In vivo gene delivery to regulatory t cells to drastically reduce the cost of regulatory t cell therapy

Ended Project Grant Awarded Last action

Award ID
R43AI179244
Recipient
Awarding agency
Obligated
$300,000

Award facts

Assistance listing
93.855 Allergy and Infectious Diseases Research
Completion date
Originally
Place of performance
California, USA

Award details come from USAspending.gov.

Description

Abstract
Project title: in vivo gene delivery to regulatory t cells to drastically reduce the cost of regulatory t cell therapy organization: gigamune INC. PI: david johnson, PH.d. Over 9,000 patients will receive liver transplants this year, and about 75% of those patients will survive at least 5 years. Long term immune suppression is required for nearly all patients, but most immune suppressants (e.g., corticosteroids) are systemic rather than targeted. Immune suppressants lead to long-term toxicities such as malignancy (5% incidence), lymphoproliferative disorder (3% incidence), and infections. About 50% of patients never tolerate the transplant to the point where immune suppressants can be removed. Adoptive cell therapies wherein chimeric antigen receptors (cars) or t cell receptors (tcrs) are engineered into autologous t cells EX vivo have shown strong clinical efficacy and safety. Regulatory t cells engineered with cars ("car tregs") are a relatively new concept wherein cars are used to direct antigen-specific immune suppression. Car-tregs are under preclinical and clinical investigation for indications such as transplant and autoimmunity, with the goal of inducing long-term tolerance without toxicities. However, the cost of manufacturing cell therapies is hundreds of thousands of dollars per patient, driving total per-patient costs to up to $1 million per patient. Recent pioneering work used lipid nanoparticles (lnps) and lentivirus (LVS) to deliver cars to t cells in vivo, eliminating the need for manufacture of engineered cells EX vivo. Gigamune has developed a novel next- generation lentivirus platform (gigalentitm) for in vivo delivery of cars and tcrs specifically to t cells in vivo. The specific aim of this phase i sbir project is to use in vitro models to assess the translational potential of chimeric antigen receptor gene delivery to regulatory t cells for liver transplant tolerance, at drastically reduced cost compared to conventional cell therapy.

Transactions

Action Obligated Date
Amendment $0
Original award $300,000

Quick answers

Who received award R43AI179244?

R43AI179244 was awarded to Gigamune, Inc. by National Institute of Allergy and Infectious Diseases (National Institutes of Health) on April 19, 2023.

How much is award R43AI179244 worth?

$300,000 has been obligated on R43AI179244.

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