
CRISPR Therapies- Pipeline Insight, 2025
Description
DelveInsight’s, “CRISPR Therapies- Pipeline Insight, 2025” report provides comprehensive insights about 25+ 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
CRISPR Therapies: Overview
CRISPRs are specialized stretches of DNA. ""CRISPR"" stands for ""clusters of regularly interspaced short palindromic repeats."" It is a specialized region of DNA with two distinct characteristics: the presence of nucleotide repeats and spacers. Repeated sequences of nucleotides — the building blocks of DNA — are distributed throughout a CRISPR region. Spacers are bits of DNA that are interspersed among these repeated sequences. CRISPRs play a role in regulating bacterial immunity.
CRISPR-Cas9 offers a high degree of fidelity and relatively simple construction. It depends on two factors for its specificity: the target sequence and the protospacer adjacent motif (PAM) sequence. The target sequence is 20 bases long as part of each CRISPR locus in the crRNA array. The PAM sequence on the host genome is recognized by Cas9. Cas9 cannot be easily modified to recognize a different PAM sequence. Once these sequences have been assembled into a plasmid and transfected into cells the Cas9 protein with the help of the crRNA finds the correct sequence in the host cell's DNA.
Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9 in which patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology. Casgevy, a cell-based gene therapy, which is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises.
The genomes of various organisms encode a series of messages and instructions within their DNA sequences. Genome editing involves changing those sequences, thereby changing the messages. This can be done by inserting a cut or break in the DNA and tricking a cell's natural DNA repair mechanisms into introducing the changes and CRISPR-Cas9 provides a means to do so. CRISPR/Cas9 is a simple two-component system used for effective targeted gene editing. The first component is the single-effector Cas9 protein, which contains the endonuclease domains RuvC and HNH. RuvC cleaves the DNA strand non-complementary to the spacer sequence and HNH cleaves the complementary strand. Together, these domains generate double-stranded breaks (DSBs) in the target DNA. The second component of effective targeted gene editing is a single guide RNA (sgRNA) carrying a scaffold sequence which enables its anchoring to Cas9 and a 20 base pair spacer sequence complementary to the target gene and adjacent to the PAM sequence. This sgRNA guides the CRISPR/Cas9 complex to its intended genomic location. The editing system then relies on either of two endogenous DNA repair pathways: non-homologous end-joining (NHEJ) or homology-directed repair. Now, as a gene-editing tool, CRISPR/Cas9 has revolutionized biomedical research and may soon enable medical breakthroughs in a way few biological innovations. The recent advent of CRISPR technology in clinical trials has paved way for the new era of CRISPR gene therapy to emerge.
Several concerns exist for the implementation of CRISPR for gene editing. First, there are concerns of off-target effects; although some studies have shown that there are insignificant or even undetectable levels of off-target effects, others have clear documentation of large insertions or deletions that may result in unintended consequences. Second, immunogenicity against CRISPR proteins would need to be monitored given observations of pre-existing serum antibodies to Cas9 in some donors or prevalence of Cas9-reactive T cells in some patients Finally, most CRISPR-mediated gene therapy has focused on ex vivo treatment, namely editing of stem cells outside the body and subsequent reintroduction of corrected cells back into patients.
""CRISPR Therapies- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the CRISPR Therapies pipeline landscape is provided which includes the disease overview and CRISPR Therapies treatment guidelines. 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
This segment of the CRISPR Therapies report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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
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:
CRISPR Therapies: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase 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
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
CRISPR Therapies: Overview
CRISPRs are specialized stretches of DNA. ""CRISPR"" stands for ""clusters of regularly interspaced short palindromic repeats."" It is a specialized region of DNA with two distinct characteristics: the presence of nucleotide repeats and spacers. Repeated sequences of nucleotides — the building blocks of DNA — are distributed throughout a CRISPR region. Spacers are bits of DNA that are interspersed among these repeated sequences. CRISPRs play a role in regulating bacterial immunity.
