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METHOD FOR INCREASING GENE THERAPY EFFICACY AND SAFETY

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43CA090097-01A1
Agency Tracking Number: CA090097
Amount: $100,000.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: N/A
Solicitation Number: N/A
Timeline
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
BIOTRANSPLANT, INC. BLDG 75, 3RD AVE, NAVY YARD
BOSTON, MA 02129
United States
DUNS: N/A
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 JULIAN DOWN
 (617) 241-5200
 JULIAN.DOWN@BIOTRANSPLANT.COM
Business Contact
 LISA LINEHAN
Phone: (617) 241-5200
Email: LISA.LINEHAN@BIOTRANSPLANT.COM
Research Institution
N/A
Abstract

DESCRIPTION (provided by the applicant): The aim of this proposal is to develop
a non-myeloablative conditioning protocol with increased safety and efficacy
for the induction of specific immune tolerance to genetically modified cells.
Potential applications of gene therapy in the treatment of genetic and
autoimmune disorders are widespread, but progress has suffered from a severe
reduction in efficacy due to immune reactions raised against therapeutic
transgene products. Induction of specific immune tolerance through mixed
hematopoietic chimerism is an established methodology that can effectively
re-educate the immune system to eliminate immune rejection. To achieve
engraftment of allogeneic stem cells, conditioning requirements are associated
with toxicities, and graft-versus-host disease is a common side effect. Studies
have shown that molecular chimerism can be used to induce tolerance. Our
preliminary data suggest that specific immune tolerance can be induced to a
single immunogenic protein expressed on autologous cells, and that conditioning
requirements for transplantation of molecularly modified autologous cells are
less than those required for allogeneic transplants. We believe that the
effectiveness of gene therapy can be greatly improved by inducing specific
immune tolerance to foreign proteins to be transferred and that this can be
accomplished using non-toxic protocols.

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

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