Lentiviral Vector for the treatment of Fanconi Anemia

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