CRISPR-Cas9 offers a high degree of fidelity and relatively simple construction. It depends on two factors for its specificity: the target sequence and the protospacer adjacent motif (PAM) sequence. The target sequence is 20 bases long as part of each CRISPR locus in the crRNA array. The PAM sequence on the host genome is recognized by Cas9. Cas9 cannot be easily modified to recognize a different PAM sequence. Once these sequences have been assembled into a plasmid and transfected into cells the Cas9 protein with the help of the crRNA finds the correct sequence in the host cell's DNA.
Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9 in which patients’ hematopoietic (blood) stem cells are modified by genome editing using CRISPR/Cas9 technology. Casgevy, a cell-based gene therapy, which is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises.
The genomes of various organisms encode a series of messages and instructions within their DNA sequences. Genome editing involves changing those sequences, thereby changing the messages. This can be done by inserting a cut or break in the DNA and tricking a cell's natural DNA repair mechanisms into introducing the changes and CRISPR-Cas9 provides a means to do so. CRISPR/Cas9 is a simple two-component system used for effective targeted gene editing. The first component is the single-effector Cas9 protein, which contains the endonuclease domains RuvC and HNH. RuvC cleaves the DNA strand non-complementary to the spacer sequence and HNH cleaves the complementary strand. Together, these domains generate double-stranded breaks (DSBs) in the target DNA. The second component of effective targeted gene editing is a single guide RNA (sgRNA) carrying a scaffold sequence which enables its anchoring to Cas9 and a 20 base pair spacer sequence complementary to the target gene and adjacent to the PAM sequence. This sgRNA guides the CRISPR/Cas9 complex to its intended genomic location. The editing system then relies on either of two endogenous DNA repair pathways: non-homologous end-joining (NHEJ) or homology-directed repair. Now, as a gene-editing tool, CRISPR/Cas9 has revolutionized biomedical research and may soon enable medical breakthroughs in a way few biological innovations. The recent advent of CRISPR technology in clinical trials has paved way for the new era of CRISPR gene therapy to emerge.
Several concerns exist for the implementation of CRISPR for gene editing. First, there are concerns of off-target effects; although some studies have shown that there are insignificant or even undetectable levels of off-target effects, others have clear documentation of large insertions or deletions that may result in unintended consequences. Second, immunogenicity against CRISPR proteins would need to be monitored given observations of pre-existing serum antibodies to Cas9 in some donors or prevalence of Cas9-reactive T cells in some patients Finally, most CRISPR-mediated gene therapy has focused on ex vivo treatment, namely editing of stem cells outside the body and subsequent reintroduction of corrected cells back into patients.
""CRISPR Therapies- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the CRISPR Therapies pipeline landscape is provided which includes the disease overview and CRISPR Therapies treatment guidelines. 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.
This segment of the CRISPR Therapies report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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
- LBP-EC01: Locus Biosciences
- NTLA-2002: Intellia Therapeutics, Inc
- CB-010: Caribou Biosciences, Inc
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
- Phases
- 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
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
- Molecule Type
- Monoclonal antibody
- Small molecule
- Peptide
- Product Type
CRISPR Therapies: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase 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
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
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?
- Locus Biosciences
- Intellia Therapeutics, Inc
- Caribou Biosciences, Inc
- Tango Therapeutics
- KSQ Therapeutics
- CRISPR therapeutics
- Emendo biotherapeutics
- Beam Therapeutics
- LBP-EC01
- NTLA-2002
- CB-010
- TNG260
- KSQ-4279
- CTX 130
- EMD-101
- BEAM-301
Table of Contents
80 Pages
- Introduction
- Executive Summary
- CRISPR Therapies: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- CRISPR Therapies– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II/III)
- Comparative Analysis
- LBP-EC01: Locus Biosciences
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- NTLA-2002: Intellia Therapeutics, Inc
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- CRISPR Therapies Key Companies
- CRISPR Therapies Key Products
- CRISPR Therapies- Unmet Needs
- CRISPR Therapies- Market Drivers and Barriers
- CRISPR Therapies- Future Perspectives and Conclusion
- CRISPR Therapies Analyst Views
- CRISPR Therapies Key Companies
- Appendix
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