Sarepta Acquires RNA Drugs for $500 Million

Sarepta Therapeutics has inked an exclusive licensing and collaboration agreement with Arrowhead Pharmaceuticals under which Sarepta will obtain exclusive global rights to multiple clinical, preclinical, and discovery-stage programs for rare, genetic diseases of the muscle, central nervous system (CNS), and lungs. The agreement will add meaningfully to Sarepta’s mid- and early-stage pipeline, complementing the Company’s existing leadership in Duchenne muscular dystrophy and gene therapy, while adding new indications and expanding into adjacent therapeutic areas.

The clinical programs use Arrowhead’s proprietary Targeted RNAi Molecule platform, which is designed to deliver siRNA to multiple tissue and cell types throughout the body to initiate the RNA interference mechanism and induce rapid and durable knockdown of target genes. The preclinical programs covered under the agreement will leverage Arrowhead’s CNS delivery platform designed for subcutaneous administration.

Additionally, Sarepta and Arrowhead have entered into a discovery collaboration for up to six additional muscle, cardiac, and/or CNS targets, using Arrowhead’s novel delivery technologies. As part of the collaboration, Sarepta has an exclusive license to Arrowhead’s technology to develop therapeutics against a broad range of skeletal muscle gene targets.

According to Sarepta President and CEO Doug Ingram, “…this broad siRNA collaboration with Arrowhead provides a synergistic platform to complement Sarepta’s gene therapy and gene editing engine. Through a strategic deployment of capital, we are able to access Arrowhead’s leading RNAi platform and will work to rapidly advance new treatments for devastating genetic diseases where there is significant unmet need. The agreement affords multiple potential blockbuster opportunities, serves our strategic priorities for the remainder of the decade and beyond, and diversifies our business model across one-time therapies and chronic treatments allowing for long-term growth and success.” (Source: Sarepta Website, 26 November, 2024)