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AVGN7, a Novel Gene Therapeutic for Treating Cancer Cachexia

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R44CA221539-01
Agency Tracking Number: R44CA221539
Amount: $1,867,191.00
Phase: Phase II
Program: SBIR
Solicitation Topic Code: 102
Solicitation Number: PAR14-088
Solicitation Year: 2014
Award Year: 2017
Award Start Date (Proposal Award Date): 2017-09-25
Award End Date (Contract End Date): 2021-02-28
Small Business Information
Rockville, MD 20850-3638
United States
DUNS: 079995027
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (208) 596-9807
Business Contact
Phone: (208) 596-9807
Research Institution

PROJECT SUMMARYThe skeletal muscle wasting that occurs with cancer cachexia compromises quality of life and is both directly and indirectly responsible for cancer mortalitiesTumor derived and tumor responsive factors as well as many therapeutics themselves contribute to the cachectic statealthough nutritional support has little if any positive effect on restoring striated muscle mass or functionThusnovel tools for preventing muscle wasting in cancer patients could transform their treatment and significantly improve their quality of lifeOur research objective is to test a novel gene therapeutic for enhancing muscle regeneration in a mouse model of tumorand chemotherapy induced cachexia and in additionto complete the GLP toxicology studies required for IND filingWe hypothesize that attenuating the intracellular signaling pathways responsible for muscle atrophy and impaired muscle regeneration will in turn restore muscle mass and function and significantly delay mortalityIn factPhase I equivalent data indicate that attenuating ActRIIB and Smadsignaling with rAAVSmada k aAVGNsignificantly enhances muscle mass and function in wild type mice and can completely prevent muscle atrophy in different mouse models of cancer cachexiaMost importantlythis therapeutic does not produce the serious off target effects that have compromised development of competing technologies that have either been shown to compromise blood vessel integrity or to possess this potentialOur specific aims are toitest the hypothesis that rAAVSmadcan prevent cancerand chemotherapyinduced muscle wastingiitest the hypothesis that rAAVSmadreduces cancer mortality andiiicomplete murine and non human primate toxicology studies with rAAVSmadThe proposed approach is truly innovative as it utilizes a novel gene therapeutic and state of the art tools to comprehensively assess muscle function at different scalesThese studies are also highly significant as they will provide a better mechanistic understanding of how tumorand chemotherapy induced muscle wasting are independently affected by ActRIIB and SmadsignalingMost importantlythese translational studies have the very real potential to impact clinical medicine and to advance clinical trials of rAAVSmadRELEVANCE TO PUBLIC HEALTHThe striated muscle wasting that occurs with cancer cachexia compromises quality of life and is both directly and indirectly responsible for cancer mortalitiesThe proposed studies will develop a novel gene therapy for preventing this wastingwhether it results from tumor burden or chemotherapyThese studies will also advance the gene therapy towards clinical trials by completing the required toxicology studies

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

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