A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
Diabetic retinopathy and diabetic kidney disease are frequent microvascular complications of diabetes, both related to exacerbated vascular permeability coming from microvascular barrier malfunctioning.
Graviton Bioscience Corp. has announced a strategic investment from Sanofi SA. Under the agreement, Sanofi receives a right of first negotiation to license compounds across various indications, including immunological and metabolic syndrome indications.
Vivet Therapeutics SAS has been awarded financing of €4.9 million (US$5.3 million) from the French government to advance the development of a gene therapy for the treatment of the neurodegenerative disease cerebrotendinous xanthomatosis (CTX).
Humans love to think of our species as unique. But on a genetic level, such uniqueness is surprisingly hard to find. And while that may be a blow to the ego, it also means that an evolutionary lens is one way to search for insights into human diseases. Animals are “adapted to use the same genes that you and I have, but in very different ways,” Ashley Zehnder told BioWorld. Zehnder is co-founder and CEO of Fauna Bio Inc., which uses comparative genomics to identify gene networks that underlie disease resistance in different animal species.
Kupffer cells are macrophages located in the liver that play a key role in liver pathology, concretely in metabolic dysfunction-associated steatotic liver disease (MASLD; formerly nonalcoholic fatty liver disease or NAFLD), but the mechanisms behind this are poorly understood.
Neuralace Medical Inc. tied up a second U.S. FDA clearance for its Axon therapy, adding painful diabetic neuropathy (PDN) to the chronic nerve pain indication it received in 2021. An ‘electroceutical’ device, Axon employs non-invasive magnetic peripheral nerve stimulation (mPNS) to provide relief without leads, injections or implants.
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders.
Kissei Pharmaceutical Co. Ltd. has described 3,4-dihydroquinolin-2(1H)-one compounds acting as thyroid-stimulating hormone (thyrotropin) receptor (TSHR) antagonists reported to be useful for the treatment of hyperthyroidism, Graves’ disease and thyroid-associated ophthalmopathy.
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