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Repression of Sodium Channels via a Gene Therapy for Treatment of Chronic Neuropathic Pain

Award Number: R43NS112088
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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Repression of Sodium Channels via a Gene Therapy for Treatment of Chronic Neuropathic Pain - Project Summary/Abstract 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 – NaV1.7 – 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 NaV1.7 in normal humans could recapitulate the phenotype of CIP. However, the high homology of human NaV 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.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2023 ( Subtotal = $0 )
2023	2019	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	8/22/2023	NEW	$0
														Subtotal = $0

Issue Date FY: 2021 ( Subtotal = $0 )
2021	2019	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	9/3/2021	NEW	$0
														Subtotal = $0

Issue Date FY: 2020 ( Subtotal = $95,050 )
2020	2020	NAVEGA THERAPEUTICS, INC.	2400 TORREY PINES RD	LA JOLLA	CA	92037	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	2/7/2020	SUPPLEMENT FOR EXPANSION	$95,050
														Subtotal = $95,050

Issue Date FY: 2019 ( Subtotal = $529,006 )
2019	2019	NAVEGA THERAPEUTICS, INC.	2400 TORREY PINES RD	LA JOLLA	CA	92037	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	9/16/2019	NEW	$529,006
														Subtotal = $529,006

Grand Total All Awards = $624,056

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Repression of Sodium Channels via a Gene Therapy for Treatment of Chronic Neuropathic Pain

Award Number: R43NS112088

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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