A packaged nanoparticle that delivered a relaxin-encoding gene therapy with microRNA reduced liver fibrosis, researchers in the laboratory of Leaf Huang at the University of North Carolina, Chapel Hill, Eshelman School of Pharmacy described in the Jan. 21, 2021, issue of Nature Nanotechnology.

Sarepta Therapeutics Inc.’s miss on a key phase II ambulatory endpoint in its Duchenne muscular dystrophy (DMD) trial may have been caused by a dramatic disparity in functional ability at baseline among older vs. younger patients afflicted with the progressively worsening disorder. In any case, Wall Street had knives out, carving 51% of the value away from shares (NASDAQ:SRPT) of the Cambridge, Mass.-based firm, which closed at $82.29, a loss of $86.66, or 51%.

A third-quarter progress report from the international advocacy group Alliance for Regenerative Medicine (ARM) has determined that the regenerative medicine and advanced therapy (RMAT) sector established a highwater mark of $15.9 billion in global financings, breaking the previous annual record of $13.5 billion that was set in 2018.

Novartis AG is expanding its position in the optogenetics space with the acquisition of Vedere Bio Inc., bringing with it a program aimed at vision loss prevention and treatment. Shareholders of Vedere, created in June 2019 through the Atlas Venture incubator, received $150 million up front and are eligible for up to $130 million in milestone payments, bringing the total to $280 million.

LONDON – Sparingvision SAS has raised €44.5 million (US$52.5 million) to advance SPVNo6, a gene therapy designed to be effective for all retinitis pigmentosa patients regardless of the underlying mutation, with a phase I dose-ranging study now due to start in 2021.

In case you haven't heard, Tessera Therapeutics Inc. is working on techniques to write genes into the genome of patients. Tessera, which was developed in Flagship Pioneering Inc.'s Flagship Labs and became a stand-alone incorporated company two years ago, recently came out of stealth mode to highlight its Gene Writing platform based on mobile genetic elements, such as transposons and retrotransposons.

CYBERSPACE – At the virtual annual meeting of the American Society of Gene and Cell Therapy, CAR T cells were the subject of both historical overviews and cutting-edge research alike.

By delivering the protein follistatin via gene therapy, researchers at Washington University in St. Louis were able to increase skeletal muscle mass, decrease fat, and reverse obesity-related arthritis in mice who developed osteoarthritis as a result of a high-fat diet.

Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.