Drug Development
The Phase III trial met its primary endpoint, a significant reduction of phenylalanine levels in the blood, with no severe adverse events reported, PTC Therapeutics reported Wednesday.
FEATURED STORIES
Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
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OptiNose announced positive top-line results from its Phase III ReOpen2 clinical trial testing OptiNose’s XHANCE, a fluticasone propionate nasal spray for chronic sinusitis.
BioSpace looks at several companies developing new gene therapies in the wake of the FDA recommending two of bluebird bio’s lentiviral vector gene therapies.
Versanis Bio’s data reveals the results of a pooled analysis, showing that its lead asset, bimagrumab, improves body composition in both diabetic and non-diabetic patients.
Ahead of a scheduled Advisory Committee meeting this week, the FDA released favorable remarks about the Pfizer-BioNTech and Moderna COVID-19 vaccines for children under the age of five.
While a handful of companies have dominated the COVID-19 vaccine market in the U.S. and Europe, a number of biotechs are continuing to develop vaccines to use as booster shots.
The approval of AMX0035, which will be marketed under the brand name Albrioza, marks the first new ALS drug approved in Canada since 2018.
Genmab’s Phase II expansion cohort of the EPCORE NHL-1 clinical trial revealed that its investigational bispecific antibody epcoritamab induced a durable response in patients with LBCL.
CRISPR Therapeutics shared positive initial data from its Phase I COBALT-LYM trial of CTX130 for cancer which demonstrated safety and efficacy when used to target CD70.
For the second straight day, the FDA’s Cell, Tissue and Gene Therapies Advisory Committee voted unanimously to approve a bluebird lentiviral vector (LVV) gene therapy.
Cogent Biosciences shared its Phase II APEX clinical trial on bezuclastinib for patients with AdvSM and showed promise in treating the rare blood disorder.