Vertex partners with Orum for $945M gene editing deal

Vertex Pharmaceuticals has entered into a substantial agreement worth up to $945 million with Orum Therapeutics to leverage Orum's dual-precision targeted protein degradation (TPD²) platform. This collaboration aims to develop conditioning agents that prepare patients for gene editing therapies. As part of the agreement, Vertex will make an initial payment of $15 million. However, Orum stands to receive additional option fees and milestone payments that could accumulate to $310 million for each of the three potential targets. Furthermore, Orum could earn tiered royalties based on future global annual net sales.

Orum Therapeutics, a South Korean biotech firm, will be harnessing its TPD² technology for this collaboration. The TPD² platform is designed to combine antibody specificity with the strong action of targeted small-molecule protein degraders. This innovative approach aims to overcome the challenges associated with conventional protein degraders by enhancing cellular selectivity and expanding the therapeutic window. The technology works by utilizing antibodies to deliver molecular glues to specific cells, where they bind to target proteins and recruit enzymes for protein degradation, ultimately leading to the destruction of cancer cells.

Vertex Pharmaceuticals, renowned for its pioneering work in the development of innovative medicines, including the first FDA-approved CRISPR/Cas9 gene-edited therapy, is expected to conduct research using Orum's technology. Upon the completion of the research phase for each target, Vertex will have the option to acquire worldwide, exclusive licenses for further development and commercialization of the therapies derived from this platform.

Orum's CEO, Sung Joo Lee, expressed optimism about the collaboration, highlighting the potential of their TPD² platform to open new avenues in the treatment of diseases. Lee noted that this partnership with Vertex offers the possibility to address novel indications through Orum's leading targeted protein degradation approach, which is set to pave the way for a new class of degrader-antibody conjugates.

Orum has previously engaged in significant collaborations in the biopharmaceutical field. In November of the previous year, Bristol Myers Squibb acquired Orum's anti-CD33 antibody-enabled GSPT1 degrader, ORM-6151, for an upfront payment of $100 million. The total potential value of this agreement could reach $180 million. This earlier collaboration was focused on developing treatments for acute myeloid leukemia and high-risk myelodysplastic syndromes.

The Vertex-Orum collaboration marks a significant step forward in the field of gene editing therapies and targeted protein degradation. By combining their expertise and innovative technologies, both companies are poised to make substantial advancements in the treatment of serious illnesses, potentially offering new hope to patients through cutting-edge therapeutic approaches.