REQUEST FOR PROPOSALS DEMONSTRATION PROJECTS IN OH-ALIVE 2014

Transcription

1 REQUEST FOR PROPOSALS DEMONSTRATION PROJECTS IN OH-ALIVE 2014 RFP Issue Date: June 1, 2014 Letter of Intent Due Date: July 1, 2014 (4 chosen) Invited Proposal Due Date: August 15, 2014, 5:00 pm EST Awardees Announcement Date: October 1, 2014 (2 chosen) Description of Funding: The National Center of Regenerative Medicine (NCRM) is accepting proposals for funding of Demonstration Projects for the OH-Alive Innovator Platform: A Process and Manufacturing platform for Cell Therapy for the period November 1, 2014 January 31, Each project will have approximately 3 months to perform optimization cell culture runs. All data generated will be shared with the platform and principal investigator. New intellectual property developed for the project will be assigned to the submitting principal investigator s institution. We expect to complete at least 8 individual pilot Demonstration Projects by December Each project will be allocated $40,000 to cover supply and evaluation costs and each PI is asked to embed one part- or full-time employee (typically PhD level) towards the conductance of the process optimization, while their project is active in OH-Alive. Academic investigators are expected to leverage other project support (such as Federal Grants, Foundation support, other means) towards their respective project as it runs within the OH- Alive. An additional $20,000 can be allocated to any of the demonstration projects after completion based on its commercial merit and immediate potential for a clinical trial. Completion of the project is determined by either finishing sufficient experiments to optimize a manufacturing method or exhaustion of funds towards this goal. No funds will be used outside of the OH-Alive platform. About NCRM: The National Center of Regenerative Medicine (NCRM) is composed of investigators from Case Western Reserve University (CWRU), University Hospitals Case Medical Center (UHCMC), the Cleveland Clinic (CC), Athersys Inc, and The Ohio State University. NCRM s mission is to rapidly translate cutting-edge adult-derived stem cell technology into the clinical and commercial arenas. Currently ten different non-embryonic and two pluripotent stem cell types are being investigated in a range of therapeutic areas including cancer, musculoskeletal, orthopedic, cardiovascular, hematopoietic and neurodegenerative disorders. We encourage you to visit, as well as save to your favorites, our website Page 1 of 10

2 OH-Alive Platform Overview: The OH-Alive platform brings interested corporate and non-profit parties together in a truly shared effort, leveraging individual commitments, interests, and experience to advance new cell therapies to patients and enable new manufacturing intellectual property (IP) to be developed and buttress the clinical programs of both academic and for-profit companies. The platform consists of specific hardware that includes a dedicated innovation platform for determining process-specific parameter optimization for advanced cell culture. Integrated with this physical accelerator is the NCRM s Cellular Therapies Integrated Service (CTIS); a competency scaffold that is able to facilitate all aspects of a cell application from FDA consultancy, IND-filing support, help in filing for intellectual property regarding stem cell technologies, and to facilitate and guide researchers and clinicians with compliance matters involving the Institutional Review Board (IRB). Together, the vision for OH-Alive is to be a one-stop Stem Cell Innovator Platform that will enable companies, the academic researcher advancing newly discovered cell biology, and clinicians desiring to offer cutting-edge therapies to their patients, all to be better able to demonstrate that they can make a difference in the area of cell therapy. OH-Alive s goals are To transform medical therapy through the use of cells rather than drugs to heal tissues and organs To create the commercial and academic infrastructure in Ohio to establish a selfsufficient, vibrant industry of biotechnology and support services that are attractive to cell-therapy institutions across the US Through the Case Western Reserve University Technology Transfer Office, OH-Alive has dedicated support for managing and protecting new intellectual property (IP). OH-Alive s clients will receive the exclusive ability to benefit from commercialization outcomes emanating from utilization of the facility and OH-Alive will assign ownership to its clients any/all IP that is created as part of this service. Collaborative agreements to reduce feefor-service costs will be negotiated on a case basis but royalty stream or milestone payments will be added to this co-development situation. This gives clients flexibility, especially in the academic setting, and increased accessibility to this platform. Demonstration projects will be assumed to be paid in full and all IP transferred to the principal investigator. Of particular benefit for academic investigators and for-profit organizations is access to robotic and incubator equipment to facilitate rapid evaluation of multiple protocol parameters, analytical equipment for verification of cell type and performance, and administrative, technical and regulatory personnel, without having to duplicate resources. They will also have access to NCRM s existing competency in executing new cell therapies. OH-Alive staff will collaborate on grant applications, especially SBIR/STTR mechanisms. Together this will lead to the commercialization of cell therapies, not only resulting in advanced treatments for disease, but the establishment of multiple companies, with approved, commercial products. OH-Alive Demonstration Project Overview: The purpose of this RFP is to assist in the development of novel cell therapeutics and diagnostic platforms that will lead to both commercializable products and clinical interventions. Given the spectrum of potential uses of cells, this RFA will not restrict responses except to note that basic science projects without a readily identifiable and Page 2 of 10

