Early Translational IV

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RFA 12-07: CIRM Early Translational IV Research Awards

The CIRM Early Translational Research Initiative aims to fund and advance potentially transformative stem cell therapies towards IND-enabling preclinical and clinical development. The Early Translational IV Research Awards RFA will support projects that perform the initial stage of translational research including the conduct of studies resulting in proof of concept for a potential development candidate (a Development Candidate Feasibility Award or DCF) and/or studies to select a Development Candidate (DC). The Early Translational Research Award is core to CIRM’s mission, and the agency plans to issue this RFA every one to two years to build a strong preclinical pipeline for patient therapies and cures.

This RFA includes participation by members of CIRM’s Collaborative Funding Partner (CFP) Program. Appendices D-H describe additional requirements and procedures for applicants whose proposals include a request for funding from a CFP. 

Please see the RFA for full details:
RFA 12-07: CIRM Early Translational IV Research Awards 
(Amended 9.21.12 – NHMRC is not participating in this RFA)

Application Process

Submission of an application for the CIRM Early Translational IV RFA involves a two-step process. An eligible PI may submit one Preliminary Application (PreApp). PreApps will be evaluated by scientific specialists from outside California who are experts in specific areas of research described in the PreApp and by CIRM scientific staff, based on the scientific review criteria described in section X of this RFA. Applicants whose projects are judged as most promising, competitive, and responsive to the RFA will be invited to submit a full application. All other applicants will be deferred, with the opportunity to apply in response to a future RFA.

Preliminary Application (PreApp) Instructions

PreApps must be submitted online via the CIRM Grants Management Portal at https://grants.cirm.ca.gov/login/upgrade_browser.

Each PI may submit only a single PreApp for this RFA.
The deadline for submission is 5:00 pm (PDT) on October 26, 2012. No exceptions to this deadline will be made.

To submit a Preliminary Application:
1. Go to the Grants Management Portal (https://grants.cirm.ca.gov/login/upgrade_browser) and log in with your existing CIRM Username and Password. If you do not have a Username, Click on the “New User” link and follow the instructions to create a CIRM Username and password.

2. After logging in, look for the section labeled “RFAs and Programs Open for Applications”. Click on the “Start a Preliminary Application” link corresponding to the “Early Translational IV Research Awards RFA: Preliminary Application” section.

3. Using the drop down menu, identify the Institution from which you intend to apply and click “Save”.

4.Complete each section of the preapplication by clicking on the appropriate link and following the posted instructions. The signature page template (an html print screen) can be found and submitted in the “Upload Required Documents” section.

5. To submit your preapplication, click on the “Done with Preliminary Application” button. The “Done with Preliminary Application” button will be enabled when all required sections have been completed and the signature page uploaded. Please note that once this has been selected, you will no longer be able to make changes to your PreAppllication.

6. To confirm submission of your Preliminary Application, go back to your home page and check the list under the section labled “Your Submitted Applications”. You will see your Preliminary Application number and project title entered for RFA 12-07 Early Translational IV once the submission process has been completed.

Full Application Process

Full Applications will only be accepted from applicants who 1) submitted a PreApp and 2) are invited by CIRM to submit a full application.

The application for the CIRM Early Translational IV RFA consists of five parts:

Part A: Application Information Form (Web-based form)
Part B: Proposal (MS Word template)
Part C: Biographical Sketches and Letters of Support (MS Word template)
Part D: Licenses and Agreements (e.g. MTAs)
Part E: Budget Detail (MS Excel Template)

All five parts of the full Application for Early Translational IV Research Awards must be submitted together and received by CIRM no later than 5:00 pm (PDT) on March 20, 2013, via the CIRM Grants Management Portal.  It is the applicant’s responsibility to meet this deadline; no exceptions will be made.

Schedule of CIRM deadlines and reviews Date
Preliminary Applications due 5:00 pm (PDT), October 26, 2012
Invitations for full Applications sent out by CIRM Late January, 2013
Full Applications due 5:00 pm (PDT), March 20, 2013
Review of full Applications by Grants Working Group (GWG)  June, 2013
Review and Approval by ICOC Summer, 2013
Earliest Funding of Awards Fall, 2013

No exceptions will be made to the indicated deadlines.


For information about this RFA or the review process:

Gilberto R. Sambrano, Ph.D.
Senior Review Officer
California Institute for Regenerative Medicine
Email: gsambrano@cirm.ca.gov
Phone: (415) 396-910

ICOC Approval:
August 28, 2013
Total Awards:
Award Value:


Institution Investigator Grant Title Award Value
University of California, Los Angeles Dr. Robert Reiter MD Clinical Development of an N-cadherin Antibody to Target Cancer Stem Cells $4,075,668
Human BioMolecular Research Institute Dr. John R Cashman Improving Existing Drugs for Long QT Syndrome type 3 (LQT3) by hiPSC Disease-in-Dish Model $6,361,369
Numerate, Inc. Dr. John H Griffin Use of human iPSC-derived neurons from Huntington’s Disease patients to develop novel, disease-modifying small molecule structural corrector drug candidates targeting the unique, neurotoxic conformation of mutant huntingtin $520,015
University of California, Los Angeles Dr. Jerome A. Zack Ph.D. Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection $4,925,166
University of California, Los Angeles Dr. Gerald Lipshutz Gene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders $1,801,629
University of California, Los Angeles Dr. Donald B. Kohn Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,651,884
Salk Institute for Biological Studies Dr. Inder M. Verma Development of a cell and gene based therapy for hemophilia $2,298,634
The Scintillon Institute Dr. Stuart A Lipton Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke $1,103,185
Sanford Burnham Prebys Medical Discovery Institute Dr. Stuart A Lipton Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke $1,020,815
University of California, San Diego Professor Alysson R Muotri A drug-screening platform for autism spectrum disorders using human astrocytes $1,656,456
Cedars-Sinai Medical Center Dr. Dan Gazit Gene Targeting to Endogenous Stem Cells for Segmental Bone Fracture Healing $5,121,514
Stanford University Dr. Michele Calos Engineered iPSC for therapy of limb girdle muscular dystrophy type 2B $1,876,253
Gladstone Institutes, J. David Dr. Steve M. Finkbeiner Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases $2,049,053
University of California, Irvine Dr. Magdalene J Seiler Restoring vision by sheet transplants of retinal progenitors and retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs) $3,998,948