Opportunity Information: Apply for PA 11 186

  • The National Institutes of Health in the health sector is offering a public funding opportunity titled "Translation of Pluripotent Stem Cell Therapies for Blood Diseases (R01)" and is now available to receive applicants.
  • Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.839 Blood Diseases and Resources Research.
  • This funding opportunity was created on Jun 10, 2011 and posted on Mar 28, 2011.
  • Applicants must submit their applications by Jan 7, 2014. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
  • Eligible applicants include: Special district governments State governments Private institutions of higher education For profit organizations other than small businesses Native American tribal organizations (other than Federally recognized tribal governments) Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Small businesses Native American tribal governments (Federally recognized) City or township governments Public housing authorities/Indian housing authorities Others (see text field entitled Additional Information on Eligibility for clarification) County governments Independent school districts Public and State controlled institutions of higher education.
  • Other Eligible Applicants include the following Alaska Native and Native Hawaiian Serving Institutions Eligible Agencies of the Federal Government Faith based or Community based Organizations Hispanic serving Institutions Historically Black Colleges and Universities (HBCUs) Indian/Native American Tribal Governments (Other than Federally Recognized) Non domestic (non U.S.) Entities (Foreign Organizations) Regional Organizations Tribally Controlled Colleges and Universities (TCCUs) U.S. Territory or Possession Foreign (non U.S.) components of U.S. Organizations are not allowed.
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Opportunity Summary:

Translation of Pluripotent Stem Cell Therapies for Blood Diseases (R01) (Funding Opportunity Number: PA 11-186) is a National Institutes of Health grant opportunity led by the National Heart, Lung, and Blood Institute (NHLBI) that aims to push pluripotent stem cell science closer to real, testable cell therapies for sickle cell disease and other serious blood disorders. The opportunity supports Research Project Grant (R01) applications that bring together collaborative, multidisciplinary teams under a multi-Project Director/Principal Investigator (multi-PD/PI) structure, with the expectation that each application will name two or more PDs/PIs. The overall intent is translation-focused technology development: not just basic discovery, but the creation and refinement of methods that can realistically feed into clinical evaluation.

The FOA is built around two major technical bottlenecks that, at the time of the announcement, were limiting progress toward stem-cell-derived blood cell therapies. The first priority area is developing robust techniques to efficiently generate hematopoietic cells (blood-forming cells) in quantities and quality appropriate for clinical testing. Applicants are encouraged to address this either by differentiating human pluripotent stem cells (PSC) into hematopoietic lineages or by using cellular reprogramming approaches that convert one cell type into another with blood-forming potential. A key translational emphasis is producing sufficient numbers of cells that meet Good Manufacturing Practice (GMP) quality expectations, meaning the work should be oriented toward scalable, reproducible, and clinically compatible cell production rather than small, purely experimental batches.

The second priority area is solving the problem of efficient engraftment. Even if hematopoietic cells can be produced from PSCs or reprogramming methods, they still need to successfully engraft in a recipient and function long-term in a way that is relevant for treating disease. This FOA therefore invites projects that develop protocols, conditioning strategies, or other enabling methods that improve the ability of PSC-derived or reprogrammed hematopoietic cells to home to the right niches, persist, and contribute meaningfully to blood formation after transplantation. The underlying translational goal is to move closer to approaches that could ultimately replace or complement current transplantation strategies for disorders like sickle cell disease, where durable production of healthy blood cells is the therapeutic target.

A defining feature of this program is its explicit integration with the NHLBI Progenitor Cell Biology Consortium (PCBC). The funded projects are expected to operate in close collaboration with existing PCBC hubs, and the new awardees are also expected to collaborate extensively among themselves and with the consortium. In practical terms, this means applicants should anticipate a strong emphasis on teamwork, data and resource sharing, complementary expertise across laboratories, and coordinated progress toward shared technical goals. The FOA frames these awards as adding separate additional research projects that work alongside, and in concert with, the ongoing consortium efforts rather than operating as isolated, stand-alone studies.

