'Missing link': Bayer, Morningside help catapult a new kind of delivery tech to cell and gene therapy
Robert Millman co-founded and led Semma Therapeutics as CEO while also a managing director of MPM Capital. By the time he left the stem cell therapy pioneer — two years before it would be sold to Vertex — he had left VC life behind.
Instead, he went around scouting new technologies, visiting with tech transfer offices and academics in the Boston/Cambridge area to find worthy ideas that could benefit from his IP expertise.
And he found quite a few.
One of them is Vesigen Therapeutics, which is launching with $28.5 million to turn a new class of extracellular vesicles into packaging and delivery tools for a variety of cargos — mRNA, Cas9, base editors, PROTACs, you name it.
“The barrier to being a drug company right now is not technology or targets,” he told Endpoints News. “There’s plenty of targets, there’s plenty of technology, but there’s no way of getting the two of those into a patient.”
Vesigen’s platform stems from scientific co-founder and Harvard professor Quan Lu’s work on ARMMs, or arrestin domain-containing protein 1 [ARRDC1]-mediated microvesicles. As he wrote in Nature Communications in March 2018, ARMMs are “distinct from exosomes,” in that they lend themselves to more controlled production and cargos that can be recruited into the vesicle during the process of manufacturing rather than put together after the fact.
“I don’t understand really why a whole lot of venture firms didn’t jump on it at the time,” Millman said. “I think it’s because most were focused on exosomes and just thought that this was another exosome company, and there was already Codiak and Evox.”
But Lu showed in his paper delivery of function p53 protein to an p53 deficient animal and restoring p53 activity: “To me it was revolutionary.”
And compared to the current workhorses of genetic medicines such as viral vectors and lipid nanoparticles, ARMMs promise to package bigger and more varied payloads, get into more organs, and stay in circulation longer without triggering toxicity.
Faced with the “Willy Wonka-like” range of possibilities, Millman said the top order right now for his team of seven is to figure out a manufacturing setup that would load the cargos efficiently and be cleared by regulators.
In the three years that it will likely take to get to the cusp of the clinic, Vesigen is also collaborating with researchers to show how ARMMs can get into “tissues that no one can go to” — with implications in ocular and neurological diseases as well as cancer.
The Series A, co-led by Leaps by Bayer and Morningside Ventures and joined by Linden Lake Ventures and Alexandria Venture Investments, should fund the company until 2025, when Millman expects to have built out the team to 30.
It’s exactly the kind of projects that his group likes to invest in early, Leaps by Bayer head Jürgen Eckhardt said, especially as big believers in the cell and gene therapy space who see delivery as the “missing link.” For Vesigen, it also opens up opportunities to find partners within the pharma giant’s network down the road.
While Millman is keen not to lose focus, he also isn’t hiding ambitions to make this “the next big one.”
“I saw all of the problems — Alnylam I was there for three years, we couldn’t get delivery. I worked very early at Celera and established collaborations with Ionis and again delivery was limited. I was helping set up Verve Therapeutics and again delivery of Cas9 was a problem,” he said. “And here was an eloquent solution. I was just pleased to be at the right place at the right time.”