The private UK group could steal the thunder of rivals like Spark, Uniqure and Biomarin.
Padua gambit seems to have worked as new gene therapy looks the equal of Pfizer/Spark Therapeutics.
Novartis's $4m price target for AVXS-101 fuels concerns that too many new gene therapies could overburden payers without a hard look at how they are financed.
Crunch time nears for oral semaglutide and Novo will find out if concizumab can cut it in a crowded haemophilia market.
Early data are due for Krystal Biotech’s epidermolysis bullosa project and Uniqure’s haemophilia B candidate.
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
Companies that want to play in rare diseases need to shop for gene therapies, but price tags are looking rich.
Nightstar hopes that early gene therapy data will give it a spark, while Alnylam awaits pivotal results for one of its biggest pipeline prospects, givosiran.
Encouraging early data have persuaded the Dutch RNA researcher to push straight into a pivotal trial of its Leber’s congenital amaurosis project, raising hopes for an…