Vertex Pharmaceuticals, a U.S.-based biotech firm, has signed a $945 million deal with Orum Therapeutics, a Korean healthcare company, to use the latter's dual-precision targeted protein degradation technology for gene editing preconditioning, Orum said on Tuesday. 

The deal aims to broaden the therapeutic window of the gene editing treatment and maximize the potential of targeted protein degraders.

Orum Therapeutics is known for its pioneering TPD² technology, the world’s first TPD in ADC format combined with antibodies. The company also developed TPS² (Dual-precision Targeted Protein Stabilization) technology, which involves linking substances inhibiting E3 ligase, crucial for protein degradation, to antibodies. Also, Orum possesses various linker technologies capable of attaching multiple degraders.

The company’s key pipeline products include ORM-5029 and ORM-6151, based on the TPD² technology, aimed at treating breast cancer and acute myeloid leukemia (AML), respectively. Notably, ORM-6151, a candidate for AML treatment, was successfully licensed to Bristol Myers Squibb (BMS) in November last year for $180 million, including an upfront payment of $100 million.

Orum's proprietary dual-precision targeted protein degradation (TPD²) approach combines new targeted protein degraders with antibodies' precise tumor cell delivery mechanism, creating an innovative anti-cancer drug technology that utilizes tumor-selective TPD.

As a result, Orum Therapeutics has been recognized for its competitiveness in the field of degrader-antibody conjugates (DAC), which combine antibodies with TPD.

DACs differ from antibody-drug conjugates (ADC), which link antibodies to anti-cancer drugs. Instead, DACs combine antibodies with TPD to target cancer cells, leading to the degradation of target proteins and inducing tumor cell death.

Under the terms of the agreement, Orum will receive an upfront payment of $15 million and may earn additional option and milestone payments of up to $310 million per target for up to three targets. Orum will also receive tiered royalties on global annual net sales.

The collaboration will utilize Orum's dual-precision targeted protein degradation (TPD²) technology to develop new preconditioning agents for gene editing therapies.

Upon completion of the research period for each target, Vertex will have the option to secure an exclusive global license to develop, manufacture, and commercialize DACs developed using Orum's TPD² technology. This license includes worldwide rights for research, development, manufacturing, and commercialization.

Vertex Pharmaceuticals, known for its innovative treatments for rare diseases, will leverage Orum's TPD² technology. Vertex has a history of breakthrough therapies in rare diseases, such as the cystic fibrosis (CF) treatment market, with a revenue of $9.87 billion last year. Notably, the company received U.S. FDA approval for the world's first gene-edited treatment, Casgevy, a CRISPR/Cas9 genome-edited cell therapy for sickle cell disease (SCD) in patients aged 12 and older with recurrent vaso-occlusive crises (VOCs), in December of last year.

"Vertex is a leader in the discovery and development of innovative medicines, including having the first FDA-approved CRISPR/Cas9 gene-editing therapy," Orum Therapeutics founder and CEO Lee Sung-joo said. "We are pleased they have chosen our TPD² technology to explore new preconditioning agents." This agreement with Vertex opens new possibilities for the company's targeted protein degradation approach to treat patients across new disease areas, Lee added.