Vertex Pharmaceuticals and Orna Therapeutics have announced a three-year collaboration to develop next-generation gene therapies for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The partnership will use Orna’s lipid nanoparticle delivery technology to improve the efficiency of gene editing, potentially addressing limitations in existing therapies.
Vertex will pay Orna $65m upfront, partly through a convertible note, and Orna is eligible for up to $635m in milestone payments for SCD and TDT products. Additionally, Orna could receive up to $365m per product for up to ten additional indications, alongside royalties on future sales.
Vertex already holds a significant position in the gene therapy landscape. In late 2023, it secured US Food and Drug Administration (FDA) approval for its CRISPR-based therapy Casgevy (exa-cel) for both SCD and TDT, marking a milestone as the first approved gene therapy for these indications. Shortly after, bluebird bio received approval for its competing therapy, Lyfenia (lovo-cel).
Casgevy involves extracting a patient’s stem cells, editing them in a laboratory using CRISPR technology to correct genetic mutations, and re-infusing the modified cells back into the patient. The groundbreaking yet resource-intensive process requires months to complete and necessitates chemotherapy preconditioning, which can lead to severe side effects.
Vertex is actively exploring ways to improve on Casgevy. In July 2024, it invested $15m in South Korea’s Orum to acquire technology aimed at making preconditioning more tolerable. However, the goal is to transition to “in vivo” therapies, where genetic editing occurs directly within the body. Achieving this requires effective delivery systems to target stem cells, a challenge that Orna’s lipid nanoparticle technology could address.
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By GlobalData
“Vertex is a leader in delivering next-generation approaches to treating haemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to haematopoietic stem cells,” said Orna’s CEO Amit Munshi in the 7 January announcement.
Acquiring ReNAgade Therapeutics last year, Orna Therapeutics has been advancing its platform through significant partnerships and funding. In 2022, it raised $221m in a Series B round and partnered with MSD on its circular RNA (oRNA) platform, a technology aimed at improving the performance of mRNA-based therapies.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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