Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
Hervolution Therapeutics ApS (formerly Inprother Aps) has announced a $11.7 million series A financing to support its work developing novel human endogenous retrovirus (HERV)-targeted immunotherapies to address diseases of aging.
Netherton syndrome (NS) is caused by mutations in the serine protease inhibitor Kazal type 5 gene (SPINK5), which encodes lympho-epithelial Kazal-type-related inhibitor (LEKTI).
Podocytes are a terminally differentiated cell type located in the glomerulus. Podocyte damage and the subsequent dysregulation of podocyte proteins have been implicated in various kidney disorders. Since gene delivery to podocytes using adeno associated vectors (AAVs) has been challenging due to various technological and physiological hurdles, investigators at Purespring Therapeutics Ltd. developed an AAV gene therapy platform that allowed for effective, specific and safe delivery of transgenes to podocytes.
CTNNB1 syndrome is a rare neurodevelopmental disorder that is caused by mutations in the gene encoding β-catenin, CTNNB1, which plays a critical role in neuronal development, synapse formation and brain maturation.
Researchers from Poseida Therapeutics Inc. presented preclinical data for P-FVIII-101, a novel nonviral gene therapy being developed for the treatment of hemophilia A.
Adolore Biotherapeutics Inc. has offered an update on recent pipeline development progress as it advances nonopioid analgesic gene therapies for the treatment of chronic pain.
Investigators at Poseida Therapeutics Inc. developed P-KLKB1-101, a nonviral KLKB1 gene editing therapy, being developed for the treatment of hereditary angioedema (HAE).
Researchers from Ikarovec Ltd. have presented preclinical data for the AAV8-IKC159V, the company’s lead bicistronic gene therapy being developed for the treatment of geographic atrophy, a late stage of geographic dry AMD.
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