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The objective of this contract is to develop chemically modified mimics of microRNAs with improved activity and stability for the treatment of many diseases

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 272201800034C-0-0-0
Agency Tracking Number: N43AI180034
Amount: $305,000.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: NIAID
Solicitation Number: N/A
Solicitation Year: 2017
Award Year: 2018
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
United States
DUNS: 080160327
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 Place Robert
 (443) 712-2258
Business Contact
 Place Robert
Phone: (443) 712-2258
Research Institution

Approximatelymillion people are currently living with human immunodeficiency virusHIVinfection worldwideWith no curative therapy on the horizonpatients must be on antiretroviral therapyARTfor decades to manage their diseaseBecause ART targets only actively replicating virustreatment interruption or ART failure leads to rebound of latent virusRNA therapeutics have shown great promise as a new mechanism for treating HIVhoweverdelivery to target cells at therapeutic doses remains a clinical problemmiRecule Incproposes to develop a mimic of the anti HIV microRNA miRpmiRecule drug candidate MCencapsulated in a dendrimer that specifically targets HIV co receptor CXCRBinding of MCdendriplex to CXCRinduces receptor mediated endocytosis delivering our therapeutic microRNA mimic to the cytosol of targeted cellsSince MCsimultaneously targets several key regulators of the HIV lifecycle including viral transcriptwe predict that it will be less susceptible to the development of resistance and enable ablock and lockeradication strategy by preventing latent HIVfrom rebounding following treatment interruptionsCompletion of this phasecontract will identify a vastly durable mimic of miRp using proprietary combinations of nucleic acid modificationsdemonstrate effective delivery to CXCRlymphocytes in vivovalidate miRp mimic inblock and lockmodelsand assess potential non target toxicitiesIn phase II we will improve formulationsestablish CMC processes to support synthesize up to Phaseclinical trialsand begin IND enabling studiesi ePKADMEpivotal toxicityetcand dose range finding experiments

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

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