From an administrative and eligibility standpoint, this is a discretionary grant mechanism using the NIH R01 funding instrument within the health-related funding activity category, tied to CFDA 93.839 (Blood Diseases and Resources Research). Cost sharing or matching is not required. Eligibility is broad and includes many types of U.S.-based and non-U.S.-based organizations, such as public and private institutions of higher education, nonprofits (including 501(c)(3) and certain non-501(c)(3) entities), for-profit organizations (including small businesses), state and local governments, tribal governments and tribally controlled institutions, and a range of mission-serving institutions such as HBCUs and Hispanic-serving institutions. Foreign organizations (non-U.S. entities) are listed as eligible, but foreign (non-U.S.) components of U.S. organizations are not allowed under this announcement, which is an important distinction for applicants with international collaborations or overseas branches.

Key timeline details in the source information show it was posted on March 28, 2011, with an original closing date of October 5, 2013, a current closing date of January 7, 2014, and an archive date of February 7, 2014. The full announcement and official details were provided through the NIH Grants Guide, with an additional information link at http://grants.nih.gov/grants/guide/pa-files/PA-11-186.html, and support contacts routed through the NIH Office of Extramural Research (OER) webmaster for access or linking issues.

Overall, the opportunity is best understood as a targeted NHLBI effort to overcome practical barriers to stem-cell-based blood therapies by funding team science that can (1) reliably produce clinically relevant, GMP-grade hematopoietic cells from PSC differentiation or reprogramming pipelines, and (2) develop engraftment-enabling protocols that make those cells therapeutically viable after transplantation, all while operating in a highly collaborative framework linked to the Progenitor Cell Biology Consortium.

FAQs: Translation of Pluripotent Stem Cell Therapies for Blood Diseases (R01) (PA-11-186)

What is this funding opportunity?

Translation of Pluripotent Stem Cell Therapies for Blood Diseases (R01) is a National Institutes of Health (NIH) grant opportunity led by the National Heart, Lung, and Blood Institute (NHLBI). It supports Research Project Grant (R01) applications aimed at moving pluripotent stem cell science closer to practical, testable cell therapy approaches for sickle cell disease and other serious blood disorders.

What is the Funding Opportunity Number (FOA number)?

The Funding Opportunity Number listed for this opportunity is PA 11-186.

Which NIH Institute is leading this FOA?

The opportunity is led by NHLBI (the National Heart, Lung, and Blood Institute), which is part of NIH.

What kind of grant mechanism is used?

This opportunity uses the NIH Research Project Grant (R01) funding instrument.

What is the main goal of the program?

The main goal is translation-focused technology development. The intent is not simply basic discovery, but developing and refining methods that can realistically support clinical evaluation of stem-cell-derived or reprogrammed blood cell therapies.

What diseases or clinical areas does the FOA emphasize?

The FOA emphasizes sickle cell disease and other serious blood disorders, with a focus on therapies that aim to produce durable, healthy blood cell formation.

Does this FOA support basic research, or is it more focused on translation?

Based on the description provided, the emphasis is strongly translational: projects are expected to tackle practical barriers and produce methods that could feed into clinical evaluation, rather than remaining at the level of small-scale, purely experimental work.

What are the two major technical bottlenecks this FOA is trying to address?

The FOA is built around two main bottlenecks: (1) robust, efficient generation of hematopoietic (blood-forming) cells in quantities and quality appropriate for clinical testing, and (2) improving efficient engraftment so the cells can successfully home, persist, and function long-term after transplantation.

What types of approaches are encouraged for generating hematopoietic cells?

Applicants are encouraged to address hematopoietic cell production either by differentiating human pluripotent stem cells (PSCs) into hematopoietic lineages or by using cellular reprogramming approaches that convert one cell type into another with blood-forming potential.

What does "GMP" mean in the context of this FOA?

The FOA highlights the need for cell production that meets Good Manufacturing Practice (GMP) quality expectations. In this context, that means applicants should orient their work toward scalable, reproducible, clinically compatible production methods rather than small, one-off experimental batches.

Is scalability and reproducibility explicitly important for proposed methods?

