Cell therapy weekly: nanoparticle platform for targeted gene therapy
This week: Syntax Bio (IL, USA) announced the expansion of its Series A funding round, VectorBuilder (IL, USA) and MaxCyte (MD, USA) announced a strategic partnership to develop a novel gene delivery system for ex vivo cell engineering, and Venture capital firm Hatch BioFund (PA, USA) invested in Optimeos Life Sciences, a Princeton University spinout developing a proprietary nanoparticle platform for the intracellular delivery of therapeutic macromolecules.
The news highlights:
Syntax Bio expands Series A funding
Syntax Bio announced the expansion of its Series A funding round to further support the advancement of the company’s Cellgorithm™ platform and fund preclinical proof-of-concept for a beta cell therapy for type 1 diabetes. The additional investment increases Syntax Bio’s Series A round to US$14.4 million and pushes the company’s overall capital raised to over US$25 million.
CEO John Craighead said: “We are pleased to expand our Series A with strong support from both new and existing investors. This financing enables us to accelerate sequential, endogenous gene activation technology, advance the first cell therapy discovered using the Cellgorithm platform and build collaborations to develop scalable regenerative medicines for serious diseases.”
The Cellgorithm platform uses a CRISPR-based system that uses programmable DNA instructions to automate stem cell differentiation by triggering genes in the correct developmental sequence, mimicking natural human development to produce specific cell types consistently and rapidly.
Partnership to advance clinical-grade cell engineering
VectorBuilder and MaxCyte announced a strategic partnership to develop a novel electroporation-based gene delivery system for ex vivo cell engineering. The system will integrate VectorBuilder’s novel miniaturized plasmid backbone, MiniVec, with MaxCyte’s advanced Flow Electroporation® technology.
Preliminary data in CAR-T manufacturing has shown a 2.4-fold improvement in cell viability and a 1.4-fold improvement in gene expression compared to conventional systems.
“Cell therapy development requires delivering therapies that are manufacturable, scalable and commercially viable,” said Maher Masoud, President and CEO of MaxCyte. “By combining MaxCyte’s Flow Electroporation® technology with VectorBuilder’s MiniVec platform, we believe we can enable a new standard for non-viral gene delivery — one that enhances cell quality, improves manufacturing efficiency, and provides developers with a more streamlined path from research through commercialization.”
Nanoparticle platform for targeted gene therapy
Venture capital firm Hatch BioFund has invested in Optimeos Life Sciences, a Princeton University spinout developing a proprietary nanoparticle platform for the intracellular delivery of therapeutic macromolecules.
Optimeos’ delivery platform utilizes a patented method called Coated Inverse Nanocarriers (CINCs), which originated in Princeton University Professor Robert Prud’homme’s laboratory. The platform offers robust and scalable encapsulation of diverse payloads, including biologics, peptides, RNA, and DNA, achieving encapsulation efficiencies above 90 percent. The technology provides modular tissue targeting capabilities, adjustable immunogenicity, and enhanced dosing characteristics. These features have been demonstrated through the company’s primary program — a gene replacement therapy targeting the rare disease CTLN1 — along with additional pipeline initiatives focused on T cell engineering and pulmonary applications.
“Optimeos was founded on the conviction that the next great wave of medicine — RNA, DNA, and biologic therapies — will only reach its potential if we can deliver these molecules precisely, efficiently, and durably to the right cells and tissues,” said Shahram Hejazi, co-founder and CEO of Optimeos. “We are thrilled to have Hatch join us on our mission to address one of the most consequential bottlenecks in modern drug development: how to deliver complex therapeutic molecules safely, precisely, and at scale.”