Bringing precision medicine to two neurodegenerative diseases
CEO: Kasper Roet, Ph.D.
Based: Cambridge, Massachusetts
Clinical focus: Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, and frontotemporal dementia (FTD).
The scoop: After winning a Pfizer "golden ticket" for a slot at LabCentral in Cambridge, Massachusetts—and a spot in Johnson & Johnson's JLABS incubator—QurAlis came onto the scene in May 2020, raising a $42 million series A in the midst of a global pandemic, and two years after its initial seed funding.
Just two months after that series A, it penned an in-licensing deal with Eli Lilly, for an undisclosed number of preclinical compounds with “disease modifying potential” in ALS, working by preventing disease-induced neuronal excitotoxicity. In a COVID world, raising a healthy series A and penning a deal with a Big Pharma, itself heavily wrapped up in pandemic work, is no small feat.
QurAlis’s chief executive, Kasper Roet, Ph.D., likens where we are now with ALS to the understanding we have developed in oncology. “As a neuroscientist, I’m always jealous of the oncology field because there is so much money being pumped into it, and for good reason, but I think oncology [research budgets] is an indicator of where neuro is going to go to,” Roet said. “I think going after ALS make sense because oncology shows us precision therapies are the way to go.”
ALS can be caused by mutations in over 25 individual human genes, many of which also cause FTD. QurAlis’s strategy is to “systematically investigate treatments targeting specific disease-causing mechanisms in patient sub-populations.”
It aims to work on a “wide range” of potential treatments through the company’s platform, which uses lab-grown neuronal networks derived from cells of ALS patients.
What makes QurAlis fierce: There are currently four drugs approved by the U.S. FDA to treat ALS: Radicava, Rilutek, Tiglutik and Nuedexta, with Radicava's 2017 FDA approval the most recent. But none of these drugs are curative, and all are limited in who they can help and by how much.
QurAlis wants to move beyond that. “ALS research been a minefield in the past,” said Roet, “ but that’s because we didn’t really understand what we were doing. We were trying to throw things up hill and just hoping something would hit.”
But things are different now: “We’re really starting to understand this disease, and we are seeing there are different ALS patients and groups of patients, and these can be very different from other groups. What we now know is, these all each require a precision medicine approach.”
These groups fall into three categories: motor system hyperactivity, toxic protein aggregates and cellular waste clearance.
QurAlis aims to zero in on precision treatments by using the understanding of genetics in ALS, and new stem cell technology from Harvard University’s Kevin Eggan, Ph.D., who made the first stem cells from ALS patients and turned those into motor neurons.
“That for me was the reason to start the company,” said Roet, “because we had the genetics giving us the understanding of the different groups that exist, but we couldn’t bridge those discoveries to the clinic until the discovery of this stem cell technology.”
Roet worked on motor neuron projects with the labs of Clifford Woolf, M.D., Ph.D., co-director of the neuroscience program of the Harvard Stem Cell Institute, and Eggan, professor of stem cell and regenerative biology, who both helped found QurAlis, along with Roet and Jonathan Fleming, CEO of Eggan’s stem cell-focused Q-State Biosciences.
Roet said animal models have been “horrible” in both neuro and ALS, but the stem cell tech offers a new tool to actually make neurons and use them at a larger scale to validate ALS targets, and also to select molecules. “We can now see these translate into the clinic,” Roet said.
Investors: $42 million series A in May 2020 led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund; and co-led by Droia Ventures, with new investors including Mitsui Global Investment and Dolby Family Ventures, joined by investments from existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences.