Your missing links are here (9 October 2020)
By: Karen O'Hanlon Cohrt - Oct. 9, 2020
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Top Picks
- It’s been a massive week for CRISPR and for women in science as Emmanuelle Charpentier and Jennifer A. Doudna took home the Nobel Prize in Chemistry 2020 for their outstanding work on CRISPR. Read the official announcement from the Nobel Prize Organisation here. We also wrote a short story about CRISPR’s timeline to mark the occasion.
- Scribe Therapeutics, a spin out from Jennifer Doudna’s lab at UC Berkeley, unveiled its vision this week for highly efficient and specific in vivo genome modification for treatment of disease. Scribe grew out of interest in Huntington’s disease in 2018 and is headed by CEO and co-founder Benjamin Oakes, along with VP of Platform and co-founder Brett T. Staahl. The company recently landed $20 million in series A funding to develop its platform, and has partnered with Biogen to advance its technology for the treatment of amyotrophic lateral sclerosis (ALS).
Research
- Researchers in Spain target fusion oncogenes in cancer cells with CRISPR-Cas9. Targeting these tumour-specific genes is an attractive approach to tackling cancer, and the researchers show proof-of-concept for this strategy in mice that developed tumours following injection with a human Ewing sarcoma cell line.
Industry
- Biotech stocks surged on Wednesday following the announcement of this year’s Nobel Prize in Chemistry to CRISPR pioneers Emmanuelle Charpentier and Jennifer A. Doudna. Among the big players, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics went up by 13%, 11%, and 6% respectively, and CRISPR Therapeutics, co-founded by Emmanuelle Charpentier, was also up by more than 11% as of Wednesday morning (7th Oct).
- Sherlock Biosciences, the first company to obtain FDA approval for a CRISPR-based product (SHERLOCK) earlier this year now launches 221b Foundation to address the COVID-19 pandemic and equity in science, technology, engineering, and math (STEM). Simply put, profits from Sherlock’s (or any 3rd party) sales of SHERLOCK CRISPR COVID-19 products will be put into the new foundation to support racial and gender diversity in the STEM areas.
Delivery & Safety
- Californian genome engineering company Synthego announced its new foundational technology for standardised precision and control of CRISPR-based gene editing inside cells using light. The technology deploys a newly-developed class of guide molecules known as CRISPRoff™, which fragment in response to light, enabling precise temporal and spatial control of double-stranded DNA breaks created during gene editing. You can read the full research article in Nature Communications.
- Efficient and safe delivery of gene-editing cargo is a challenge in the development of CRISPR-based therapeutics. Viral methods are the gold standard but non-viral methods are gaining traction as an alternative. This discussion piece looks at the pros and cons of each approach and provides insights based on recent findings.
- Danish company Samplix launches new quality control service for validation of CRISPR samples. Using its proprietary genomic enrichment technology XdropTM followed by next sequencing, the company is able to reconstruct any structural rearrangements, SNPs or other unintended modifications that result from editing with CRISPR-Cas technology.
COVID-19
- A consortium of researchers in the US reports an amplification-free CRISPR-Cas13a-based mobile phone assay for direct detection of SARS-CoV-2 in 5 minutes from nasal swab RNA extracts. Authors claim the assay “can provide rapid, low-cost, point-of-care screening to aid in the control of SARS-CoV-2” and share their results on the preprint server medRxiv.
- Feng Zhang’s Lab at the Broad Institute describe a simple test for SARS-CoV-2 detection. The new test, STOP, or Sherlock testing in one pot, combines a simplified magnetic-bead RNA isolation protocol with isothermal amplification and CRISPR-Cas12a-mediated detection. The team could run the assay at a single temperature in less than 1 hour and with minimal equipment with increased sensitivity over the CDC’s gold-standard RT-PCR test. Read the findings in the New England Journal of Medicine here.
- BBC write that India’s new COVID-19 test FELUDA, which will be made by leading Indian conglomerate, Tata, “could be the world's first paper-based Covid-19 test available in the market”. The paper test gives an answer in 45 minutes and has similarities to other CRISPR-based COVID19 test developed by Mammoth and Sherlock. Read the research findings on preprint server bioRxiv.
Reviews and Opinion
- Opinion piece about CRISPR in The New York Times: “This Year’s Nobel Prize in Chemistry Honors a Revolution” by Walter Isaacson, author of the forthcoming “The Code Breaker: Jennifer Doudna, Gene Editing, and the Future of the Human Race.”
- Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors. A review article by Qatar-based researchers discussing recent efforts in developing novel non-viral delivery systems for CRISPR, with special focus on potential of peptide-based systems for cell-type specific delivery.
- Book Review of “Editing Humanity: The CRISPR Revolution and the New Era of Genome Editing” authored by Kevin Davies. Reviewed by Carl Zimmer for The New York Times.
Awards & Funding
- Gene Yeo, University of California San Diego, awarded Allen Distinguished Investigator funding for research on the mechanisms underlying neurodegenerative diseases.
Tags
CLINICAL TRIALS
IND Enabling
Phase I
Phase II
Phase III
Gastric Cancer and Colorectal Cancer, CRC, (NCT07166263)
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Acute Myeloid Leukemia, AML, (NCT06541444)
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
Sponsors:
Base Therapeutics (Shanghai) Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III







