CRISPR and Sirius Therapeutics Partner on siRNA Therapy Development

CRISPR Therapeutics, a pioneering biopharmaceutical company, has announced a strategic partnership with Sirius Therapeutics to advance the development and commercialization of innovative small interfering RNA (siRNA) therapies. This collaboration focuses on the co-development of SRSD107, a promising next-generation, long-acting siRNA targeted at Factor XI (FXI) for treating thromboembolic disorders.

SRSD107 is designed to selectively inhibit FXI, which plays a crucial role in pathological thrombosis, while having minimal impact on normal blood clotting processes. The Phase 1 clinical trials for SRSD107 have shown impressive results, with the therapy demonstrating over 93% reductions in FXI activity and more than a twofold increase in activated partial thromboplastin time (aPTT), maintaining its efficacy for up to six months after administration. These findings suggest that SRSD107 could become a leading FXI inhibitor, offering infrequent dosing and improved patient outcomes.

The collaboration between CRISPR Therapeutics and Sirius Therapeutics is structured under a 50-50 cost and profit-sharing agreement. CRISPR will make an upfront payment of $25 million in cash and $70 million in equity to Sirius. Additionally, CRISPR Therapeutics has the option to license up to two more siRNA programs, expanding its therapeutic capabilities beyond gene-editing technologies already under clinical development.

The alliance between the two companies seeks to address the significant unmet medical need in treating thrombotic diseases, which affect millions globally. Many patients at risk of thromboembolic events are inadequately treated due to concerns over bleeding risks and compliance with current therapies. SRSD107 aims to offer a safer alternative with fewer bleeding risks and better compliance due to its infrequent dosing schedule.

The therapeutic potential of SRSD107 extends to individuals with conditions such as atrial fibrillation, venous thromboembolism, cancer-associated thrombosis, and chronic cardiovascular diseases. The ongoing clinical program also includes a Phase 2 trial that will test the safety and efficacy of SRSD107 in preventing venous thromboembolism in patients undergoing total knee arthroplasty.

Sirius Therapeutics, a clinical-stage biotech company, is focused on developing groundbreaking siRNA therapies for cardiovascular and cerebrovascular diseases. Through this partnership, Sirius aims to enhance the global reach of its siRNA technology, providing transformative treatment options for patients with chronic diseases.

The collaboration is seen as a strategic move by CRISPR Therapeutics to diversify its portfolio and expand its reach into cardiovascular medicine. By leveraging Sirius's siRNA platform, CRISPR is enhancing its capability to develop a broader range of gene-based therapies that could transform the treatment landscape for various serious diseases.

Overall, the partnership between CRISPR Therapeutics and Sirius Therapeutics marks a significant advancement in the field of siRNA therapies, with the potential to offer groundbreaking treatments for thromboembolic disorders and beyond. This collaboration underscores the potential of combining complementary scientific and technological capabilities to address pressing medical challenges and improve patient outcomes globally.