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Preclinical optimization of a gene therapy for erythromelalgia and chronic pain

Award Number: U44NS122114
ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
OPDIV: NIH
AWARD CLASS: COOPERATIVE AGREEMENT
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 06/01/2021
PERIOD OF PERFORMANCE END DATE: 07/31/2023

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Preclinical optimization of a gene therapy for erythromelalgia and chronic pain - ABSTRACT Chronic pain is pain that persists past the normal time of healing. 1.5 billion people worldwide suffer from chronic pain and this number continues to increase as the elderly population grows, the prevalence of diabetes rises, and cancer survival rates improve. Chronic pain not only severely impacts daily quality of life for many patients, it also places a heavy socioeconomic burden on society. Due to the limited number of efficacious treatment options available, chronic pain is often treated with opioids despite the risk of addiction and side effects. Unfortunately, the prescribing of opioids to treat chronic pain has largely fueled the current opioid epidemic. Therefore, there is an urgent and clear unmet need for non-addictive alternative analgesics for the treatment of chronic pain. The push to develop specific and non-addictive alternative painkillers has brought interest to a particular sodium channel, NaV1.7, shown to be important for pain sensing. Gain-of-function mutations in NaV1.7 are associated with a disorder characterized by intense burning pain in the extremities: primary erythromelalgia. Conversely, loss-of function of NaV1.7 results in the inability to feel pain. Therefore, inhibiting NaV1.7 can be an effective method of reducing pain and treat erythromelalgia patients. To accomplish this, we designed epigenetic modulators to repress expression of NaV1.7. Rather than making permanent edits to the genome, these epigenetic modulators will transiently inhibit expression of NaV1.7. By targeting NaV1.7 at the DNA-level, we can achieve specific and long-lasting modulation of NaV1.7, with better pharmacokinetics prospects than RNA- and protein-targeting approaches. In this study, we propose to optimize these epigenetic modulators as well as their delivery in order to achieve high specificity and efficacy. In addition, we will evaluate our optimized modulators in small-scale manufacturing studies as well as toxicological studies in a large animal model. The result of this study will be an optimized gene therapy that is not only non-addictive and efficacious for treatment of chronic pain but also highly specific and long-lasting.


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: 2025 ( Subtotal = $0 )
2025	2022	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	4/16/2025	NON-COMPETING CONTINUATION	$0
2025	2022	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	2	4/25/2025	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2024 ( Subtotal = $1,701,459 )
2024	2024	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	3	1/29/2024	SUPPLEMENT FOR EXPANSION	$37,149
2024	2024	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	2/26/2024	SUPPLEMENT FOR EXPANSION	$37,552
2024	2024	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	004	4	6/19/2024	SUPPLEMENT FOR EXPANSION	$98,701
2024	2024	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	003	4	6/18/2024	NON-COMPETING CONTINUATION	$1,528,057
2024	2023	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	3	6/17/2024	EXTENSION WITH OR WITHOUT FUNDS	$0
														Subtotal = $1,701,459

Issue Date FY: 2023 ( Subtotal = $1,149,467 )
2023	2023	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	3	9/8/2023	EXTENSION WITH OR WITHOUT FUNDS	$1,074,268
2023	2023	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	002	3	9/12/2023	EXTENSION WITH OR WITHOUT FUNDS	$75,199
2023	2022	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	2	7/13/2023	NON-COMPETING CONTINUATION	$0
														Subtotal = $1,149,467

Issue Date FY: 2022 ( Subtotal = $730,264 )
2022	2022	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	001	2	5/27/2022	NON-COMPETING CONTINUATION	$730,264
2022	2021	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	5/19/2022	NEW	$0
														Subtotal = $730,264

Issue Date FY: 2021 ( Subtotal = $698,171 )
2021	2021	NAVEGA THERAPEUTICS, INC.	3210 MERRYFIELD ROW	SAN DIEGO	CA	92121	SAN DIEGO	USA	Extramural Research Programs in the Neurosciences and Neurological Disorders	000	1	6/1/2021	NEW	$698,171
														Subtotal = $698,171

Grand Total All Awards = $4,279,361

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Preclinical optimization of a gene therapy for erythromelalgia and chronic pain

Award Number: U44NS122114

ORGANIZATION: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

OPDIV: NIH

AWARD CLASS: COOPERATIVE AGREEMENT

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

PERIOD OF PERFORMANCE START DATE: 06/01/2021

PERIOD OF PERFORMANCE END DATE: 07/31/2023

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