Your missing links are here (13 November 2020)

Top 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

• A novel way to use a beam of light to deliver CRISPR gene therapy has been developed by researchers at University of New South Wales. The technology uses light illumination to disrupt liposomes that are loaded with the CRISPR molecules, thereby ensuring delivery at the right target and at the right time.
• American researchers have demonstrated Streptococcus pyogenes Cas9-ADAR2 fusion system that enables adenosine-to-inosine (A-to-I) editing of mRNAs. The system is as efficient and specific as previous Cas13 RNA editing versions and has the further advantage that Cas9 can act as an RNA-aptamer-binding protein.

Industry

• Beam Therapeutics has announced the advancement of a new allogenic CAR-T therapy for the treatment of T-cell acute lymphoblastic leukemia (T-ALL). The therapy, BEAM-201, is the first CRISPR-based cell therapy featuring simultaneous edits to four genes and preclinical data have demonstrated 96-99% editing efficiencies across all four targets.
• Fate Therapeuticsin San Diego, US has reported its third-quarter financial results including R&D expenses of $30.7 million and revenue of $7.6 million from collaborations with Janssen and Ono Pharmaceutical. The biopharmaceutical company has several CRISPR-based immunotherapies for cancer and immune disorders in the pipeline.
• The clinical-stage biopharmaceutical company Locus Bioscienceshas signed a contract with the global non-profit, Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) to fight the bacterial pathogen Klebsiella pneumoniae. The company has developed a CRISPR Cas3-enhanced bacteriophage (crPhage™) that has the potential to treat recurrent urinary tract infections caused by K. pneumoniae as well as E. coli.

Conferences

• The 62nd American Society of Hematology (ASH) Annual Meeting will kick-off virtually on December 5-8, 2020. You can expect no less than twelve presentations from Fate Therapeutics detailing data from their numerous gene-editing clinical trials and more. The clinical-stage biopharmaceutical company Cellectis will also present data from their Phase 1 dose-escalation study of UCART22.

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.