Looking to make donor cells obsolete, an ex-Harvard researcher gets off-the-shelf stem cell biotech off the ground
A stem cell researcher who spent time at the Harvard Stem Cell Institute, the Broad Institute and Brigham and Women’s Hospital launched a new biotech Thursday, and he’s attracted significant blue-chip investment to get things up and running.
Dhvanit Shah unveiled a $72 million Series A for Garuda Therapeutics with the goal of developing off-the-shelf blood stem cell therapies. Shah, who founded Garuda and serves as CEO, told Endpoints News that while he recognizes other companies are researching off-the-shelf cell therapies, Garuda differentiates itself by trying to eliminate the need for donor cells entirely.
“We can’t choose who our donors will be; the age, quality and quantity of cells from another always varies,” Shah told Endpoints. “There’s been nothing new in the last 50 years, and the promise is unmatched here. If someone can come up with a way to make off-the-shelf hematopoietic stem cells, to me that’s the biggest breakthrough in the field.”
Some prominent investors are backing the biotech, with Aisling Capital, Northpond Ventures and OrbiMed leading the Series A.
The field of hematopoietic stem cells has progressed to the point where something like an off-the-shelf therapy is possible, Shah added. He described a steady rise after the Nobel Prize-winning research of Shinya Yamanaka and John Gurdon changed the game in 2006, when the pair showed how mature stem cells could be reprogrammed to be “pluripotent,” or can divide into more stem cells.
It’s essentially a way for pluripotent stem cells to become self-renewing hematopoietic stem cells, Shah said, and it proves the basis of Garuda’s underlying theory. After recruiting a select group of volunteers to help generate a “bank” of pluripotent stem cells, Garuda researchers then convert these cells to the hematopoietic stem cells necessary for its therapies.
And Shah hopes having that bank on hand makes donor cells — and what can be a difficult search to find them — completely unnecessary.
“I’m done with people lining up to be a donor,” he said. “We can do the job once in a lifetime and no one ever has to look back.”
What goes into the technology behind this conversion, or how Garuda picked the most suitable volunteers, Shah isn’t saying. But he claims the biotech has some stellar preclinical data showing the technology works, evidenced by the hefty Series A and blue-chip syndicate.
But he likened the potential of Garuda’s platform to provide broad access to curative treatments to the experimental stem cell transplants given to two HIV-positive individuals. In these instances, the patients were given new cells donated from someone who had a genetic mutation resistant to HIV, allowing them to stop taking antiretroviral drugs and be declared virus-free, per a 2019 Nature article.
Those treatments were unable to be brought into the larger HIV/AIDS population, Shah noted, but when building Garuda’s cell bank, he said the biotech is looking for people whose genetic makeup is suitable for similar therapies. If successful, Garuda will be able to bring stem cell therapies to patients who normally struggle to access them, such as racial and ethnic minorities.
Garuda is developing a pipeline of drugs as well, with initial focuses in hematologic malignancies, sickle cell disease, beta-thalassemia and bone marrow failure diseases. But Shah said the immediate next step is to begin building out manufacturing capabilities to ensure Garuda can meet what he expects will be significant demand.
If everything works out, the biotech expects to launch its first in-human studies within the next two to three years, Shah said. But he cautioned that the estimate is just a ballpark figure.
“I don’t want to overpromise and underdeliver,” he said.
In addition to the lead investors, Thursday’s Series A saw funding from Cormorant Asset Management, Ridgeback Capital Investments, Monashee Investment Management, Sectoral Asset Management, National Resilience, Inc. (Resilience) and Mass General Brigham Ventures, among others.
Clarification: This article has been updated to clarify how pluripotent stem cells become self-renewing.