CMN Weekly (10 September 2021)

Top picks

• A team of researchers led by Dr. Stanley Qi at Stanford University has developed CasMINI, an engineered multifunctional Cas protein that is much smaller than those currently in clinical development. The work was published in Molecular Cell on Friday. In an interview with CRISPR Medicine News, Stanley Qi explains how Cas:MINI was engineered and why it makes a difference to the genome-editing field.
• The business of editing genes to battle disease is bringing in record funding. In this piece for Marketplace, Janet Lambert, CEO of the Alliance for Regenerative Medicine, discusses CRISPR, the surge of investment and the progress being made in developing treatments.
• Wageningen University & Research, Netherlands, announced this week that it will provide non-profit organisations with free licences to use its CRISPR–Cas gene-editing technology for non-commercial applications. Read more about this in a Nature Editorial published shortly afterwards.

Clinical

• Editas Medicine plans to share initial clinical data from its BRILLIANCE trial of EDIT-101 for the inherited retinal disease Leber congenital amaurosis (LCA10) at the International Symposium on Retinal Degeneration in September. The oral presentation will include patient safety assessments and a preliminary analysis of secondary endpoints relating to signals of gene editing and clinical benefit.
• EdiGene announces clinical sites activation and enrolment of the first patient in it multicenter Phase I trial for ET-01, an investigational gene-editing haematopoietic stem cell therapy for transfusion-dependent β-thalassemia.

Industry

• Caribou Biosciences announces publication of data demonstrating high specificity genome editing with its proprietary chRDNA technology that the company is advancing for a pipeline of allogeneic CAR-T cell therapies. The findings were published recently in Molecular Cell.
• Caribou Biosciences also recently shared a business update and reported second quarter 2021 financial results. Highlights include an update on the ANTLER Phase 1 clinical trial of CB-010 in relapsed or refractory B cell non-Hodgkin lymphoma and details of the company's successful IPO round.
• Mammoth Biosciences raises $195 million to build next-generation CRISPR products in therapeutics and diagnostics. The funding will be used to expand the company's novel CRISPR platform, including its proprietary ultra-small Cas14 and Casɸ CRISPR systems for in vivo gene-editing therapeutics.
• Precision BioSciences and Tiziana Life Sciences announce exclusive license agreement to evaluate Foralumab, a novel, fully human Anti-CD3 monoclonal antibody, in conjunction with allogeneic CAR-T candidates for cancer
• iECURE launches in close collaboration with University of Pennysylvania with $50 million Series A financing to develop in vivo gene insertion approaches for devastating diseases with unmet need.
• Precision BioSciences and iECURE announce license and collaboration agreement to develop ARCUS-based gene- editing therapies. Under the agreement, iECURE will license Precision’s PCSK9-directed ARCUS nuclease and pursue four gene-insertion programmes focused on liver diseases. Precision will retain the righs to all indications except for those licensed iECURE.
• Eli Lilly is partnering with ProQR Therapeutics in a programme that's centered on the Dutch biotech company's technology for editing RNA. The two companies will use ProQR's "Axiomer" platform, which is designed to allow editing of single nucleotides within RNA, and the collaboration will encompass five potential treatments for liver and nervous system conditions.

Research

• Scientists from multiple institutions in the US have stablished an in vivo strategy to evolve and stringently select capsid variants of adeno-associated viruses (AAVs) that enable potent delivery to desired tissues. Using this approach, the team has identified a class of RGD motif-containing capsids that transduces muscle with superior efficiency and selectivity after injection into mice and non-human primates. Experiments in mouse models of genetic muscle disease revealed that the engineered vectors had enhanced potency and therapeutic efficacy compared to naturally occurring AAV vectors. The work was published in Cell yesterday.
• Researchers from the US and Switzerland demonstrate programmable RNA targeting in mammalian cells with the single-protein CRISPR effector Cas7-11, a class 1 CRISPR–Cas system that originates from the fusion of a putative Cas11 domain and multiple Cas7 subunits. The findings were published in Nature earlier this week.
• Researchers at University of California San Diego have developed pgSIT, a new scalable genetic control system that uses a CRISPR-based approach to engineer deployable mosquitoes that can suppress populations of malaria-carrying mosquitoes. The findings were published in Nature Communications today.
• Research led by Feng Zhang at Massachusetts Institute of Technology has uncovered a widespread class of transposon-encoded RNA-guided nucleases which they name OMEGA (Obligate Mobile Element Guided Activity), with strong potential for biotechnological development. The team made the discovery while exploring the evolutionary origins of an enzyme used in the CRISPR genome-editing system. The findings were published in Science yesterday.

Podcasts and webinars

• Base Editing for Therapeutics: Beam Therapeutics CEO John Evans talks to Luke Timmerman on the latest episode of the The Long Run podcast.

Reviews

• Digging into the lesser-known aspects of CRISPR biology. This review summarises the present knowledge on the diversity, molecular mechanisms and biology of CRISPR-Cas systems, and pays special attention to recent findings regarding CRISPR biology.

COVID-19

• An article published in Science Advances this week describes a universal bacteriophage T4 nanoparticle platform to design multiplex SARS-CoV-2 vaccine candidates by CRISPR engineering. Using bacteriophage T4, the researchers behind the work engineered a pipeline of vaccine candidates by incorporating various viral components into appropriate compartments of the phage nanoparticle. Without any adjuvant, the vaccine stimulated robust immune responses in animals.
• Researchers in China have developed a new CRISPR-Cas12a-based diagnostic platform through a Raman transducer generated by Raman enhancement effect. The new platform is called SERS-CRISPR (S-CRISPR), and it uses high-activity noble metallic nanoscopic materials to increase the sensitivity of detection of SARS-CoV-2 RNA nucleic acids, without the need for amplification. The findings wre published in Journal of Nanotechnology this week.

Jobs

• Looking for a job in CRISPR? Then look no further than CMN Jobs where we regularly add new positions within CRISPR medicine, gene therapy and cell therapy. Check out the current listings right here.