Words by GOLD newsdesk
Orna Therapeutics has entered into a three-year strategic research collaboration with Vertex Pharmaceuticals, which will see the latter leverage Orna's lipid nanoparticle (LNP) delivery technology.
This collaboration is set to enhance Vertex’s development of next-generation gene editing therapies for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
"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," commented Amit Munshi, Chief Executive Officer, Orna.
Hematopoietic stem cells, also known HSCs, are an immature cell that can develop into all types of blood cells, including white blood cells, red blood cell and platelets—making them a crucial target for gene editing therapies designed to address severe blood disorders such as SCD and TDT.
As part of the deal, Orna will receive an initial payment of $65m and has the potential to receive up to $635m upon the achievement of commercial milestones related to preclinical studies, research efforts, development progress and regulatory approvals for SCD and TDT products.
Ansbert Gadicke, Chairman, Orna and Managing Partner, MPM BioImpact, spoke to the broader implications of the partnership, noting that: “This deal further solidifies Orna's leadership in next generation RNA medicines and has the potential to deliver large-scale impact to patients.”
