CRISPR Therapeutics is paying $25 million upfront to work on multiple siRNA targets with Sirius Tx with an early focus on the next-gen thrombotic disease asset SRSD107.
That drug, Sirius’ lead program, works as a long-acting, next-generation anticoagulant for thromboembolic disorders which, after passing early phase 1 tests, is gearing up for midstage trials.
The deal involves a $25 million upfront fee as well as an extra $70 million in equity from CRISPR to Sirius and allows the pair to work together on SRSD107 under a 50-50 cost and profit-sharing structure.
CRISPR will lead sales work in the U.S. while Sirius will be responsible for commercialization in greater China.
CRISPR can also pick up to two other siRNA targets for research work and, for each target, the biotech “will fund research and retain opt-in rights to lead clinical development and commercialization,” it said in a May 19 release, though it did not give any further details on targets.
Sirius, meanwhile, is in line for future milestone payments as well as tiered royalties on drugs that make it to market.
SRSD107 is a long-acting siRNA designed to selectively inhibit Factor XI (FXI), a key driver of pathological thrombosis with “minimal impact on normal hemostasis,” according to Sirius. Small interfering RNA is a type of RNA molecule that plays a crucial role in gene regulation with the promise of silencing disease-related genes.
Alnylam has been leading the charge in siRNA research with six FDA approvals across a range of disorders using the tech. This began in 2018 with the first-ever green light for an siRNA therapy in the form of Onpattro for polyneuropathy of hATTR amyloidosis.
This new deal dovetails with CRISPR’s work on its phase 1 asset CTX310, created out of its in vivo gene editing platform. The therapy is going after ANGPTL3, a gene that encodes for a key protein involved in the regulation of low-density lipoprotein and triglyceride levels, both known risk factors for atherosclerotic heart disease.
“We are excited to partner with Sirius, and broaden our cardiovascular medicine portfolio, on the heels of promising top-line data that we recently shared for CTX310, which targets ANGPTL3,” said Samarth Kulkarni, Ph.D., chair and CEO of CRISPR, in a statement.
“SRSD107, which targets Factor XI, has the potential to be a best-in-class therapy, offering infrequent dosing and improved patient outcomes,” Kulkarni added. “Sirius’ siRNA platform complements our existing capabilities and expands our therapeutic toolkit, enabling us to develop a broader range of transformative gene-based medicines.”
Analysts at Jefferies said in a note to clients that “the deal makes sense as it expands [CRISPR’s] toolkit capabilities beyond gene editing core, aiming for both reversible and permanent approaches to disease.”
The firm adds that the deal “adds versatility” to the biotech’s pipeline, and a potentially large list of targets.
This includes atrial fibrillation; venous thromboembolism (VTE); cancer-associated thrombosis; chronic coronary artery disease; chronic Peripheral Vascular Disease; ESRD requiring hemodialysis; and for patients with undergoing major orthopedic surgery.
It’s been a good month for Sino-American biotech Sirius, coming several weeks after the company raised $50 million in a series B2, bringing its total investment to $150 million.
The biotech, founded in 2021, has three clinical-stage programs, which include SRSD107, as well as a preclinical pipeline.