- Megakaryocyte-derived EVs show preferential biodistribution to bone marrow, bypassing the liver
- Safety and tolerability data demonstrate the potential for repeat dosing
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, presented the first preclinical data on their novel delivery platform that uses megakaryocyte-derived EVs to target hematopoietic stem/progenitor cells (HSPCs) in the bone marrow at the American Society of Hematology (ASH) annual meeting being held in San Diego, California, December 9 – 12, 2023.
Study results demonstrate that megakaryocyte-derived EVs preferentially target bone marrow in mice and non-human primates. The EVs selectively targeted and delivered pDNA cargo to HSPCs and, following intravenous delivery, led to reporter protein expression almost exclusively in the bone marrow. Safety and tolerability data in non-human primates demonstrated amenability of the platform to repeat dosing. Collectively, these data describe a platform that can be used to develop and deliver targeted gene therapies in vivo and that will be safe for repeat dosing.
“The data we presented at ASH describe an advanced gene therapy platform that can target bone marrow following in vivo administration in both mice and non-human primates,” said Laura Goldberg, M.D., Ph.D., Vice President of R&D at STRM.BIO. “This approach offers the potential to replace current ex vivo approaches to treat rare blood disorders and provides the possibility of repeat dosing as required for successful clinical outcomes and to expand the landscape of diseases that could be treated with this approach.”
“We are leveraging our platform to develop gene therapies for rare blood diseases. 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,” said Jonathan Thon, Ph.D., CEO and Founder of STRM.BIO. “We are extremely encouraged by our preclinical data, and we look forward to continuing development of our gene delivery platform and moving this platform into the clinic.”
Source – PR Newswire