BioMarin updated several of the company’s gene therapy programs on February 17 following a September 2021 update, when the drug manufacturer reported the U.S. Food and Drug Administration had placed a clinical hold on the BMN 307 Phearless Phase I/II trial.
Novartis announced Phase II primary endpoint data showing the investigational, first-in-class, oral, targeted factor B inhibitor iptacopan reduced protein in the urine – an increasingly recognized surrogate marker correlating with progression to kidney failure – and showed promise in stabilizing kidney function in patients with IgA nephropathy (IgAN).
Amryt inked an all-stock acquisition deal with Chiasma. The combined company will focus on rare and orphan diseases with three marketed products and a strong development pipeline.
Syneos Health announced an agreement to acquire Synteract, a full-service contract research organization (CRO) focused on the biopharmaceutical industry.
With $30 million in Series A funding, Tranquis Therapeutics launches with a mission of revolutionizing the management of neurodegenerative and aging-related diseases through the advancement of a novel immuno-neurology treatment approach.
Shares of Dova Pharmaceuticals were up more than 38 percent after Sweden-based Sobi announced the intended acquisition of the North Carolina-based company for $915 million.
Rare Cancer Treatments and the Power of Checkpoint Inhibitors Among Hot Topics at ASCOAmerican Society of Clinical Oncology (ASCO), Blockbusters, Cancer, Checkpoint Inhibitors, Clinical Studies, Clinical Trials, Data, Desmoid Tumors, Endocrine Therapy, Gastroenteropancreatic neuroendocrine tumors, Lung Cancer, Nobel Prize, Nobel Prize in Physiology or Medicine, Orphan Diseases, PD-L1 Inhibitor, PD-L1 protein, PIK3CA mutations, R&D, Rare Cancers, Rare Sarcomas, Tenosynovial Giant Cell Tumor (TGCT)
With the books closed on another ASCO meeting, it’s clear to see that checkpoint inhibitors keep showing a promise.
Global healthcare spending is expected to increase dramatically to more than $10 trillion by 2022, driven primarily by an increase in spending on data that will increase value and create a sustainable culture.
Patients are becoming increasingly knowledgeable about their personal health and about the healthcare options available to them – and that is a good thing.
Two Artificial Intelligence Companies Partner to Take on DMD and Orphan DiseasesArtificial Intelligence, Biologicals, Biology, Drug Discovery, Duchenne Muscular Dystrophy (DMD), Dystrophin, Generative Adversarial Networks (GANs), Ligands, New Ventures, Orphan Diseases, Rare Diseases, Small Molecules
Two companies that specialize in AI-backed drug development – Insilico Medicine and A2A Pharmaceuticals – are teaming up to take on the rare disease known as Duchene Muscular Dystrophy.