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Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 2R42HL062715-02
Agency Tracking Number: HL062715
Amount: $0.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: N/A
Solicitation Number: N/A
Solicitation Year: N/A
Award Year: 2002
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
WESTON, MA 02493
United States
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (617) 638-4173
Business Contact
Phone: (617) 638-5639
Research Institution
BOSTON, MA 02215
United States

 Nonprofit College or University

DESCRIPTION (provided by applicant): Sickle cell disease (SCD) and
beta-thalassemia are genetic disorders caused by molecular mutations affecting
the genes for adult hemoglobin. With a world-wide birth prevalence of 2/1,000,
hemoglobin disorders are the most common genetic diseases in the world (360,000
patients/year - 250,000 with sickling disorders and 110,000 with thalassemias.
These conditions can be ameliorated by reactivating production of fetal
hemoglobin (Hb F) in the patient?s blood. Pharmacologic re-induction of fetal
hemoglobin has been achieved in these diseases using a first generation
intravenous therapeutic, a short-chain fatty acid (SCFA) salt, and these
treated patients experienced both biochemical and clinical improvement in their
diseases, with excellent safety profiles. During the Phase I STTR, the
applicant organization developed third-generation SCFAs with substantial
advantages over the first generation agent, including enhanced Hb F-and
cellular growth stimulatory activity, oral-bioavailability, and prolonged
biological half-lives. These agents have proven effective and safe in a baboon
model for Hb F induction.

In this Phase II application, we propose to conduct the preclinical development
studies necessary to obtain Investigational New Drug status for a new lead
compound (ST-20) for human clinical trials. The goals of this proposal are: I)
To prepare a stable medicinal formulation of a sodium salt of ST20, a synthetic
SCFAD which stimulates Hb F production; ii) To refine oral dosing-regimens for
ST20 for application to patients; iii) To prepare an IND Application for ST20;
iv) To evaluate additional SCFADs for activity in induction of Hb F expression,
as back-up compounds.

PROPOSED COMMERCIAL APPLICATION: Sickle cell disease and thalassemia are the two most common genetic diseases in the world, killing millions of people. No safe definitive therapy exists for these diseases. The applicant organization has developed an oral version of a first-generation therapeutic, which must be given intravenously. Upon completion of preclinical studies, Investigational New Drug status will be obtained and the new therapeutic agent will be tested in human clinical trials.

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

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