CRISPR therapies - Pipeline Insight, 2024

This “CRISPR Therapies - Pipeline Insight, 2024,” 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

• Global coverage

CRISPR Therapies Understanding

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. In the case of bacteria, the spacers are taken from viruses that previously attacked the organism. They serve as a bank of memories, which enables bacteria to recognize the viruses and fight off future attacks. 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.

CRISPR Therapies - Pipeline Insight, 2024 report 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.

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 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

CTX001: CRISPR Therapeutics CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from ß-thalassemia and sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen carrying hemoglobin that is naturally present at birth, and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion-requirements for ß-thalassemia patients and painful and debilitating sickle crises for sickle cell patients. CTX001 a CRISPR/Cas9-based treatment to advance from a research program is jointly conducted by CRISPR Therapeutics and Vertex Pharmaceuticals under the companies’ collaboration aimed at the discovery and development of new gene editing treatments that use the CRISPR/Cas9 technology. In May 2020, the FDA granted the therapy the designation of regenerative medicine advanced therapy (RMAT) for treating severe sickle cell disease and transfusion-dependent beta-thalassemia. CTX 001 also received Orphan Drug status for Sickle cell anaemia in USA and Priority Medicine (PRIME) status for Sickle cell anaemia in European Union.

EDIT-101: Editas Medicine EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells. The BRILLIANCE Phase I/II clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. The preliminary results showed that EDIT-101 has the potential to provide meaningful benefits to people living with CEP290-related retinal degeneration or LCA10. A positive safety profile has been observed through up to 15 months, with mostly mild adverse events primarily related to the procedure of retinalinjection.

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. 25+ 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. Phase I/II include, CRISPR therapeutics.

Phases

This 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
• Parenteral
• Intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• 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 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?

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