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Sun, Jul 20, 2025

Gene therapy

  • Gene therapy
    • CAR T
Home » Topics » Science » Gene therapy
  • Gene therapy
    May 26, 2025

    Latus Bio’s LTS-101 is a potential therapeutic approach for Batten disease

    Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
  • Stock table gold arrow pointing down
    March 18, 2025
    By Randy Osborne

    CMV history cited in DMD liver death on Sarepta’s Elevidys

    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.
  • Dollar sign in light bulb on yellow background
    Dec. 24, 2024

    Series A to advance Hervolution’s HERV-targeted immunotherapies

    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.
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