Speed—Rapid Manufacturing Cycle and Vein-to-Vein Time
In contrast to other patient-specific cell therapies, such as CAR-T therapies and gene-modified allogeneic cell therapies, the manufacturing of our shielded transplant is a rapid and elegant process that fits into the standard HSC transplant process. The primary reason we can produce a shielded transplant so quickly is the lack of a need for cell expansion. Our approach to creating eHSCs also does not involve the insertion of new genetic material, thereby avoiding complications related to the use of delivery modalities necessary for gene insertion, such as the viral vectors used in CAR-T therapies. The relatively simple and streamlined process of creating our shielded transplant provides significant advantages in the required manufacturing infrastructure, and we are continuing to develop in-house clinical Current Good Manufacturing Practices (cGMP) capabilities to support our planned clinical trials. We believe the efficiency and low capital expenditure of our cell therapy manufacturing process should translate into higher scalability, a lower cost of goods and easy integration into routine transplant practice.
Investment in our own internal cell therapy manufacturing facility
In September 2022, we initiated operations at our new in-house clinical manufacturing facility in Cambridge, Massachusetts to support our development of potentially transformative shielded transplants and CAR-T therapeutic product candidates for patients with blood cancers. The facility will provide us with end-to-end oversight over drug product for our planned clinical trials. With this new facility, our manufacturing teams are seamlessly integrated within our wider organization, a crucial component of our strategy as we continue to enroll our clinical studies. The facility has been designed to support clinical manufacturing for our cell therapy programs, including both shielded transplants and CAR-T therapeutic candidates, and to be cGMP compliant. By integrating our internal research, process development, analytical development, manufacturing, and quality control testing capabilities under one roof, we aim to achieve flexible manufacturing capacity and to reduce the time and cost required to manufacture complex cell therapy clinical product candidates.