Bayer AG is joining forces with Acuitas Therapeutics Inc. Acuitas' LNP technology will support Bayer's in vivo gene editing and protein replacement programs with the goal of specifically delivering RNA payloads to the desired target organ, the liver. LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Acuitas' proprietary LNP technology is used in multiple vaccines and therapeutics in clinical development and was also used in some of the COVID-19 vaccines that were approved and administered to people in 180 countries.

This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells. In addition to mRNA, Acuitas LNP can be used to deliver a range of different nucleic acid therapeutics including small interfering RNA (siRNA), antisense oligonucleotides and DNA. Through the development and option for license agreement, Bayer and its gene therapy focused affiliate Asklepios BioPharmaceutical (AskBio) will gain access to Acuitas' high potency ionizable lipid technology and LNP carriers to develop efficient, targeted, and transient delivery of gene editing RNA components to the liver.

Market-maturity combined with demonstrated manufacturing scalability will drive the development and has the potential to accelerate the path to the clinic of Bayer and AskBio's first in vivo gene editing programs. Financial details were not disclosed. Gene editing is the targeted manipulation of genetic material. It enables a range of edits to the DNA allowing a diverse range of therapeutic applications.

Gene editing can be applied inside and outside the patient to treat a variety of diseases and provide diverse clinical benefits. Gene editing as therapeutic treatment of genetic diseases can be used ex vivo to treat genetic dysfunctions, e.g., sickle cell anemia where a patient's cells are ex vivo modified and afterwards re-administered, or in vivo, where modifications are made directly within the human body. To deliver the different components needed for in vivo gene editing to the right place in the patient's body, transport vehicles, such as LNPs can be used.