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 today it has been awarded a Small Business Innovation Research (SBIR) grant for approximately $2,100,000 from the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). The award will allow the company to further advance its proprietary EV technology for use as a novel non-viral gene therapy delivery platform.
The specific objective of this NIH funding opportunity is to support the development and evaluation of innovative approaches to deliver genome editing machinery into somatic cells, with the goal of enabling the use of genome editing therapeutics to treat human disease. STRM.BIO has developed a proprietary, large capacity EV-based system for in vivo nucleic acid and protein delivery that specifically targets hematopoietic stem cells (HSCs) in bone marrow, is amenable to large scale commercial manufacture, and presents with low immunogenicity which confers unique potential for repeat dosing.
“It is critical to the future of the field that the next generation of gene therapies be delivered as simple injections in standard clinical settings,” said Jonathan Thon, CEO and Founder of STRM.BIO. “Current ex vivo approaches, in which a patient’s cells are edited in culture and then transplanted, are not a sustainable model. Patients undergo harsh conditioning before receiving an ex vivo gene therapy treatment, which often has severe side effects and can be fatal. These treatments also require specialized facilities and training and are too expensive to be supported by payers as a routine option. Our EV-based delivery system has the potential to efficiently deliver gene editors safely to the bone marrow following intravenous injection—specifically the long-term HSCs in the bone marrow we all strive to target for durable gene correction. This is big. This precision targeting creates promising new options to treat rare blood diseases and represents a paradigm shift over HSC transplant.”
This SBIR award will enable the company to optimize procedures for loading their proprietary EVs with cargo, further characterize the biodistribution and delivery pattern of cargo-loaded EVs, and verify feasibility of STRM.BIO EVs for in vivo cargo delivery in a pre-clinical model of human genetic hematologic disease. With this grant, the company aims to expand pre-clinical proof-of-concept support for the use of this novel system as a non-viral, in vivo genome editor delivery system for the treatment of inherited hematologic diseases.
Source – PR Newswire