CRISPR Therapeutics Expands Pipeline: Gene Editing & RNAi Show Promise Beyond Sickle Cell

By Patrick Walker

Cambridge, MA – CRISPR Therapeutics is rapidly evolving from a gene-editing pioneer focused on blood disorders to a diversified biopharmaceutical company with a burgeoning pipeline targeting cardiovascular disease, genetic deficiencies, and thrombosis. While the company celebrates the commercial success of Casgevy for sickle cell disease and beta thalassemia, a deeper look reveals a strategic push into new therapeutic areas, fueled by both its core gene-editing technology and innovative RNA interference (RNAi) approaches.

Beyond Casgevy: A Multifaceted Pipeline

CRISPR Therapeutics’ expansion isn't a pivot away from gene editing, but rather an application of that expertise – alongside strategic collaborations – to address a wider range of unmet medical needs. The company’s pipeline now encompasses programs using its core CRISPR-Cas9 technology, alongside RNAi therapies developed in partnership with Sirius Therapeutics, and even novel platforms like its SyNTase™ editing approach.

“The initial success of Casgevy has validated the potential of CRISPR-based therapies, but it’s just the beginning,” notes an industry analyst familiar with the company’s strategy. “CRISPR Therapeutics is aiming to build a sustainable business by diversifying its pipeline and tackling diseases with significant market potential.”

Pioneering Precision Editing in Alpha-1 Antitrypsin Deficiency

One area of intense focus is Alpha-1 Antitrypsin Deficiency (AATD), a genetic disorder that can lead to severe lung and liver disease. Unlike current treatments that address symptoms, CRISPR Therapeutics’ CTX460 aims to correct the underlying genetic defect. Utilizing the novel SyNTase™ editing platform, CTX460 seeks to precisely correct the faulty SERPINA1 gene in liver cells.

Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress showed remarkable results. The data highlighted near-complete correction of the AATD mutation in both mouse and rat models, leading to a five-fold increase in serum AAT levels. “The durability of these edits, maintained for weeks in preclinical models, is particularly encouraging,” states a researcher following the program. The company expects to initiate clinical trials for CTX460 in mid-2026, hoping to offer a potential one-time curative therapy.

Addressing Cardiovascular Disease with Gene Editing & RNAi

Cardiovascular disease remains a leading cause of death worldwide, and CRISPR Therapeutics is pursuing multiple approaches to address this complex area. Its lead program, CTX310, utilizes CRISPR-Cas9 to knock out the ANGPTL3 gene in the liver. By reducing ANGPTL3 levels, the therapy aims to lower LDL cholesterol and reduce the risk of heart attack and stroke. This program is directly competitive with Verve Therapeutics’ similar approach, highlighting the growing interest in in vivo gene editing for cardiovascular disease.

Beyond gene editing, CRISPR Therapeutics is collaborating with Sirius Therapeutics on SRSD107, a long-acting siRNA targeting Factor XI (FXI). This partnership expands the company’s reach into RNA-based medicines and offers a complementary approach to preventing thromboembolic disorders. Phase 2 clinical trials for SRSD107 are now underway, examining its ability to prevent blood clots following total knee arthroplasty. The long-acting nature of SRSD107 – potentially requiring infrequent dosing – represents a significant advantage over existing anticoagulant therapies.

“The strategic collaboration with Sirius Therapeutics is a smart move,” says an industry observer. “It allows CRISPR Therapeutics to leverage RNAi technology, complementing its gene editing expertise and broadening its cardiovascular portfolio.”

The Patent Landscape & Competitive Pressures

The CRISPR patent landscape remains complex, with ongoing disputes between the Broad Institute, the University of California, Berkeley, and other institutions. While these legal battles continue, CRISPR Therapeutics has established a strong patent portfolio protecting its core gene editing technologies and its specific applications.

The company faces increasing competition from other gene editing companies like Verve Therapeutics and Beam Therapeutics, as well as from traditional pharmaceutical companies developing RNAi therapies. “The gene editing space is becoming increasingly crowded,” notes an analyst. “CRISPR Therapeutics will need to continue innovating and demonstrating clinical success to maintain its competitive edge.”

Looking Ahead: Diversification and Long-Term Growth

CRISPR Therapeutics’ expansion beyond sickle cell disease is a testament to the versatility of its technology platform and the company’s strategic vision. By diversifying its pipeline, the company is reducing its dependence on a single therapy and positioning itself for long-term growth. The combination of gene editing, RNAi therapies, and innovative platforms like SyNTase™ creates a robust and multifaceted pipeline with the potential to address a wide range of unmet medical needs.

“CRISPR Therapeutics is no longer just a gene editing company; it’s becoming a diversified biopharmaceutical company with a broad portfolio of innovative therapies,” concludes a source close to the company. “The next few years will be crucial as the company advances its pipeline and demonstrates the clinical benefits of its transformative technologies.”

While challenges remain, CRISPR Therapeutics is well-positioned to lead the next wave of innovation in genetic medicine and deliver life-changing therapies to patients around the world.