Brooklyn ImmunoTherapeutics LLC (NYSE American: BTX) ("Brooklyn") today announced it has acquired an exclusive license for mRNA gene editing and cell therapies technology of Factor Bioscience Limited and Novellus Therapeutics Limited pursuant to an exercise of a previously announced option.
The license includes use of an extensively patented process to develop gene editing compounds using mRNA, which preclinical data suggest demonstrate a high degree of efficiency, as well as being non-immunogenic and non-mutagenic.
The licensed platform includes mRNA cell reprogramming, which is considered to be the highest efficiency and footprint-free technology that can be applied to both allogeneic and autologous cells, and is combined with mRNA-based gene editing – along with a proprietary gene editing protein – to eliminate off-target effects. It also includes the proprietary ToRNAdo lipid delivery system that provides efficient delivery of mRNA ex vivo and in vivo to skin, brain, eye and lung tissue.
"As a result of the license acquisition, Brooklyn is now poised to become a key player among companies exploring gene editing for cell therapies. This mRNA gene editing technology has the potential to be disruptive given its high efficiency and relatively low manufacturing costs. We look forward to continuing the work begun by Factor Bioscience and Novellus with this platform and to reporting our progress as our products advance towards the clinic," commented Howard J. Federoff, M.D., Ph.D., Chief Executive Officer and President of Brooklyn. "Our licensing of this technology represents a significant advancement in our approach to treating cancers, blood and other disorders, and transforms us from a single therapeutic company with multiple potential indications to a platform company with multiple products in a pipeline of next-generation engineered cellular medicines."
Brooklyn intends to initiate pre-clinical development programs for sickle cell anemia, other inherited monogenic disorders, and solid and liquid tumors, with the intention of being at the Investigational New Drug (IND) stage for at least one of these disorders by 2024.
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