Partnership to ensure supply of EDIT-301 gene-editing therapy

Phase 1/2 RUBY trial of EDIT-301 now recruiting patients in US, Canada

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Editas Medicine has expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, its experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia.

If approved, the scaling up of EDIT-301 needs to follow current good manufacturing practice (cGMP) standards — set to ensure that batches of a medicine are produced with consistent high quality.

“Azzur Cleanrooms on Demand has been a trusted and reliable partner of Editas Medicine for nearly three years, providing us with the dedicated, cGMP-compliant space necessary to support our clinical manufacturing and quality management, which are critical components to making medicines,” Harry Gill, senior vice president of operations at Editas Medicine, said in a company press release.

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Manufacturing of EDIT-301 will take place in Massachusetts facility

This manufacturing of EDIT-301 will be conducted in cleanroom space and labs services at Azzur’s COD site in Devens, Massachusetts. The building is slated to open by spring 2024.

“The Azzur COD model helps our partners accelerate time to clinic and commercialization with on-demand cleanrooms supported by GMP wraparound services, allowing our customers to focus on their science while we focus on compliance,” said Ravi Samavedam, chief innovation officer at the Azzur Group, which includes Azzur COD.

SCD is caused by mutations that lead to the production of an abnormal form of adult hemoglobin, the protein responsible for transporting oxygen in red blood cells. As a result, red blood cells acquire a sickle-like shape and die prematurely, resulting in fewer cells available to carry oxygen throughout the body. These misshapen cells are also more prone to form clumps that obstruct blood flow.

EDIT-301 is an experimental cell therapy that involves the collection of a patient’s hematopoietic stem cells, the cells in the bone marrow that can give rise to mature blood cells. After collection, the cells are genetically engineered to make high levels of fetal hemoglobin. The cells are delivered back to the patient in one single infusion where they are expected to repopulate the body with cells that can compensate for the faulty adult protein.

Fetal hemoglobin is produced during embryonic development. After birth, this form of hemoglobin is replaced by the adult version of the protein, which is not as effective at carrying oxygen.

EDIT-301 is currently being investigated in the Phase 1/2 open-label RUBY trial (NCT04853576). This trial aims to test the safety and effectiveness of a single infusion of the therapy in individuals ages 18-50 who have severe SCD.

With locations across the U.S. and Canada actively recruiting participants, the trial will follow patients for two years after the infusion.

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Promising early data from first two patients treated with EDIT-301

Early data from the first two patients infused with EDIT-301 showed that the therapy engrafted as intended, which means that the transplanted stem cells reached the bone marrow, where they began to make new blood cells. Also, the levels of total hemoglobin have reached the normal range in the first patient. According to the company, the levels of fetal hemoglobin were high enough to stop the red blood cells from sickling.

EDIT-301 was granted orphan drug status and a rare pediatric disease designation for the treatment of both SCD and beta thalassemia, a disease where the body does not make enough hemoglobin.

The EdiTHAL Phase 1/2 trial (NCT05444894) is testing the safety and efficacy of EDIT-301 in patients with beta thalassemia across seven currently-recruiting clinical sites in the U.S.

“As our partnership has grown, Azzur has continued to support us and our evolving needs to manufacture clinical supply to support the advancement of our RUBY trial and EdiTHAL trial of EDIT-301,” Gill said. “The new facility in Devens will further support and enable us to advance our programs through the clinic and towards commercialization as we work to achieve our mission to deliver transformative new medicines to people living with serious diseases.”