Orna Therapeutics develops fully engineered circular RNA (oRNA®) therapeutics and lipid nanoparticle (LNP) delivery systems to enable targeted treatment of diseases beyond the liver, including oncology and autoimmune disorders. Their technology addresses the limitations of traditional mRNA therapies by providing simplified production and enhanced protein expression, facilitating direct treatment options for conditions like sickle cell disease and beta-thalassemia.
Funding
$321.6M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
MKFounders
Product
Problem
Traditional mRNA therapies face limitations in protein expression and delivery, particularly when targeting tissues beyond the liver, which restricts their effectiveness in treating a wide range of diseases. Current cell engineering therapies for genetic diseases often require harsh preconditioning regimens, posing safety risks and limiting patient eligibility.
Solution
Orna Therapeutics is developing a new class of fully engineered circular RNA (oRNA®) therapeutics designed to overcome the limitations of traditional mRNA. Their oRNA technology offers simplified production, improved formulation into lipid nanoparticles (LNPs), and enhanced protein expression, enabling targeted treatment of diseases throughout the body. Orna's LNP delivery systems facilitate treatment of diseases beyond the liver, including oncology, autoimmune disorders, and certain genetic diseases like sickle cell disease and beta-thalassemia directly in the bone marrow. This approach allows for immediate, off-the-shelf treatment options without the need for harsh preconditioning regimens associated with traditional cell engineering therapies.
Target Audience
The primary target audience includes patients with cancer, autoimmune disorders, and genetic diseases, as well as researchers and clinicians seeking advanced RNA-based therapeutics and delivery systems.
Features
- Fully engineered circular RNA (oRNA®) therapeutics with enhanced protein expression
- Industry-leading LNP-based delivery systems for targeted delivery beyond the liver
- panCAR™ in vivo CAR therapies for oncology and autoimmune diseases
- SiTu Editing in the Marrow (STEM) platform for in vivo CRISPR editing
- Repeat dosing capabilities, which is not currently feasible for viral-based delivery approaches
- High-throughput barcoding screening approach in non-human primates (NHPs) to identify LNPs that demonstrated tropism to a rare population of CD34+ HSPCs that reside in the bone marrow