Vesigen Therapeutics has raised $28.5 million from groups including Bayer. The series A round sets the Harvard University spinout up to build on technology that could redefine how RNAi, mRNA and CRISPR/Cas9 are delivered. 

In 2018, Harvard University’s Quan Lu co-authored a Nature paper on the use of ARRDC1-mediated microvesicles (ARMMs) to get macromolecules into cells. The paper showed the vehicles can deliver macromolecules including the tumor suppressor p53 protein, RNA and CRISPR-Cas9 into human cells. The macromolecules were biologically active in the cells, enabling them to drive effects such as DNA damage-dependent cell death in mice.

Now, Vesigen, the startup co-founded by Lu, has raised $28.5 million to develop candidates based on ARMMs. Leaps by Bayer and Morningside Ventures led the round with assists from Linden Lake Ventures and Alexandria Venture Investments.

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Vesigen will use the money to build out its platform and take multiple candidates into preclinical and beyond. Further details are yet to emerge. The nature of the technology means Vesigen may have a long list of potential opportunities to choose from.

Lu and his collaborators showed ARMMs can get the p53 protein, RNA and CRISPR-Cas9 into human cells, making the technology applicable to a range of payloads. Vesigen also has plenty of possible targets to go after. More than 80% of validated targets are found inside cells, but issues with existing delivery vehicles including low specificity and high immunogenicity have held back efforts to drug them.

ARMMs are produced endogenously, suggesting immunogenicity may be less of a problem, and can be engineered to present cell-surface molecules that enable targeted delivery to specific cell types. Lu explained some of the options in a statement.

“If you want to deliver a therapeutic to the muscle, this vesicle can be engineered with specific surface molecules to target muscle cells. Injecting locally into the retina might avoid some of the unwanted immune responses and toxicity associated with viral delivery methods,” Lu said. 

The potential of the concept attracted Robert Millman, who co-founded Vesigen and has taken up the CEO role. Millman played similar roles at Semma Therapeutics and CoStim Pharmaceuticals, which were bought by Vertex and Novartis, respectively. 

On LinkedIn, Millman said Vesigen wants to “become the next big player in RNAi, mRNA and editing.” Companies such as Alnylam, Moderna and CRISPR Therapeutics got to those fields first, but Millman thinks Vesigen has “the killer system to deliver what these companies and pharma needs and has wanted to do for years.”



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