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| | Your Missing Links are HereTop Picks - The ultimate guide to guide RNAs helps you flatten the gRNA learning curve and select the best one for your choice of experiment. This piece in Nature Methods gives you a wealth of information and recommendations on how to design your gRNA for both general and specific purposes like CRISPR interference (CRISPRi), base editing, prime editing etc.
Research Industry Conferences Clinical - Intellia Therapeutics was recently awarded the green light to begin a clinical trial of an experimental CRISPR-based treatment for the rare, inherited disease transthyretin amyloidosis (ATTR). It is an in vivo that is potentially curative, and it has been dubbed NTLA-2001. Now, the small Phase 1 study has been kicked off, and the very first patient has been injected intravenously with NTLA-2001 to assess its safety, tolerability, pharmacokinetics and pharmacodynamics.
- The clinical-stage biopharmaceutical company Cellectis has announced, that the company will soon present data from their Phase 1 dose-escalation study of UCART22, a TALEN-based product to treat adult patients with relapsed/refractory CD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL). The data will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting that kicks off virtually on December 5, 2020. You can read more about the clinical study here.
- Fate Therapeutics has started enrollment of their multi-centre Phase 1 study of FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy for the treatment of relapsed/refractory acute myeloid leukaemia (AML). The announcement was presented in the company's financial report for the 3rd quarter of 2020.
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