Orna Therapeutics, through its wholly-owned subsidiary ReNAgade Therapeutics Inc., announced a three-year strategic research collaboration with Vertex Pharmaceuticals Incorporated to utilise Orna's novel and proprietary LNP delivery solutions to enhance Vertex's efforts in developing next-generation gene editing therapies for patients with SCD and TDT.

Under the terms of the agreement, Orna will receive upfront payments of USD 65 million, including an investment in the form of a convertible note, and is eligible to receive up to USD 635 million based upon the achievement of specified pre-clinical, research, development, regulatory and commercial milestones related to SCD/TDT products.

Additionally, Orna is further eligible to receive up to USD 365 million in additional option fees and milestones per product for up to ten additional products if Vertex options rights in additional indications. Orna will be eligible to receive tiered royalties on future net sales of any products that may result from this collaboration. Vertex is funding the three-year research collaboration and holds an option to extend the research collaboration term.

Commenting on the development, Amit Munshi, Chief Executive Officer of Orna said, “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. 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.”

Ansbert Gadicke, Chairman of Orna and Managing Partner of MPM BioImpact added, “Today's collaboration leveraging Orna's industry-leading non-viral HSC delivery represents our second major partnership and is a testament to the breadth and potential of the company's platform. We look forward to partnering with Vertex to develop transformative therapies for SCD and TDT, while simultaneously advancing our lead panCAR pipeline programs in autoimmune and oncology. This deal further solidifies Orna's leadership in next-generation RNA medicines and has the potential to deliver large-scale impact to patients.”