CRISPR Therapies Pipeline Insight Summary
DelveInsight’s “CRISPR Therapies Pipeline Insight 2026” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in the 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. This intelligence resource equips stakeholders – from pharmaceutical researchers to investors – with the actionable data needed to navigate the evolving CRISPR Therapies genome editing landscape confidently.
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CRISPR Therapies Overview
CRISPRs are specialized stretches of DNA, with “CRISPR” standing for clusters of regularly interspaced short palindromic repeats. This specialized DNA region is defined by two distinct characteristics: repeated nucleotide sequences and the spacer sequences interspersed among them, and CRISPRs play a role in regulating bacterial immunity.
CRISPR-Cas9 offers a high degree of fidelity along with relatively simple construction, relying on both a target sequence and a protospacer adjacent motif (PAM) sequence for its specificity. The target sequence spans 20 bases as part of each CRISPR locus in the crRNA array, while Cas9 recognizes the host genome’s PAM sequence and cannot easily be modified to recognize an alternate one. Once these sequences are assembled into a plasmid and transfected into cells, the Cas9 protein, guided by the crRNA, locates the correct sequence within the host cell’s DNA.
Casgevy stands as the first FDA-approved CRISPR/Cas9-based therapy, a cell-based gene therapy in which patients’ hematopoietic stem cells are modified through genome editing, approved for treating sickle cell disease in patients 12 years of age and older who experience recurrent vaso-occlusive crises.
Genome editing works by altering the sequences encoded within an organism’s DNA, thereby changing its instructions. CRISPR/Cas9 achieves this through a simple two-component system: the single-effector Cas9 protein, containing RuvC and HNH endonuclease domains that together generate double-stranded breaks in the target DNA, and a single guide RNA (sgRNA) that anchors to Cas9 and directs the complex to the intended genomic location via a 20 base pair spacer sequence. The editing process then relies on one of two endogenous DNA repair pathways – non-homologous end-joining or homology-directed repair. This technology has revolutionized biomedical research and continues to drive new clinical breakthroughs.
Several concerns remain around CRISPR-based gene editing. Off-target effects are a key consideration, as some studies report insignificant or undetectable levels while others document large insertions or deletions with unintended consequences. Immunogenicity against CRISPR proteins also requires monitoring, given observed pre-existing serum antibodies to Cas9 or Cas9-reactive T cells in certain patients. Additionally, most CRISPR-mediated gene therapy to date has centered on ex vivo treatment, editing stem cells outside the body before reintroducing corrected cells into patients.
CRISPR Therapies Emerging Drugs
The CRISPR Therapies drugs in development represent a new generation of genome-editing therapeutics designed to address serious unmet needs across infectious disease, hereditary conditions, and oncology. Below are three notable pipeline candidates:
- LBP-EC01 – Locus Biosciences
LBP-EC01 is a Locus crPhage® therapy in development for treating urinary tract infections and other infections caused by Escherichia coli (E. coli). It is a bacteriophage cocktail engineered with a CRISPR-Cas3 construct targeting the E. coli genome, working through a dual mechanism combining the natural lytic activity of the bacteriophage with the DNA-targeting activity of CRISPR-Cas3. Laboratory tests and small animal models have shown LBP-EC01 to be significantly more effective at killing E. coli than corresponding natural bacteriophages, and it met all primary and secondary endpoints while demonstrating safety and tolerability in a Phase 1b trial. LBP-EC01 is currently being evaluated in a registrational Phase II/III trial for UTIs caused by E. coli.
- NTLA-2002 – Intellia Therapeutics, Inc.
NTLA-2002 is a wholly owned, single-dose investigational CRISPR therapeutic designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes prekallikrein, the precursor to kallikrein. It is Intellia’s second investigational CRISPR therapeutic administered systemically via intravenous infusion to edit disease-causing genes inside the body with a single dose. Intellia’s proprietary non-viral platform uses lipid nanoparticles to deliver a two-part genome editing system to the liver – a guide RNA specific to the disease-causing gene and messenger RNA encoding the Cas9 enzyme – together carrying out precision editing. NTLA-2002 is currently in the Phase I/II stage of development.
