Description:
Lipid 10a-26 is an ionizable lipid developed by Orna Therapeutics for lipid nanoparticle (LNP) formulations. It features a biodegradable ester backbone and an ionizable headgroup, enabling efficient encapsulation and delivery of circular RNA (oRNA). Experimental data show that Lipid 10a-26 mediates robust protein expression in hepatocytes and immune cells (e.g., T cells), with strong liver-targeting specificity observed in vivo. Its optimized hydrolysis profile ensures stable oRNA delivery and reduced immunogenicity. For instance, LNPs formulated with Lipid 10a-26 (molar ratio 50:10:38.5:1.5) demonstrate high transfection efficiency in splenic B cells and sustained therapeutic protein production.The lipid’s design balances efficacy and safety, making it ideal for applications like CAR-T therapy and hepatic protein replacement.
Handling:
Providing storage is as stated on the product vial and the vial is kept tightly sealed, the product can be stored for up to 24 months.
Wherever possible, you should prepare and use solutions on the same day. However, if you need to make up stock solutions in advance,
we recommend that you store the solution as aliquots in tightly sealed vials at -20°C. Generally, these will be useable for up to one
month. Before use, and prior to opening the vial we recommend that you allow your product to equilibrate to room temperature for at
least 1 hour.
Reference:
Circular RNA compositions for targeted and efficient therapeutic protein expression in immune cells for treating cancer or autoimmune disease
By: Wesselhoeft, Alex; Goodman, Brian-WO2021189059 A2 2021-09-23