Orna Therapeutics Enters Strategic Collaboration with Vertex for Next-Gen Gene Therapies for Rare Blood Diseases

Rare Daily Staff
Orna Therapeutics’ ReNAgade Therapeutics subsidiary entered into a strategic research collaboration with Vertex Pharmaceuticals to use Orna’s lipid nanoparticle delivery solutions to enhance Vertex’s efforts in developing next-generation gene editing therapies for patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The goal is to develop an in vivo alternative to existing SCD and TDT gene therapies, which today require taking cells from a patient and genetically modifying them before returning them to the body. This complex process requires the use of chemotherapy to precondition patients and can cause a variety of side effects that can be difficult for patients to tolerate.
Under the terms of the agreement, Orna will receive upfront payments of $65 million, including an investment in the form of a convertible note, and is eligible to receive up to $635 million based upon the achievement of specified pre-clinical, research, development, regulatory, and commercial milestones related to SCD/TDT products.
If Vertex options rights in additional indications, Orna is also eligible to receive up to $365 million in additional option fees and milestones per product for up to ten additional products. Orna will receive tiered royalties on future net sales of any products that may result from the collaboration.
Vertex is funding the three-year research collaboration and holds an option to extend the research collaboration term.
“Vertex is a leader in delivering next-generation approaches to treating hemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to HSCs,” said Amit Munshi, CEO of Orna. “Today’s collaboration further validates our industry-leading extra-hepatic LNP delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines.”