DelveInsight’s “Crispr Therapies – Pipeline Insight, 2023” report provides comprehensive insights about 5+ companies and 5+ 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.
Key Takeaways from the Crispr Therapies Pipeline Report
-
Leading Crispr Therapies companies developing novel drug candidates are Caribou Biosciences, Intimia Biosciences, Excision Biotherapeutics, and many others.
-
Promising Crispr Therapies pipeline therapies in various stages of development include CB-011, BD111, Cyclophosphamide, Fludarabine, and others
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 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 the way for a new era of CRISPR gene therapy to emerge.
Discover more about the emerging Crispr Therapies drugs@ Crispr Therapies Treatment Drugs
Crispr Therapies Pipeline Therapies and Key Companies
CTX001: CRISPR Therapeutics
CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from β-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 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 β-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 anemia in the USA and Priority Medicine (PRIME) status for Sickle cell anemia 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 retinal injection.
Scope of the Crispr Therapies Pipeline Report
-
Coverage: Global
-
Key Crispr Therapies Companies: Caribou Biosciences, Intimia Biosciences, Excision Biotherapeutics, and others.
-
Key Crispr Therapies Pipeline Therapies: CB-011, BD111, Cyclophosphamide and others.
.Find out more about the Crispr Therapies treatment options in development @ Crispr Therapies Clinical Trials
Table of Contents
1. Introduction
2. Executive Summary
3. Overview
4. Pipeline Therapeutics
5. Late-Stage Products (Phase III)
6. Mid-Stage Products (Phase II)
7. Early Stage Products (Phase I/II)
8. Preclinical Stage Products
9. Discovery Stage Products
10. Therapeutic Assessment
11. Inactive Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Unmet Needs
14. Market Drivers and Barriers
15. Appendix
16. About DelveInsight
Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Adya Kaul
Email: Send Email
Phone: 919650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/consulting/due-diligence-services