GC Therapeutics has entered gameplay with the mission of unlocking a new generation of cell therapies, uploading with $75 million for its “plug-and-play” tech developed in the lab of—and with cells from—George Church, Ph.D.
“George donated his skin cells, which were reprogrammed into the stem cells that were used. So, he truly has skin in the game,” GCTx co-founder and Chief Scientific Officer Alex Ng, Ph.D., told Fierce Biotech. “Along with this deep contribution, we're really proud to have his initials on our door. Of course, GC Therapeutics could also mean gene and cell therapeutics, or the letters of DNA.”
The biotech has capped off a $65 million series A financing led by Cormorant Asset Management with participation from Mubadala Capital and Andreessen Horowitz (a16z) Bio + Health, among others. That brings the company’s total capital raised since its 2019 inception to $75 million, according to a Sept. 19 release.
From science fiction to reality
GCTx is based around TFome, a cellular programming platform built to “plug and play”—that is, to deliver off-the-shelf induced pluripotent stem cell (iPSC)-based medicines up to 100 times faster than conventional methods. The single-step process combines synthetic biology, gene editing, cell engineering and machine learning in efforts to boost potency, efficiency and quality, according to the company.
“This approach harnesses the transformative potential of transcription factor biology to guide iPSC cell fate into any differentiated cell type in a single, seamless step,” GCTx co-founder Church said in the company release. “By transcending the limitations of natural processes, it holds the promise to create cells that surpass the capabilities of those found in nature, generating a groundbreaking new class of SuperCell medicines to address diverse disease areas and reduce healthcare burdens.”
Church, a professor of genetics at Harvard Medical School and director of synthetic biology at the Wyss Institute, is a pioneer in genomics and synthetic biology. The scientist is tied to a slew of biotechs, including Nebula Genomics, Ally Therapeutics, Rejuvenate Bio and Manifold Bio.
This time, Church formed GCTx alongside CEO Parastoo Khoshakhlagh, Ph.D., and CSO Ng.
“Professor George Church's lab is the hot spot for people like me,” Khoshakhlagh told Fierce. “He does bring science fiction to reality.”
“The reason that I immediately fell in love with the project … was because of the significant contrast between the current state of the field and what the TFome platform can actually achieve,” the CEO continued. “Traditional cell therapies often take weeks to months to develop, and they typically have low quality and efficiency. Now, in contrast, our technology enables the production of the same cell type in a four-day process—and a single-step process—and with up to 99% efficiency and really high quality.”
TFome is designed to go beyond traditional developmental biology by isolating one single biological process specific to cell fate, a process that is actually governed by the key transcription factors, or what Khoshakhlagh calls “the genetic master knobs,” which act in concert to program cells to become a specialized cell type.
The biotech is shortcutting the entire process, a streamlining effort that also cuts down manufacturing timelines. The approach allows for a more direct and precise control over cell fate, according to Ng.
GCTx has validated its programming approach across diverse cell types both in vitro and in vivo, Ng said in the company's release, adding that the company has also engineered cells into SuperCells to tailor them for specific disease applications.
These SuperCells are designed in two different ways, Khoshakhlagh explained. The first way is by improving existing functions the cells already have, while the second route is introducing new functions that the cell doesn’t typically have. The ultimate goal for SuperCells is having a higher potency and thereby reducing the number of cells a patient needs transplanted, according to the CEO.
GCTx’s initial focus will be on gastrointestinal, neurological and immunological diseases to broaden access to cell therapies.
“These are the disease areas that really we believe that are waiting to be transformed by cell therapy,” Khoshakhlagh said.
While the 35-member team hasn’t yet revealed development timelines for the programs, Khoshakhlagh said the biotech is moving “full speed ahead with a sense of urgency based on the strength of the platform and also the robustness of our preclinical data.”
“We really want to ensure that we are bringing the safest option to patients as soon as we can, because we know that patients are waiting,” the CEO said. “My cousin, who has a progressive form of MS, he literally texts me every week, if not every day … asking ‘When? When are therapies like ours going to be available to patients who have no other options?’ And this is what we are working towards—giving hope to patients with no options.”
'Two unmarriable concepts'
The Cambridge, Massachusetts-based biotech could be the first iPSC-based cell therapy company to pursue multiple cell products from one starting point using a similar manufacturing strategy for more accelerated development.
The platform’s versatility and efficiency have allowed GCTx to build out a rich repertoire of cell types, which subsequently allows the biotech to unlock strategic business development opportunities, according to Khoshakhlagh. The company is open to having “selective partnerships” in biotech and pharma that accelerate certain programs into the clinic, she said.
“GC Therapeutics’ groundbreaking approach to cell therapy has the potential to redefine the industry,” Alaa Halawa, executive director and head of U.S. ventures at Mubadala Capital, said in the company release. “By accelerating the production of iPSC-based therapies, the TFome platform unlocks unprecedented efficiency and scalability, which could lead to faster patient access to life-changing treatments.”
Halawa has joined GCTx’s board, alongside Cormorant Asset Management’s Ray Kelleher, M.D., Ph.D.; a16z’s Vineeta Agarwala, M.D., Ph.D.; Alnylam founding CEO and JMM Innovations CEO John Maraganore, Ph.D.; plus former Karuna Therapeutics CEO and past Eli Lilly exec Steve Paul, M.D.
“Our platform really allows us to re-envision cell therapy manufacturing and marry the two unmarriable concepts, improving our quality while simultaneously increasing cost efficiency,” Khoshakhlagh said. “These are the challenges that we believe have been delaying cell therapy to become a more widespread modality.”
“I can envision a world where we have banks of cell products and no patients are suffering due to lack of accessibility to these types of advanced treatment options,” the CEO concluded. “That's my dream.”