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

Award Number: R43EY025905
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: 08/31/2017

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Ambient light activatable opsin based therapy for age-related macular degeneration - ¿ DESCRIPTION (provided by applicant): Severe loss of vision occurs due to age-related macular degeneration (AMD) and approximately 11 million people in the US have some form of AMD which is expected to double by 2050. Most of the current clinical treatments are primarily focused on slowing down the progression of the disease, as there is neither a cure that can stop the degeneration nor a therapy, other than retinal prostheses, that can restore vision lost due to the degeneration. Current systems, however, are limited by poor resolution (higher electrode density requiring more current, leading to heat production), retinal damage over a time period and cellular overgrowth due to surgical implantation. Use of optogenetic sensitization of retinal cells has several advantages over electrical stimulation such as single-cell resolution, cellular specificity and minimal-invasiveness. However, clinical translation of optogenetic enabled vision restoration suffers from the drawback of active stimulation by blue light source having intensity order of magnitude higher than ambient light owing to the narrow spectral sensitivity of single opsins. To allow ambient-light based stimulation, we propose to utilize (via intravitreal injection lenti-viral vector carrying multi- color opsins (VMCO) to sensitize retinal ganglion cells in photodegenerated AMD retina to different colors of light, allowing broad spectral excitability and thus, significantly higher sensitivity to ambient light. The overall objective of this Phase I SBIR project is to demonstrate behavioral restoration of vision in mice models of late-stage AMD with ambient-level white-light stimulation of retinal ganglion cells (RGCs), sensitized by VMCO. Towards this goal we have following aims: (i) Evaluation of long-term viability and functioning of virally-delivered multi-color opsin sensitized degenerated-retina of AMD mice model; and (ii) Behavioral restoration of vision in mice models of late stage AMD after multi-color opsin sensitization of the degenerated-retina. This proposal is a collaborative effort between NanoScope Technologies, Retina Foundation, and Drs. Mohanty and Kim's laboratories at University of Texas. Success of this proposal will lead to a new clinical approach for treating patients with AMD by conventional intravitreal injection of virus carrying opsin constructs. We believe that such treatment will lead to restoration of high-resolution vision in AMD patients by white-light stimulation of multi-color opsin sensitized RGCs at ambient light level. Upon completion of the Phase I we envision a Phase II SBIR targeted at (i) optimizing the final product design and method, (ii) identifying AMD-population for treatment, (iii) submission of orphan drug license application to FDA, and (iv) providing core marketing material for commercializing the product and method.


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: 2018 ( Subtotal = $0 )
2018	2015	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	1	2/27/2018	NEW	$0
														Subtotal = $0

Issue Date FY: 2016 ( Subtotal = $35,000 )
2016	2016	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	1	9/8/2016	SUPPLEMENT FOR EXPANSION	$35,000
														Subtotal = $35,000

Issue Date FY: 2015 ( Subtotal = $224,925 )
2015	2015	NANOSCOPE TECHNOLOGIES LLC	2519 RADCLIFFE DR	ARLINGTON	TX	76012	TARRANT	USA	Vision Research	000	1	8/17/2015	NEW	$224,925
														Subtotal = $224,925

Grand Total All Awards = $259,925

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

Award Number: R43EY025905

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: 08/31/2017

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