DelveInsight’s, “CRISPR Therapies Pipeline Insight, 2023,” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in CRISPR Therapies pipeline landscape. It covers the CRISPR Therapies pipeline drug profiles, including CRISPR Therapies clinical trials 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.
In the CRISPR Therapies pipeline report, a detailed description of the drug is given which includes the mechanism of action of the drug, CRISPR Therapies clinical trials studies, CRISPR Therapies 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.
Key takeaways from the CRISPR Therapies Pipeline Report
- DelveInsight’s CRISPR Therapies pipeline report depicts a robust space with 25+ active players working to develop 30+ pipeline therapies for CRISPR Therapies treatment.
- The leading CRISPR Therapies Companies includes Vertex Pharmaceuticals; Excision Biotherapeutic; CRISPR Therapeutics; Intellia Therapeutics; Editas Medicine; Thermo Fisher Scientific; Integrated DNA Technologies, Inc. (Danaher); GenScript Biotech Corporation; GeneCopoeia; Applied StemCell; Casebia Therapeutics; Modalis Therapeutics; Mustang Bio; Sarepta Therapeutics; Origene Technologies; ASC Therapeutics; Emendo Biotherapeutics; Precision Biosciences; Horizon Discovery; Synthego Corporation; Agilent Technologies; Evotec A.G; Beam Therapeutics; New England Biolabs; Novartis; Caribou Biosciences; Addgene among others.f
- Promising CRISPR Pipeline Therapies includes BD111 Adult single group Dose, GPH101 Drug Product, and others.
- The CRISPR Pipeline companies and academics are working to assess challenges and seek opportunities that could influence CRISPR Therapies R&D. The CRISPR pipeline therapies under development are focused on novel approaches to treat/improve CRISPR Therapies.
To explore more information on the latest breakthroughs in the CRISPR Therapies Pipeline treatment landscape of the report, click here @ CRISPR Therapies Pipeline Outlook
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.
Recent Development Activities in the CRISPR Therapies Treatment Landscape
- In November 2019, Vertex Pharmaceuticals entered into a strategic research collaboration with Molecular Templates (MTEM) to discover and develop targeted conditioning regimens to enhance the haematopoietic stem cell transplant process, including transplants conducted as part of treatment with ex vivo CRISPR/Cas9 CTX 001 gene-editing therapy. The collaboration seeks to discover a new conditioning regimen utilizing MTEM’s engineered toxin body (ETB) platform designed to specifically target and remove specific cells to enable successful engraftment of new cells.
- Gene editing technologies such as CRISPR produces good results in the screening phase of drug discovery by using the CRISPR–associated protein 9 (Cas9) enzyme and guide RNA to edit the genome at a particular point. In November 2019, German drug discovery and development company, Evotec SE received the CRISPR gene-editing technology license from Merck to discover and test new drugs.
- CRISPR Therapeutics has announced its participation in the 20th Annual Needham Virtual Healthcare Conference on April 12, 2021, at 9:30 a.m. ET.
- Scribe Therapeutics recently announced a USD 100 million series B funding round to help the company achieve its mission in developing molecules through its “Crispr by design” platform. The company has also inked a deal with Biogen to design Crispr-based genetic medicines for diseases such as amyotrophic lateral sclerosis (ALS).
- Intellia Therapeutics announced the European Commission (EC) approval of orphan drug designation to its NTLA-2001, being developed for a rare condition transthyretin amyloidosis (ATTR).
- Editas Medicine plans to advance its landmark Brilliance trial, the first-ever in vivo gene editing program investigating EDIT-101 for Leber Congenital Amaurosis 10 (LCA10).
CRISPR Therapies Emerging Drugs Profile
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 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 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 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 retinal injection.
CRISPR Therapies Pipeline Therapeutics Assessment
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.
For further information, refer to the detailed CRISPR Therapies Unmet Needs, CRISPR Therapies Market Drivers, and Market Barriers, click here for CRISPR Therapies Ongoing Clinical Trial Analysis
Scope of the CRISPR Therapies Pipeline Report
- Coverage- Global
- CRISPR Therapies Companies- Vertex Pharmaceuticals; Excision Biotherapeutic; CRISPR Therapeutics; Intellia Therapeutics; Editas Medicine; Thermo Fisher Scientific; Integrated DNA Technologies, Inc. (Danaher); GenScript Biotech Corporation; GeneCopoeia; Applied StemCell; Casebia Therapeutics; Modalis Therapeutics; Mustang Bio; Sarepta Therapeutics; Origene Technologies; ASC Therapeutics; Emendo Biotherapeutics; Precision Biosciences; Horizon Discovery; Synthego Corporation; Agilent Technologies; Evotec A.G; Beam Therapeutics; New England Biolabs; Novartis; Caribou Biosciences; Addgene; and many others.
- CRISPR Pipeline Therapies: CTX 120; CTX 110; OTQ 923; EDIT 301; NTLA 2001; ASC 618; EBT-101; LCA10 program; LBP-EC01; CTX-130; EDIT-101 among others.
- CRISPR Therapies Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration
Dive deep into rich insights for drugs for CRISPR Therapies Market Drivers and CRISPR Therapies Market Barriers, click here @ CRISPR Therapies Unmet Needs and Analyst Views
Table of Content
- Introduction
- Executive Summary
- CRISPR Therapies: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Late Stage Products (Phase III)
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- CTX001: CRISPR Therapeutics
- Drug profiles in the detailed report…..
- Early stage products (Phase I)
- LBP-EC01: Locus Biosciences
- Drug profiles in the detailed report…..
- Inactive Products
- 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
Got Queries? Find out the related information on CRISPR Therapies Mergers and acquisitions, CRISPR Therapies Licensing Activities @ CRISPR Therapies Emerging Drugs, and Recent Trends
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