Intellia has established high profile collaborations with Regeneron and Novartis, which will boost its ability to navigate a successful route to market and provide the company with the necessary infrastructure and experience in drug development and commercialisation.Įditas Medicine has the smallest market cap of the three, and currently only has a single agent in clinical development: EDIT-101. Similar to CRISPR Therapeutics, Intellia is also considering sickle cell disease as a target indication, but the company is slightly behind CRISPR in terms of clinical development, potentially indicating a future second to market disadvantage. Intellia’s NTLA-2001 uses an in vivo approach to tackle a rare hereditary disorder, transthyretin amyloidosis, for which only chronic treatment options are currently available. In vivo describes the introduction of the CRISPR technology into the patient directly via an intravenous route of administration, while ex vivo describes the removal of stem cells for gene editing followed by the re-introduction of these repaired cells into the patient. Intellia’s approaches to clinical development can be separated into an in vivo and ex vivo approach. Doudna, the other joint winner of the Nobel Prize in Chemistry 2020, co-founded Intellia. Furthermore, CRISPR Therapeutics was co-founded by Dr Emmanuelle Charpentier, joint winner of the Nobel Prize in Chemistry 2020 for CRISPR-Cas9, whose expertise will further support the successful direction of the company’s R&D programs. The development of allogeneic CAR-T cells may circumvent issues with manufacturing and the costs associated with autologous CAR-T cells, which have already demonstrated impressive durable responses in patients with haematological malignancies. The company has also initiated early stage clinical trials for its immuno-oncology program, which is based on the development of allogeneic chimeric antigen receptor T (CAR-T) cells targeting well-characterised targets in haematological malignancies, such as CD19+ and B cell maturation agent. CRISPR Therapeutics has already published promising data on the use of CRISPR in β-thalassemia and sickle cell disease in the New England Journal of Medicine. Expanding our services to encompass screening of both primary T and B cells is another example of our commitment to apply decades of gene editing experience in support of drug discovery and development for the treatment of human disease.CRISPR Therapeutics has the largest market cap of the three, at $10.9B, with a clinical development program that is more advanced than those of Intellia and Editas. Terry Pizzie, CEO, Horizon Discovery said: “The interest in harnessing the immune system for effective therapies continues to grow, with the global cell therapy market predicted to reach $8.21bn by 2025*. The new B cell screening service, the first of its kind in the market, will enable researchers to identify genes that affect the function of B cells and examine how this impacts other immune cell types, particularly in infectious diseases, cancer, and auto-immune disorders, such as COVID-19, Burkitt’s lymphoma and multiple sclerosis respectively. Horizon has already applied its gene editing and cell culture expertise to maintain the viability of primary human T cells to enable functional genomic screens and has been delivering data-rich information to customers working in drug discovery and development. However, working with these cells brings scientists one step closer to healthy or diseased micro-environments, enabling them to better understand disease etiology and therapeutic mechanisms, and thereby advance drug discovery and development programs. Primary human cells, which are cells that are freshly isolated from donors, are known to be difficult to study in the lab. CAMBRIDGE, England-( BUSINESS WIRE)-Horizon Discovery Group plc (LSE: HZD) (“Horizon”, “the Company” or “the Group”), a global leader in the application of gene editing and gene modulation for cell line engineering, today announced the addition of an arrayed CRISPR knockout screening service for primary human B cells to its cell-based screening services.
0 kommentar(er)
