20 Jul STRM.BIO
Jonathan Thon, Ph.D., CEO
Oct. 11 | 10:00am | Rentschler ATMP Ballroom
STRM.BIO is developing extracellular vesicles to deliver gene therapies in vivo. Our platform is differentiated by: (1) a natural tropism and preferential targeting to long-term hematopoietic stem cells (earliest progenitor desired for durable correction), (2) tunable loading that supports nucleic acid and protein cargo delivery in vivo, and (3) ability to repeat dose. The company’s lead therapeutic programs are targeting rare blood diseases. We recently completed mouse in vivo protein expression and pilot NHP biodistribution, tolerability and repeat dosing studies. In mice, reporter protein was expressed preferentially in bone marrow cells following in vivo delivery of pDNA by EVs. In NHPs, EVs preferentially targeted and pDNA-loaded EVs preferentially delivered pDNA cargo to bone marrow. pDNA loading did not alter biodistribution profile in either animal model. Mouse safety studies and the NHP tolerability study confirms unloaded EVs are non-toxic and amenable to repeat dosing.