CAMBRIDGE, Mass.--(BUSINESS WIRE)--GC Therapeutics, Inc. (GCTx), announced its launch today to scale and unlock the next generation of cell therapy. Invented and developed by a world-class team of scientists in the lab of professor George Church, Ph.D., at Harvard Medical School and the Wyss Institute, GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™ (Transcription-Factor-ome, pronounced as tee-eff-ome), integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx completed a $65 million Series A financing led by Cormorant Asset Management with participation from Mubadala Capital, Andreessen Horowitz (a16z) Bio + Health, Medical Excellence Capital, Cercano Management and Pear VC, bringing total capital raised by GCTx to $75 million.
“Cell therapies have revolutionized the treatment of certain disease areas; however, challenges in scaling these therapies and generating high quality products limit their full potential and patient accessibility,” said Parastoo Khoshakhlagh, Ph.D., co-founder and chief executive officer of GCTx. “Our novel cell programming approach enables the rapid and efficient production of fully optimized iPSC-based cell therapies through a streamlined, single-step process. This method enhances potency, efficiency, quality and cost-effectiveness compared to conventional approaches. We are excited to launch GCTx to broaden the accessibility of cell therapy and advance our pipeline of potential best-in-class treatments in various therapeutic areas starting with gastrointestinal, neurological and immunological diseases.”
“GCTx’s cell programming platform represents a major scientific advancement,” said George Church, Ph.D., professor of genetics at Harvard Medical School, core faculty member and synthetic biology lead at the Wyss Institute, and co-founder and senior advisor of GCTx. “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. 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.”
TFome: The world’s first “plug-and-play” iPSC cellular programming platform
iPSC differentiation into distinct cell types depends on various signaling pathways that ultimately converge onto transcription factors (TFs), which control the sets of genes that are active in a cell. Cell fate is determined by specific TFs acting in concert.
GCTx has built the world’s first complete collection of human TFs. Through sophisticated multidimensional screening modalities that integrate genome-scale experimental testing and powerful next-generation machine learning, TFome can identify fully optimized TF combinations to differentiate iPSCs into virtually any functional cell type. This allows for a single-step, four-day stem cell differentiation process with >90% efficiency with no requirement for cell type-specific optimization for microenvironmental factors. This has not been achieved previously via conventional small-scale testing.
Through TF-based cell fate programming, TFome is designed to yield first-in-class and best-in-class off-the-shelf cell therapy products up to 100 times more rapidly than conventional methods of cell differentiation with improved potency, efficiency, quality and cost (SuperCells™). GCTx is positioned to be the first company in the iPSC-based cell therapy space to pursue multiple cell products from one starting point using a similar manufacturing strategy for more accelerated development and timelines.
“Unlike conventional iPSC approaches, TFome goes beyond traditional developmental biology by pushing the limits of the principle that cell identity is determined by the active gene regulatory networks governed by TFs. This approach enables a more direct and precise control over cell fate, thereby setting a new standard in cell programming,” said Alex Ng, Ph.D., co-founder and chief scientific officer of GCTx. “We have validated our single-step cell fate programming approach across diverse cell types both in vitro and in vivo and have further engineered cells into SuperCells to tailor them for specific disease applications. This programming-centric paradigm achieves scalability, cost-effectiveness and streamlined clinical development while maintaining quality, safety and efficacy.”
Originating from professor Church’s lab, Harvard’s Blavatnik Biomedical Accelerator (BBA) supported the project to advance the research toward commercialization opportunities. Harvard’s Office of Technology Development (OTD) protected the intellectual property associated with the founding research and licensed the technology exclusively to GCTx for further development.
Building a leading, fully integrated cell therapy company with multiple pipeline programs advancing toward the clinic
GCTx is committed to leading the field with first-in-class and best-in-class off-the-shelf cell therapies targeting gastrointestinal, neurological and immunological diseases. The versatility and efficiency of TFome also enables GCTx to broaden its reach beyond its core pipeline areas, unlocking strategic business development opportunities and harnessing the expansive capabilities of the company’s portfolio.
“GC Therapeutics’ groundbreaking approach to cell therapy has the potential to redefine the industry. 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. We are proud to partner with the GCTx team as they push the boundaries of what is possible in the development of next-generation cell therapies,” said Alaa Halawa, executive director and head of US Ventures at Mubadala Capital.
Assembled world-class team of thought leaders in biopharma industry
GCTx co-founders
- Parastoo Khoshakhlagh, Ph.D., chief executive officer
- Alex Ng, Ph.D., chief scientific officer
- Cory Smith, Ph.D., scientific co-founder and head of discovery platforms
- George Church, Ph.D., professor of genetics at Harvard Medical School and core faculty member and synthetic biology lead at the Wyss Institute
GCTx board of directors and advisers
- Ray Kelleher, M.D., Ph.D., managing director, Cormorant Asset Management
- Vineeta Agarwala, M.D., Ph.D., general partner, a16z Bio + Health
- Alaa Halawa, MBA, executive director and head of U.S. ventures, Mubadala Capital
- John Maraganore, Ph.D., CEO and principal, JMM Innovations, LLC, founding CEO, Alnylam Pharmaceuticals, Inc.
- Steve Paul, M.D., former CEO, Karuna Therapeutics, former EVP, Eli Lilly and Company, co-founder and board chair, Rapport Therapeutics and Seaport Therapeutics
“GCTx’s TFome platform represents one of the most innovative approaches in the cell therapy space and has the potential to yield a revolutionary class of new SuperCell medicines that are tailored for specific patients and diseases,” said Ray Kelleher, M.D., Ph.D., managing director, Cormorant Asset Management. “We are thrilled to support this exceptional team of scientists and company builders as we work to create the industry’s next leading cell therapy company.”
About GC Therapeutics
GC Therapeutics (GCTx) is a biopharmaceutical company created to scale and unlock the next generation of cell therapy-based medicines. GCTx’s induced pluripotent stem cell (iPSC) programming platform, TFome™ (tee-eff-ome), integrates the latest advancements in synthetic biology, gene editing, cell engineering and machine learning to overcome the therapeutic development and scaling complexities associated with cell therapy and improve patient access across a broad range of disease areas. GCTx’s initial focus is on developing first-in-class and best-in-class cell therapies for gastrointestinal, neurological and immunological diseases. Founded in 2019, GCTx is based in Cambridge, MA and has raised $75 million to date from leading life sciences investors. For more information, please visit us at www.gc-tx.com and follow us on LinkedIn and X.
