Background & Purpose

Please click on the “RFA Announcement” link above for complete information.

Description

JDRF aims to support the advancement of early preclinical stage hypoimmunogenic pluripotent stem cells (PSCs) for beta cell replacement in type 1 diabetes (T1D) into late preclinical or pilot clinical testing. Here late preclinical testing refers to experiments in relevant preclinical models (pre-IND) and/or IND-enabling studies. Prior investments in genetic engineering of renewable PSC sources of beta/islet cells has resulted in the development and validation of modifications which impart cells with immune-evasive properties. However, these technologies are still relatively new and require further validation and development. This funding opportunity aims to support the further development of such cell sources. Therefore, JDRF is soliciting Letters of Intent (LOI) from investigators who have an early- to late-stage preclinical beta/islet cell source fitting these criteria.

Proposed experiments may use preclinical animal models such as porcine, NHP, or humanized mouse models to demonstrate immune evasive properties with a focus on long term survival and protection from allo- and/or auto-rejection. Simple immune protection experiments (e.g., teratoma formation, in vitro assays, and/or allogeneic transplant in mice) should have previously demonstrated success and be included as preliminary data. PSC sources must be able to demonstrate functional competence in beta cell/islet differentiation and insulin production before and after genetic modification. The scaling and manufacturing of previously validated hypoimmunogenic cell sources for beta cell replacement are also of interest. The discovery of new genes to evade the immune system that require extensive in vitro and in vivo testing are NOT of interest. Our primary objective is to further develop established genetically modified beta/islet cell derived from PSCs with immune evasive properties through late preclinical validation, scaling, and clinical testing which will ultimately lead to T1D therapies.

Informational Webinar and Q&A

JDRF will hold an announcement introduction meeting via Zoom on August 8, 2023, from 1-2 pm Eastern Time to which all prospective applicants are invited. JDRF scientists will give an overview of the goals of this initiative, explain the application process, and answer initial questions on applications.

Registration Link for Webinar (please register by August 4, 2023): https://jdrf.zoom.us/webinar/register/WN_SXvdMt2cRHCGnhU0IUpSkA