Vertex Pharmaceuticals and Orum Therapeutics have entered into an agreement worth up to $945m aimed at developing targeted conditioning agents to prepare patients for gene editing.
The deal gives Vertex the rights to conduct research using Orum’s dual-precision targeted protein degradation technology to develop novel degrader-antibody conjugates (DACs).
Vertex will then have the option to obtain a global, exclusive licence to research, develop, manufacture and commercialise the resulting DACs, following the research period for each target.
In exchange, Orum will receive $15m upfront and will be eligible for additional option and milestone payments of up to $310m for each of the three potential targets included under the agreement, as well as tiered royalties on future global net sales.
Sung Joo Lee, Orum’s founder and chief executive officer, described Vertex as a “leader” in discovering and developing innovative medicines, citing the US Food and Drug Administration’s approval its CRISPR Therapeutics-partnered Casgevy (exagamglogene autotemcel) gene therapy in December last year.
The authorisation made Casgevy, which is now indicated to treat certain cases of sickle cell disease and beta thalassaemia, the first CRISPR-based gene-editing therapy to be approved in the US.
Orum’s approach is designed to combine the power of targeted protein degradation with the precision of antibody-drug conjugates technology, while overcoming the limitations of each modality.
“This agreement with Vertex creates the potential to treat patients in a novel indication space with our… targeted protein degradation approach for an exciting new therapeutic class of DACs,” Lee said.
The announcement came on the same day that Genomics said it had extended its collaboration with Vertex to accelerate the discovery and development of precision medicines using human genetics and machine learning.
The partners have been working together since 2018 to support Vertex’s efforts to develop medicines for serious diseases, with the collaboration now set to run until 2026.
Though financial details of the latest deal have not been disclosed, the healthcare company said it will now expand the variety of genomic insights it uses to support its work under the alliance and increase the number of therapeutic areas under study.