative contact between CZI and all other co-PIs on the grant. The Coordinating PI must submit the application on behalf of all PIs (including the Lead Patient Organization PI, see below) and ensure that the collaborative team has the necessary skills to deliver on the aims of the project.

The Coordinating PI must be affiliated with the academic/research institution submitting the application, and grant funds will be awarded to that institution, which will take responsibility for distributing funds to all other research institutions (patient funds will be awarded directly to the patient organization).

Note that institutions outside the U.S. may not subcontract to U.S. institutions, so please be mindful when selecting the Coordinating PI/institution. Lead Patient Organization PI: Will represent the patient organization. The research team should coordinate with one patient-led rare disease organization.

If the project requires collaboration with more than one patient-led rare disease organization, please reach out to sciencegrants@chanzuckerberg. com to discuss. Up to three additional co-PIs: Teams may designate up to three additional research co-PIs from one or more research institutions.

Co-PIs may be from the same or different research institutions as the Coordinating PI. The strongest applications will incorporate interdisciplinary expertise and perspectives. Teams may also include additional collaborators and contractors but only 3 individuals can be listed as research co-PIs.

Each team must include at least one clinician with expertise relevant to the application and experience working with patients in this disease area. It is allowable for the clinician to either be located at a research institution or be designated as the Lead Patient Organization PI. Diversity, Equity, & Inclusion We believe that the strongest teams incorporate a wide range of voices.

Those underrepresented in science and technology are strongly encouraged to apply. This includes but is not limited to women, those with disabilities, underrepresented racial and ethnic groups, LGBTQ individuals, and organizations representing disease areas that disproportionately impact underrepresented or underserved communities.

Researchers and patient organizations from the Global South and low-to-middle income countries, in particular those with populations that have historically been underrepresented in biomedical research, are strongly encouraged to apply and to be included as members of international collaborative networks.

International collaborations between investigators and rare disease organizations in the Global North and South that leverage regional and technological expertise and strengths are encouraged. It is the expectation that international collaborations will follow guidelines for conducting research in an equitable and mutually beneficial manner.

Collaboration and Open Science All projects will be evaluated based on their potential for scientific output (productivity), tool and resource dissemination (reach), inclusion of representative donors and communities, and collaboration among the team. We are looking for investigators and groups who will enthusiastically contribute to and benefit from a collaborative, dynamic, and interdisciplinary approach.

For examples of evidence of productivity, reach, and collaboration, please see the CZI statement of values . In addition to providing funding, CZI functions as a scientific partner to grantees to help build open and accessible tools and datasets that serve as references for healthy and disease states.

Investigators will have the opportunity to learn from, collaborate with, and interact with the community of investigators and groups across all Networks, as well as with CZI computational biologists and software engineers. CZI supports collaboration. All PIs listed on the application are expected to participate in project meetings, reports, and other events.

Networks should be genuine collaborative projects with responsibility and participation distributed across participating research groups and organizations. Members of funded labs with key roles in the project—such as students, postdocs, and staff—will also participate in scientific meetings, hackathons, and other activities, and relevant members of patient organizations will also be included in such events.

The Rare As One team will also expand access to some of its relevant training and support program offerings to both the researchers and organizations that are listed as co-PIs on this grant, and will develop targeted trainings to support this specific collaborative opportunity. CZI’s mission is at the interface of technology and science.

Working in collaboration with, and guided by, other grantees in the Network and the wider Human Cell Atlas (HCA) community, we aim to develop technology-based tools and approaches to support and accelerate the scope and impact of tissue atlases and the HCA community. CZI supports open science values and principles.

To accelerate scientific discovery and collaboration, CZI supports a consent, sharing, and publication policy for open and rapid dissemination of research results and a policy for software development that maximizes accessibility, reuse, and shared development. CZI believes that the people most affected by problems should be centered in developing solutions to those problems.

We work with patient communities to ensure they are heard, and recognize patients and patient communities and organizations as key partners in research. Applications should be submitted by the Coordinating PI at a research institution as described below.

Patient organizations and the Lead Patient Organization PI must be included in project conception, execution, and analysis as detailed by the application submitted by the Coordinating PI.

If selected for funding, research funds will be disbursed separately to 1) the research institution of the Coordinating PI, who will be responsible for distributing funds as appropriate to support the work of co-PIs in the same or other research institutions, and 2) to the patient organization. Applicants may submit only ONE application to this RFA.

