It’s not enough for a cell or gene therapy to reach a cell, it must also get inside it to access most of the validated drug targets. Vesigen Therapeutics is developing technology that could best other approaches at penetrating the cellular membrane, and it launched on Wednesday with $28.5 million to advance its research.
The Series A financing was led by Leaps by Bayer and Morningside Ventures. Linden Lake Venture Capital and Alexandria Venture Investments also invested in Cambridge, MA-based Vesigen, which licensed its technology from Harvard University.
Biologic drugs are large molecules that need help crossing a cell’s membrane. Some large molecule drugs currently available, and many still in development, use engineered viruses or lipid nanoparticles to deliver their therapeutic payloads. These medicines rely on endocytosis, the cellular mechanism for absorbing external substances.
While this approach works, Harvard says that most of what enters in this manner goes to the lysosome, cellular machinery that breaks down old or worn out parts for reuse. Quan Lu, a professor of environmental genetics and physiology at the Harvard T.H. Chan School of Public Health, and his team discovered another cellular component that could be used for drug delivery. Tiny particles called extracellular vesicles carry material into and out of cells. Lu’s team focused on one particular type of extracellular vesicle called arrestin domain containing protein 1-mediated microvesicles (ARMMS). Though the initial research centered on how cells use ARMMS to communicate, Lu saw their potential for drug delivery.
Using ARMMS to bring therapeutic molecules into a cell offers advantages over viruses or lipid nanoparticles, according to Harvard research published in 2018 in the journal Nature Communications. These particles are already found in humans so they’re less likely to prompt an immune response or pose toxicity problems. Once an ARMMS particle reaches its target, the researchers say it’s taken up by a cell in a way that avoids the lysosome. And an ARMMS particle can be engineered with peptides and antibodies on its surface enabling targeted delivery to specific tissues or cells.
The Harvard scientists evaluated ARMMS as way to deliver a number of different types of drugs including RNA, protein, and gene-editing therapies. In lab tests and in animals, they found that ARMMS could be used to package and deliver these molecular payloads into cells.
“If you want to deliver a therapeutic to the muscle, this vesicle can be engineered with specific surface molecules to target muscle cells,” Lu said in a statement from Harvard. In the case of eye disease, he said “injecting locally into the retina might avoid some of the unwanted immune responses and toxicity associated with viral delivery methods.”
Vesigen isn’t the only company tapping into cellular particles for drug delivery. Cambridge-based Codiak Biosciences focuses its research on extracellular vesicles called exosomes to try to address “undruggable” targets. The biotech recently inked a research partnership with Sarepta Therapeutics (NASDAQ: SRPT), which is interested in using Codiak exosomes to deliver genetic medicines into muscles as a way of addressing inherited muscular disorders.
Lu’s early research received support from Harvard’s Blavatnik Biomedical Accelerator. Vesigen has an exclusive license to the ARMMS technology. The company is led by co-founder and CEO Robert Millman, a former executive at Semma Therapeutics and CoStim Pharmaceuticals. Lu is staying at Harvard but will serve on Vesigen’s scientific advisory board. The company says it will use its new capital to further develop the technology and advance “numerous therapeutic agents” into preclinical and clinical development. Vesigen did not disclose which diseases it is researching.
With the financing, Morningside Ventures’ Gerald Chan (the son of T.H. Chan, the namesake of Harvard’s school of public health) has been named chairman of the startup’s board of directors. Stephen Bruso, also of Morningside, and Juergen Eckhardt and Jak Knowles of Leaps by Bayer are also joining the Vesigen board.
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