Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drugThe first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the Share XIntellia, Regeneron ace first trial with ‘in vivo’ CRISPR drughttps://pharmaphorum.com/news/intellia-regeneron-ace-first-trial-with-in-vivo-crispr-drug/
Biogen’s ambitions in gene therapy hit by another failed pivotal trialWhen Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that Share XBiogen’s ambitions in gene therapy hit by another failed pivotal trialhttps://pharmaphorum.com/news/biogens-ambitions-in-gene-therapy-are-hit-by-a-second-failed-phase-3-trial/
How the patient voice is becoming vital for drug approvalRebecca Sanders from Lipodystrophy UK tells us how the patient voice helped convince NICE to approve a much-needed Share XHow the patient voice is becoming vital for drug approvalhttps://deep-dive.pharmaphorum.com/magazine/market-access-2021/patient-voice-drug-approvals-lipodystrophy/
EHA21: Novartis trumpets iptacopan as oral alternative to antibodies in PNHThe mainstay of therapy for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) is Alexion’s infusion therapy Soliris, Share XEHA21: Novartis trumpets iptacopan as oral alternative to antibodies in PNHhttps://pharmaphorum.com/news/eha21-novartis-trumpets-iptacopan-as-oral-alternative-to-antibodies-in-pnh/
EHA21: Sanofi builds case for rare disease drug sutimlimab after FDA rejectionSanofi’s hopes of a speedy approval of first-in-class C1s inhibitor sutimlimab in rare disorder cold agglutinin disease (CAD) Share XEHA21: Sanofi builds case for rare disease drug sutimlimab after FDA rejectionhttps://pharmaphorum.com/news/eha21-sanofi-builds-case-for-rare-disease-drug-sutimlimab-after-fda-rejection/
Sanofi’s venglustat bombs again, but isn’t dead yetShortly after failing a Parkinson’s disease study, Sanofi’s venglustat has missed the mark in another trial in autosomal Share XSanofi’s venglustat bombs again, but isn’t dead yethttps://pharmaphorum.com/news/sanofis-venglustat-bombs-again-but-isnt-dead-yet/
CHMP backs bluebird bio’s gene therapy for rare disease ALDUS biotech bluebird bio has had a challenging time in the last few months, so a recommendation for Share XCHMP backs bluebird bio’s gene therapy for rare disease ALDhttps://pharmaphorum.com/news/chmp-backs-bluebird-bios-gene-therapy-for-rare-disease-ald/
Apellis set to take on Alexion as FDA clears PNH drug EmpaveliApellis Pharma has secured FDA approval for its complement C3 inhibitor Empaveli as a treatment for paroxysmal nocturnal Share XApellis set to take on Alexion as FDA clears PNH drug Empavelihttps://pharmaphorum.com/news/apellis-set-to-take-on-alexion-as-fda-clears-pnh-drug-empaveli/
Scotland backs NHS use of Alexion’s Ultomiris for rare disease aHUSNHS patients in Scotland with the rare disease atypical haemolytic uremic syndrome (aHUS) will now be able to Share XScotland backs NHS use of Alexion’s Ultomiris for rare disease aHUShttps://pharmaphorum.com/news/scotland-backs-nhs-use-of-alexions-ultomiris-for-rare-disease-ahus/
