Merck has entered into an exclusive licensing agreement with US-based Vertex handing back rights to two DNA-dependent protein kinase (DNA-PK) inhibitors – M9831 (formerly known as VX-984) and an additional preclinical compound – in the field of gene editing for six specific genetic disease indications.

Preclinical studies have shown that DNA-PK inhibitors can enhance CRISPR/Cas9-mediated gene editing. CRISPR/Cas9 is a technology used to modify genetic sequences and is being investigated for the treatment of various genetic disorders.

Under the agreement Merck will retain the right to both compounds in all other disease areas, including oncology, whilst also retaining the rights to utilise and license this technology in gene editing outside of the six specified disease areas.

“This transaction illustrates our determination to maximise value creation from our pipeline,” said Belén Garijo, member of the executive board and chief executive, Healthcare, Merck. “We are rapidly advancing our leading-edge DDR portfolio in oncology and are delighted to see the potential benefit of DNA-PK in genetic diseases through the enhancement of CRISPR/Cas9-mediated gene editing.”

Merck is also currently investigating four DDR molecules, including two ATR inhibitors, an 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.