20 Jul STRM.BIO
Michael Luther, Ph.D., Chief Executive Officer and Board of Directors
Oct. 7 | 3:15pm | FLW Ballroom G
Cambridge, MA
(Private)
STRM.BIO is developing megakaryocyte-derived extracellular vesicles (MVs) to deliver gene therapies in vivo. Our platform is differentiated by: (1) a striking tropism to bone marrow and HSPCs while bypassing the liver; (2) tunable loading that supports nucleic acid and protein cargo delivery in vivo; and (3) ability to repeat dose. The company’s lead programs are targeting rare blood diseases. We recently completed mouse in vivo protein expression studies in several reporter systems. In mice, reporter protein is expressed preferentially in bone marrow cells following in vivo delivery of nucleic acid by MVs; no cargo-derived expression was observed in the liver. In prior NHP repeat dosing/tolerability and biodistribution studies, pDNA-loaded MVs preferentially delivered cargo to bone marrow. Cargo loading did not alter biodistribution profile in either animal model. Mouse safety studies and the NHP tolerability & repeat dosing study confirms unloaded EVs are non-toxic and amenable to repeat dosing.
