Vertex licenses back CRISPR compounds from German Merck

US pharma Vertex has licensed back two gene editing compounds that can be used with CRISPR/Cas9 technology, from Germany’s Merck KGaA.

The German pharma said that it had granted Vertex a licence for one clinical and one preclinical molecule for gene editing applications in six specific genetic disease areas.

Merck bought both molecules in a licensing agreement from Vertex in 2017, and are part of Merck’s portfolio of DNA Damage Response (DDR) inhibitors.

This class of drugs has been shown to enhance CRISPR/Cas9 gene editing, which is already used in genetic engineering and is in the very early stages of development as a therapeutic treatment.

Before Christmas the FDA gave Vertex and the US biotech CRISPR Therapeutics permission to begin trials of a CRISPR/Cas9 therapy, CTX001, for beta-thalassemia and sickle cell disease.

The latest deal covers two DNA-dependent protein kinase (DNA-PK) inhibitors – M9831 (formerly known as VX-984) and an additional pre-clinical compound – in the field of gene editing for six specific genetic disease indications.

Darmstadt-based Merck retains rights to both compounds in all other disease areas, including oncology, and outside of the six specified disease areas.

Merck will receive an upfront payment in addition to milestones and royalties on future net sales and retains the rights to both assets in all other disease areas including oncology, with the ability to develop both these compounds in-house, or to license them to future partners in the gene editing field.

Vertex has the option to add indications to the license grant.

Merck said it is investing in DNA damage response (DDR) and has the objective of becoming one of the leading players in this therapeutic area.

The company is currently investigating four DDR molecules, including two ataxia telangiectasia and rad3-related (ATR) kinase inhibitors, an ataxia telangiectasia mutated kinase (ATM) inhibitor and an investigational small-molecule of DNA-PK.

DNA-PK is a key enzyme that could potentially enhance the efficacy of many commonly used DNA-damaging agents such as radiotherapy and chemotherapy.

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