Educational Webinar September 9 2011 Ellen G Feigal MD Vice President Research and Development California Institute for Regenerative Medicine Webinar objective is to help address your questions as you prepare your application ID: 779503
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September 9 2011
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RFA 10-05: Disease Team Therapy Development Research Awards Educational WebinarSeptember 9, 2011
Ellen G. Feigal, M.D.
Vice President, Research and Development
California Institute for Regenerative Medicine
Slide2Webinar objective is to help address your questions as you prepare your application
We will review the following:Goals and intent of the RFA
Context of this initiative within CIRM’s portfolio Eligibility criteria e.g. of therapeutic candidate, Principal investigator, and InstitutionReview Criteria by which the award will be assessedTemplates to guide your organization of the material to help ensure a complete submissions packageSeptember 9, 2011
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Slide3Webinar objective is to help address your questions as you prepare your application
We will review the following:Exceptions pathway for
for-profits, other special issuesOpportunity with collaborative funding partners from Canada (CSCC), Germany (BMBF), and Spain (Andalucia)Time frame for submission, exceptions request, supplemental information, Grants Review Group review, and anticipated ICOC assessmentSeptember 9, 2011
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Slide4Purpose is to advance preclinical and/or early clinical development of stem cell-based therapies
Purpose of this RFA is to advance preclinical and/or early clinical development of novel therapies, derived from or targeting stem cells or utilizing direct reprogramming, that may lead to new and more efficacious treatments for patients with debilitating disease or serious injury.
Goal of the DTTD is to achieve, within the 4 year time frame of the research award, one or more:Submit a well-supported IND for a clinical studyComplete a Phase I or Phase I/II studyComplete a Phase II study
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September 9, 2011
Slide5Where does this RFA fit in CIRM’s current
p
ortfolio
p
rograms
Fundamental Biology
Early Translational Research I,II
Disease Team Research I
Disease Team Research II
Targeted Clinical Development
File IND
CandidateDiscovery
Research
Phase 1 Clinical Research
Phase 2 Clinical Research
Basic Research
Research
Preclinical Research
Preclinical Dev.
Select Development
Candidate (DC)
Preclinical Proof of Concept (POC)
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Translational
Portfolio, Current
September 9, 2011
Slide6CIRM’s translational portfolio is growing
44 grants (Early Translation I, II; Disease Teams I and Targeted Clinical Development)
1 Early stage clinical trial14 (Disease Team I) target an Investigational New Drug (IND) filing
20 (Early Translational I, II) target identification and selection of a Development Candidate (DC)
9 (Early Translational II) conduct a subset of the studies to identify a development candidate or a feasible development candidate
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September 9, 2011
Slide7Provide compelling evidence for your approach
CIRM has made substantive investments across a broad number of therapeutic areas, with largest in neurological diseases, cancer, HIV/AIDS and eye diseases – see appendix A
Applicants proposing a project substantially similar to one already represented in CIRM’s translational portfolio must provide compelling evidence for their approachSeptember 9, 2011
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Slide8Scope criteria - must be cell-based, single therapeutic candidate
Cell therapy candidate derived from/utilizing
hESCs, hiPSCs, neural stem cells, neural progenitor cells, or reprogrammed/genetically-modified stem cellsSmall molecule or biologic candidate characterized or generated using stem cell types aboveCandidate that targets cancer stem cells or endogenous stem cells in vivoEngineered functional tissue candidate for transplantationOutside of Scope and Specifically Excluded: minimally manipulated bone marrow cells;
mesenchymal
stem cells; umbilical cord blood stem cells; adipose-derived stem cells; and hematopoietic stem cells
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September 9, 2011
Slide9Therapeutic candidate must meet criteria
Suitable for use in humans, completed all the research necessary to initiate IND-enabling preclinical development required for regulatory approval for testing in humans. Projects further along in the development pipeline are also eligible
Suitable for use in humans (i.e., must use human, not animal cells);Compelling, statistically significant, reproducible disease modifying activity with adequate controls in (multiple) relevant in vitro and in vivo models;Preliminary assessment of potency, dose, formulation, stability and safety (includes immunogenicity, if applicable) completed;Evidence for potential mechanism of action;Research assays developed to characterize the candidate (e.g., for identity, purity and activity);
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September 9, 2011
Slide10Therapeutic candidate must meet criteria
Methods developed for reproducible production of a defined therapeutic candidate (including viral vector, if applicable) at yields adequate to conduct IND enabling studies;
Candidate compatible with cGMP (Current Good Manufacturing Practices) (e.g. for a cell therapeutic, derivation and maintenance adequately documented);Site, mode and method of delivery selected and/or under development.10
September 9, 2011
Slide11Eligibility criteria for PI and institutions
Only PIs and applicant institutions who have received a Planning Award are eligible to apply, with two exceptions: For-profit applicant institutions and recipients of Disease Team I Research Awards (RFA 09-01) may apply for an exemption from the Planning Award requirement, provided that they meet specific criteria
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Slide12For-profit applicants – exception pathway
For-profit entities without a funded planning award may request permission to apply. To qualify for consideration, they must meet the following criteria:
Therapeutic candidate must be in scope and evidence is provided that it meets the eligibility criteria Evidence is provided that a multidisciplinary team (which may all be employed by the for-profit applicant) containing all appropriate functional groups for the proposed project has been assembled. All necessary partners (Co-PI, Partner PI), and collaborators have been identified and are willing to participate in the project if recommended for funding.September 9, 2011
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Slide13For-profit applicants – exception pathway
Proposals seeking CIRM funding for clinical trials are highly encouraged to provide evidence that additional funds (minimum of 50% of the total funds) for the proposed project have been secured or will be secured if recommended for funding.
