Autologous cell therapy with SMC modified MAPCs

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$99,850.00
Award Year:
2004
Program:
SBIR
Phase:
Phase I
Contract:
1R43HD046177-01
Award Id:
71373
Agency Tracking Number:
HD046177
Solicitation Year:
n/a
Solicitation Topic Code:
n/a
Solicitation Number:
n/a
Small Business Information
ATHERSYS, INC., 3201 CARNEGIE AVE, CLEVELAND, OH, 44115
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
n/a
Principal Investigator:
JOHN HARRINGTON
(216) 431-9900
JHARRINGTON@ATHERSYS.COM
Business Contact:
GIL BOKKELEN
(216) 431-9900
GILVB@ATHERSYS.COM
Research Institution:
n/a
Abstract
DESCRIPTION (provided by applicant): The objective of the current proposal is to establish genetic modification of Multipotent Adult Progenitor Cells (MAPCs) with Synthetic Microchromosome vectors (SMCs) as a strategy for application in autologous cell based therapy of Hurler's Syndrome (Mucopolysaccharidosis Type 1). Multipotent Adult Progenitor Cells (MAPCs) are a potentially revolutionary, pluripotent stem cell population isolatable from adult human bone marrow, capable of differentiation along multiple developmental lineages in a controlled, definable manner. BAC (Bacterial Artificial Chromosome) based Synthetic Microchromosomes (SMCs) are a proprietary, non-integrative, gene therapy platform potentially capable of bypassing the technical and operational challenges inherent in conventional retroviral based gene therapy vectors. The specific experimental aims of the program are as follows: 1) Establish the efficiency of de novo microchromosome formation in MAPCs using BAC-based SMC vectors. Molecular techniques will be utilized to establish the presence, structural integrity and stability of the microchromosome. 2) Determine the impact of the microchromosome on the stem cell-like characteristics and differentiation properties of the host MAPC. 3) Demonstrate long term genetic complementation of MAPCs isolated from Hurler's patients using a synthetic microchromosome vector engineered to express a-L-iduronidase under the control of its endogenous regulatory elements. Synergy of the SMC platform with the MAPC technology will likely provide unique solutions to the difficulties inherent in both gene and cell therapy by permitting non-integrative, mitotically stable genetic modification of the patient's own stem cells with non-immunogenic vectors capable of delivering and expressing any gene of therapeutic significance regardless of size or genomic structure. Success in this Phase 1 study as defined by completion of Aims 1-3 above will trigger Phase 2 studies to evaluate the long-term therapeutic potential of genetically modified human MAPCs in vivo in an immunosuppressed Hurler's mouse model.

* information listed above is at the time of submission.

Agency Micro-sites


SBA logo

Department of Agriculture logo

Department of Commerce logo

Department of Defense logo

Department of Education logo

Department of Energy logo

Department of Health and Human Services logo

Department of Homeland Security logo

Department of Transportation logo

Enviromental Protection Agency logo

National Aeronautics and Space Administration logo

National Science Foundation logo
US Flag An Official Website of the United States Government