Sarepta Therapeutics, a global biotech company based in Cambridge, Mass, USA, has announced a global licensing agreement with Arrowhead Pharmaceuticals, a company focused on leveraging the development of RNAi technology to target genetic diseases.

Under the terms of the agreement, Sarepta will obtain exclusive global rights to multiple clinical, preclinical, and discovery-stage programmes for rare, genetic diseases of the muscle, central nervous system (CNS) and the lungs. The agreement is intended to broaden Sarepta’s mid- and early-stage pipeline, supporting the company’s existing presence in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and gene therapy, while adding new indications and expanding into adjacent therapeutic areas.

The clinical programmes covered under the agreement will use Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform, which is designed to deliver siRNA to multiple tissue and cell types throughout the body. This aims to initiate the RNA interference mechanism and reduce the expression of target genes. In addition, Sarepta and Arrowhead intend to research up to six additional muscle, cardiac and CNS targets, utilising Arrowhead’s delivery technologies. As part of this joint venture, Sarepta has an exclusive license to Arrowhead’s technology for the development of therapies directed at skeletal muscle gene targets.

Arrowhead is to receive an initial payment of $500 million plus potential milestone payments and royalties to follow, as well as a $325 million equity investment at a 35% premium. Sarepta’s Board of Directors has also independently approved a share repurchase authorisation of up to $500 million.