An early-intervention gene-editing therapeutic for pulmonary arterial hypertension
Ended Project Grant Awarded Last action
- Award ID
- R43HL167478
- Recipient
-
Hunterian Medicine LLC UEI MCVJGPMKCAL1
- Awarding agency
-
National Heart Lung and Blood Institute
National Institutes of Health, Department of Health and Human Services
- Obligated
- $350,000
Award facts
- Assistance listing
- 93.837 Cardiovascular Diseases Research
- Completion date
- Place of performance
- Maryland, USA
Award details come from USAspending.gov.
Description
- Abstract
- Pulmonary arterial hypertension (PAH) is a rapidly progressing pulmonary vascular disease that leads to right heart failure and premature death. The hallmark features of PAH are increased pulmonary arterial pressure, vascular remodeling, and right ventricle hypertrophy. There are no curative treatments for PAH and approximately 1,000 new cases of PAH are diagnosed in the U.S. Each year with a median survival of 6 years. Novel therapies are urgently needed. Hypoxia-inducible factors (HIF) are critical mediators of the oxygen sensing and adaptive pathways, and strong evidence supports HIF-2A pathway activation in PAH. Patients with PAH have elevated HIF-2A levels, and pathway activation is well supported by preclinical models. Conversely, reduced pathway activity through either pharmacologic inhibition or conditional knockouts is protective in multiple animal models. A role for HIF-2A is further supported by genome-wide studies of high-altitude populations with low pulmonary arterial pressure that carry HIF-2A variants with reduced activity. We hypothesize that gene- editing represents a novel therapeutic approach to suppress HIF-2A pathway activation that underlies PAH etiology. Hunterian's proprietary technology enables packaging of crispr components into a single AAV virus. In this phase i, we develop a gene-editing approach to target HIF-2A activation.
Transactions
| Action | Obligated | Date |
|---|---|---|
| Amendment | $0 | |
| Original award | $350,000 |
Quick answers
Who received award R43HL167478?
R43HL167478 was awarded to Hunterian Medicine LLC by National Heart Lung and Blood Institute (National Institutes of Health) on September 1, 2023.
How much is award R43HL167478 worth?
$350,000 has been obligated on R43HL167478.
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