Abdominal aortic aneurysm is a life-threatening cardiovascular disease characterized by chronic inflammation and irreversible dilatation of the abdominal aorta and is asymptomatic at the early stages of the disease, with diagnosis usually relying on imaging techniques and surgery being the main approach for treating it.
Myocardial hypertrophy is a condition characterized by thickening of the ventricular wall and commonly associated with progression to heart failure. It develops when the heart is subjected to biomechanical stress or neurohormonal or hemodynamic stimuli.
The Cystic Fibrosis Foundation has agreed to provide Prime Medicine Inc. an additional investment of up to $24 million to continue the development of a gene editing therapy for people with cystic fibrosis. Prime Medicine uses a gene editing technology called prime editing, which enables a wide range of modifications to the DNA with a high degree of precision.
Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute SCI, a single injection of AMFX-200 into the spinal cord enabled motor neurons from the brain to regrow past the injury site.
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload.
The U.S. FDA has cleared Genetic Leap’s IND application for GL-IL2-138, a small-molecule RNA drug that modulates natural IL-2, allowing for downregulating or upregulating of the immune system to fight diseases.
Idiopathic pulmonary fibrosis (IPF) is a progressive disease characterized by lung scarring, fibrosis and finally respiratory failure. Currently, few treatment options are available for IPF and they only slow down disease progression and do not reverse fibrosis. There is a need for new therapeutic targets that aid in the management of the disease.
Illimis Therapeutics Inc. raised ₩58 billion (US$42 million) in a series B financing round. The funds will support development of ILM-01, its lead bispecific fusion protein candidate, into preclinical development for Alzheimer’s disease by the second half of 2025, along with the company’s neuroimmunology portfolio.
Akebia Therapeutics Inc. has identified new derivatives of ([1,2,4]triazolo[5,1-a]isoquinoline-5-carbonyl)glycinate acting as prolyl 4-hydroxylase (PHD) inhibitors.
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