Sana Biotechnology has a new $50 million tool in its toolbox.
Sana signed an agreement with Beam Therapeutics for non-exclusive commercial rights to the Cambridge, Mass.-based biotech’s gene editing system, called CRISPR-Cas12b.
Under the terms of the agreement, Sana will pay Beam an upfront payment of $50 million. Beam may also receive additional payments, for instance upon the achievement of development and sales milestones.
“Gene editing technology is a key component in developing engineered cells as medicines, and we are pleased to have the ability to use the Cas12b system as part of a number of our ex vivo engineered cell programs,” said Steve Harr, Sana’s president and CEO in a statement.
Gene editing is central to the Seattle biotech’s therapeutic goals, as stated in its SEC filing prior to the company’s $587 million IPO this February.
“Our long-term aspirations are to be able to control or modify any gene in the body, to replace any cell that is damaged or missing, and to markedly improve access to cellular and gene-based medicines,” stated the company. Sana aims to develop therapeutics for conditions as diverse as cancer, diabetes and cardiovascular disorders.
It’s critical when working with human cells to have the most accurate and specific gene editing technology. The original gene editing system discovered several years ago, CRISPR-Cas9, is plagued with issues such as “off-target” cutting of DNA sequences.
Beam’s gene editing technology is more specific, leading to fewer gene cuts outside of the intended target region. That’s according to a study in Nature Communications led by CRISPR pioneer Feng Zhang at the Broad Institute of MIT and Harvard, which has licensed its technology to Beam.
CRISPR-Cas12b is also more compact: its DNA is shorter and easier to fit into the delivery systems that researchers use to put such gene editors into cells.
A high-quality and specific gene editing system could support a wide range of Sana’s preclinical programs.
SANA is generating therapies from pluripotent stem cells, according to its SEC filing. The company aims to genetically modify the stem cells so that the resulting cell products can “hide” from a patient’s immune system and evade rejection. The stem cells can then be prompted to turn into specific cell types, such as the pancreatic cells damaged in diabetes.
The company also has programs to modify T cells, key immune cells that can be leveraged to fight cancer. Cancer-fighting CAR T cells, for instance, are generated through genetic modification of such cells.
Sana has a program to develop “allogeneic” T cells taken from a donor. The company aims to genetically modify such cells so they can also be hidden from the immune system and used in an unrelated patient.
“The specificity and efficiency of Cas12b make it appealing for Sana’s allogeneic T cell as well as gene-edited pluripotent stem cell programs,” said Harr. “We intend to incorporate this platform into multiple product candidates.”
SANA intends to file its first investigational new drug application incorporating the new gene editing platform as early as next year, said Harr. The application with the U.S. Food and Drug Administration would be a key step towards a clinical trial.