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Optogenetic control over transgene expression for the therapy of brain and spine

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43MH110273-01A1
Agency Tracking Number: R43MH110273
Amount: $220,083.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: 101
Solicitation Number: PA15-269
Solicitation Year: 2015
Award Year: 2017
Award Start Date (Proposal Award Date): 2017-08-01
Award End Date (Contract End Date): 2019-03-31
Small Business Information
Rochester, NY 14618-2722
United States
DUNS: 078731533
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (585) 204-0507
Business Contact
Phone: (585) 204-0507
Research Institution

Optogenetic control over transgene expression for the therapy of brain and spine
Gene modified stem cells are a powerful tool for the production of secretable therapeutic proteins in the body Yet
there is a valid concern over their safety since there is no way to control them once they have been transplanted We
propose to utilize optogenetic control of transcription ORT to externally control secretion of the therapeutic protein
using red light ORT would improve the efficacy and safety of the therapy by repeated remote activation of transgene
synthesis with red light delivered non invasively For a specific disease application we propose to use intrathecally
delivered ORT driven stem cells to produce anti inflammatory anti TNF hybrid antibodies in the subarachnoid cavity
for the therapy of neuroinflammation in the brain The ORT option would allow us to externally induce production of
the anti inflammatory antibody using red light as needed At this initial phase of the project we propose the
conceptual evaluation of the ORT system genetically embedded into bone marrow stem cells This testing is proposed
in cell culture Aim and the mouse cerebral subarachnoid cavity Aim The proposed method aims to improve the
safety and efficiency of the therapy of chronic CNS disorders with episodic neuroinflammatory components by
providing the benefit of remote control of the timing and dosage of the therapy Narrative
Gene modified stem cells offer promise for the therapy of central nervous disorders However the
current methods of delivery and dosage adjustment are not sufficiently safe This proposal offers to
test a novel concept of producing a biological drug in the subarachnoid cavity of the brain under
repetitive induction by red light In this phase we propose to perform the initial evaluation of this
technology in cell culture and a mouse model The option of control over gene modified cell activity
would improve the efficacy and safety of central nervous system therapy opening new opportunities
for treatment of brain and spinal cord disorders The proposed technology has the potential to
facilitate the therapy s dosage adjustment and administration timing thus improving the quality of
life and comfort of neuropsychiatric patients

* Information listed above is at the time of submission. *

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