3 immediate translational regenerative medicine application; pharmacological testing and assay development; and cell expansion projects that do not require testing and optimization would in general be non-responsive. Projects using cells of non-human origin are not considered. Demonstration projects which have strong commercialization potential, a clinical application strategy with a realistic timeline, are desired. Engagement of commercial partners or a stated entrepreneurial approach to the emerging technology will strengthen the application. Our staff can be approached to assist in expanding these options. Funds are to be used to offset the costs of optimization and validation of cell expansion, treatments, purifications and release criteria that would move product development substantially forward. Some funds could be used as needed to offset costs for using the Cellular Therapies Integrated Service (CTIS). NCRM s CTIS is a competency scaffold that is able to facilitate all aspects of a cell application from FDA consultancy, IND-filing support, help in filing for intellectual property regarding stem cell technologies, and to facilitate and guide researchers and clinicians with compliance matters involving Institutional Review Board (IRB). Confidentiality of product development will be maintained with appropriate documentation, as indicated in the application. Proprietary projects should submit applications with explicit statements of stating the need for confidentiality so reviewers and participants can sign confidentiality agreements (executed by the CWRU Technology Transfer Office). Description of Equipment Available: Project feasibility is determined by instrumentation compatibility, and we encourage interested investigators to read the platform capabilities below carefully, and, when in doubt, contact OH-Alive personnel to determine technical fit. The OH-Alive platform consists of an environmentally isolated and controlled X-Evo system (X-Vivo enclosure + Evo150 liquid handling instrumentation + carousel incubator) for manual and automated cell culturing, and a QuantStudio instrument set up with OpenArray capabilities for gene expression analysis. The X-Vivo enclosure is a modular solution replacing the more traditional clean room setting. It allows for complete environmental control of cellular conditions putting the cells rather than the scientist first. Oxygen, carbon dioxide, humidity and temperature can be controlled on a modular or global scale to eliminate cell shock during cell culturing and processing. Manual processing chambers and built-in incubators allow for manual cell expansion. A light microscope, centrifuge and cell counter are enclosed in the system to limit exposure to non-ideal conditions during basic cell evaluation procedures. Buffer chambers allow items to be introduced into the system with minimum particulates and contaminants. Licensed design of experiments (DoE) software is used to create an optimized experimental design to meet the desired outcomes for the project. Factors to be manipulated are entered into the program and an experimental design meeting the breadth of testing is generated. Resultant experimental data (gene and imaging) can then be entered back into the software for data analysis. Multivariate analysis from this software allows deep understanding of the experimental space, including sweet spot detection of a desired outcome using the input factors. The Evo liquid handling instrument along with the carousel incubator are also enclosed within the X-Vivo enclosure and allow for automated processing of a wide range of Page 3 of 10