Applicants are welcome to apply to both this opportunity and the opportunity titled “ Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease RFA” ; however, the application and scope of work must be distinct.

Researchers/Research Institutions Applications may be submitted by domestic and foreign nonprofit organizations, public and private institutions, such as colleges, universities, hospitals, laboratories, units of state and local government, and eligible agencies of the federal government. For-profit organizations are not eligible to receive funding but may be involved in projects as a collaborator.

All grants will be awarded to institutions, not individuals. Research institutions may be based in any country. More than one application will be accepted from each research institution.

Each Coordinating PI and patient organization may only submit one application to this RFA. Each application should designate one Principal Investigator (PI) as the Coordinating Principal Investigator (Coordinating PI). The Coordinating PI will act as the administrative contact between CZI and all other PIs on the grant (co-PIs).

The Coordinating PI must submit the application on behalf of all PIs. The Coordinating PI must be affiliated with the institution submitting the application, and grant funds will be awarded to that institution, which will take responsibility for distributing funds to all other research institutions.

Note that institutions outside the U.S. may not subcontract to U.S. institutions, so please be mindful when selecting the Coordinating PI/institution. Principal Investigators may only serve as funded Principal Investigators on one application. They can be involved in multiple applications as unfunded collaborators, but should not be named as Coordinating PIs or co-PIs on multiple applications.

PIs/co-PIs on one application may be employed at the same or at different research institutions. PIs/co-PIs must have an academic appointment and be in an independent faculty position or equivalent at an accredited college, university, medical school, or other research facility at the time of grant start. Independence is demonstrated by institutional support for independent research activities.

Collaborators from companies are permitted as long as no funds are requested to support them or their work and there is full compliance with CZI policies regarding open science. Early-career investigators are strongly encouraged to apply as Coordinating PIs as well as co-PIs. Meta employees, including employees of any subsidiary Meta entities, as well as employees of Chan Zuckerberg Initiative, LLC, are not permitted to apply.

CZI reserves the sole right to decide if an applicant and applicant institution meet the eligibility requirements. CZI reserves the right to request budget changes prior to award.

Prior to award, all grant applications will be reviewed for compliance with the United States Treasury Department’s Office of Foreign Asset Control (OFAC) sanctions program, the United States Department of Commerce’s export administration regulations, the Foreign Corrupt Practices Act (FCPA), any other applicable U.S. laws and regulations, and any corresponding laws and regulations in the country where the applicant is based.

All grant agreements will also require the grantee to comply with these laws and regulations. For additional information please refer to: the U.S. Treasury Department’s resources , the International Trade Administration’s website on US Export Controls , and the Department of Justice’s website on the FCPA .

Patient organizations must be patient-led rare disease organizations (advocacy groups, disease foundations or organizations that represent patients, engage patients in key leadership roles—e.g., as founder, executive director, board member, key staff, etc.—and are patient-centered in their programming).

Patient organizations must be organizations that are tax-exempt under section 501(c)(3) of the United States Internal Revenue Code, OR have a valid fiscal sponsor that is tax-exempt under section 501(c)(3) of the Internal Revenue Code, OR a 501(c)(3) charity/non-profit organization equivalent for non-US based organizations. Non-US based organizations will be subject to Equivalency Determination.

Patient organizations must be focused on a rare disease, disorder, or syndrome, or group of closely related rare diseases, disorders, or syndromes (defined, for instance, as a condition that affects fewer than 200,000 people in the U. S . , or no more than 1 in 2,000 people in the European Union).

This RFA is intended to support early stage research on rare diseases with sparse scientific research to date. It is not intended to further already mature areas of research, such as those supported by large organizations with extensive research programs or areas funded by governmental funding (i.e., NIH or the equivalent). We will prioritize support for patient organizations with annual research budgets less than $5 million.

Each patient organization may only be represented in one application to this RFA. Lead Patient Organization PIs may only be named on one application. Meta employees, including employees of any subsidiary Meta entities, as well as employees of Chan Zuckerberg Initiative, LLC, are not permitted to apply.

CZI reserves the sole right to decide if an applicant and applicant organization meet the eligibility requirements. CZI reserves the right to request budget changes prior to award.

