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Antisense gene therapy with tumor-targeted nanocapsules

Award Information

Agency:
Department of Health and Human Services
Branch:
N/A
Award ID:
65461
Program Year/Program:
2003 / SBIR
Agency Tracking Number:
CA099366
Solicitation Year:
N/A
Solicitation Topic Code:
N/A
Solicitation Number:
N/A
Small Business Information
GENESEGUES, INC.
GENESEGUES, INC. 3180 HIGH POINT DRIVE CHASKA, MN 55318-9476
View profile »
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No
 
Phase 1
Fiscal Year: 2003
Title: Antisense gene therapy with tumor-targeted nanocapsules
Agency: HHS
Contract: 1R43CA099366-01
Award Amount: $245,059.00
 

Abstract:

DESCRIPTION (provided by applicant): Antisense oligonucleotides have generated enormous interest as therapeutic molecules since their initial demonstration in 1978, but their full utilization as effective human therapies has been hampered by inadequate drug delivery to the correct cellular compartment of the correct tissue. We have designed a water-based, ultra-small nanocapsule formulation for targeted intracellular delivery of biologics to solid tumors. This delivery system takes advantage of caveolar endocytosis for highly efficient transit of nanocapsules and their cargo to the target cell's nucleus. The extremely small size of these particles (< 50 nm, "s50") enables high tissue penetration and transdermal delivery via topical application offering creative options for drug delivery to pressurized solid tumors. Possession of a tumor-targeted delivery for biologics enables us to consider manipulation of extremely sensitive and potentially very effective targets for tumor eradication. The enzyme Casein Kinase 2 (CK2) is such a target as it: i) is consistently unregulated in all cancers studied to date; ii) correlates with poor clinical outcome; iii) is essential for cellular proliferation activity; and, iv) regulates apoptosis suppression as well as other validated chemotherapeutic targets, such as Topoisomerase 2 and histone deacetylase. In this proposal, building on promising initial animal studies, we will test feasibility of the s50 nanocapsule antisense CK2 therapeutic for clinical development in the treatment of recurrent squamous cell carcinoma of the head and neck (SSCHN), a grim disease with an approximate 30% five-year survival rate. In these studies, we will optimize the choice of backbone chemistry for the CK2 antisense molecule, then conduct in vitro studies and in vivo animal studies with the s50 nanoencapsulated molecule. Our focus in these studies will be on more aggressive SCCHN tumors; in particular, those tumors not expected to be treatable by experimental therapies currently in development.

Principal Investigator:

Gretchen M. Unger
9524433798
GMU@GENESEGUES.COM

Business Contact:

Robert Unger
9524433798
RTU@GENESEGUES.COM
Small Business Information at Submission:

GENESEGUES, INC.
GENESEGUES, INC. 3180 HIGHPOINT DR CHASKA, MN 55318

EIN/Tax ID: 411955104
DUNS: N/A
Number of Employees: N/A
Woman-Owned: No
Minority-Owned: No
HUBZone-Owned: No