4 experimental parameters as defined by DoE designs. The system is uniquely designed for optimization protocols exploring cell growth and differentiation conditions using complicated media compositions, including multi-stage differentiation. Barcode scanning allows for chain of custody tracking of plates and ultimately the experimental conditions within those plates. The automated system can handle cell culture plates ranging from 6 well up to highthroughput 96 well plates. For data analysis, the QuantStudio OpenArray platform (Life Technologies) is available to provide gene expression data. The Investigator can customize up to 56 genes to analyze for each experiment. These 56 genes will include internal standards and target genes. For a typical project, minimum custom orders consist of 10 OpenArray plates, each plate testing 48 samples x 56 genes. This allows each experiment to generate 26,880 gene expression data points which are imported back into the DoE software for data analysis of experimental conditions. OH-Alive staff can provide investigators with guidance on selecting appropriate genes to meet their experimental design or designing the OpenArray plate. Additional services can include use of a Cellomics ArrayScan High Content Analysis Reader to analyze the cell cultures for phenotypic characteristics via fluorescent markers, and nanofiber coating for 3D cell culture (Nanofiber Solutions). Example Demonstration Projects (not a comprehensive listing) Process optimization to determine optimal cell culture conditions of an existing biologic. A basic media formulation is currently being used; however this has never been optimized to increase culture stability, yield, or maximizing known performance parameters for the cellular material. Differentiation of a specific cell fate from a stem/stem-like state, where the resulting fate has clear clinical therapeutic relevance. The input material could be induced pluripotent stem cells, and the desired fate a more terminal one. Expansion and maintenance of adult primary cell cultures, otherwise difficult to grow ex-vivo. Identification and minimization of stray fate in human cell culture. Demonstration Project Process and Deliverables: Pipeline of OH-Alive projects: Academic or for-profit owned projects are evaluated based on preliminary data strength, commercialization ability, and match to the abilities of the OH-Alive platform Upon entry, process optimization will occur for a period of 3 months, which includes the validation of the production process New project IP will be filed with the aid of the OH-Alive personnel if applicable If desired, the OH-Alive can help facilitate the investigational new drug (IND) process If desired, OH-Alive and its business development partners can help with business startup, raising investment capital, and/or raising federal funds through R-type, or SBIR/STTR grants OH-Alive Deliverables: DoE-based experimentation to determine optimum cell culture conditions Other endpoints outlined and approved in the proposal Production of cellular material suitable for GLP studies Enable protocols to be transferred to a GMP production facility Study report that applies Quality by Design fundamentals Page 4 of 10

5 Development and validation of analytical methods required for release of cellular materials for human-use Support for preparation of IND documentation and regulatory guidance Business development support for spinout companies Important Dates: June 1, 2014 Issue RFP for Demonstration Project funding. July 1, 2014 Letter of Intent due date July 15, 2014 Invitation for full proposals sent to submitters August 15, 2014, 5:00 pm EST Proposals due to NCRM administration via to Michael Gilkey, October 1, 2014 OH-Alive Steering Board will choose awardees based scientific and commercialization merit and NCRM will announce awardees in a press release. October 2, 2014 Preparation for Demonstration project 1 will begin. November 1, 2014 Demonstration project 1 to commence. December 1, 2014 Preparation for Demonstration project 2 will begin. This date may change. January 1, 2015 Demonstration project 2 to commence. This date may change. Page 5 of 10