For-profit entities seeking permission to apply under these terms must submit:an Exception Request Form (Adobe PDF), available on CIRM’s web site (http://www.cirm.ca.gov/RFA_10-05) and a letter (not exceeding 5 pages) briefly describing the therapeutic candidate, target indication/patient population, proposed preclinical and/or clinical trial(s) and address all eligibility criteria . September 9, 2011
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Slide14Disease Team 1 applicants - exception pathway
DT1 applicants must have completed an IND filing that is ready to begin Phase I clinical trials by summer of 2012 with the therapeutic candidate that is the subject of the DT 1 award.
Evidence is provided that a multidisciplinary team containing all appropriate functional groups for the proposed project has been assembled. All necessary partners (Co-PI, Partner PI), and collaborators have been identified and are willing to participate in the project if recommended for funding.September 9, 201114
Slide15Disease Team 1 applicants - exception pathway
Proposal seeking CIRM funding for clinical trials are highly encouraged to provide evidence that additional funds (minimum of 50% of the total funds) for the proposed project have been secured or will be secured if recommended for funding.
The therapeutic candidate must meet all scope requirements and provide evidence that the therapeutic candidate proposed meets all required criteria.September 9, 201115
Slide16Disease Team 1 applicants - exception pathway
Disease Team I applicants seeking permission to apply under these terms must submit: an Exception Request Form (Adobe PDF) available on CIRM’s web site (http://www.cirm.ca.gov/RFA_10-05) and a letter (not exceeding 5 pages) briefly describing the therapeutic candidate, target indication/patient population, proposed clinical trial(s) and address all eligibility criteria.
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Slide17Reviewer criteria to assess the application
Applications will be evaluated in six key areas:
Significance and Impact Reasonable draft Target Product Profile (TPP), Clinical Competitiveness and Impact, Responsiveness to RFARationaleStrong scientific rationale supported by compelling preclinical studies for the proposed therapeutic intervention in the target disease or injuryTherapeutic Development ReadinessThe project is sufficiently mature and its status is such that there is reasonable expectation that the stated project objective(s) can be achieved within 4 years of the project start date
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Slide18Reviewer criteria to assess the application
Feasibility of the Project Plan
Plan and goals feasible and adequate to meet the objectives of this RFAPlan proposes studies that address specific metrics (success criteria) defined for attributes of the TPP appropriate for the stage of developmentProject milestones describe key activities and deliverables. The project milestones are reliable indicators of the project's progress. The criteria for Go/No Go decisions are adequately defined and provide quantifiable measures of the project's performance. Project timeline is complete, highlights key progress and Go/No Go decision milestones and is realistic. September 9, 2011
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Slide19Reviewer criteria to assess the application
Principal Investigator and Development Team
Possess relevant experience in regulated translational research and therapy development, with appropriately assembled team with key expertise, appropriate structure, function and plan for execution of project, and budget with appropriate rationaleCollaborations, Resources and EnvironmentNecessary facilities, major equipment, and services are available; relevant assets available e.g., IP, licenses; collaborations and/or partnerships appropriate for the success of the projectSeptember 9, 2011
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Slide20Templates to guide the organization of your submission
Templates are located in Part B of the application
Section 2 – CIRM Target Product Profile (TPP) Section 3 – Summary of Nonclinical TestingSection 4 – CIRM Clinical Protocol Synopsis Section 5 – CIRM Manufacturing Plan Synopsis September 9, 2011
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Slide2121
CIRM’
s Collaborative Funding Partners (CFP): Leveraging Expertise and Resources with Germany, Canada and Andalucia in this RFA
Canada
Victoria
India
Japan
UK
Spain
Germany
China
Maryland
NYSCF
Andalucia
Australia
JDRF
France
September 9, 2011
Slide22Interested in a CFP project? See appendices for details
To apply for a collaboratively funded project involving CIRM and a Collaborative Funding Partner (CFP), applicants must satisfy both the CIRM requirements and any additional requirements established by the applicable CFP.
For more details on CFP requirements, please see Appendices C, D, or E.September 9, 201122
Slide23CIRM’s Governing Board (ICOC) allocated up to $240 million for 12 awards
4 year
awards, allocated up to a total of 12 awards up to a total of $240 millionFor-profit applicants seeking CIRM funding for clinical trials are highly encouraged to provide evidence that additional funds (minimum of 50% of the total funds) for the proposed project have been secured or will be secured if recommended for funding. Nonprofit applicants proposing clinical trials are encouraged to engage in partnership(s) with industry to leverage expertise and additional funds for the proposed project.
These strategies are intended to leverage CIRM’s funds.
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Slide24CIRM Key dates to remember*
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Exceptions RequestOctober 4, 2011
(CIRM decision by November 15, 2011)
Award Applications due
Jan 25, 2012
Limited supplemental info due
March 13, 2012
GWG Review
April 2012
ICOC Consideration
June 2012
Earliest Funding
August
2012
*CFPs may have additional requirements
that impose some different dates on their applicants
September 9, 2011
Slide25If you have additional questions, here are the people to contact
For information about this RFA:Sohel Talib, Ph.D.
Science OfficerCalifornia Institute for Regenerative MedicineEmail: stalib@cirm.ca.govPhone: (415) 396-9137For information about the review process:Gilberto R Sambrano, Ph.D.Senior Review OfficerCalifornia Institute for Regenerative Medicine
Email:
gsambrano@cirm.ca.gov
Phone: (415) 396-9103
September 9, 2011
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