Currently more than 100 million Americans and 1.5 billion people worldwide suffer from chronic pain. This is more than cancer, diabetes, and heart disease combined. Voltage-gated sodium channels are responsible for the transmission of pain signals. Nine genes have been identified, each having unique properties and tissue distribution patterns. Genetic studies have correlated a hereditary loss-of-function mutation in one human Na+ channel isoform ? ?Na?V?1.7 ? with a rare genetic disorder known as Congenital Insensitivity to Pain (CIP). Individuals with CIP are not able to feel pain without any significant secondary alteration. Thus, selective inhibition of ?Na?V?1.7 in normal humans could recapitulate the phenotype of CIP. However, the high homology of human ?Na?V proteins have frustrated most efforts to develop selective inhibitors. We have developed a non-permanent gene therapy to target pain that is non-addictive (because it targets a non-opioid pathway), highly specific (only targeting the gene of interest), and long-term lasting (around 3 weeks in preliminary assays in mice). During this Phase I SBIR, we will 1) test additional pain targets ?in vitro?, and 2) evaluate the new targets ?in vivo ?in mice models of inflammatory and neuropathic pain. In addition, we will initiate our toxicology studies in mice. At the end of this Phase I work, we will know the spectrum of activity and safety of our optimized candidate to perform IND-enabling toxicology studies in Phase II.

Public Health Relevance

Chronic pain affects more than 100 million people in the U.S. with over 25 million Americans living with daily chronic pain. Therapeutic options for these patients consist mainly of opioid narcotics, which come with side-effects and risk of addiction. Navega Therapeutics? proposal aims to develop a non-addictive gene therapy for chronic pain, to improve the current paradigm of pain treatment.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Small Business Innovation Research Grants (SBIR) - Phase I (R43)
Project #
1R43NS112088-01A1
Application #
9845353
Study Section
Special Emphasis Panel (ZRG1)
Program Officer
Hudak, Eric Michael
Project Start
2019-09-30
Project End
2020-08-31
Budget Start
2019-09-30
Budget End
2020-08-31
Support Year
1
Fiscal Year
2019
Total Cost
Indirect Cost
Name
Navega Therapeutics, Inc.
Department
Type
DUNS #
081266832
City
La Jolla
State
CA
Country
United States
Zip Code
92037