CRISPR-Cas9 gene editing technology-based therapeutic / Vertex, CRISPR Therap - LARVOL DELTA

Exagamglogene Autotemcel for Severe Sickle Cell Disease. (PubMed, N Engl J Med) - P2/3 | "Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.)."

Journal • Genetic Disorders • Hematological Disorders • Oncology • Sickle Cell Disease • Transplantation • CD34

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. (PubMed, N Engl J Med) - P2/3 | "Treatment with exa-cel, preceded by myeloablation, resulted in transfusion independence in 91% of patients with transfusion-dependent β-thalassemia. (Supported by Vertex Pharmaceuticals and CRISPR Therapeutics; CLIMB THAL-111 ClinicalTrials.gov number, NCT03655678.)."

Journal • Beta-Thalassemia • Genetic Disorders • Oncology • Transplantation • CD34

Bacterial genome engineering using CRISPR-associated transposases. (PubMed, Nat Protoc) - "Clustered regularly interspaced short palindromic repeats (CRISPR)-associated transposases have the potential to transform the technology landscape for kilobase-scale genome engineering, by virtue of their ability to integrate large genetic payloads with high accuracy, easy programmability and no requirement for homologous recombination machinery...We further describe a computational CRISPR RNA design algorithm to avoid potential off-targets, and a CRISPR array cloning pipeline for performing multiplexed DNA insertions. The method presented here allows the isolation of clonal strains containing a novel genomic integration event of interest within 1-2 weeks using available plasmid constructs and standard molecular biology techniques."

Journal • Review • Infectious Disease

Uncovering the roles of dihydropyrimidine dehydrogenase in fatty-acid induced steatosis using human cellular models. (PubMed, Sci Rep) - "Dihydropyrimidine dehydrogenase (DPYD) is an enzyme responsible for uracil and thymine catabolism, and DPYD human genetic variability affects clinically observed toxicity following 5-Fluorouracil administration. Additionally, a gain-of-function mutation of DPYD, created through clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR-Cas9) engineering, led to an increased lipid burden, which was associated with altered mitochondrial functionality in a hepatocarcionma cell line. The studies presented herein describe a novel role for DPYD in hepatocyte metabolic regulation as a modulator of hepatic steatosis."

Journal • DPYD

CRISPR Therapeutics Provides Business Update and Reports First Quarter 2020 Financial Results (GlobeNewswire, CRISPR Therapeutics AG) - "...collaboration agreement with Vertex to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1), CRISPR Therapeutics received a payment of $25 million from Vertex related to the achievement of a research milestone in the DM1 program. CRISPR Therapeutics is eligible to receive additional milestone payments from Vertex of up to $800 million for these two programs."

Grant • Licensing / partnership • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy • Myotonic Dystrophy