The nonprofit foundation, n-Lorem developed personalized medicine for a child with rare leukodystrophy.

Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.

Both Tabrizi and Yang think that knocking down MSH3 could stave off HD pathology. Tabrizi noted that MSH3 also modifies other run-on repeats, including those causing fragile X syndrome, myotonic ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Under terms of the deal, Stoke will spearhead global development of zorevunersen, while Biogen will receive exclusive rights ...

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

Biogen and Stoke Therapeutics have announced a partnership aimed at advancing a disease-modifying treatment for Dravet ...

Biogen will receive rights to commercialize zorevunersen outside the US, Canada, and Mexico, paying $165 million upfront and other milestone payments.

Biogen receives exclusive rights to commercialize zorevunersen outside U.S., Europe, and Japan.

Market OverviewThe Global RNA Therapeutics Market is valued at USD 13.3 Billion in 2023 and is projected to reach a value ...

Researchers from the Huntington's Disease Center, UCL Queen Square Institute of Neurology, and UK DRI at UCL have taken major ...