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The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of advanced gene editing technology for therapeutic applications.

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of a stable cell line for the production of human Retinoschisin (RS1) for therapeutic applications in ocular diseases.

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of an AAV gene therapy for Leber Congenital Amaurosis caused by CEP290 mutations.

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of a transgenic mouse model for X-linked retinoschisis (XLRS).

Summary:

 The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) is seeking potential licensees interested in further developing or utilizing these Casq2 mouse strains. As a research tool, patent protection is not being pursued for this technology. More information to access these strains can be found here: https://www.jax.org/strain/036291 and https://www.jax.org/strain/036290.

Traf3ip2-/- C57/BL6 mice are a genetically modified mouse model in which the Traf3ip2 gene, responsible for encoding the CIKS adaptor protein essential for IL-17 cytokine signaling, has been disrupted. These mice offer a robust platform for research in autoimmune and inflammatory diseases, as well as potential applications in cancer studies. By eliminating IL-17 signaling and cross-interactions with other pathways, they provide a unique opportunity for drug discovery and proof-of-principle studies, shedding light on disease mechanisms and therapeutic development.

The technology at hand involves a rabbit polyclonal antibody specifically designed for the human Sun1 inner nuclear envelope protein, even though it is directed against the mouse Sun1 inner nuclear membrane protein. Sun1 is known to be an inner nuclear envelope protein, and defects in such proteins can lead to debilitating conditions like Emery-Dreifuss muscular dystrophy and Hutchinson Gilford Progeria Syndrome. Importantly, the antibody serves as a valuable tool for diagnostic and analytical studies concerning cells afflicted with nuclear envelope defects.

The described technology pertains to the development of bispecific antibodies targeting the Ebola virus glycoprotein. These antibodies, mAb114 and either S1-4-A09 or its engineered variant S1-4-A09 A80P, demonstrate specificity towards the Ebola virus (EBOV) glycoproteins from different strains, including Kikwit and Bundibugyo. The variant S1-4-A09 A80P retains binding specificity and activity but lacks a glycosylation motif, enhancing manufacturability without compromising function.

This innovative HIV immunization strategy relies on mRNA technology to prime and boost immune responses, using full-length or minimally truncated Env proteins to maintain native conformation for effective membrane expression. Co-formulating Env and Gag proteins promotes virus-like particle (VLP) production in vivo, mimicking HIV's structure for better immune recognition. The regimen involves an intensive schedule of 8+ sequential immunizations and includes boosting with diverse Envs from different HIV-1 clades to broaden the immune response.

Novel CD8+ T cell receptors (TCRs) isolated from elite HIV controllers show unprecedented affinity for conserved HIV epitopes, offering new avenues for direct immunotherapies and T cell engineering. These TCRs exhibit potential for cytotoxicity mediation against HIV-infected cells, with prospects for use in toxin-coupled treatments or as part of engineered T cell therapies. Collaborations, such as with Altor Biosciences, are enhancing these TCRs with proprietary molecules like IL-15, to bolster therapeutic efficacy.