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Prime Medicine and Cimeio Therapeutics Join Forces for Genetic Therapies Development

Prime Medicine and Cimeio Therapeutics, two renowned biotechnology companies, have announced a research collaboration. Their shared goal is to merge their respective technologies: Prime Medicine's Prime Editing platform and Cimeio's Shielded Cell and Immunotherapy Pairs (SCIP) platform. The goal is to enhance the safety and efficacy of hematopoietic stem cell (HSC) transplants and to introduce new therapeutic possibilities that could extend the utility of HSC transplants and in vivo genetic therapies.

The Aims of the Collaboration

The collaboration aims to reduce the toxicity of conditioning regimens in the treatment of genetic diseases and specific malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Moreover, it aims to enable the in vivo selection of edited HSCs, potentially eliminating the need for transplantation altogether.

HSC transplant offers a potentially curative approach for many debilitating and life-threatening diseases, such as AML and rare genetic diseases like Gaucher's and Hurler's. However, the clinical utility of this approach is currently limited by the necessity for myeloablative conditioning regimens and the risk of post-transplant progression of malignant disease.

Promising Technologies

Cimeio's SCIP platform, when combined with Prime Editing, may significantly improve the accessibility, eligibility, and outcomes for patients who could benefit from HSC transplant. These combined technologies may also enable the selection of in vivo edited HSCs, which could permit the treatment of genetic diseases without a transplant.

Under the terms of the agreement, Prime Medicine will develop a Prime Editor for Cimeio's CD117 shielding variant. CD117 is a receptor tyrosine kinase expressed on normal HSCs and on leukemia cells, making it a promising target for an antibody-based conditioning therapy for patients in need of a stem cell transplant or new treatment options for AML or MDS.

Licensing Terms

If the research collaboration proves successful, the companies will grant each other exclusive license options for their technologies. Prime Medicine will have the exclusive option to license Cimeio's cell shielding technology for CD117-shielded HSC transplant and for in vivo editing of CD117-shielded HSCs for genetic diseases, with the exception of sickle cell disease. On the other hand, Cimeio will have the exclusive option to license Prime Medicine's Prime Editing technology for its CD117-shielded allogeneic HSC product, potentially a second shielding protein for use in AML and MDS. If the companies exercise their exclusive license options, they will each be eligible to receive economics on net sales of licensed products.

Immunotherapies and Prime Editing

Cimeio's proprietary investigational immunotherapies are designed to selectively deplete diseased HSCs, while its novel CD117 protein variant, which can be introduced into HSCs using Prime Editing, could protect the healthy transplanted HSCs from depletion. This allows them to engraft while maintaining the normal function of the CD117 receptor. Prime Editing, due to its versatility, precision, and efficiency, is uniquely suited to introduce these novel protein variants into HSCs.

Potential Impacts

"We believe Prime Editing is an incredibly powerful technology that could impact the care and treatment of a wide range of diseases," said Keith Gottesdiener, M.D., Chief Executive Officer of Prime Medicine. He also expressed optimism about the collaboration with the Cimeio team, who are experts in the fields of cell therapy, gene editing, and HSC transplant. He looks forward to evaluating the synergistic potential of their technologies.

Proof of Concept

In December 2022, at the American Society of Hematology (ASH) Annual Meeting, Cimeio presented results from a preclinical study demonstrating proof-of-concept for its CD117-shielded cells. The data showed that its shielded cells expressing a genetically engineered variant of CD117 were fully functional in vitro and contributed to engraftment in vivo, similar to unmodified HSCs expressing the wild-type receptor.

Unique Shielding Technology

"Our recently disclosed data continues to show that our unique shielding technology and paired immunotherapies have the potential to deliver transformative therapies for patients with many types of diseases," said Thomas Fuchs, Chief Executive Officer of Cimeio Therapeutics. Through this collaboration, the goal is to eliminate the need for toxic chemotherapies and radiation, and enable new therapeutic approaches post-transplant.

About Prime Medicine

Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is leveraging its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated, one-time, potentially curative genetic therapies.

About Cimeio Therapeutics

Cimeio is an applied gene editing and immunotherapy company developing a portfolio of Shielded-Cell & Immunotherapy Pairs™ (SCIP), which has the potential to transform hematopoietic stem cell transplant. Cimeio's technology platform is based on the design and expression of modified variants of naturally occurring cell surface proteins in HSCs. This technology has significant therapeutic potential, which Cimeio is using to develop curative treatments for patients with genetic diseases, hematologic malignancies, and severe autoimmune disorders.

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