Using a knock-in mouse model of Brugada syndrome (BrS) with an SCN5A mutation identified in multiple BrS families (Scn5aG1746R/+), a team of scientists from various research institutions evaluated the effects of upregulation of MOG1 (ran guanine nucleotide release factor, RanGNRF) via adeno-associated virus serotype 9 (AAV9) vector delivery on BrS features.
Researchers from Cantargia and Johns Hopkins University School of Medicine presented data from a preclinical study evaluating an IL-1 receptor accessory protein (IL-1RAP)-blocking antibody, CAN-10, in a model of acute coxsackievirus B3 (CVB3) myocarditis.
Robocath SAS said Cathbot, its joint venture set up in 2020, has enrolled the final patient for its clinical study in China to evaluate the safety and the efficacy of its robotic platform for percutaneous coronary intervention (PCI). “The completion of our PCI robotic multicenter trial in China is a crucial milestone in our development in this part of the world,” Philippe Bencteux, president and founder of Robocath, told BioWorld.
Scientists will investigate whether cutting-edge technology such as base editing could be used to cure inherited heart muscle conditions after an international team co-led by Harvard Medical School won a research challenge. The $36 million Big Beat Challenge, run by the British Heart Foundation, is one of the largest non-commercial awards ever given and will focus on inherited heart muscle diseases known as genetic cardiomyopathies.
Although Pfizer Inc. has the only drugs approved in the U.S. to treat a rare, progressive heart disease, the U.S. Court of Appeals for the Second Circuit agreed this week with the Department of Health and Human Services, and a lower court, that Pfizer’s proposed copay assistance program for middle-income Americans covered by Medicare would violate the federal Anti-Kickback Statute – even if the company has no “corrupt” intent.
Barely a month after signing a €1 billion-plus deal with Menarini Group for cholesterol-lowering drug obicetrapib, Newamsterdam Pharma BV has struck a $235 million SPAC merger deal that will see the biotech list on Nasdaq in late 2022. The deal will finance phase III development and potential regulatory filings of the drug once dropped by Amgen Inc. as big pharma turned away from the cholesteryl ester transfer protein inhibitor drug class around five years ago.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
Stem cell therapy company Mesoblast Ltd. said that for patients with chronic heart failure with reduced ejection fraction, treatment with rexlemestrocel-L, its allogeneic product candidate, resulted in greater improvement in a prespecified analysis of left ventricular ejection fraction at 12 months relative to controls in the phase III DREAM-HF trial.
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