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Development of Astrocyte-Restricted Precursors for Cell Therapy and Drug…

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
96461
Program Year/Program:
2010 / SBIR
Agency Tracking Number:
NS067719
Solicitation Year:
N/A
Solicitation Topic Code:
NINDS
Solicitation Number:
N/A
Small Business Information
Q THERAPEUTICS, INC.
Q THERAPEUTICS, INC. 421 WAKARA WAY, STE 201 SALT LAKE CITY, UT 84108
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2010
Title: Development of Astrocyte-Restricted Precursors for Cell Therapy and Drug Screenin
Agency: HHS
Contract: 1R43NS067719-01
Award Amount: $118,355.00
 

Abstract:

DESCRIPTION (provided by applicant): The long-term objective of this research is to develop a cell therapy for the treatment of neurological disorders that are due to dysfunctional astrocytes. Current approaches to cure or manage these diseases by small mo lecule drugs are limited due to the polygenic and multifactorial nature of the disease. In addition, the blood brain barrier creates a significant hurdle for these drugs to target the diseased cell population. Using a targeted strategy of cell-transplant b ased therapy, diseases as diverse as lysosomal storage disorders and leukodystrophies, Amyotrophic Lateral Sclerosis (ALS; Lou Gehrig's Disease), and Parkinson's Disease may all be approachable. Unlike small molecule therapeutics, natural proteins and comp ounds may be expressed at sufficient levels, at appropriate times, and at proper organ location by the transplanted cells to counteract the dysfunctional imbalance of the endogenous diseased cells. This proposal describes development of a human derived ast rocyte-restricted precursor (ARP) cell population to meet the long-term objectives stated above. This population of cells has been defined in the CNS of rodents and we intend to build on this knowledge for isolation of the human cellular ortholog. We will first define optimal purification and growth conditions for ARPs using fully defined medium conditions, and then assess the ability of ARPs to differentiate into astrocytes after transplantation into the CNS of an animal model. Successful completion of the se studies will form the basis for additional pre-clinical analysis of these cells in a rat model for ALS. In addition to their use as a therapy, Invitrogen and several other pharmaceutical companies have expressed interest in obtaining these cells for dru g screening purposes. Another goal of this project is to provide a pure and consistent astrocyte population derived from ARPs to these companies. PUBLIC HEALTH RELEVANCE: Recent data suggest that dysfunctional astrocytes lead to disease progression in Amyotrophic Lateral Sclerosis (ALS; Lou Gerhig's Disease). Development of an astrocyte-restricted precursor cell population may hold promise as a therapy for this disease.

Principal Investigator:

Robert W. Sandrock
8015825400
RSANDROCK@QTHERA.COM

Business Contact:

Steven J. Borst
sborst@qthera.com
Small Business Information at Submission:

Q THERAPEUTICS, INC.
615 ARAPEEN DR Suite 102 SALT LAKE CITY, UT 84108

EIN/Tax ID: 146049049
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No