The landscape of CRISPR therapies is undergoing significant evolution, marked by a surge in innovation driven by over 25 companies actively engaging in the development of advanced gene-editing solutions. Among these innovators are notable firms such as Tango Therapeutics, KSQ Therapeutics, CRISPR Therapeutics, Emendo Biotherapeutics, and Beam Therapeutics. The report from DelveInsight provides an in-depth analysis of the ongoing and prospective developments within the CRISPR Therapies market, indicating a promising trajectory for precision medicine.
According to DelveInsight's detailed publication titled "CRISPR Therapies Pipeline Insight, 2025," the report outlines a comprehensive examination of the current clinical landscape as well as anticipated growth within the domain. Evidence suggests that there are more than 30 pipeline drugs under development, showcasing the robust nature of the CRISPR therapies sector. The analysis includes vital information regarding disease insights, clinical treatment guidelines, and an extensive evaluation of the pipeline stages ranging from preclinical trials to marketed products.
Key players highlighted in the report include prominent companies like Locus Biosciences, Intellia Therapeutics, and Caribou Biosciences, which are expanding the CRISPR therapies treatment spectrum. Noteworthy pipeline therapies in various development stages encompass LBP-EC01 from Locus Biosciences, NTLA-2002 from Intellia Therapeutics, and CB-010 from Caribou Biosciences, among others.
Significant milestones within this emerging field have also been recorded. Notably, in March 2025, Intellia Therapeutics received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for NTLA-2001, an investigational CRISPR-based therapy aimed at treating transthyretin amyloidosis with cardiomyopathy. This therapy is intended to provide a potentially curative solution with a single administration.
Further exploring the advancements, the report highlights Arbor Biotechnologies, which gained both Orphan Drug and Rare Pediatric Disease designations from the FDA for ABO-101—a novel CRISPR-Cas12i2 gene-editing therapy designed for primary hyperoxaluria type 1 (PH1). Additionally, HuidaGene Therapeutics made headlines in November 2024 by achieving FDA clearance for its IND application concerning HG202, described as the world’s first CRISPR/Cas13 RNA-editing therapy, demonstrating a focus on eye health and targeting neovascular age-related macular degeneration.
The applications of CRISPR extend into various treatment avenues, evidenced by developments such as Modalis Therapeutics securing Rare Pediatric Disease designation for MDL-101, a CRISPR-GNDM®-based therapy aimed at treating congenital muscular dystrophy type 1A (LAMA2-CMD).
The report details the mechanics of CRISPR technology, emphasising that it utilises the CRISPR-Cas9 system, which consists of the Cas9 protein and single guide RNA (sgRNA). This mechanism allows for targeted genome modifications by introducing double-stranded breaks in DNA, followed by cellular repair processes that can lead to desired genetic alterations.
Despite the advances, the application of CRISPR technology is not without challenges. Concerns are noted regarding potential off-target effects, immunogenicity linked to pre-existing immune responses to Cas9, and the limitations of in vivo applications, as many therapies predominantly involve ex vivo editing of cells.
Casgevy has emerged as a notable success story within the CRISPR framework, being the first FDA-approved CRISPR/Cas9 therapy designated for the treatment of sickle cell disease in patients aged 12 and older. This therapy exemplifies the evolution of CRISPR from experimental technology to a viable therapeutic option.
With the growing landscape of CRISPR therapies demonstrating broad potential in treating genetic disorders and other conditions, ongoing research and development efforts are likely to yield further innovations in the medical field.
Source: Noah Wire Services