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Lentiviral Vector for the treatment of Fanconi Anemia

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 2R42HL099150-02A1
Agency Tracking Number: R42HL099150
Amount: $1,278,659.00
Phase: Phase II
Program: STTR
Solicitation Topic Code: NHLBI
Solicitation Number: PA14-072
Solicitation Year: 2014
Award Year: 2015
Award Start Date (Proposal Award Date): 2015-08-14
Award End Date (Contract End Date): 2019-06-30
Small Business Information
351 W 10TH ST STE 503
Indianapolis, IN 46202-4100
United States
DUNS: 828836614
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (317) 885-6137
Business Contact
Phone: (317) 370-7293
Research Institution
United States

 Nonprofit College or University

DESCRIPTIONprovided by applicantIn this Phase II STTR application Rimedion seeks to commercialize a novel treatment for patients suffering from Fanconi anemia and request funding for a Phase I II clinical trialThe product will utilize a HIVbased lentiviral vector expressin the Fanconi anemia A proteinThe strong scientific support for this approached is strengthened by a successful Phase I STTRThe proposal is highly responsive to the PAPHSOmnibus Solicitation of the NIH for Small Business Technology Transfer Grant ApplicationsParent STTR RRand the area of interest for NHLBIHLSThe clinical trial will be open at Indiana University and brings together a combined experience in the biology of Fanconi anemiagene therapyand hematopoietic stem cell transplantationFanconi AnemiaFAis a heterogeneous genetic disorder that is characterized by progressive bone marrow failure and cancer predisposition due to a deficiency in DNA repairAnimal models of FA are available and have shown that introduce a wild type copy of the defect gene into hematopoietic stem cells can prevent marrow failureThis Phase II proposal has the following aimsSpecific AimComplete Method ValidationGenerate Clinical Vector Productand Perform Additional In Vitro Immortalization AssaysSpecific AimConduct a Phase I trial in subjects with Fanconi anemia A by introducing a functioning FANCA gene into autologous CDstem and progenitor cellsCells will be treated ex vivo and reinfused into the circulation through the intravenous route

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

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