Factor Bioscience Granted Second U.S. Patent for mRNA Vectorization of TALENs Technology

November 10, 2020

Factor Bioscience Inc., a Cambridge-based biotechnology company that develops cell-engineering technologies, today announced that the United States Patent and Trademark Office has granted Factor Bioscience U.S. Patent No. 10,829,738 covering methods for producing gene-edited cells using mRNA encoding transcription activator-like effector nucleases (TALENs). This patent is the second Factor has been granted for the mRNA vectorization of TALENs in 2020.

Factor’s patented methods have wide-ranging applications in the development of engineered cell therapies. Both this patent, and its earlier patent, U.S. Patent No. 10,662,410, are already being used in the development of the most advanced allogeneic CAR T-cell therapies. In these therapies, the patented methods are used to inactivate the endogenous T-cell receptor to prevent therapeutic T cells from causing graft-versus-host-disease (GvHD) and to perform other key cell-engineering steps.

Other applications of the patented methods include the generation of allogeneic stem cell-derived therapies in which mRNA encoding TALENs is used to inactivate components of the human leukocyte antigen (HLA) complex to render the cells immuno-nonreactive (i.e., “stealth”) and to insert donor sequences into defined genomic loci to enable controlled expression of exogenous genes. The patented methods enable higher efficiency gene editing, including in primary cells, than other approaches, without relying on viruses or DNA-based vectors that may cause unwanted mutagenesis.

“Nearly ten years ago, we recognized the potential of mRNA to change the way we approach therapeutic development,” said co-inventor Christopher Rohde, Ph.D., Co-Founder, and Chief Technology Officer. “We found that mRNA, when used appropriately, can be an ideal vector for expressing gene-editing proteins such as TALENs. We are so pleased to see our continued discoveries in this area being used to develop life-saving therapies,” he said.

“This second patent covering mRNA vectorization of TALENs provides additional protection—and underscores Factor’s contribution to the development of clinically useful gene-editing technologies,” said co-inventor Matt Angel, Ph.D., Co-Founder, Chairman, and CEO. Factor currently holds more than ninety patents in 14 countries covering foundational technologies in mRNA, gene editing, cell reprogramming, and nucleic-acid delivery. “Our growth is accelerating, and we are hard at work on the development of new technologies,” Matt Angel stated. “We are on track to add more than 40 patents to our portfolio this year.”

Factor’s portfolio includes granted patents covering methods and compositions for reprogramming and gene editing cells, as well as disease-focused technologies for the development of treatments for cancer, HIV, and certain genetic diseases. Factor has also been granted four U.S. patents covering novel chemical substances that are exceptionally effective at delivering nucleic acids, including mRNA, to cells both ex vivo and in vivo. Factor actively licenses its technologies to entities wishing to conduct commercial research, sell tools, reagents, and other products, perform commercial services for third parties, and develop human and veterinary therapeutics.

Factor currently licenses its technology to its affiliate, Novellus Therapeutics, a company headquartered in Cambridge, Massachusetts that is developing engineered cellular medicines to treat a wide range of diseases. Novellus recently exclusively out-licensed its induced mesenchymal stem cells (iMSCs) for the treatment of acute respiratory conditions, including Acute Respiratory Distress Syndrome (“ARDS”) associated with COVID-19.

About Factor Bioscience
Founded in 2011, Factor Bioscience develops technologies for engineering cells to advance the study and treatment of disease. Factor Bioscience is privately held and is headquartered in Cambridge, Massachusetts. For more information, visit www.factorbio.com.

The content of this press release is the sole responsibility of the authors and does not necessarily represent the official views of the USPTO.