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Start-Up Developing Inside-Cell Delivery, Gains $28.5M

Vesicles

(Needpix.com)

22 July 2020. A new company spun-off from Harvard University is developing techniques for delivering advanced genetic and protein therapies directly inside cells. Vesigen Therapeutics in Cambridge, Massachusetts, is licensing research from the environmental genetics lab of Quan Lu at Harvard’s public health school, the company’s scientific founder, and raising $28.5 million in its first venture funding round.

Lu’s lab studies basic cell signaling processes, with their research leading to discoveries that show signals between neighboring cells and tissues are packaged and delivered through cell membranes in tiny containers called vesicles. These vesicles are produced with a protein called arrestin domain containing protein 1 or ARRDC1 that enables formation of the tiny containers, thus the researchers call them ARRDC1-mediated microvesicles, or ARMMs. Further research by the lab shows ARMMs are a key mechanism in the spread of viruses such as Ebola and HIV between cells, and can be engineered to deliver macromolecules like therapeutic proteins, Crispr-edited genes, and RNA molecules.

Current methods for gene therapy delivery, such as adeno-associated viruses and lipid nanoparticles, say the researchers, often degrade when crossing from one cell to another. ARMMs, according to a 2018 paper by Lu and colleagues in Nature Communications, can deliver macromolecules and retain their therapeutic potency.

“We can harness the capability of these vesicles,” says Lu in a university statement, “to first package and then deliver therapeutic cargoes to the targeted tissues. That’s the ultimate goal of this, to enable next-generation therapeutics to reach their full potential in combating a wide range of diseases.”

“Our mission,” adds Robert Millman, Vesigen’s co-founder and CEO in a company statement, “is to realize the therapeutic potential of many of the new modalities, such as RNA interference, mRNA replacement, and gene editing, that have been used to identify and validate targets, but have proven difficult to translate into functional therapeutics due to delivery barriers.”

Vesigen Therapeutics plans to develop ARMM-delivered therapies for conditions where delivery inside cells is difficult or impossible, including neurological disorders, cancer, and eye diseases. “Injecting locally into the retina,” notes Lu, “might avoid some of the unwanted immune responses and toxicity associated with viral delivery methods.”

The company is raising $28.5 million in its first venture funding round, led by Leaps by Bayer, the venture investment arm of drug maker Bayer, and Morningside Ventures, a technology and life science investment company. Morningside Ventures, in Boston and Shanghai, is the venture arm of the Chan family that endowed Harvard’s public health school, where Lu is a faculty member. Linden Lake Ventures and Alexandria Venture Investments also took part in the funding round.

Vesigen says it plans to apply the proceeds from the round to building the ARMM technology and advancing a number of therapies into preclinical and clinical stages.

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