2024 IMA Cell and Gene Therapies Request for Proposals (RFP)

The Stanford Innovative Medicines Accelerator (IMA) is accepting proposals for projects that address two major challenges in drug development: identification of drug candidates and optimization towards clinical gene and cell therapy products. Through this request for proposals, the IMA is soliciting Letters of Intent (LOI) for projects that fall under the cell and gene therapies module. The IMA is dedicated to accelerating the drug development of innovative translational gene and cell therapies that target unmet clinical needs. Basic research is outside the scope of the current RFP solicitation.

Competitive projects will have 1) a strong therapeutic hypothesis, 2) a novel biological target or mode-of-action that is well-differentiated from other ongoing translational programs in academia or the biopharma industry, and 3) available in vitro assays, and/ or in vivo models to support drug development.

Faculty members who are uncertain about whether their project aligns with this RFP are encouraged to contact Dr. Fabrizia Urbinati (urbinati@stanford.edu) for advice.

Support Provided:

• Cell and Gene Therapy: Support from the IMA Cell and Gene Therapy (CGT) module can include but is not limited to access to AI supported vector design, development, and research scale production services, along with cell-based assay screening and characterization, including 3D and organ on chip technologies.
• Protein Engineering: IMA’s Protein Engineering module can provide support for protein or antibody engineering on which the proposed cell or gene therapy is predicated.
• Preclinical Pharmacology: Additionally, selected projects may also access the IMA’s Preclinical Pharmacology module for evaluating efficacy and/or pharmacokinetics/toxicology of the most promising drug candidates in a suitable in-vitro and in-vivo disease model.
• Stanford’s Clinical and Translational Research Unit (CTRU): for accessing one or more patient-derived reagents that may be necessary to enable prototyping of the target cell or gene therapy.
• External resources: Depending on the nature and requirements of each project, the IMA can also provide access to strategic alliances and qualified contract research organizations (CROs) to support awarded projects. Upon mutual agreement between the PI and the IMA, we will also help identify potential partners (pharma/VC) for the project exit.
• Project management: IMA module leads in CGT, Protein Engineering and Preclinical pharmacology will collaboratively help the PI formulate a goal- and milestone-driven project plan. In addition, the IMA Project Management team will support the project in planning, budgeting, and identifying outsourcing opportunities.
• Financial Support: Awarded projects can be supported for 12–18 months and the specific level of support will vary by project needs. Details regarding specific roles, responsibilities, and financial allocations will be elaborated in individual award letters issued to selected projects. The IMA will not provide salary support for members of the PI’s lab. The PI-lab is expected to contribute the equivalent of one full-time FTE to the project.

Collaboration: It is generally expected that the projects are run in a collaborative and fully transparent manner including decision-making processes, data generation and handling, experimental design, and external collaborations or resources. The IMA will act in a fully confidential manner and guarantee long-term data storage and accessibility.

Deadline: All application materials must be received by 5 p.m. on Friday, August 30, 2024 (PDT).

Eligibility: All Stanford faculty with PI status are eligible to apply. The support of the IMA is limited to one active project per PI at a time. However, PIs with an active IMA collaboration can be listed as a co-PI.

Application Instructions: Proposals should be submitted directly through the SlideRoom portal found at Sarafan ChEM-H SlideRoom Portal.

Proposals should be submitted as single PDF files containing the following materials in the order indicated below. All documents must be single-spaced and Arial 11-point font with 1-inch margins.

1. Title page (1 page max.):

• Project title, investigator name(s), department, address, phone number, email address, and a project summary for a lay audience (150 words max).

2. Introduction (2 pages max.)

• Target disease including cause and biology, age of onset, symptoms, prevalence, severity, and standard of care.
• Proposed therapeutic intervention and justification for why a cell or gene therapy holds promise for qualitatively superior clinical outcomes over alternative therapeutic modalities.
• Describe any new (i.e., non-standard) technology whose implementation at scale will be necessary for prototyping the proposed cell or gene therapy, if applicable.
• Biological tractability including previous work indicating the disease phenotype is addressable by gene/cell therapy. Include the target tissue/cells as well as 5-10 references and relevant preliminary data.

3. Research plan (2 pages max.)

• Define the goals of the project. The research goals should be achievable within 12-24 months.
• Identify critical milestones aligned with the proposed project goals and key dependencies.
• Provide a timeline with estimated start and completion dates for each phase of the project.
• List in vitro and/or animal models that will be used in this project and include defined endpoints to reach the goals.
• Indicate any regulatory submission that is either planned during or resulting from the proposed project.
• Indicate desired exit strategy (e.g., clinical candidate identification, IND submission, spinout, licensing, etc.).

4. Potential challenges (1⁄2 page max.):

• Identify any potential challenges that may impact the development of the therapy/product (i.e., potential toxicity, animal model availability, manufacturability, IP consideration, competitive landscape, resources, etc.) and outline strategies to overcome them effectively.

5. Future plan (1⁄2 page max.):

• Describe your vision for how the project will exit from the university. Identify recent comparables in the field that form the basis for this expectation.

6. Intellectual Property:

• Please provide a list of any OTL docket numbers associated with relevant IP filings related to this proposal.

You do not need to submit your applications to your Research Process Manager (RPM) in RMG or through your Office of Sponsored Research (OSR) Contract and Grant Officer (CGO) for their approval at this time.

Selection Process, Timeline, and Expectations: Each LOI will go through a three-step due diligence process.

1. The most meritorious LOIs will be identified based on review by a panel (internal and external) knowledgeable in gene and cell therapy translational research and evaluated according to the following criteria:

• Novelty of the therapeutic hypothesis
• Significance of the unmet medical need
• Feasibility of support with the available technologies and resources at the IMA
• Competitive landscape analysis and freedom to operate.

2. Based on the rankings, shortlisted projects will be invited to present an oral pitch in October 2024. The IMA review committee will use this pitch to select the awardees for this RFP.

3. Mutual due diligence meetings will be scheduled between the IMA and PI to address any outstanding questions or concerns.

The awardees will be notified by the end of 2024, and collaborative project planning will begin in January 2025.

IMA Contacts

For questions about the Cell and Gene Therapies module of the IMA, please contact:

Fabrizia Urbinati, Ph.D., urbinati@stanford.edu

Michael Fischbach, Ph.D., fischbach@fischbachgroup.org

For questions about the funding opportunity, please contact:

Kristina Woodruff, Ph.D., kwoodruf@stanford.edu

For questions about the application submission process, please contact:

Katrina Barajas, kbarajas@stanford.edu