news
Novartis to advance novel Huntington’s disease therapy
As part of the agreement with Novartis Pharmaceuticals, PTC will receive an initial $1 billion to develop the potential first-in-class oral therapy.
List view / Grid view
PTC Therapeutics
article
What gene therapy manufacturers can gain from collaboration
In this article, Neil Almstead, Chief Technical Operations Officer at PTC Therapeutics, shares his view on why collaboration is key to streamlining manufacture of gene therapies.
article
Bioprocessing/Bioproduction In-Depth Focus 2023
In this In-Depth Focus are articles on the potential of AAV gene therapies and what gene therapy manufacturers can gain from collaboration.
article
European Pharmaceutical Review Issue 2 2023
EPR Issue 2 includes articles on the future of cell and gene therapy, the potential of AAV gene therapies, NMR relaxometry and more…
news
MHRA approves first gene therapy for infusion into the brain
Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.
news
Roche awarded 2022 Drug Discovery of the Year for Evrysdi®
The development team behind Roche’s Evrysdi® (risdiplam), the first mRNA splicing modifier to be approved, have been honoured with the BPS’s Drug Discovery of the Year Award 2022.
news
EMA accepts marketing application for Spinal Muscular Atrophy treatment
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
news
NICE allows extra time for agreement on ataluren price
4 May 2016 | By Victoria White, Digital Content Producer
NICE has allowed extra time for further discussions on access arrangements for PTC Therapeutics’ Translarna (ataluren) for Duchenne Muscular Dystrophy...
news
PTC submits EU application for Translarna for cystic fibrosis
30 September 2015 | By Victoria White
If approved, Translarna would be the first oral protein restoration treatment that targets the underlying cause of nonsense mutation cystic fibrosis...
