CRISPR Therapies Clinical Trials 2025: EMA, PDMA, FDA Approvals, Medication, Therapies, Treatment Market, Mechanism of Action, Route of Administration, and Companies by DelveInsight

(Albany, USA) DelveInsight's "CRISPR Therapies Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the CRISPR Therapies market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

The CRISPR therapies landscape is witnessing rapid growth and is powered by cutting-edge research and transformative innovations. Pioneering companies like Tango Therapeutics, KSQ Therapeutics, CRISPR Therapeutics, Emendo Biotherapeutics, and Beam Therapeutics are leading the charge-reshaping therapeutic approaches and unlocking new possibilities in genetic medicine. With their groundbreaking advancements, these leaders are paving the way for a future where CRISPR-based treatments offer renewed hope and lasting impact for patients worldwide.

For emerging CRISPR therapy drugs, the CRISPR Therapies pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

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Key Takeaways from the CRISPR Therapies Pipeline Report
• DelveInsight's CRISPR therapies pipeline analysis depicts a robust space with 25+ active players working to develop 30+ pipeline drugs for CRISPR Therapies treatment.
• The leading CRISPR therapies companies include Locus Biosciences, Intellia Therapeutics, Caribou Biosciences, Tango Therapeutics, KSQ Therapeutics, CRISPR Therapeutics, Emendo Biotherapeutics, Beam Therapeutics, and others are evaluating their lead assets to improve the CRISPR Therapies treatment landscape.
• Key CRISPR therapies pipeline therapies in various stages of development include LBP-EC01, NTLA-2002, CB-010, TNG260, KSQ-4279, CTX 130, EMD-101, BEAM-301, CTX131, CTX112, and others.
• In March 2025, Intellia Therapeutics announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2001 (nexiguran ziclumeran) for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM). NTLA-2001 is a CRISPR-based investigational therapy designed to offer a potentially curative, single-dose solution.
• In February 2025, Arbor Biotechnologies received both Orphan Drug and Rare Pediatric Disease designations from the FDA for ABO-101, a novel CRISPR-Cas12i2 gene-editing therapy targeting primary hyperoxaluria type 1 (PH1). Delivered via a lipid nanoparticle system, ABO-101 is designed as a one-time liver-directed treatment that deactivates the HAO1 gene to reduce oxalate production.
• In November 2024, HuidaGene Therapeutics announced FDA clearance of its IND application for HG202, marking the world's first CRISPR/Cas13 RNA-editing therapy to enter clinical trials. HG202 targets neovascular age-related macular degeneration (nAMD), aiming to address a major cause of vision loss worldwide.
• In September 2024, Modalis Therapeutics secured Rare Pediatric Disease designation from the FDA for MDL-101, a precision CRISPR-GNDM®-based epigenome editing therapy being developed for congenital muscular dystrophy type 1A (LAMA2-CMD), a rare and severe neuromuscular disorder.

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CRISPR Therapies Overview
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) refers to specialized DNA sequences in bacteria, containing repeats and spacers that help regulate bacterial immunity. The CRISPR-Cas9 system, derived from this mechanism, has become a powerful and precise tool for genome editing.
The system relies on two components:
1. Cas9 protein, which makes double-stranded breaks (DSBs) in DNA via its RuvC and HNH domains.
2. Single guide RNA (sgRNA), which directs Cas9 to the target DNA sequence adjacent to a PAM (protospacer adjacent motif).
Once targeted, CRISPR-Cas9 introduces cuts in DNA, triggering the cell's natural repair processes- non-homologous end joining (NHEJ) or homology-directed repair (HDR)-to modify the genome.

A major milestone in CRISPR-based therapy is Casgevy, the first FDA-approved CRISPR/Cas9 therapy for sickle cell disease in patients aged 12 and older. It involves ex vivo editing of hematopoietic stem cells to correct disease-causing mutations.

Despite its promise, CRISPR raises concerns:
• Off-target effects, potentially causing unintended genetic changes.
• Immunogenicity, due to pre-existing immune responses to Cas9.
• Limited in vivo application, as most therapies currently involve ex vivo cell editing.

Nonetheless, CRISPR/Cas9 is paving the way for groundbreaking advances in genetic medicine and holds vast potential for future therapies.

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CRISPR Therapies Treatment Analysis: Drug Profile
LBP-EC01: Locus Biosciences
LBP-EC01 is a CRISPR-enhanced bacteriophage (crPhage®) therapy developed by Locus Biosciences for treating urinary tract infections (UTIs) and other infections caused by Escherichia coli (E. coli). The therapy combines the natural lytic activity of bacteriophages with the targeted DNA-cleaving power of CRISPR-Cas3, delivering a potent dual mechanism of action. In preclinical studies and a Phase 1b trial, LBP-EC01 showed superior efficacy in eliminating E. coli compared to natural phages, along with a strong safety and tolerability profile. It is currently in a registrational Phase II/III trial for E. coli-induced UTIs.

NTLA-2002: Intellia Therapeutics
NTLA-2002 is an investigational single-dose CRISPR therapy developed by Intellia Therapeutics for the treatment of hereditary angioedema (HAE). It targets the KLKB1 gene, which encodes prekallikrein, a key protein involved in disease pathology. Delivered systemically via lipid nanoparticles, the therapy utilizes a guide RNA and messenger RNA encoding the Cas9 enzyme to achieve precise in vivo gene editing in the liver. NTLA-2002 is currently in Phase I/II clinical development.

Key CRISPR Therapies and Companies
• LBP-EC01: Locus Biosciences
• NTLA-2002: Intellia Therapeutics, Inc
• CB-010: Caribou Biosciences, Inc
• CTX131: CRISPR Therapeutics
• CTX112: CRISPR Therapeutics

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CRISPR Therapies Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Intravenous
• Subcutaneous
• Oral
• Intramuscular

By Molecule Type
• Monoclonal antibody
• Small molecule
• Peptide

Scope of the CRISPR Therapies Pipeline Report
• Coverage: Global
• Key CRISPR Therapies Companies: Locus Biosciences, Intellia Therapeutics, Caribou Biosciences, Tango Therapeutics, KSQ Therapeutics, CRISPR Therapeutics, Emendo Biotherapeutics, Beam Therapeutics, and others.
• Key CRISPR Therapies Pipeline Therapies: LBP-EC01, NTLA-2002, CB-010, TNG260, KSQ-4279, CTX 130, EMD-101, BEAM-301, CTX131, CTX112, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. CRISPR Therapies Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. CRISPR Therapies Pipeline Therapeutics
6. CRISPR Therapies Pipeline: Late-Stage Products (Phase III)
7. CRISPR Therapies Pipeline: Late-Stage Products (Phase III)
8. CRISPR Therapies Pipeline: Mid-Stage Products (Phase II)
9. CRISPR Therapies Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

Contact Us:
Ankit Nigam
Manager Marketing
info@delveinsight.com
+14699457679
https://www.delveinsight.com/ats-conference-coverage

About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

This release was published on openPR.