Orally administered anti-TNFalpha RNAi therapeutic for autoimmune disorders

Award Information
Agency: Department of Health and Human Services
Branch: N/A
Contract: 1R43AI091045-01
Agency Tracking Number: AI091045
Amount: $597,088.00
Phase: Phase I
Program: SBIR
Awards Year: 2010
Solicitation Year: 2010
Solicitation Topic Code: NIAID
Solicitation Number: PHS2010-2
Small Business Information
RXI PHARMACEUTICALS CORPORATION, 60 PRESCOTT ST, WORCESTER, MA, -
DUNS: 932221729
HUBZone Owned: N
Woman Owned: N
Socially and Economically Disadvantaged: N
Principal Investigator
 ANASTASIA KHVOROVA
 (508) 767-3861
 AKHVOROVA@RXIPHARMA.COM
Business Contact
 MITCHELL WOOLF
Phone: (508) 767-3861
Email: admin@rxipharma.com
Research Institution
N/A
Abstract
DESCRIPTION (provided by applicant): Introduction of small interfering RNAs (siRNAs) into cells results in potent and specific gene silencing by RNA interference (RNAi). Unfortunately, while siRNA-based drugs represent a potentially significant therapeutic paradigm, the ability to apply this technology to human afflictions, in particular, diseases associated with chronic inflammation, has been impeded by the absence of efficient, non-toxic and tissue-specific delivery systems. We have recently shown that P1, 3-D-Glucan particles can be efficiently employed to deliver siRNAs to macrophages via oral administration (Aouadi, Tesz et al. 2009). As low dose, oral administration of chemically synthesized oligonucleotides was previously thought to be impossible, this discovery is viewed as a significant scientific breakthrough. The objective of this proposal is to employ this technology to develop a Glucan particle formulated with TNFalpha targeting siRNAs and validate this platform's efficacy in accepted models of inflammation. Completion of this project is expected to enable rapid progression into the preclinical /clinical development of an orally administered anti-inflammatory drug, for autoimmune diseases such as inflammatory bowel disease, rheumatoid arthritis and psoriasis. PUBLIC HEALTH RELEVANCE: RNAi (RNA interference) has large potential for the treatment of human disease. Efficient delivery is a major road block for therapeutic development. We have recently shown that 1, 3-D-Glucan particles can be efficiently employed to deliver siRNAs to macrophages via oral administration (Aouadi, Tesz et al. 2009). Completion of this project is expected to enable rapid progression into the preclinical /clinical development of an orally administered anti-inflammatory drug, first for autoimmune diseases such as inflammatory bowel disease, rheumatoid arthritis and psoriasis.

* Information listed above is at the time of submission. *

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