Clinical trials in China are growing up in much the same way the R&D enterprise in China has grown up. They are evolving and becoming more sophisticated, reflecting the maturation of the R&D environment around them.
The serious adverse event in the first phase I/II patient dosed with nulabeglogene autogedtemcel (nula-cel) – a gene editing, autologous hematopoietic stem cell therapy for sickle cell disease (SCD) – may have involved the quality of the stem cells and not the conditioning regimen or characteristics of the patient at baseline. But researchers won’t know until more work is done.
Biontech SE is piggybacking on the U.K.’s lead in implementing cancer genomics in routine health care to set up a national clinical trial of personalized mRNA cancer vaccines that are based on the mutational landscape of individual tumors.
The EMA will require all new clinical trial applications to be submitted via its clinical trials information system after Jan. 31, 2023, despite pharma companies and other users raising concerns about functionality and technical failures they are experiencing with the system.
Recent news from Quralis Corp. and Xenon Pharmaceuticals Inc. highlighted the potential for targeting the Kv7 potassium channel, of special interest to a handful of developers lately.
Unlike amphibians, mammals do not regenerate appendages. Except when they do. “If you amputate one of the branches off of the antler [of a reindeer], it will also regenerate,” Jeff Biernaskie told BioWorld. Even without amputation, the antlers of both male and female reindeer regenerate annually, including their skin. That regeneration is “the only large mammal model of true skin regeneration,” he said.
Geron Corp. executives highlighted a potential billion-dollar market opportunity for imetelstat on the back of positive phase III data for the telomerase inhibitor in patients with lower-risk myelodysplastic syndromes (MDS), news that sent shares (NASDAQ:GERN) rising 36% and could portend the first regulatory win for a scientific approach the company has pursued since the 1990s.
After Vera Therapeutics Inc.’s disappointing 24-week data from the Origin phase IIb study with atacicept in immunoglobulin A nephropathy (IgAN), Wall Street is looking forward to more results at 36 weeks, due later this year.
Newron Pharmaceuticals SpA has reported positive six-month data for its selective sodium channel blocker, evenamide, as an add-on therapy to antipsychotic drugs in chronic, treatment-resistant schizophrenia. Patients who had responded at the initial six-week evaluation showed continued improvements in symptoms of psychosis, while others who had not responded at six weeks did so by six months.
New research published in BMJ Open claims there are inconsistencies in Amgen Inc.’s phase III Fourier study for cholesterol lowering. Cardiac deaths were numerically higher in those taking the PCSK9 inhibitor Repatha (evolocumab) combined with a statin than those in the placebo group, the research noted.
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