An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.
Intellia Therapeutics has early clinical trial data showing that its CRISPR-based therapy for a rare disease can edit genes inside the body safely and effectively. The trial is ongoing, but the preliminary results suggest that Intellia’s approach could offer a cure.
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Vertex Pharmaceuticals is collaborating with Obsidian Therapeutics to develop up to five new gene editing therapies. Obsidian’s technology precisely controls protein expression, a capability the partners plan to apply to several undisclosed serious diseases.
Vertex Pharmaceuticals is amending its collaboration with CRISPR Therapeutics, paying $900 million now for chance to grab a bigger share of a gene-editing therapy’s profits later---if the treatment is approved. Clinical trials evaluating the CRISPR-based therapy are underway in sickle cell disease and beta thalassemia.
The prize went to Jennifer Doudna of the University of California Berkeley and Emmanuelle Charpentier of the Max Planck Institute. Charpentier published her research on the biology behind the technology in 2011 and collaborated with Doudna. The two discovered it was possible to control the mechanism.
Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
A team at UPenn led by CAR-T pioneer Carl June used CRISPR/Cas9 to engineer T cells for treating multiple myeloma and sarcoma. Data on three patients were published in Science. All the patients' cancers ultimately progressed, though none had cytokine release syndrome.
The company was founded to develop base editing, a form of gene editing that alters genes at the single-letter level rather than using the "cutting" technique typical of CRISPR/Cas9. Beam had filed to go public in September, aiming at raising $100 million.
One of the four sites of the trial, in a rare form of inherited blindness, is currently listed as open for enrollment, and the companies plan to dose the first patient soon.
The company, partnered with Beam Therapeutics, Verily Life Sciences and academic institutions, plans to use gene editing to tackle coronary artery disease. However, experience with gene therapy indicates cost could be a hurdle.
A new report from Relatient, A Data-Driven Guide to Patient Access Succes, highlights how focusing on data accuracy and relevance can enhance the performance of healthcare practices.
The company aims to develop faster, lower-cost diagnostics using the gene-editing technology that could be used in oncology, infectious diseases, low-resource settings and other areas.
NPR reported that the two patients, one with multiple myeloma and one with sarcoma, had received the gene-edited cell therapy as part of a Phase I trial.
The 18 signatories emphasized that the moratorium wouldn't constitute a ban or include gene editing for research or treatment of diseases, but called on nations to develop a framework to govern the practice.
The company is developing means using CRISPR/Cas9 to edit genomes at the single-letter level, as opposed to the DNA- and RNA-cutting method usually employed.
The Phase I/II trial is in beta-thalassemia, a rare blood disorder. Another Phase I/II study of the same drug, in sickle cell disease, is expected to infuse its first patient in mid-2019.