Ipsen, a global biopharmaceutical firm that specializes in neuroscience, oncology and rare diseases, partnered with Exicure, a clinical-stage biotech company that develops treatments for neurological, inflammatory and genetic disorders, to create new therapies for Angelman syndrome (AS) and Huntington’s disease (HD).
Roche is calling it quits on a late-stage trial of the company’s Huntington’s disease hopeful tominersen, the Swiss drugmaker said on March 22, a blow for sufferers of the rare, debilitating disease with no treatments to stop or reverse it.
ProQR Therapeutics N.V. announced that the company received Fast Track designation from the Food and Drug Administration for QR-421a for Usher syndrome type 2.
PureTech Health plc entered into a multiyear collaboration with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to advance PureTech’s milk-derived exosome platform technology for the oral administration of Roche’s antisense oligonucleotide platform.
