The Sickle Cell Solution

Editas Medicine and Azzur Cleanrooms on Demand have expanded their multi-year contract to support the scaling of manufacturing for EDIT-301 – a gene editing medicine under investigation for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). SCD is caused by a single mutation in the HBB gene that causes sickling of red blood cells, leading to anemia, hemolysis, and vaso-occlusive episodes. According to Editas, SCD is a strong candidate for a gene editing medicine.

The agreement includes compliant cleanroom space and lab services at Azzur’s site in Devens, Massachusetts. “The Devens Cleanrooms on Demand site will help Azzur’s partners, such as Editas Medicine, accelerate time to clinic and commercialization with on-demand cleanrooms supported by GMP wraparound services and lab services,” said Ravi Samavedam, Chief Innovation Officer at Azzur Group. “These services include materials management, storage, asset management, personnel training, QC testing, and quality management.”

Traditional manufacturing models of build or outsource generally have long lead times, making manufacturing capacity for the emerging field of gene-edited therapies a challenge. Editas is working with Azzur to increase the company’s manufacturing capacity for its RUBY and EdiTHAL trials of EDIT-301, which was developed using the company’s proprietary AsCas12a technology. 

In June, Editas shared positive initial clinical safety and efficacy data from the RUBY trial, which signaled that EDIT-301 has the potential to be a clinically differentiated, one-time, durable medicine. The trial remains on track to dose 20 total SCD patients, and the company plans to provide an additional RUBY clinical update before the year is out.