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Small Biz Grant Funds Non-Viral Gene Editing Delivery

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(Public Domain Pictures, Pixabay)

26 July 2022. National Institutes of Health is financing a delivery method for gene-editing therapies to blood-forming stem cells with natural lipid bubbles instead of viruses. The company STRM.Bio Inc. in Cambridge, Massachusetts is receiving a $2.1 million award from National Center for Advancing Translational Sciences or NCATS, an agency of NIH, for the two-year project.

STRM.Bio is a three year-old biotechnology enterprise developing a delivery mechanism for gene therapies, including gene editors, using tiny lipid or natural oil bubbles secreted from cells called from extracellular vesicles. These excreted vesicles were up to recently considered maintenance vehicles for cells to remove excess or waste proteins and RNA. But because of their ability to interact with other cells, extracellular vesicles have drawn attention for their cellular signaling functions and potential for delivering gene therapies.

Most gene therapies today, including gene editing treatments, are delivered with benign viruses, such as adeno-associated viruses, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, and may generate a mild immune response. In some sick or weakened patients, however, even mild immune responses can be dangerous, particularly if a treatment requires repeat doses. Also, says the company, engineered cell therapies addressing hematopoietic or blood-forming stem cells often require harsh conditioning pre-treatments for the immune system, which can be dangerous as well to patients.

Delivery of gene-editing cargos

STRM.Bio is developing extracellular vesicles into an alternative process for gene therapy delivery to blood forming stem cells. Jonathan Thon, the company’s founder, was formerly a founder and chief scientist for Platelet BioGenesis in Watertown, Mass., a company spun-off from Boston Children’s Hospital and Harvard Medical School that engineered stem cells into blood platelets to perform therapeutic functions other than blood coagulation. Thon says he was one of the researchers that studied the underlying platelet processes licensed by the company. Science & Enterprise reported on Platelet BioGenesis’s second venture funding round in Nov. 2021.

For NCATS, STRM.Bio proposes to demonstrate delivery of therapeutic gene-editing cargos to hematopoietic stem cells or HSCs in bone marrow with engineered extracellular vesicles. The project, says the company, will show how gene editing treatments are loaded into extracellular vesicles, and describe the distribution and delivery patterns of those vehicles. And STRM.Bio plans to verify the feasibility of delivering gene editing therapies with extracellular vesicles, or EVs, in lab mice induced with human blood diseases. The company says the demonstration will show the technology is safe, can find specific targets in bone marrow, and efficiently deliver its gene-editing cargo.

“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,” says Thon in a company statement released through Cision. Thon adds, “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.”

The award is a Small Business Innovation Research or SBIR grant made under NIH’s small business programs that set aside a part of the agency’s research funding for U.S.-based and owned companies. Most SBIR grants are made in two parts: a first phase to determine technical and commercial feasibility, and a second phase to develop and test a working prototype or prepare for clinical trials. This is a second-phase project.

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