Dive Brief:
- Vertex Pharmaceuticals will work together with Orna Therapeutics over the next three years to develop technology that would help enable “in vivo” gene therapies for sickle cell disease and beta thalassemia.
- Under the deal announced Tuesday, Vertex will pay Orna $65 million upfront, a sum that includes an investment via a convertible note. Orna could receive up to $635 million more in milestone payments related to work involving the two blood diseases, while Vertex holds an option to collaborate in other indications.
- Vertex, which markets the CRISPR therapy Casgevy for sickle cell and beta thalassemia, has been exploring ways to improve on the treatment. In July, the company paid Orum Therapeutics $15 million for rights to technology that could make for more tolerable “preconditioning” before a therapy like Casgevy. Currently, patients are treated with chemotherapy before infusion, which can cause severe side effects.
Dive Insight:
Casgevy’s approval was a major milestone for Vertex and partner CRISPR Therapeutics. The gene editing therapy won Food and Drug Administration clearance at the end of 2023, but has had a slow rollout since due to complexity of treatment, its price and caution around its side effects.
Casgevy is constructed from patient stem cells. Cells are collected, edited in a central laboratory using CRISPR and, once modified to detour around mutations caused by sickle cell or beta thalassemia, re-infused back into each patient. It’s a laborious process that takes several months to complete.
Easier would be an “in vivo” approach, which would involve editing stem cells while they remain inside the body. To do that, Vertex needs to find ways of getting its CRISPR gene editing machinery to the target stem cells.
That’s where Orna could help. Its lipid nanoparticles are designed to reach tissues outside of the liver, where therapies that are infused generally end up.
“We are excited to collaborate with [Vertex] to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to [hematopoietic stem cell]s," said Amit Munshi, Orna’s CEO, in a statement.
If the research partnership is successful, Vertex could be in better position to develop a gene editing option that bypasses both the toxic preconditioning currently used and the time-intensive manufacturing process.
Orna acquired some of its technology through the buyout of startup ReNAgade Therapeutics last year.
Vertex is funding the three-year collaboration with Orna and holds an option to extend the deal.
