Key findings
- Cellares is partnering with Stanford Medicine to automate manufacturing and release testing for gene-edited hematopoietic stem cell (HSC) therapies.
- Initial automation work is underway using a “safe harbor knock-in” gene-editing approach, with the goal of supporting HIV and more than 19 rare diseases.
Cellares has entered a collaboration with the Stanford Medicine Center for Definitive and Curative Medicine (CDCM) and the Stanford Innovative Medicines Accelerator (IMA) to automate both manufacturing and release testing (the final quality checks required before a product is cleared for patient use) for gene-edited hematopoietic stem cell (HSC) therapies.
Gene-edited HSCs are being developed as durable, potentially one-time treatments intended to rebuild a patient’s blood and immune system with corrected cells. The collaboration is focused, at least initially, around programs for HIV and rare inherited diseases, which often lack effective treatment options and can be difficult to support at scale if manufacturing is complex or inconsistent.
What the collaboration is building
Under this collaboration, Cellares will:
- Establish a standardized, platform manufacturing process on the Cell Shuttle™ , its automated, end-to-end cell therapy manufacturing system.
- Develop platform release assays on the Cell Q™, its automated in-process and release quality control system.
The goal is to design a platform approach that can be applied across multiple indications, rather than building a bespoke process for each disease program.
Gene-editing automation underway
Cellares says automation efforts are already underway using the HARBOR KNOCK (safe harbor knock-in) gene-editing approach. (In this context, “safe harbor” generally refers to inserting a therapeutic gene into a region of the genome intended to support stable expression without disrupting essential genes.)
Additionally, Matthew Porteus, MD, PhD, Director of the CDCM at Stanford University School of Medicine, and his lab, have developed an HSC gene-editing approach that can be applied across HIV and a wide range of monogenic diseases.
Now partnered together, the groups hope to scale Cellares’ technology to remove barriers and make therapies more cost effective/scalable.
Company snapshot
Cellares is described as the first Integrated Development and Manufacturing Organization (IDMO) and focuses on automated cell therapy manufacturing. The company is headquartered in South San Francisco and operates a commercial-scale facility in Bridgewater, New Jersey, with additional facilities under construction in Europe and Japan.
More information: cellares.com
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