CRISPR therapies Pipeline Summary

DelveInsight’s, “CRISPR therapies Pipeline Insight, 2026” report provides comprehensive insights about 20+ companies and 30+ pipeline drugs in CRISPR therapies pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

CRISPR therapies: Understanding

CRISPR therapies: Overview

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) therapies are a new class of gene-editing medicines designed to precisely modify disease-causing DNA sequences or regulate gene expression. Most therapeutic platforms use the CRISPR-associated protein Cas9, guided by a single-guide RNA (sgRNA), to recognize and edit specific genomic targets. Following DNA cleavage, endogenous cellular repair pathways non-homologous end joining (NHEJ) or homology-directed repair (HDR) introduce targeted gene disruption or precise sequence correction.

Newer systems, including base editing and prime editing, enable single-nucleotide modifications or targeted insertions without creating double-strand DNA breaks, potentially improving editing precision and reducing unwanted genomic alterations. CRISPR components can be delivered ex vivo by editing patient-derived cells before reinfusion or in vivo using viral vectors, lipid nanoparticles, or other targeted delivery platforms.

CRISPR therapies have broad therapeutic applications across inherited genetic disorders, hematologic diseases, cancer, infectious diseases, ophthalmic disorders, and selected metabolic and neurological conditions. The first approved CRISPR-based therapy demonstrated that editing the BCL11A enhancer in autologous hematopoietic stem cells can restore fetal hemoglobin production, providing durable clinical benefit in sickle cell disease and transfusion-dependent β-thalassemia. Numerous investigational programs are evaluating CRISPR-mediated correction or disruption of disease-causing genes for conditions such as transthyretin amyloidosis, hereditary angioedema, Duchenne muscular dystrophy, cystic fibrosis, inherited retinal diseases, and chronic viral infections.

CRISPR is also being integrated with cell therapies by engineering CAR-T and CAR-NK cells to enhance anti-tumor activity, persistence, and immune evasion. Beyond permanent gene editing, CRISPR-based epigenome editing and CRISPR interference/activation (CRISPRi/CRISPRa) are being explored to reversibly regulate gene expression without altering the DNA sequence.

Despite its transformative potential, CRISPR therapy faces important scientific and clinical challenges, including efficient tissue-specific delivery, off-target editing, immune responses to Cas proteins, mosaic editing, and long-term safety monitoring. Advances in high-fidelity Cas enzymes, optimized guide RNA design, transient editing systems, and non-viral delivery technologies have significantly improved editing specificity and reduced unintended genomic changes. Regulatory agencies require extensive preclinical and long-term clinical follow-up to assess durability, genotoxicity, and potential delayed adverse effects. Ongoing research is expanding the therapeutic scope through multiplex genome editing, RNA-targeting CRISPR systems, programmable gene insertion, and next-generation editors with greater precision. As these technologies continue to mature, CRISPR-based therapies are expected to become an increasingly important component of precision medicine for both rare genetic disorders and more common complex diseases.

"CRISPR therapies- Pipeline Insight, 2026" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the area A detailed picture of the CRISPR therapies pipeline landscape is provided which includes the overview and CRISPR therapies applications. The assessment part of the report embraces, in depth CRISPR therapies commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, CRISPR therapies collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence CRISPR therapies R&D. The therapies under development are focused on novel approaches to treat/improve CRISPR therapies.

CRISPR therapies Emerging Drugs Chapters

This segment of the CRISPR therapies report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

CRISPR therapies Emerging Drugs

NTLA-2002: Intellia Therapeutics

NTLA-2002 (lonvoguran ziclumeran [lonvo-z]) is an investigational in vivo CRISPR-Cas9 gene-editing therapy developed by Intellia Therapeutics for the treatment of hereditary angioedema (HAE). It uses lipid nanoparticles (LNPs) to deliver Cas9 mRNA and a guide RNA to hepatocytes, where it permanently inactivates the KLKB1 gene, resulting in sustained reductions in plasma kallikrein and bradykinin levels that drive HAE attacks. Designed as a single-dose therapy, NTLA-2002 aims to provide durable prevention of recurrent swelling episodes and reduce the need for lifelong prophylactic treatment. Based on positive Phase III results, Intellia initiated a rolling Biologics License Application (BLA) submission to the US FDA in 2026 for lonvoguran ziclumeran as a potential one-time treatment for HAE.

