STRM.BIO, a pre-clinical, VC-backed biotechnology company that is leveraging extracellular vesicles (EVs) to deliver gene therapies and developing new therapeutics for rare blood diseases, announced that it will present preclinical data on their novel delivery platform for in vivo gene therapy that uses megakaryocyte-derived EVs to target hematopoietic stem cells in the bone marrow at the 65th American Society of Hematology (ASH) Annual Meeting being held in San Diego, California, December 9 – 12, 2023.
The data to be presented demonstrates that megakaryocyte-derived EVs:
- Bypass the liver and preferentially target bone marrow
- Selectively deliver pDNA and mRNA cargo to hematopoietic stem and progenitor cells to drive reporter protein expression almost exclusively in bone marrow following intravenous administration
- Represent a platform to develop and deliver targeted gene therapies in vivo that are safe for repeat dosing
Based in Cambridge MA, STRM.BIO is a pre-clinical, VC-backed biotechnology company leveraging extracellular vesicles as a platform to develop and deliver targeted gene therapies in vivo that are safe for repeat dosing. Our vision is to open the door to the future of medicine for patients living with rare diseases worldwide and bring gene therapy to life. Please visit strm.bio to meet our growing team of partners and collaborators and stay up to date on our progress.
SOURCE: PRNewswire