Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Amsterdam Molecular Therapeutics has announced that it obtained a license from La Sapienza University in Rome, Italy, to their advanced small nuclear RNA-based exon-skipping technology for the treatment of Duchenne muscular dystrophy.
Subscribe to our email newsletter
The combination with Amsterdam Molecular Therapeutics’s (AMT) adeno-associated virus (AAV) gene therapy platform potentially makes up a long-term treatment for this seriously debilitating disease with a single administration of the product.
Ronald Lorijn, CEO of AMT, said: “Access to La Sapienza’s RNA technology perfectly complements our gene and vector therapy platform. It adds a project to AMT’s R&D pipeline that will start its preclinical phase this year.”