CMN Weekly (7 May 2021)

Some of the best links we picked up around the internet

By: Karen O'Hanlon Cohrt - May. 7, 2021

Top picks

  • Researchers in Germany have developed a multiplexable diagnostic platform known as LEOPARD (Leveraging Engineered tracrRNAs and On-target DNAs for PArallel RNA Detection). LEOPARD allows simultaneous detection of RNAs from different viruses in one test and could distinguish SARS-CoV-2 and its D614G variant with single-base resolution in patient samples. The findings were published in Science last week.
  • CRISPR’s Next Frontier: Treating Common Conditions. Popular piece from Wall Streeet Journal that discusses how gene editing might not just help people with rare disorders but millions of others with heart disease, chronic pain and other conditions, in the decade to come.

Industry

Quarter 1 industry update highlights

  • Editas Medicine: Initial clinical data for the first in vivo CRISPR therapy EDIT-101 in Phase 1/2 BRILLIANCE trial for the treatment of LCA10 is expected later this year. The company also plans to progress clinical studies for Cas12-edited EDIT-301 in haemoglobinopathies this year.
  • Fate Therapeutics: Highlights include a positive financial update, and that Phase 1 data from the FT516 and FT538 programmes in relapsed and refractory acute myeloid leukaemia (AML) is expected in conjunction with the ASGCT meeting next week.
  • Intellia Therapeutics expects to submit an IND or equivalent for its CRISPR-edited T cell therapy NTLA-5001 for AML in mid-2021. Initial safety and activity data from a Phase 1 trial for NTLA-2001 is also expected mid-2021. NTLA-2001, developed in partnership with Regeneron, is the first systemically delivered CRISPR-based therapy, and is designed as a single-dose cure for the rare genetic disease transthyretin amyloidosis.
  • Sangamo Therapeutics: The company received fast track designation from the FDA for SAR445136 (formerly known as BIVV003) in Q1. SAR445136 is a zinc finger nuclease (ZFN)-edited cell therapy candidate for the treatment of sickle cell disease, developed in collaboration with Sanofi. The European Medicines Agency (EMA) has also granted orphan designation to SAR445136 based on early clinical data from three treated patients.
  • Allogene Therapeutics: Data expected on ALLO-501 ALPHA and ALLO-501A ALPHA-2 trials at the American Society of Clinical Oncology (ASCO) Annual Meeting in June. Allogene has also initiated its first solid tumour trial (TRAVERSE) for ALLO-316 (anti-CD70 CAR T) in advanced or metastatic renal cell carcinoma.

Research

  • Scientists from the Wyss Institute at Harvard have created a new gene-editing tool called retron library recombineering (RLR) that might rival CRISPR. RLR is a high-throughput method with multiplex capabilities that exploites the targeted reverse-transcription activity of retrons to produce ssDNA in vivo. The method was shown to incorporate edits at >90% efficiency. The findings were published in PNAS this week. You can also catch a writeup of the study at Wyss Institute's website.
  • Researchers at Duke University retrospectively examined sequence features that impact gRNA activity in 39 published data sets, and uncovered strong evidence that the genomic context greatly influences differences in gRNA-dependent activity in CRISPR editing. The authors defined context as the DNA content outside of the gRNA/target sequence and hope that the findings will contribute to better understanding of Cas9 activity and optimisation of gRNAs and Cas variants. The results were shared on bioRxiv yesterday.
  • A team in China has developed TB-QUICK, a DNA detection platform for Mycobacterium tuberculosis that combines loop-mediated isothermal amplification (LAMP) and CRISPR-Cas12b detection. Initial results reveal potential for rapid and sensitive detection in pulmonary and plasma samples but the clinical application needs to be assessed further. The results were published in the Journal of Infection this week.
  • Researchers demonstrate ligand-induced activation of CRISPR-Cas9 function by integrating a theophylline aptamer into protein-unrecognised regions of gRNA. This design of allosteric regulation creates an opportunity for conditional control of CRISPR-Cas9 function using specially designed gRNAs. The findings were published in Chemical Communications earlier this week.

COVID-19

Delivery

  • Overview and Update on Methods for Cargo Loading into Extracellular Vesicles: Review that summarises and discusss the pros and cons of methods used for cargo loading into extracellular vesicles, including CRISPR/Cas9, mRNAs, proteins, and othe cargoes. Extracellular vesicles are naturally-occuring phospholipid bilayer-enclosed vesicles secreted from all cell types. They interact intrisically with the plasma membrane and are gaining much attention as a natural drug carrier.

Webinars

Conferences

Awards

Ethics

  • He Jiankui´s gene-editing experiment and the non-identity problem: a bioethics analysis of He Jiankui's case in relation to one of the most difficult problems in procreative ethics (or the ethics of future generations): the non-identity problem. The non-identity problem was introduced by British philosopher Derek Parfit in the 1980s and it is said to arise when our actions in the present could change which people will exist in the future, for better or worse.

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