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

May 26, 2020

– Patented methods form the foundation of clinical-stage allogeneic CAR T-cell therapies

– Other applications include allogeneic stem cell-derived therapies and therapeutic gene editing for the treatment of genetic diseases

– Additional patents pending in the U.S. and in other countries

CAMBRIDGE, Mass., May 26, 2020 /PRNewswire/ — Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing therapeutic cell-engineering technologies, today announced that the United States Patent and Trademark Office has granted Factor Bioscience U.S. Patent No. 10,662,410 covering methods for producing a gene-edited cell using mRNA encoding transcription activator-like effector nucleases (TALENs).

The technology covered by this patent has wide-ranging applications in the development of engineered cell therapies and is already being used in the most advanced allogeneic CAR T-cell therapies, which are currently undergoing clinical testing. 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.

“mRNA can be a challenging molecule to work with,” said co-inventor Christopher Rohde, Ph.D., Co-Founder and Chief Technology Officer. “However, we found that when used appropriately, the ability to express proteins transiently, without a risk of insertional mutagenesis, together with the ability to produce a large amount of an encoded protein, can make mRNA an ideal vector for expressing gene-editing proteins such as TALENs. It is incredibly rewarding to see our discoveries in this area being translated into powerful therapies that can improve so many lives.”

“The granting of this patent represents an important milestone in work that began at Factor soon after its founding in 2011,” said co-inventor Matt Angel, Ph.D., Co-Founder, Chairman and CEO. “Our science and our company have come a long way over the last decade, with over 70 patents in 14 countries covering foundational technologies in the fields of mRNA, gene editing, cell reprogramming, and nucleic-acid delivery. We are very pleased to contribute to advancing these fields and to promoting the successful development of therapeutic products.”

Factor’s patent 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. In addition, Factor has been granted three 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.

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.