Prior to award, all grant applications will be reviewed for compliance with the United States Treasury Department’s Office of Foreign Asset Control (OFAC) sanctions program, the United States Department of Commerce’s export administration regulations, the Foreign Corrupt Practices Act (FCPA), any other applicable U.S. laws and regulations, and any corresponding laws and regulations in the country where the applicant is based.

All grant agreements will also require the grantee to comply with these laws and regulations. For additional information please refer to: the U.S. Treasury Department’s resources , the International Trade Administration’s website on US Export Controls , and the Department of Justice’s website on the FCPA .

CZI suggests that you consult your home institution to determine eligibility to apply for this grant and your institutional policy on indirect costs. For questions about eligibility for this award or the application process, please contact us in advance of the proposal deadline at sciencegrants@chanzuckerberg. com .

Deadline extensions will not be granted. All applications must be completed and submitted through CZI’s online grants management portal at https://apply. chanzuckerberg.

com . It is recommended that applicants familiarize themselves with this portal well in advance of the application deadline. Detailed application instructions are available below in the Detailed Application Instructions section, as well as in the grants management portal .

Applications due by 5 p. m. Pacific Time Earliest notification of decisions (subject to change) Expected start date of award period Award period and start date: The award period for each RFA is for an initial two years of funding, followed by an additional two years, pending evaluation.

The expected start date is December 1, 2022. Budget: Total project budgets should be $500,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $2,000,000 total costs for 48 months. We anticipate funding research institutions for $400,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $1,600,000 USD over a 48 month period.

Indirect costs cannot exceed 15 percent of direct costs. Budgets should include allocations for each patient organization for $100,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $400,000 total costs over a 48 month period. This will be awarded separately and directly to the patient organization and is in addition to the $400,000 to the research institution per year.

Detailed Application Instructions The Chan Zuckerberg Initiative uses SurveyMonkey Apply (SMApply) as its grants management portal. All applications must be submitted through this portal ( https://apply. chanzuckerberg.

com ). SMApply is configured to work best using the Google Chrome browser. It is recommended that you familiarize yourself with this portal well in advance of any deadlines.

Deadline extensions will not be granted. Eligibility: Please refer to the Eligibility section. Award Period: The award period for each RFA is for an initial two years of funding, followed by an additional two years, pending evaluation.

The expected start date is December 1, 2022. Number of Principal Investigators (PIs): Multi-disciplinary project teams composed of members with the necessary skill sets and engagement with patient communities are required to maximize the success of these projects.

Each project must have a designated Coordinating Principal Investigator that will serve as an administrative point of contact for all research activities and help coordinate progress within the group as well as a Lead Patient Organization PI. All other participants will be co-PIs and are expected to contribute to the project. A clinician must be included in each collaborative project.

It is allowable for the clinician to either be located at a research institution or be designated as the Lead Patient Organization PI. Teams should have a minimum of two PIs and a maximum of five PIs to fulfill the eligibility criteria. Total project budgets should be $500,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $2,000,000 USD total costs for 48 months.

We anticipate funding research teams for $400,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $1,600,000 USD over a 48 month period. Indirect costs cannot exceed 15 percent of direct costs.

Budgets should include allocations for the patient organization for $100,000 USD total costs (inclusive of up to 15 percent indirect costs) per year for a total of $400,000 USD total costs per institution over a 48 month period. This will be awarded separately and directly to the patient organization and is in addition to the $400,000 USD to the research institution per year.

Institutional sign-off by the research institution is required at the time of submission.

The application consists of the following sections (called tasks in the grants portal): Coordinating PI Details, Research Institution Details for Coordinating PI, Lead Patient Organization PI Details, Patient Organization Details, Equal Opportunity & Diversity, Project Details, Project Proposal, Budget Description, and Biosketches for Coordinating PI/Lead Patient Organization PI/Co-PIs.

Coordinating PI Details: Complete all fields in this task; all fields are required. The information entered should be for the Coordinating Principal Investigator (Coordinating PI), who will be the person submitting the application on behalf of the team. The Coordinating PI will take responsibility for managing the group collaboration and be the administrative point of contact for CZI and any partners.

The Coordinating PI must be affiliated with the academic/research institution submitting the application. Note that institutions outside the U.S. may not subcontract to U.S. institutions, so please be mindful when selecting the Coordinating PI/institution. Information about the Co-Principal Investigator(s) on the proposal should be entered where requested in the Project Details part of the application.

