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Vertex and Orna Partner to Develop In Vivo Gene Therapies for Blood Disorders

NextEdge Admin

08 Jan 2025

2 min 20 sec

Orna Therapeutics,sickle cell disease,beta thalassemia,CRISPR

Vertex Pharmaceuticals and Orna Therapeutics have partnered on a three-year collaboration to advance “in vivo” gene therapies for sickle cell disease and beta thalassemia. Under the agreement, Vertex will pay Orna $65 million upfront, including an investment via a convertible note, with potential milestone payments reaching $635 million. Vertex also retains an option to expand the collaboration to other indications.

Vertex currently markets the CRISPR-based therapy Casgevy for sickle cell and beta thalassemia but is seeking improvements. The treatment requires extracting a patient’s stem cells, modifying them in a lab using CRISPR, and reinfusing them—a complex, months-long process. Patients also undergo chemotherapy-based preconditioning, which carries significant side effects.

An “in vivo” approach, which edits stem cells directly within the body, could simplify treatment. To achieve this, Vertex needs an efficient delivery system for its gene-editing tools. Orna’s proprietary lipid nanoparticle technology is designed to reach hematopoietic stem cells beyond the liver, making it a promising solution.

“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.

A successful partnership could lead to a more accessible and less toxic gene editing therapy by eliminating chemotherapy preconditioning and the labor-intensive manufacturing process. Orna strengthened its technology through the acquisition of ReNAgade Therapeutics. Vertex has the option to extend the collaboration beyond the initial three years.