University of Massachusetts

Shire Viropharma Incorporated

Takeda Pharmaceutical Company Limited

Marc E Uknis

Timothy F Kowalik

Colin Broom

The present invention, at least in part, relates to the discovery of efficacious delivery of an RNAi agent (in preferred aspects of the invention, an siRNA) to a transplantable tissue. Organ rejection, transplantation-mediated transmission of viral infection, and triggering of apoptosis in transplanted tissues can each be minimized by the methods and compositions of the instant invention. The RNAi agent(s) of the instant invention can be delivered as 'naked' molecules, or using liposomal and other modes of delivery, to transplantable tissues. Such delivery can occur via perfusion of the RNAi agent in solution through the vasculature of a whole or partial organ; or tissues including transplantable cells and cell lines may be bathed, injected or otherwise treated with RNAi agents. Preferred transplantable tissues include, for example, pancreas, liver, kidney, heart, lung, and all cells and cell lines derived from such tissues (e.g., pancreatic islet cells that may, e.g., be transplanted as a treated population).

Therapeutic alteration of transplantable tissues throughorexposure to RNA interference molecules

Therapeutic alteration of transplantable tissues through in situ or ex vivo exposure to RNA interference molecules

A method and composition for treating or preventing antibody-mediated rejection (AMR) of a transplanted organ are provided.

Methods of treating antibody-mediated rejection in organ transplant patients with C1-esterase inhibitor

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The present invention, at least in part, relates to the discovery of efficacious delivery of an RNAi agent (e.g., an siRNA) to a transplantable tissue. The agent may be used to minimize organ rejection, transplantation-mediated transmission of viral infection, and triggering of apoptosis in transplanted tissues. The RNAi agent(s) can be delivered as 'naked' molecules, or using liposomal and other modes of delivery, to transplantable tissues. Such delivery can occur via perfusion of the RNAi agent in solution through the vasculature of a whole or partial organ; or tissues including transplantable cells and cell lines may be bathed, injected or otherwise treated with RNAi agents. Preferred transplantable tissues include, for example, pancreas, liver, kidney, heart, lung, and all cells and cell lines derived from such tissues (e.g., pancreatic islet cells that may, e.g., be transplanted as a treated population).

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Chadds Ford, PA, United States of America

Marc E Uknis