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.
Korro Bio Inc. has established a collaboration with Novo Nordisk A/S to advance the discovery and development of new genetic medicines, initially to treat cardiometabolic diseases. The collaboration brings together Novo Nordisk’s deep cardiometabolic disease understanding and drug development experience with Korro’s proprietary platform to develop RNA editing product candidates for two undisclosed targets.
Voyager Therapeutics Inc. has announced the selection of a development candidate in a gene therapy program for the treatment of an undisclosed neurological disease under its collaboration with Neurocrine Biosciences Inc.
A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.
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