Cell and Gene Therapies

The Stanford Innovative Medicines Accelerator (IMA) is dedicated to accelerating the prototyping of innovative translational gene and cell therapies that target unmet clinical needs. Basic research is outside the scope of the current RFP solicitation. While new technology development per se is also outside the scope of the RFP, leveraging an emerging technology to therapeutically address a specific clinical need is encouraged.

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

Support Provided:

Projects selected through this call for proposal will receive support primarily through the IMA Cell and Gene Therapies module. Support can include but is not limited to access to vector design, development, and research scale production services, along with cell-based assay screening and characterization. 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. Additionally, selected projects may also access (1) the IMA’s Preclinical Pharmacology module for evaluating efficacy and/or pharmacokinetics/toxicology of the most promising drug candidates in a suitable disease model; (2) the IMA’s Protein Engineering module for protein or antibody engineering on which the proposed cell or gene therapy is predicated; and (3) 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.

The IMA’s Cell and Gene Therapies module, under the direction of Dr. Fabrizia Urbinati, will collaborate with the PIs of selected projects to formulate goal- and milestone-driven project plans. Selected projects will be supported for 12-24 months with critical milestone decision points defined for this project period.

Deadline:

All application materials must be received by 5 p.m. on Friday, September 8, 2023 (PDT). Applications must be submitted through SlideRoom (instructions below).

Eligibility:

All Stanford faculty with PI eligibility are eligible to apply. Proposals should only be submitted to the IMA and cannot receive funding from multiple seed grant programs.

Application Instructions:
Proposals should be submitted directly through the 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.

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).

Introduction (2 pages max.): Briefly describe –

• 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.

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.).

Potential challenges (½ page max.):

• Identify any potential challenges that may have an impact on 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.

Future plan (½ 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.

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:
Proposals will be reviewed by a panel knowledgeable in translational research and evaluated according to the following criteria:

• Novelty and significance of the therapeutic hypothesis and the severity of the unmet need.
• Feasibility of the path to clinic or commercialization.
• Goal-oriented research plans that work toward achieving milestones and key deliverables within the proposed time frame.
• Feasibility of support via the available IMA mechanisms/resources.

Based on the rankings, shortlisted projects will be invited to present an oral pitch in October 2023 (date TBD).

Project Execution:
Selected proposals will go through a planning process to finalize a detailed 12-24-month work plan, including timelines, resources needed, mutual responsibilities, data and reagent management, intellectual property considerations, competitive landscape analysis, and an interim assessment and communication plan to ensure effective collaboration throughout the project’s duration. Project-specific details agreed upon through this planning process will be elaborated in individual award letters issued to selected projects. The specific level of support will vary by project need.