SBIR/STTR Research Award
Enzyme Replacement Therapy for GM1 Gangliosidosis Lysosomal Rare Disease
Award amount
$225,000Program and phase
SBIR · Phase IAward year
2014Award details
- Awarding agency
- Department of Health and Human Services
- Branch
- National Institutes of Health
- Recipient company
- BIOSTRATEGIES LC
- Proposal award date
- Not reported
- Contract end date
- Not reported
- UEI
- PE6MEQZRFW63
- Solicitation number
- PA13-234
- Topic code
- NINDS
Research abstract
DESCRIPTION (provided by applicant): The goal of this proposal is to develop an effective enzyme replacement therapy (ERT) for patients suffering from GM1 Gangliosidosis by integrating a novel protein fusion ERT cell delivery strategy under development atBioStrategies LC and by exploiting safety, supply, and cost advantages of new plant-based enzyme therapeutics bio-manufacturing systems. GM1 Gangliosidosis is a rare genetic lysosomal storage disorder affecting less than 200,000 people in the U.S. It is caused by a defect in the gene encoding the enzyme ?-galactosidase and is characterized by progressive degeneration of normal childhood development especially in brain function leading to death at an early age. Current treatment options are limited to management of disease symptoms and development of an effective ERT drug has been hindered by challenges of delivering these drugs to the brain and central nervous system. This research project is a collaboration between BioStrategies LC, a company focused
Technology keywords
Not reported
Other awards by this recipient
Verified by the same UEI. These are other public SBIR/STTR award pages, not a merged company record.
- Treatment of GM1 gangliosidosis CNS manifestations by AAV systemic expression of lectin-enhanced enzyme
- Morquio A therapy integrating gene transfer with lectin-enhanced enzyme delivery to treat multisystemic clinical impairments of rare metabolic childhood diseases
- Gene Therapy that Systemically Produces Brain-penetrating Replacement Enzyme for MPS IIIA (Sanfilippo A Syndrome)
- Krabbe disease therapy integrating gene transfer with lectin-enhanced enzyme delivery to treat pathologies of the CNS
- Enzyme Replacement Therapeutics for Rare Childhood Genetic Diseases: An ERT Delivery System that Mitigates Immune-sensitization
Awards with the same topic code
Verified by an exact topic code match. This is not a text-similarity match.
- Enhancing Healthcare Delivery and Patient Outcomes: Noninvasive Wireless Sensors to Improve Ventricular Shunt Monitoring, Reduce Imaging, and Alleviate the Burden on Providers, Patients, and Families
- Commercialization Readiness Pilot for Flow Acceleration for Stroke Thrombolysis (FAST) System
- 3D in vitro model of genetic and sporadic FTD: accelerating novel therapeutic development
- Tacrolimus-eluting nerve guidance conduit for enhanced regeneration in critical-sized peripheral nerve injuries
- Promoting tissue repair and functional recovery after spinal cord injury by targeting the microtubule regulatory protein Fidgetin-like 2: Supplement for Imaging System
Research funding history
22 SBIR/STTR records share this UEI (2007–2025). Reported award amounts total $18,287,965. Reported amounts are not federal obligations.
Phases: Phase I, Phase II
Agencies: Department of Health and Human Services, Department of Agriculture