Interim data from two early stage Friedreich’s ataxia (FA) cardiomyopathy studies from Lexeo Therapeutics Inc. hit the mark by reducing heart muscle thickness, a key cause of death among patients with the rare disease. The results came from the Sunrise-FA phase I/II study and an investigator-initiated phase Ia study of LX-2006, an adeno-associated virus-mediated gene therapy encoding the human frataxin gene. The drug is designed to improve frataxin protein expression to improve mitochondrial cell function.

Edwards Lifesciences Corp. said it has exercised its option to buy Innovalve Bio Medical Ltd., an early stage transcatheter mitral valve replacemen company, for $300 million in cash following its initial investment in 2017. Since that time, Edwards said Innovalve has demonstrated progress in its program with promising early clinical experience.

Edwards Lifesciences Inc. struck three deals totaling €15 million (US$16.3 million) with Affluent Medical SAS to gain access to its mitral valve technology. Edwards secured an exclusive option to acquire Affluent subsidiary Kephalios, which makes the Kalios adjustable mitral ring, for €5 million (US$5.44 million); paid €5 million more for global, non-exclusive licensing of Affluent’s intellectual property related to its biomimetic cardiac mitral valve replacement technology; and invested a further €5 million to acquire 9.21% of Affluent.

Sirnaomics Ltd. has completed IND-enabling studies for STP-125G, a single-stranded siRNA therapeutic targeting apolipoprotein C3 (ApoC3), based on its proprietary Galahead mxRNA technology.

Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.

Nearly 80% of people in Australia and the U.S. that used Genetic Technologies Ltd.’s Genetype multi-risk assessment test showed an elevated risk for at least one disease covered by the test.