Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
At the 30th Annual Congress of the European Society for Gene and Cell Therapy in Brussels this week, researchers presented both preclinical and clinical strategies for applying gene therapy to a functional HIV cure. At a Wednesday session on Infectious Diseases & Vaccines, Alessio Nahmad, of Tabby Therapeutics Ltd., described using B cells edited to express broadly neutralizing antibodies (bnAbs) 3BNC117 to deliver high titers of antibodies in mice.
Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).
Oxford Nanopore Technologies plc secured a £70 million (US$85 million) investment from Biomérieux SA. The investment comes on the back of a partnership formed between the two companies earlier in the year. The funds will support the development of products in Oxford Nanopore’s portfolio to serve in vitro diagnostics (IVD) markets.
Activated phosphoinositide 3-kinase δ syndrome type 1 (APDS1) is a rare disease caused by gain-of-function (GOF) mutations in the PIK3CD gene that presents with combined immunodeficiency due to decreases in IgA, IgG, naive CD4 and naive CD8 cells. Nearly all APDS1 patients suffer from recurrent respiratory tract infections with most presenting with bronchiectasis and chronic viral infections.
Bloomsbury Genetic Therapies Ltd. has announced U.S. and E.U. orphan drug designations for BGT-NPC, an investigational gene therapy for the treatment of Niemann-Pick disease type C (NPC). BGT-NPC is an investigational AAV9 gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid.
Rejuvenate Bio Inc. has released new preclinical efficacy data for its gene therapy, RJB-0402, in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
Kriya Therapeutics Inc. has entered into an exclusive license, collaboration and supply agreement with Everads Therapy Ltd. to advance Kriya’s portfolio of gene therapies for retinal diseases using Everads’ suprachoroidal delivery device.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
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