Search Results - TherapeuticArea+%3e+Rare%2fNeglected+Diseases

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RS-1 Knockout Mouse Model for X-Linked Retinoschisis (XLRS) Research

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). This technology includes a transgenic mouse model specifically designed to study X-linked retinoschisis (XLRS) disease by interrupting exon 1 of the Rs1 gene. XLRS is a genetic condition...

Published: 6/24/2026 | Updated: 6/24/2026 | Inventor(s): Paul Sieving, Yong Zeng, Ronald Bush

Keywords(s):

Category(s): Application > Research Materials, Application > Therapeutics, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Collaboration Sought > Materials Available, ResearchProducts > Animal Models, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

Gene Therapy for Leber Congenital Amaurosis

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. This technology includes an innovative gene therapy approach for treating Leber congenital amaurosis (LCA) caused by CEP290 mutations. LCA is a severe inherited...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Zhijian Wu, Anand Swaroop, Suddhasil Mookherjee, Suja Hiriyanna

Keywords(s):

Category(s): Application > Therapeutics, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors, ResearchProducts > Sequences, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

Innovative Gene Therapy for Retinal Diseases

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of innovative gene therapy for retinal diseases. This technology includes an innovative gene therapy approach aimed at treating retinal diseases, which are a leading cause of vision loss. Retinal diseases, such as age-related macular degeneration...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Susan Gentleman, Thomas Redmond

Keywords(s):

Category(s): Application > Therapeutics, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors, ResearchProducts > Virus/Bacteria, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

Advanced Gene Editing Technology for Therapeutic Applications

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of advanced gene editing technology for therapeutic applications. This technology includes advanced gene editing techniques aimed at developing targeted therapies for genetic disorders. Current treatments for many genetic conditions are limited,...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Ruchi Sharma, Kapil Bharti

Keywords(s):

Category(s): Application > Therapeutics, Application > Research Materials, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors, ResearchProducts > Sequences, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

Stable Cell Line for Large Scale Production of Human Retinoschisin for Ocular Therapy

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. This technology includes the development of a stable cell line for the large-scale production of human Retinoschisin (RS1),...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Paul Sieving, Alaknanda MIshra, Vijayasarathy Camasamudram, Lisa Wei

Keywords(s):

Category(s): Application > Research Materials, Application > Therapeutics, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, ResearchProducts > Human Cell Lines, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

RPGR Gene Therapy for Retinitis Pigmentosa

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of RPGR gene therapy for Retinitis Pigmentosa. This technology includes an abbreviated version of the RPGR gene that is effective in gene replacement therapy for treating X-linked Retinitis Pigmentosa (XLRP). This condition is a genetic disorder...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Michael Sandberg, Basil Pawlyk, Xinhua Shu, Robin Ali, Alan Wright, Tiansen Li

Keywords(s):

Category(s): Application > Therapeutics, Application > Research Materials, Collaboration Sought > Collaboration, Collaboration Sought > Licensing, ResearchProducts > Plasmids/Vectors, ResearchProducts > Sequences, ResearchProducts > Virus/Bacteria, TherapeuticArea > Ophthalmology, TherapeuticArea > Rare/Neglected Diseases

Targeted Disruption of Rpe65 Gene for Retinal Disease Research

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of a targeted disruption of the Rpe65 gene in mice, a novel model for studying retinal diseases and testing potential therapies. This technology includes the targeted disruption of the Rpe65 gene in mice, which serves as a crucial model for...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Thomas Redmond, Karl Pfeifer

Keywords(s):

AI Based Workflow for Single Cell Analysis

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for single-cell RNA sequencing (scRNA-seq) analysis software that leverages existing large language models (LLM) to simplify and enhance data analysis by providing intelligent recommendations and interpretations. This technology includes SCassist, an innovative...

Published: 6/18/2026 | Updated: 6/16/2026 | Inventor(s): Rachel Caspi, Vijayaraj Nagarajan

Keywords(s):

MmabΔ3-7/Δ3-7 mouse model of metabolism of cobalamin associated B (MMAB) for the study of methylmalonic acidemia (MMA)

Isolated Methylmalonic Acidemia (MMA) comprises a relatively common and heterogeneous group of inborn errors of metabolism. One cause of isolated MMA is genetic deficiency of the enzyme ATP:cob(I)alamin adenosyltransferase encoded by the metabolism of cobalamin associated B (MMAB) gene. MMAB deficiency, also called the cobalamin B type (cblB) of MMA...

Published: 5/28/2026 | Updated: 5/28/2026 | Inventor(s): Charles Venditti, Eun-Young Choi

Keywords(s):

Category(s): Application > Research Materials, TherapeuticArea > Rare/Neglected Diseases, ResearchProducts, ResearchProducts > Animal Models, Collaboration Sought, Collaboration Sought > Licensing, Collaboration Sought > Collaboration, Collaboration Sought > Materials Available

Gene Editing for ALPK1 p.Thr237Met

ROSAH syndrome is a rare genetic disease caused by a mutation in the human alpha kinase 1 (ALPK1) gene (p.Thr237Met), leading to vision loss, swollen optic nerves, dry mouth, enlarged spleen, and frequent headaches. Researchers in the Laboratory of Clinical Immunology and Microbiology (LCIM) at the National Institute of Allergy and Infectious Diseases...

Published: 3/26/2026 | Updated: 3/23/2026 | Inventor(s): Christina Kozycki, Colin Sweeney, Uimook Choi

Keywords(s):

Category(s): Application, Collaboration Sought, ResearchProducts, TherapeuticArea, TherapeuticArea > Rare/Neglected Diseases

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