ARTICLE | Discovery & Translation
BioCentury’s roundup of translational innovations
By Danielle Golovin, Senior Biopharma Analyst
March 9, 2024 2:38 AM UTC
Elixirgen showed it was able to deliver a linear mRNA encoding a full-length human dystrophin protein, avoiding concerns that miniaturized versions of the gene may lack some critical functionality.
Most of the Duchenne muscular dystrophy (DMD) gene therapies in development deliver only a portion of the dystrophin gene, since it is too large to fit in a conventional adeno-associated viral (AAV) vector. Elixirgen Therapeutics Inc.’s full-length version was delivered via a nanoparticle, and it restored muscular function in a mouse model of Duchenne muscular dystrophy, as shown at the MDA Clinical & Scientific Conference this week…
- SEO Powered Content & PR Distribution. Get Amplified Today.
- PlatoData.Network Vertical Generative Ai. Empower Yourself. Access Here.
- PlatoAiStream. Web3 Intelligence. Knowledge Amplified. Access Here.
- PlatoESG. Carbon, CleanTech, Energy, Environment, Solar, Waste Management. Access Here.
- PlatoHealth. Biotech and Clinical Trials Intelligence. Access Here.
- Source: https://www.biocentury.com/article/651690/science-spotlight-elixirgen-s-full-length-dystrophin-gene-therapy-insilico-s-tnik-inhibitor-and-more