LBP-EC01: Locus Biosciences, Inc

LBP-EC01 is an investigational CRISPR-Cas3-enhanced bacteriophage therapy developed by Locus Biosciences for the treatment of acute uncomplicated urinary tract infections (uUTIs) caused by antimicrobial-resistant and multidrug-resistant Escherichia coli. The therapy consists of a recombinant bacteriophage cocktail engineered with a CRISPR-Cas3 system that selectively targets and degrades the bacterial genome, combining the natural lytic activity of bacteriophages with programmable CRISPR-mediated bacterial killing. LBP-EC01 is administered in combination with trimethoprim/sulfamethoxazole (TMP/SMX) to improve bacterial eradication while minimizing disruption of the normal microbiome. The therapy is currently being evaluated in the ELIMINATE Phase II clinical trial, where early results have demonstrated favorable safety, pharmacokinetics, and promising microbiological and clinical efficacy against drug-resistant E. coli infections.

CTX310: CRISPR Therapeutics

CTX310 is an investigational in vivo CRISPR-Cas9 gene-editing therapy developed by CRISPR Therapeutics for the treatment of refractory dyslipidemias, including hypercholesterolemia, hypertriglyceridemia, and familial hypercholesterolemia. It is formulated as a lipid nanoparticle (LNP) containing Cas9 mRNA and a guide RNA that selectively edits the ANGPTL3 gene in hepatocytes following a single intravenous infusion. By permanently disrupting ANGPTL3 expression, CTX310 aims to produce durable reductions in LDL cholesterol, triglycerides, and other atherogenic lipids, offering a potential one-time treatment for patients inadequately controlled with existing lipid-lowering therapies. CTX310 is currently being evaluated in a first-in-human, open-label Phase I clinical trial assessing its safety, tolerability, pharmacodynamics, and preliminary efficacy.

Further product details are provided in the report……..

CRISPR therapies: Therapeutic Assessment

This segment of the report provides insights about the different CRISPR therapies drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in CRISPR therapies
• There are approx. 20+ key companies which are developing the therapies for CRISPR therapies. The companies which have their CRISPR therapies drug candidates in the most advanced stage, i.e. Pre-registration include, Intellia Therapeutics.

• Phases

DelveInsight’s report covers around 30+ products under different phases of clinical development like

• 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

• Route of Administration

CRISPR therapies pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

CRISPR therapies: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses CRISPR therapies therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging CRISPR therapies drugs.

CRISPR therapies Report Insights

• CRISPR therapies Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

CRISPR therapies Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing CRISPR therapies drugs?
• How many CRISPR therapies drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of CRISPR therapies?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the CRISPR therapies therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for CRISPR therapies and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Intellia Therapeutics
• Locus Biosciences, Inc
• CRISPR Therapeutics
• Epicrispr Biotechnologies, Inc.
• Editas Medicine
• Beam Therapeutics
• Caribou Biosciences
• Emendo biotherapeutics
• KSQ Therapeutics, Inc.
• HuidaGene Therapeutics
• Suzhou Maximum Bio-tech
• YolTech Therapeutics
• Precision BioSciences
• Scribe Therapeutics

Key Products

• NTLA-2002
• LBP-EC01
• CTX310
• EPI-331
• EDIT-401
• BEAM-302
• CB-011
• EMD-101
• KSQ-001EX
• HG202
• MT027
• YOLT-203
• PBGENE-DMD
• Research Program