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Human T cell Lymphotropic Virus Vaccine development

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41AI165061-01A1
Agency Tracking Number: R41AI165061
Amount: $588,155.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: NIAID
Solicitation Number: PA21-262
Solicitation Year: 2021
Award Year: 2022
Award Start Date (Proposal Award Date): 2022-02-01
Award End Date (Contract End Date): 2024-01-31
Small Business Information
1951 NW 7TH AVE STE 600
Miami, FL 33136-1128
United States
DUNS: 002058614
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (305) 646-8103
Business Contact
Phone: (305) 243-3808
Research Institution
CORAL GABLES, FL 33146-2926
United States

 Nonprofit College or University

PROJECT SUMMARY: For this proposal we intend to develop a novel vaccine to prevent and possibly treat
Human T cell Leukemia Virus type-1 (HTLV-1) associated diseases. HTLV-1 is a human retrovirus that is
the causative agent of a malignant CD4+ T cell lymphoproliferation referred to as Adult T cell leukemia/lymphoma
(ATLL), as well as several inflammatory disorders with the most problematic being human myelopathy/tropical
spastic paraparesis (HAM/TSP). HTLV-1 infection is endemic in many areas around the world including southern
Japan, the southern United States, central Australia, the Caribbean, South America, equatorial Africa, and the
Middle East. Over 10 million people may be infected worldwide. It is estimated that approximately 5% of HTLV-
1 positive individuals will develop ATLL, and 2% HAM/TSP. Seropositive rates in certain areas reach 20–40%
among people aged over 50 years. With millions affected worldwide, HTLV-1 is a major problem in endemic
communities and remarkably, there are no effective vaccines to prevent associated disease or treatment
options for ATLL or HAM/TSP afflicted individuals. HTLV-1 has three modes of transmission: mother-to-child,
mainly linked to prolonged breast-feeding; sexual, predominantly occurring from male to female; and via
transplantation of organs and blood components. Surprisingly, unlike Human Immunodeficiency Virus type-1
(HIV-1), HTLV-1 possesses significant genetic stability. Furthermore, viral amplification via clonal expansion of
infected cells and cell-cell fusion (syncytia) formation, rather than viral lytic replication and release is responsible
for viral spread. Accordingly, ATLL is a clonal malignant disease. HAM/TSP, in contrast, is thought to be caused
by viral-triggered inflammatory damage to the nervous system, by mechanisms that remain unclear. It is also
unclear why certain patients may remain infected for years without clinical features, while others develop
HAM/TSP and/or ATLL. Given this information, and with improved knowledge of innate immune signaling and
vaccine development, we aim to develop and test the efficacy of a novel vaccine to prevent HTLV1-mediated
disease. To achieve this, we have generated a vaccine vector based on Vesicular Stomatitis Virus (VSV) and
have assembled an experienced team with significant knowledge in innate immunity, HTLV-1, VSV manipulation
and manufacture at the GMP level and Phase I clinical trial design. We request resources to manufacture VSV
expressing HTLV-1 viral proteins (VSV-gp62-∆HT) at a level sufficient for FDA relevant preclinical studies with
facilities at the Mayo Clinic Rochester led by Dr. Mark Federspiel a long-standing collaborator of Dr. Barber.
These preclinical studies will produce large-scale VSV-gp62-ΔHT virus stocks with characteristics
comparable to the future GMP clinical-grade products, acceptable by the FDA for use in final efficacy,
toxicology and pharmacology studies supporting Pre-IND and IND applications. We have patented our
intellectual property through the University of Miami’s Office of Technology Transfer (OTT) and this has now
been licensed to STINGINN LLC for development, as a collaboration.

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

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