In 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 trial. The product will utilize a HIV-1 based lentiviral vector expressin the Fanconi anemia A protein. The strong scientific support for this approached is strengthened by a successful Phase I STTR. The proposal is highly responsive to the PA-13- 235 PHS 2014-02 Omnibus Solicitation of the NIH for Small Business Technology Transfer Grant Applications (Parent STTR[R41/R42]) and the area of interest for NHLBI (HLS13-04). The clinical trial will be open at Indiana University and brings together a combined experience in the biology of Fanconi anemia, gene therapy, and hematopoietic stem cell transplantation. Fanconi Anemia (FA) is a heterogeneous genetic disorder that is characterized by progressive bone marrow failure and cancer predisposition due to a deficiency in DNA repair. Animal 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 failure. This Phase II proposal has the following aims:
Specific Aim 1. Complete Method Validation, Generate Clinical Vector Product, and Perform Additional In Vitro Immortalization Assays.
Specific Aim 2 : Conduct a Phase I trial in subjects with Fanconi anemia A by introducing a functioning FANCA gene into autologous CD34+ stem and progenitor cells. Cells will be treated ex vivo and reinfused into the circulation through the intravenous route.
In 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 trial at Indiana University. The product will utilize a HIV-1 based lentiiral vector with a novel foamyviral envelope to correct the genetic defect in autologous hematopoietic stem cells.