- CB-010 – Caribou Biosciences, Inc.
CB-010 is the lead clinical-stage candidate from Caribou’s allogeneic CAR-T cell therapy platform, being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma in the ongoing ANTLER Phase I trial, and in patients with lupus nephritis and extrarenal lupus in the GALLOP Phase 1 trial. CB-010 is notable as reportedly the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to limit premature CAR-T cell exhaustion, and reportedly the first anti-CD19 allogeneic CAR-T therapy evaluated in the second-line large B cell lymphoma setting. For relapsed/refractory B-NHL, it has been granted Regenerative Medicine Advanced Therapy, Fast Track, and Orphan Drug designations by the FDA. CB-010 is currently in the Phase I stage of development.
Major CRISPR Therapies Players
There are approximately 25+ key CRISPR Therapies companies actively developing genome-editing therapies across infectious disease, rare disease, and oncology indications. The companies which have their CRISPR Therapies drug candidates in the most advanced stage, i.e., Phase III, include Locus Biosciences. These organizations are collectively pushing the boundaries of gene-editing science and therapeutics, driving meaningful progress in the treatment of complex and previously intractable conditions.
- Locus Biosciences’ LBP-EC01 has advanced into a registrational Phase II/III trial after meeting all primary and secondary endpoints in its Phase 1b study
- Intellia Therapeutics’ NTLA-2002 continues Phase I/II development as a systemically administered, single-dose in vivo editing approach delivered via lipid nanoparticles
- Caribou Biosciences’ CB-010 is advancing through the ANTLER and GALLOP Phase I trials, holding RMAT, Fast Track, and Orphan Drug designations from the FDA
- Growing interest in allogeneic CAR-T and in vivo lipid nanoparticle-based CRISPR platforms observed among emerging biotech players
Key companies in the CRISPR Therapies space include:
- Locus Biosciences
- Intellia Therapeutics, Inc.
- Caribou Biosciences, Inc.
- Tango Therapeutics
- KSQ Therapeutics
- CRISPR Therapeutics
- Emendo Biotherapeutics
- Beam Therapeutics
- And others.
CRISPR Therapies Clinical Trial Phases
DelveInsight’s report covers around 30+ products under different phases of clinical development as part of ongoing CRISPR Therapies clinical trials:
- Late-stage products (Phase III): Advanced candidates with pivotal trial data, targeting regulatory approval
- Mid-stage products (Phase II): Candidates demonstrating early efficacy and safety with expanded cohort trials
- Early-stage products (Phase I): First-in-human studies assessing safety and dosing profiles
- Pre-clinical and Discovery stage candidates: Exploratory molecules in laboratory and animal model testing
- Discontinued & Inactive candidates: Products that have been halted or deprioritized, providing strategic competitive intelligence
CRISPR Therapies Drug Route of Administration
The CRISPR Therapies pipeline report provides the therapeutic assessment of the pipeline drugs by Route of Administration. Products have been categorized under various ROAs such as:
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
CRISPR Therapies Product Molecule Type
Products have been categorized under various molecule types such as:
- Monoclonal antibody
- Small molecule
- Peptide
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Conclusion
The CRISPR Therapies pipeline is rapidly evolving, with a robust set of candidates spanning phage-based genome editing, in vivo lipid nanoparticle delivery, and allogeneic CAR-T platforms across multiple clinical stages. The growing involvement of 25+ CRISPR Therapies companies and the depth of ongoing CRISPR Therapies clinical trials signal a highly active and competitive landscape. With advanced candidates like LBP-EC01 progressing through registrational trials and next-generation editing approaches like NTLA-2002 and CB-010 advancing through early-to-mid stage development, the pipeline holds strong promise for improving patient outcomes globally. Continued investment, cross-sector collaboration, and strategic pipeline monitoring will be critical to address unmet medical needs and expand the reach of precision genome editing across infectious disease, rare disease, and oncology.
About DelveInsight
DelveInsight is a leading Healthcare Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharmaceutical companies by providing comprehensive end-to-end solutions to improve their performance. It also offers healthcare consulting services that leverage market analysis to accelerate business growth and overcome challenges with practical approaches.
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