Yes. The FOA explicitly emphasizes producing sufficient numbers of cells and doing so in a way that is scalable, reproducible, and compatible with clinical expectations (including GMP-oriented considerations).

What does the FOA mean by "engraftment" challenges?

Engraftment refers to the ability of transplanted hematopoietic cells to successfully home to the correct biological niches in a recipient, persist over time, and contribute meaningfully to blood formation. The FOA calls for methods that improve these outcomes for PSC-derived or reprogrammed hematopoietic cells.

What kinds of engraftment-related solutions does the FOA invite?

The FOA invites projects that develop protocols, conditioning strategies, or other enabling methods that improve homing, persistence, and long-term function of PSC-derived or reprogrammed hematopoietic cells after transplantation.

How is collaboration structured for this program?

A defining feature is integration with the NHLBI Progenitor Cell Biology Consortium (PCBC). Funded projects are expected to operate in close collaboration with existing PCBC hubs, collaborate among themselves, and coordinate progress toward shared technical goals.

What is the NHLBI Progenitor Cell Biology Consortium (PCBC) role here?

The FOA frames new awards as additional research projects that work alongside, and in concert with, ongoing PCBC efforts. Applicants should expect strong emphasis on teamwork, sharing of data and resources, complementary expertise across laboratories, and coordinated progress.

Are applications expected to be stand-alone projects?

No. The FOA describes these awards as operating in a highly collaborative framework aligned with the PCBC, rather than as isolated, stand-alone studies.

Does the FOA require a multi-PD/PI structure?

Yes. The FOA expects applications to use a multi-Project Director/Principal Investigator (multi-PD/PI) structure, with the expectation that each application will name two or more PDs/PIs.

How many PDs/PIs should be named on an application?

The FOA expectation stated in the provided information is that each application will name two or more PDs/PIs.

Is cost sharing or matching required?

No. The provided information states that cost sharing or matching is not required.

What is the CFDA number associated with this opportunity?

The opportunity is tied to CFDA 93.839, identified as Blood Diseases and Resources Research.

Who is eligible to apply?

Eligibility is described as broad. It includes many types of U.S.-based and non-U.S.-based organizations, including public and private institutions of higher education, nonprofits (including 501(c)(3) and certain non-501(c)(3) entities), for-profit organizations (including small businesses), state and local governments, tribal governments and tribally controlled institutions, and mission-serving institutions such as HBCUs and Hispanic-serving institutions.

Are foreign (non-U.S.) organizations eligible?

Yes. Foreign organizations (non-U.S. entities) are listed as eligible in the provided information.

Are foreign (non-U.S.) components of U.S. organizations allowed?

No. The provided information states that foreign (non-U.S.) components of U.S. organizations are not allowed under this announcement. This is distinct from the eligibility of foreign organizations themselves.

Where can applicants find the official announcement?

The official details were provided through the NIH Grants Guide. The additional information link given is: http://grants.nih.gov/grants/guide/pa-files/PA-11-186.html

What are the key dates listed for this FOA?

The source information lists: posted on March 28, 2011; original closing date of October 5, 2013; current closing date of January 7, 2014; and archive date of February 7, 2014.

Is this opportunity still open?

Based on the dates provided, the FOA has a closing date of January 7, 2014 and an archive date of February 7, 2014, indicating it is not a current open opportunity according to the listed timeline.

Who should be contacted for access or linking issues?

The provided information indicates support contacts are routed through the NIH Office of Extramural Research (OER) webmaster for access or linking issues.

What does "translation-focused technology development" mean here?

In this FOA, it means developing practical methods and enabling technologies that can realistically advance toward clinical evaluation, especially around producing clinically relevant hematopoietic cells (including GMP-oriented production) and improving engraftment after transplantation.

What is the overall theme of the funded work?

The overall theme is overcoming practical barriers to stem-cell-based blood therapies through team science that (1) reliably produces clinically relevant hematopoietic cells from PSC differentiation or reprogramming pipelines and (2) develops engraftment-enabling protocols, all within a consortium-linked collaborative framework.

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