In August, a press release from HHS announced the cancellation of 22 vaccine research projects based on mRNA, the latest available technology aimed at developing therapies for viral infections, cancer, and genetic conditions. What happens to mRNA innovation when funding dries up? This series explores how reductions in funding could impact mRNA technology, affecting innovation, research and future therapies.

Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.

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.