NEW YORK – Orna Therapeutics on Tuesday said it has inked a deal with Vertex Pharmaceuticals valued at more than $1.07 billion, in which Vertex will use its drug delivery technology to develop new gene-editing treatments for patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Vertex already sells a CRISPR-based treatment developed with CRISPR Therapeutics for SCD and TDT, called Casgevy (exagamglogene autotemcel). Vertex is aiming to further bolster its gene-editing therapy pipeline by using Orna's lipid nanoparticle delivery technologies during their three-year research collaboration. Boston-based Vertex is funding the research collaboration and holds an option to extend its length.
Under the terms of the deal, Vertex will pay Watertown, Massachusetts-based Orna $65 million upfront, which includes making an investment in Orna in the form of a convertible note. Orna is also eligible to receive up to $635 million in preclinical, R&D, regulatory, and potential commercial milestones related to products that arise from this collaboration, as well as $365 million in option fees and milestones for up to 10 additional products if Vertex exercises its options to advance additional indications. Orna will also be eligible to receive tiered royalties on future net sales.
Orna entered into the research collaboration through its wholly owned subsidiary, Renagade Therapeutics, an RNA therapeutics company it acquired last year.
"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 [hematopoietic stem cells]," Orna CEO Amit Munshi said in a statement.