Name and email (auto-filled): To edit your name or email, please do so in your account information by clicking your name in the upper right corner and clicking My Account in the dropdown menu. Institution, Title/Position, Department or equivalent. Career status: Select early-career (0 to 6 years), mid-career (6+ to 10 years), or neither.

Note: Early- or mid- career status is not required to be eligible for this RFA, although we encourage participation and leadership from early-career researchers. Early-Career Definition: In the context of this RFA, an early-career investigator is someone who has been in an independent position for zero to six years at the time of application, i.e. have started their first independent position between May 24, 2016 and May 24, 2022.

Mid-Career Definition: In the context of this RFA, a mid-career investigator is someone who has been in an independent position for more than six to 10 years at the time of application, i.e. have started their first independent position between May 24, 2012 and May 23, 2016. Short narrative biography of the Coordinating PI (maximum of 100 words). ORCID iD: Enter in format XXXX-XXXX-XXXX-XXXX.

ORCID iDs are unique, digital identifiers that distinguish individual scientists and unambiguously connect their contributions to science over time and across changes of name, location, and institutional affiliation. ORCID iDs will be used to streamline reporting in our applications and grant reports to reduce the burden on grantees. For more information, please visit https://orcid.

org/register (please contact us at sciencegrants@chanzuckerberg. com if you wish to opt out). Research Institution Details for Coordinating PI: Complete all fields in this task; all fields are required.

The information entered should be for the research institution of the Coordinating Principal Investigator (Coordinating PI), who will be the person submitting the application on behalf of the team. The Coordinating PI must be affiliated with the institution listed, and research grant funds will be awarded to this institution, which will take responsibility for distributing funds to the institutions of the other research team members.

Patient organization funds will be awarded separately. Institution name/Street address/City/State or Province/Country/Website. Type of Institution (Academic, Other Non-profit, Government, Other).

Tax ID: Enter your institution’s Employer Identification Number (EIN), as assigned by the Internal Revenue Service in the 9-digit format (XX-XXXXXXX; 10 characters total). Foreign institutions or others who do not have an EIN should enter 44-4444444. Institutional / Administrative Contact: List the name and contact information for the administrative contact to discuss additional information needed, if selected for award.

First name, Last name, Title/Position, Email. Signing Official: List the name and contact information for the person authorized to sign on behalf of your institution. First name, Last name, Title/Position, Email.

First name, Last name, Title/Position, Email. Institutional Approval Form : Upload as a single PDF.

This form should be reviewed and signed by a person authorized to sign on behalf of your institution agreeing to the stated institutional and investigator requirements and commitments on data, resource sharing, and publication policies, as well as endorsing/verifying your application materials and confirming their ability to receive funding for the proposal.

In the event of an award, all funds will be awarded to the Coordinating PI institution as the prime institution, and the Coordinating PI institution will be responsible for ensuring compliance of all of the terms, including compliance of all partners/subcontract institutions. These policies are non-negotiable so this form should only be signed if the organization is able to comply with the terms as stated.

While CZI does not require sign-off by all of your partner institutions, please refer to what your institution requires. Note: digital signatures are permitted as long as the document is not encrypted or password-protected.

Lead Patient Organization PI Details: The information entered should be for the Lead Patient Organization PI (Executive Director, Chief Scientific Officer, Research Coordinator, etc) who will be taking responsibility for the grant from the patient organization (as the Lead Patient Organization PI) and will be the administrative point of contact for the patient organization for CZI. Title/Position at organization.

Short narrative biography of the Lead Patient Organization PI (maximum of 100 words). Patient Organization Details: Eligibility requirements: In an effort to ensure your patient organization meets the eligibility requirements to apply, this eligibility questionnaire must be completed. Is the organization a patient-led organization?

This is defined as an advocacy group/disease foundation or organization that represents patients, employs patients in key leadership roles (e.g., Founder, Executive Director, Board of Directors) and is patient-centered in its programming. Is the organization focused on a rare disease, disorder, or syndrome, or group of closely related rare diseases, disorders, or syndromes?

For the purposes of this application: as defined in the U.S. as a condition that affects fewer than 200,000 people in the U.S., or