CMN Weekly (5 August 2022) - Your Weekly CRISPR Medicine News
By: Gorm Palmgren - Aug. 5, 2022
- Prime editing and somatic cell nuclear transfer (SCNT) has been used to correct a pathogenic mutation in dogs and to produce two cloned puppies carrying the fixed mutation. Researchers from South Korea used the gene-editing method in fibroblasts from a purebred Labrador retriever to correct a T to C point mutation in the BICF2S23030416 locus believed to cause canine hip dysplasia (HD). This disease excludes dogs from breeding programs. The corrected mutation was confirmed in both puppies, while no off-target mutations were identified.
- Chinese researchers have used CRISPR-Cas9 gene editing of the BCL11A enhancer to alleviate pediatric transfusion-dependent β-thalassemia. The research is part of an ongoing clinical trial and involves transplantation of BCL11A enhancer-edited, autologous, hematopoietic stem and progenitor cells into two diseased children. Both children achieved transfusion independence for >18 months after treatment, and their haemoglobin concentration increased.
- American researchers have developed a CRISPR-dCas9-based modular epigenetic toolkit that can modify multiple epigenetic marks in a target-specific manner. Researchers can use the toolkit to understand the contributions of specific histone marks on gene expression by screening a large promoter region and identifying differential outcomes with high base-pair resolution.
- A Chinese study describes an optimised strategy for targeting de novo DNA methylation with CRISPR-Cas9. The method uses the homology-directed repair (HDR) pathway to target the vital epigenetic biomarker, O6-methylguanine-DNA methyltransferase (MGMT).
- SNIPR Biome has been granted a new patent (US11,400,110) that covers lytic phages armed with CRISPR gene editing systems. The patent supports the company’s pipeline and lead program (SNIPR001). It is a CRISPR-armed bacteriophage cocktail that targets antibiotic-resistant E. coli infections in haematological cancer patients.
- The FDA has granted Cellectis clearance for its Investigational New Drug (IND) application to initiate a Phase 1/2a clinical trial of UCART20x22 for patients with relapsed or refractory Non-Hodgkin Lymphoma (r/r NHL). The company plans to enrol patients in the study (NatHaLi-01) in the year's second half.
- Poseida Therapeutics has announced a global strategic collaboration with Roche to develop allogeneic CAR-T therapies for hematologic malignancies. Under the agreement, Roche will receive from Poseida either exclusive rights or options to develop and commercialise a number of allogeneic CAR-T programs. In addition, Poseida will receive $110 million upfront and potentially up to $110 million more.
- Poseida Therapeutics also announced the pricing of an underwritten public offering of 20,000,000 shares of its common stock at a public offering price of $3.50 per share, totalling approximately $70.0 million.
- Several CRISPR companies have announced financial results for the second quarter of 2022: Cellectis, bluebird bio, Sangamo, Fate Therapeutics and Intellia Therapeutics.
- Researchers in South Korea have developed a CRISPR-Cas12a-based diagnostic method for multiple genotypes of severe fever with thrombocytopenia syndrome virus (SFTSV). The technique utilises reverse transcription recombinase polymerase amplification (RT-RPA) to diagnose SFTSV infections in blood plasma in 50 min without cross-reactivity to other viruses.
- A new method uses CRISPR-Cas12a for single-molecule counting fat mass and obesity-associated protein (FTO) in human breast tissues. The procedure is based on rolling circle transcription amplification and can accurately discriminate between healthy persons and breast cancer patients.
- A mini-review by researchers from Pakistan describes the CRISPR-Cas12c system that other scientists recently described. Cas12c is a noncleaving DNA binder with minimal PAM requirement.
- A Chinese review looks at the development and mechanism of CRISPR/Cas9 and its applications and challenges in gene therapy for eye diseases. In addition, strategies to control Cas9 activity for improved safety and the general risks of genome editing are discussed.
- Researchers in Australia have authored a review about the recent CRISPR applications for Duchenne muscular dystrophy (DMD). The study focuses on the various CRISPR-Cas9 strategies for correcting mutations in the disease-causing Dmd gene and discusses using CRISPR-generated animal models of DMD.
- Researchers from China study the current efforts to use CRISPR-Cas9 gene editing in obesity management. The review elaborates on the molecular basis of obesity, risk factors, types of gene therapy, possible mechanisms, and advantages of the CRISPR–Cas9 system over other methods.
- In a Perspective in the CRISPR Journal, American researchers discuss how CRISPR has impacted several aspects of the drug discovery workflow. The authors also look at the future of this gene-editing technology.
News from CRISPR Medicine News
- Thursday, we wrote about the recent announcement from Editas Medicine that they have successfully engrafted the first patient dosed with the CRISPR-based therapeutic candidate EDIT-301 for treating sickle cell disease (SCD). EDIT-301 works by reactivating foetal haemoglobin (HbF) production, compensating for the lack of functional adult haemoglobin in individuals with SCD.
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