CMN Weekly (4 July 2021)
By: Karen O'Hanlon Cohrt - Jul. 4, 2021
- CRISPR reached a massive milestone this past week, with the first ever clinical data from Intellia Therapeutics and Regeneron demonstrating safety and efficacy for an in vivo CRISPR therapy in humans. The new therapy is NTLA-2001, which is being developed for hereditary transthyretin (TTR) amyloidosis with polyneuropathy (ATTRv-PN). Read our coverage of the story here.
- Patient story from one of the partipicants of Intellia's and Regeneron's NTLA-2001 trial for transthyretin amyloidosis.
- Researchers from Harvard University’s Weiss Institute have developed a CRISPR-based wearable diagnostics technology that enables SARS-CoV-2 detection through a face mask. The findings were published in the journal Nature Biotechnology on Monday and CRISPR Medicine published an interview with two of the lead authors on the same day.
- Precision BioSciences and SpringWorks Therapeutics dose the first patient in an expanded Phase 1/2 trial to evaluate PBCAR269A with Nirogacestat in patients with relapsed or refractory multiple myeloma. PBCAR269A is an allogeneic BCMA-targeted CAR T cell therapy developed using Precision’s meganuclease genome-editing platform, Arcus.
- Precision BioSciences also reports dosing the first patient in a Phase 1 for its allogeneic immune evading stealth cell CAR T candidate PBCAR19B for relapsed or refractory Non-hodgkin lymphoma.
- Apellis and Beam Therapeutics announce 5-year research collaboration to apply Beam’s proprietary base-editing platform to six research programmes for tissues modulated by the complement system, including the eye, liver, and brain. The collaboration brings together Apellis’ complement expertise and Beam’s expertise in base editing.
- The FDA has granted fast track designation to Allogene Therapeutics for ALLO-605, the first allogeneic TurboCAR™ T cell therapy, for the treatment of relapsed or refractory multiple myeloma.
- Dublin-based ERS Genomics Limited and Japan-based Nippon Gene Co., Ltd announced a non-exclusive license agreement that sees Nippon Gene gain access to ERS Genomics’ CRISPR-Cas9 patent portfolio. Nippon Gene is a manufacturer of genetic engineering research reagents and diagnostic reagents for humans, animals, and plants, The deal will allow Nippon to develop, manufacture, and sell research reagents using CRISPR-Cas9 technology
- Graphite Bio announces closing of upsized initial public offering and full excercise of underwriters’ option to purchase additional shares.
- Intellia Therapeutics announces pricing of public offering of common stock.
- Berkeley and California-based biotech drug company Caribou Biosciences files for IPO to help fund CRISPR-based drug development studies for cancer treatments, and plans to list under the ticker “CRBU” on the Nasdaq.
- A team of U.S. scientists has developed a CRISPR-based method with the goal of studying how eukaryotic cells repair DNA in space. The rationale behind the work is that astronauts travelling beyond outside the Earth’s magnetosphere are at an increased risk of DNA damage from ionising radiation. The new method uses Cas9 to create precise DNA breaks so that DNA repair pathways can be studied in more detail than would be possible using random DNA damage protocols such as radiation. The results were published in PLOS ONE this week.
- A team at Brigham and Women’s Hospital (Boston) and collaborators has developed a designer yeast probiotic to help the management of inflammatory bowel disease. The probiotic was developed using CRISPR-Cas9 to introduce genes that could sense inflammation and respond to it by secreting an enzyme with anti-inflammatory activity. Preclinical results with the new probiotic, dubbed Y-bots' (yeast robots), were published in Nature Medicine this week.
- Scientists at the University of Texas at Austin have developed a series of PEGylated chitosan/CRISPR-Cas9 dry powders for pulmonary delivery via thin-film freeze-drying. The results were published online ahead of print in the International Journal of Pharmaceutics.
Webinars and events
- In case you missed them, you can catch CRISPR Medicine News webinars on-demand with a CMN+ subscription. Check out our webinar list here and read more about CMN+ benefits here.
- CRISPR Tsunami: A 1-day introductory course to computational Cas9-gRNA design takes place virtually on 10th September 2021. The course is run by the University of Copenhagen, Denmark, doesn’t assume prior knowledge about gRNA design and is free of charge. Read more about it here.
- CRISPR-Associated (CAS) Effectors Delivery via Microfluidic Cell-Deformation Chip. This review focuses on the recent advancements of microfluidic systems to deliver CRISPR-Cas systems in clinical and therapy investigations.
- CRISPR-Cas, a Revolution in the Treatment and Study of ESKAPE Infections: Pre-Clinical Studies. This review summarises the various strategies for the treatment and study of molecular mechanisms of antimicrobial-resistant bacteria in the so-called ESKAPE group of pathogens (Enterococcus spp., Staphylococcus aureus, Klebsiella pneumoniae, Acinetobacter baumannii, Pseudomonas aeruginosa and Enterobacter spp.) based on CRISPR and related genome-editing technologies.
Podcasts and tidbits
- In the latest episode of The Readout LOUD,” STAT’s biotech podcast, George Yancopoulos, head scientist at Regeneron Pharmaceuticals, talks about the future of CRISPR genome editing.
- Fighting Cancer with CRISPR: A snappy 10 min video from Bloomberg TV explaining the transformation currently taking place in healthcare and biotech industries involving CRISPR.
Transfusion Dependent Beta-Thalassaemia, TDT, (NCT04925206)
EdiGene (GuangZhou) Inc.
EdiGene (GuangZhou) Inc.
Multiple myeloma, MM (NCT04960579)
Poseida Therapeutics, Inc.
Poseida Therapeutics, Inc.
View all clinical trials
Acute Myeloid Leukemia, AML, (NCT04849910)