6 Proposal Guidelines and Format: This is an open and competitive process. Limited feedback from the OH-Alive Steering Board will be sent out to all applicants by December 1, (Refer to the section Proposal Format and Scoring Criteria as well as the Score Sheet.) All grants submitted will be reviewed. A brief summary of the review will be provided. Applicants must have investigator status: if commercial, be identified as a program leader, if academic be assistant professor or independent grant eligible project scientist or above. Collaborators must submit only one proposal; mirror proposals from multiple institutions will not be considered. Letters of Intent are due no later than 5 p.m. EST, July 1, will be invited for full proposals. Individuals may submit multiple LOIs, if desired. However, each proposal must be submitted separately and must match program criteria. Invited proposals are due no later than 5 p.m. EST, August 15, Proposals received after this date and time may not be considered for funding. Proposals must be submitted in electronic format using either Word or PDF files to Michael Gilkey, meg14@case.edu. Proposals not conforming to submission guidelines will not be considered for funding. Format: Proposals must use type size no smaller than 10 point and at least 1 margins The document must be saved in a Word or PDF titled as follows: your last name (period) first word of title (excluding articles, i.e. JONES.STEM). Proposal will have a maximum of 7 pages not including the NIH Approved Biographical Sketch and will be ordered as follows: 1. Title page. 2. Up to 2 pages background and results to date. 3. Up to 2 pages for program plan. 4. Up to 1 pages commercial potential, plan, timeline and milestones towards commercialization and commercial progress to date. 5. Up to 1 page matching funds and budget forecast detail note provisions for grantee funding of portions of the project 6. NIH Approved Biographical Sketch. 7. Departmental or corporate leadership signoff. This is essential to verify matching funds assignment to this project Confidential Information: We prefer to receive non-confidential information in both the LOI and full proposal (if invited). If it is necessary to include this information, please contact Michael Gilkey directly so appropriate steps can be taken to insure confidentiality. Page 6 of 10

7 Letter of Intent (LOI): The LOI is a one page description of the project you wish to submit. Describe the protocol you wish to optimize, including cell type, reagents, and specialized equipment needed to perform the studies. Will you provide the starting cells and/or reagents? Which cellular manufacturing parameters do you wish to test reagents, temperature, gas concentrations, etc? What tests, especially imaging markers, need to be run to determine the results of your differentiation process? What genes should be run on the Quantstudio OpenArray? Who from your group will be sent to the OH-Alive facility to oversee and direct the protocols? What are the expected outcomes of the work? If you are not sure about any of these questions, please indicate that so we can assist. Full Proposal Format and Scoring Criteria: The funding to be awarded in this program is underwritten by the Ohio Third Frontier. Its 10-year objectives with regard to this program are to: Build world-class research capacity. Support early stage capital formation and the development of new products. As such, projects funded by this program must be based on solid science and innovation and must have an identifiable and measurable clinical or commercial goal. To this end, project proposals must include the following information: Program background, scientific rationale, results and progress to date (2 pages): Information should include prior data and results that support the scientific plan being proposed, clinical rationale and significance, potential clinical need and impact, and competing work to be considered. Program Plan (2 pages): Describe in detail the protocol you wish to optimize, including cell type, reagents, and specialized equipment needed to perform the studies. Will you provide the starting cells and/or reagents? Which cellular manufacturing parameters do you wish to test reagents, temperature, gas concentrations, etc? What tests, especially imaging markers, need to be run to determine the results of your differentiation process? What genes should be run on the Quantstudio OpenArray 56 are available for each sample? Who from your group will be sent to the OH-Alive facility to oversee and direct the protocols? What are the expected outcomes of the work? Commercialization Potential, Plan and Progress to date (1 pages): What is the roadmap to commercialization for this technology? Has a Product been defined? Have any Intellectual Property filings (invention disclosure, patent, or license) been completed or planned? What is the regulatory pathway for this technology? Provide details for any substantive interactions with commercial entities or your current status as a commercial entity (basic company background and current investments). Matching Funds and Budget Forecast (1 page): Matching funds are highly encouraged though do not need to exceed personnel and travel costs for you or your lab person to help direct the experiments on site. Please provide detail on your matching funds to apply to the demonstration project; these might include personnel, supplies, and outside services. If you know the costs of the materials or services the platform will need to purchase for your project, please provide as much detail as possible so we can estimate the cost of your project. Please also remember, no funds will go outside of OH-Alive unless to a required core facility to support the project. Please send proposals and direct questions to meg14@case.edu or Page 7 of 10

