Project Grant R44NS141514
- This federal Project Grant award of $500,000.00 from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, is supporting research by Tega Therapeutics Inc. to develop new clinical biomarkers for GM1 gangliosidosis, a lysosomal storage disease. The project aims to confirm whether levels of specific glycan metabolites in urine and/or cerebrospinal fluid reflect changes in...
- This federal Project Grant award of $206,739 from the National Eye Institute (NEI) under the Vision Research program (CFDA 93.867) supports research to develop a novel adeno-associated virus (AAV) gene therapy approach for treating inherited retinal diseases (IRDs). The key objectives are to test an innovative pre-retinal AAV administration method combined with insulin delivery to accelerate uptake of the gene therapy vector into retinal cells. This aims to address limitations with current...
- This $870,675 Project Grant was awarded on September 15, 2025 by the National Eye Institute (NEI), as part of the Vision Research federal grant program (CFDA 93.867), to Atsena Therapeutics, Inc. for the "Manufacturing Development and Commercial Readiness of an Adeno-Associated Virus-based Gene Therapy for the Treatment of Leber Congenital Amaurosis Type 1". The project aims to conduct the necessary manufacturing development studies to yield a Phase 3 and commercially-ready gene...
- The National Eye Institute (NEI) awarded a $250,500 Project Grant (CFDA 93.867 - Vision Research) to The General Hospital Corporation, doing business as Massachusetts General Hospital (MGH), for the project "A ONE AND DONE THERAPEUTIC STRATEGY TO CORRECT ELP1 SPLICING DEFECT IN FAMILIAL DYSAUTONOMIA". The funding will support the development and customization of base editors to precisely correct the ELP1 mutation that causes Familial Dysautonomia, a neurodegenerative disease leading to...
- This Project Grant award from the National Eye Institute (NEI) under the Vision Research federal grant program (CFDA 93.867) is funding research to develop an innovative gene therapy approach using self-amplifying and self-splicing RNA technologies. The $346,500 award, spanning September 2024 to September 2026, aims to evaluate the longevity, immunogenicity, and potential in vivo side effects of this novel mRNA-based therapy for treating inherited retinal dystrophies. The award recipient, The...
- This Project Grant award from the National Eye Institute (NEI), part of the U.S. Department of Health and Human Services, provides $385,000.00 in funding to Wayne State University to conduct research on genetic and glial support mechanisms in acute and chronic models of photoreceptor degeneration. The research aims to understand, prevent, and treat vision-related conditions and diseases, a focus area of NEI's Vision Research federal grant program (CFDA 93.867). The study will compare...
- This Project Grant award from the National Eye Institute (CFDA 93.867 - Vision Research) provides $306,814 to Piximune, Inc. to develop an antibody-based, gene-agnostic treatment for inherited retinal degeneration, such as retinitis pigmentosa. The proposed approach aims to augment natural mechanisms that eliminate toxic cellular debris from dead and dying photoreceptor cells, thereby protecting surrounding functional cells and preventing secondary retinal damage independent of the causal...
- This Project Grant award for $679,159 from the National Eye Institute (NEI), under the Vision Research federal grant program (CFDA 93.867), supports research on in vivo epigenetic reprogramming of retinal ganglion cells (RGCs) in aging mice with glaucoma. The research aims to identify new therapeutic targets for glaucoma that can protect and rejuvenate RGCs, which are specifically targeted and damaged in this disease leading to vision loss. The 4-year project, awarded to The Schepens Eye...
- This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $461,701 to Tega Therapeutics Inc. to develop and validate an intracerebroventricular (ICV) enzyme replacement therapy for the treatment of GM1 gangliosidosis, a rare lysosomal storage disease affecting the central nervous system. The key objectives are to engineer cell lines to...
- This Project Grant award from the National Eye Institute (CFDA 93.867 - Vision Research) provides funding to Human Cell Co, a for-profit biotechnology research company, to develop a combination therapy for glaucoma. The award, totaling $360,897, will support research to determine if topical application of a nerve growth factor (NGF) mutein (HC201) in combination with oral nicotinamide (NAM) supplementation can more effectively improve retinal ganglion cell function compared to either treatment...
This federal Project Grant award from the National Eye Institute (NEI) at the Department of Health and Human Services provides $1,850,000 in funding to Biostrategies LC to advance the development of BIOS-GM1, a novel gene therapy and enzyme replacement therapy for the treatment of GM1 gangliosidosis. The grant supports Biostrategies' efforts to complete IND-enabling studies for BIOS-GM1, which combines an adeno-associated virus (AAV) gene therapy approach with a lectin-enhanced enzyme delivery system. This innovative therapy aims to systematically deliver corrective enzyme across the blood-brain barrier to prevent the debilitating neurological, psychomotor, and ultimately fatal effects of GM1 gangliosidosis, a rare genetic disorder. If successful, this could represent the first effective treatment for this devastating disease and potentially serve as a breakthrough for delivering enzymes to the brain and body for other rare genetic disorders. The project is funded under the NEI's Vision Research program (CFDA 93.867), which supports comprehensive research to understand, prevent, and treat vision-related conditions and diseases. The award has an ultimate completion date of August 30, 2027.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $350.0k | 9/19/25 | ||
| Not listed | $1.5m | 9/15/25 |