It has been previously demonstrated that intranasal treatment with COG-201, an AAV9-shRNA designed to target the down-regulation of the 5-HT2A receptor, significantly decreased anxiety and improved memory in mice and rats.
Engene Holdings Inc.’s recent disclosure of pivotal phase II data with nonviral gene therapy detalimogene voraplasmid (also known as detalimogene, and previously as EG-70) – along with a planned protocol refinement – added intrigue to the non-muscle invasive bladder cancer space, which continues to percolate.
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR.
Prime Medicine Inc. has announced its plans to strategically focus its efforts on a set of high value programs as it advances its pipeline of next-generation gene editing therapies.
Precision Biosciences Inc. has submitted its first clinical trial applications (CTAs) to initiate a phase I study evaluating PBGENE-HBV, an in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV).
In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, GENE-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.
In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, Gene-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.
Genespire Srl has closed a €46.6 million (~$52 million) series B financing to support its work developing off-the-shelf gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients with genetic diseases.
Functionally active hepatitis B virus (HBV) DNA is open and accessible, while the action of an epigenetic editor turns it into functionally inactive DNA, which is closed and inaccessible. Chroma Medicine Inc. has presented data regarding their epigenetic editor CRMA-1001, which is delivered by lipid nanoparticles.
Nanovation Therapeutics Inc. has established a multiyear partnership with Novo Nordisk A/S to advance the development of novel genetic medicines targeting cardiometabolic and rare diseases. The partnership brings together Nanovation’s proprietary long-circulating lipid nanoparticle (lcLNP) technology for RNA delivery to cells outside of the liver, and Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation.
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