8 Scientific and Commercialization Scoring Criteria: Scoring of submitted pilot grants will be performed by the OH-Alive Steering Board. This will be an expedited review but is based on current NIH criteria and each domain s weighting is shown out of a total of 100 points. Scientific Scoring (5 domains to be considered): Soundness of Scientific plan and Originality of the proposed Project: Is the proposed work based upon sound and well developed scientific principles? Is the experimental design and rational for the work proposed clearly articulated? Does the technology and approach represent a significant innovative step? (25 Points) Clinical Significance: Does this address a relevant clinical need? How effective are the current treatment options? What is the opportunity for this new technology to make a significant difference in either the standard of care, the total cost of care, or the patient experience? How many patients could be positively affected by this technology? (25 Points) Environment & Timeline: Is there adequate additional support through institutional resources, NCRM or other collaborators, complementary projects and funding sources? Is the project team the right one to get the work done? Does the on-site person working directly with the OH-Alive team have the ability to perform the proposed project within the time frame allotted? (25 points) Scope and Budget: Are the matching funds sufficient to run the project? Will the contribution made by an OH-Alive investment significantly accelerate the work and/or take it to a substantially higher level of value or fundability? (15 points) Leverage of NCRM, Ohio, or Regional Resources: Does this project involve collaborations that take advantage of or advance regional capabilities? Are downstream investments by other funding agencies likely if the project is successful? (10 points) Institutional or corporate commitment Commercialization Scoring (5 domains to be considered): Commercial ( Productization ) Feasibility: Are there existing disclosures, filings, issued patents or copyrights? Is it likely that the project will bear new patentable matter? How likely is it that a commercial product can be developed from this technology? Is there a product configuration anticipated? Are there significant hurdles to overcome from a cost, production, or regulatory standpoint? (30 points) Regulatory Feasibility: Is the proposed regulatory pathway correct and feasible to navigate by the applicant and its partners? What is the regulatory timeline and necessary pathway for safety and efficacy studies? How has the FDA handled products of this sort in the past? Is this a technology approach that the FDA has not encountered before? (25 points) How many years before this technology could be brought to market? Overall Market Impact: What is the size of the market? Is this a new platform technology or an incremental improvement over existing products? If the project is successful, does it have the potential to capture significant market share? (15 points) Page 8 of 10

9 Commercial Readiness: Have potential commercial partners been identified? Is there a relationship in place already? If applicant is a for-profit company, how well is the company positioned to commercialize the technology? (20 points) Disruptive index : To what extent will this technology create a fundamental shift in the market, versus simply creating an incremental improvement over existing technology? Will this development allow a dramatic market expansion or create access to a patient population that could not be served previously? Does it represent a dramatic cost reduction over current approaches? (10 points) Enthusiasm for each of these criteria will be graded by the reviewer based on the score guide below. Score Descriptor Additional Guidance on Strengths/Weaknesses 1 Exceptional Exceptionally strong with essentially no weaknesses 2 Outstanding Extremely strong with negligible weaknesses 3 Excellent Very strong with only some minor weaknesses 4 Very Good Strong but with numerous minor weaknesses 5 Good Strong but with at least one moderate weakness 6 Satisfactory Some strengths but also some moderate weaknesses 7 Fair Some strengths but with at least one major weakness 8 Marginal A few strengths and a few major weaknesses 9 Poor Very few strengths and numerous major weaknesses Minor Weakness: An easily addressable weakness that does not substantially lessen impact Moderate Weakness: A weakness that lessens impact Major Weakness: A weakness that severely limits impact Page 9 of 10

10 Project: PI: OH-Alive Demonstration Project Score Sheet Reviewer Score Comments Scientific Scoring (1-9) Soundness of Scientific plan and Originality of the project Clinical Significance Environment & Timeline Scope and Budget Leverage of NCRM/Regional Resources Scientific Composite Score Commercialization Scoring (1-9) Commercialization ( Productization ) Feasibility Overall Market Impact Regulatory Feasibility Commercial Readiness Disruptive Index Commercial Composite Score Overall Composite Score Reviewer Comments: Page 10 of 10