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Ambient light activatable opsin based therapy for age-related macular degeneration

Award Number: R44EY025905
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2015
PERIOD OF PERFORMANCE END DATE: 02/29/2024

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Ambient light activatable opsin based therapy for age-related macular degeneration - Severe loss of vision occurs due to retinal degeneration as in dry age-related macular degeneration (dry-AMD) in which dysfunction of retinal cells occurs leading to blocking of the visual transduction cycle. Retinal prostheses offer the possibility of restoring limited vision. Current systems, however, are limited by poor resolution, retinal damage over time. Optogenetic sensitization of retinal cells has potential as an interim solution until the regenerative medicine is successful. In addition to higher resolution, optogenetics has advantages such as cellular specificity and minimally invasive intravitreal injection. With advent of Ambient- light activatable, fast and broadband Multi-Characteristic Opsin (MCO), the hurdle in clinical translation of optogenetic vision restoration is removed. The phase-II SBIR support has enabled us to complete rigorous IND-enabling pre-clinical studies including evaluation of efficacy, pharmacokinetics and GLP toxicity and Biodistribution of intravitreally-injected vMCO in small and large animals. These studies were guided by inputs from FDA during our pre-IND meeting and follow-up communications. Our phase-II SBIR studies demonstrate that process-developed vMCO remains stable and potent for > 1 year leading to the efficient expression in bipolar cells. MCO-dose dependent expression was highly correlated with improved visually guided behavior in radial water maze and optomotor response at ambient light illumination. Further, we found intravitreal injection of vMCO drives MCO expression in bipolar cells of two different mice models of retinal degeneration and large animals. The safety of vMCO in mice was confirmed by no detectable retinal phototoxicity after chronic exposure with intense light for four months. Further, no significant increase of pro and anti- inflammatory cytokines in plasma or vitreous humor was observed six months after vMCO injection. Based on this success and feedback from FDA, we aim to further develop the product (vMCO) for restoring vision in dry- AMD subjects with retinal dystrophies in ambient light environment. The objective of this phase-IIb SBIR project will be accomplished through three aims. Aim 1: Robust controlled production of vMCO with high yield and comparable performance; Aim 2: Evaluation of transduction efficacy and visual function using electrophysiology in dry-AMD model of NHPs; and Aim 3: A Phase 1 Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intraocular vMCO injection in Patients with Advanced dry-AMD. This collaborative effort brings together complementary expertise to address a challenging problem in retinal degenerative diseases. Upon completion of the phase IIb SBIR we envision to advance: (i) Initiation of Phase- 2 trial for evaluating efficacy, and (ii) enhance our partnership with private-equity/venture capital firms and pharmaceutical company for commercialization. Success of this proposal will lead to a new clinical approach for treating patients with dry-AMD at ambient light. This gene-agnostic approach will allow treatment of other retinal degenerative diseases such as retinitis pigmentosa, Stargardt and cone-rod dystrophy.


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: 2024 ( Subtotal = $0 )
2024	2022	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL	BEDFORD	TX	76022	TARRANT	USA	Vision Research	000	5	9/17/2024	NON-COMPETING CONTINUATION	$0
														Subtotal = $0

Issue Date FY: 2022 ( Subtotal = $737,805 )
2022	2022	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL	BEDFORD	TX	76022	TARRANT	USA	Vision Research	001	5	7/29/2022	NON-COMPETING CONTINUATION	$737,805
2022	2021	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL STE A	BEDFORD	TX	76022	TARRANT	USA	Vision Research	000	4	4/4/2022	COMPETING CONTINUATION	$0
														Subtotal = $737,805

Issue Date FY: 2021 ( Subtotal = $742,339 )
2021	2021	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL STE A	BEDFORD	TX	76022	TARRANT	USA	Vision Research	002	4	9/9/2021	COMPETING CONTINUATION	$749,517
2021	2020	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL STE A	BEDFORD	TX	76022	TARRANT	USA	Vision Research	001	3	7/19/2021	SUPPLEMENT FOR EXPANSION	-$7,178
2021	2019	NANOSCOPE TECHNOLOGIES LLC	1312 BROWN TRL STE A	BEDFORD	TX	76022	TARRANT	USA	Vision Research	000	3	3/31/2021	NON-COMPETING CONTINUATION	$0
														Subtotal = $742,339

Issue Date FY: 2020 ( Subtotal = $77,597 )
2020	2020	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	3	2/15/2020	SUPPLEMENT FOR EXPANSION	$77,597
														Subtotal = $77,597

Issue Date FY: 2019 ( Subtotal = $1,059,573 )
2019	2019	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	001	3	9/2/2019	NON-COMPETING CONTINUATION	$950,373
2019	2019	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	2	1/24/2019	SUPPLEMENT FOR EXPANSION	$109,200
														Subtotal = $1,059,573

Issue Date FY: 2018 ( Subtotal = $1,031,182 )
2018	2018	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	2	9/20/2018	COMPETING CONTINUATION	$1,031,182
														Subtotal = $1,031,182

Grand Total All Awards = $3,648,496

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Ambient light activatable opsin based therapy for age-related macular degeneration

Award Number: R44EY025905

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

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

PERIOD OF PERFORMANCE START DATE: 09/01/2015

PERIOD OF PERFORMANCE END DATE: 02/29/2024

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