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.

Public Health Relevance

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.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Small Business Technology Transfer (STTR) Grants - Phase II (R42)
Project #
5R42HL099150-04
Application #
9304267
Study Section
Special Emphasis Panel (ZRG1)
Program Officer
Qasba, Pankaj
Project Start
2010-07-01
Project End
2019-06-30
Budget Start
2018-07-01
Budget End
2019-06-30
Support Year
4
Fiscal Year
2018
Total Cost
Indirect Cost
Name
Rimedion, Inc.
Department
Type
DUNS #
828836614
City
San Francisco
State
CA
Country
United States
Zip Code
94102