CMN Weekly (14 May 2021)

Top pick

• Efficient and gentle delivery of Cas9–sgRNA RNPs into cells has been obtained by a new technology called progressive mechanoporation (PM). PM is based on a polymer biochip that contains a series of microchannels where each microchannel is narrower than the previous one. When cells are passed through these channels, pores are temporarily formed in their cell membranes. The method is also applicable for the introduction of drugs and other small molecules.

Research

• Researchers in China have developed two new Cas13d orthologs for sensitive analysis of single nucleotide variation in RNA. The new method can detect RNA in the picomolar range and might be helpful for efficient molecular diagnostics.
• A novel genetic sensor called a "CopyCatcher" uses CRISPR-based gene drive technology to detect instances in which a genetic element is copied precisely from one chromosome to another throughout cells in the body. The method has been tested in fruit flies and human cells and might eventually allow scientists to make desired precise genome edits throughout the body.

Industry

• Cellectis and Sanofi have partnered on using the anti-CD52 monoclonal antibody alemtuzumab to develop further Cellectis' CAR T cell programme employing the TALEN gene-editing technology.
• Vertex Pharmaceuticals and CRISPR Therapeutics have announced that they will report new clinical data on their CTX001 CRISPR-based treatment for sickle cell disease and β-thalassemia. The data will be presented in an e-poster at the Annual European Hematology Association Virtual Congress on 11 June.
• Precision Biosciences has reported promising results from its proprietary ARCUS gene-editing technology to treat transthyretin amyloidosis (ATTR). ATTR is a rare disease that leads to peripheral neuropathy and cardiomyopathy.
• Poseida Therapeutics has presented encouraging preclinical data across its CAR T and gene therapy programmes. The company employs a high-precision Cas-CLOVER gene-editing technology that utilises an inactivated Cas9 fused to a nuclease called Clo51.

Quarter 1 industry update highlights

• Precision BioSciences has reported first-quarter 2021 financial results and business updates. The company has begun its PBCAR19B phase 1 study on allogeneic CAR T cells and is advancing on three other phase 1/2a clinical studies. It is also about to file an IND application for treatment of R/R multiple myeloma.
• Beam Therapeutics reports a substantial increase in first-quarter R&D spending of $190.1 million, up from $21.5 in 2020. The company also reported new promising data on its proprietary lipid nanoparticle (LNP) formulation and base editing programmes.

COVID-19

• Sherlock Biosciences has licensed its COVID-19 diagnostic tests using CRISPR technology to Rokline Health Concepts. Rocklin Health Concepts will develop, manufacture and commercialise the tests for worldwide distribution.

Reviews

• The use of CRISPR for epigenetic modifications to endogenous DNA and histones and to manipulate the architecture of native chromatin is the subject of a review in Trends in Biotechnology. The authors discuss technical improvements in the field and how researchers could improve these technologies for greater future utility.
• A new review in the Journal of Analysis and Testing highlights the role of nucleic acid nanotechnology in CRISP-Cas9 systems. The review addresses delivery of Cas proteins by DNA nanovehicles, conditional CRISPR-Cas system based on dynamic RNA nanotechnology, and coupling of CRISPR-Cas and DNA origami.
• Another review focuses on CRISPR-based diagnostics assays assisted by nanotechnology hardware. Among the included technologies are nanoprobes, nanochips, nanodevices, microfluidic technology, and lateral flow strips.

Conferences

• The 24th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) ends today, and you can access all 898 abstracts as well as a recorded 'fireside chat' with Jennifer Doudna on the conference website.

Opinion and People

• The online media NPR has interviewed two of the first patients who received a CRISPR based treatment for Leber congenital amaurosis. This disease destroys light-sensing cells in the retina and causes blindness. It is still too early to say if the treatment will restore vision, but the two patients are very optimistic. The treatment was part of the Phase 1/2 clinical trial LCA10 sponsored by Editas Medicine.
• An article on WebMD addresses a disability activist's fear of CRISPR. Rebecca Cokley sidelines CRISPR with eugenics that has historically been embraced to weed out 'undesirable traits'. She asks if autism and a potential gay gene will be the next targets for gene editing. She also questions the distinction between disabilities and differences and points out that not all people with disabilities want to be cured.