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Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.
PhaseBio is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapies to treat orphan diseases, with an initial focus on cardiopulmonary indications. Our lead product candidate is PB2452, a novel reversal agent for the antiplatelet drug ticagrelor, and our second product candidate is PB1046, a once-weekly fusion protein for the treatment of pulmonary arterial hypertension. PB1046 utilizes our elastin-like polypeptide technology, which also serves as the engine for our preclinical pipeline. Our proprietary technology platform uses recombinant elastin-like polypeptide (ELP) biopolymers to control the half-life, bioavailability and physical characteristics of molecules for ease of administration. The compounds are engineered for a specific rate of absorption to enhance efficacy and reduce side effects, which may lead to less-frequent dosing and better patient compliance. PB1046 is a vasoactive intestinal peptide (VIP) receptor agonist that leverages our ELP technology for once-weekly dosing. PB1046 is being evaluated in clinical studies in pulmonary arterial hypertension (PAH) and in preclinical studies for the treatment of cardiomyopathy associated with dystrophinopathies. PhaseBio recently concluded a Phase 2a trial of PB1046 in patients with heart failure with reduced ejection fraction. We are also evaluating our development candidate PB2452, a reversal agent for the antiplatelet therapy ticagrelor, in clinical trials. PB2452 was exclusively licensed from MedImmune, the global biologics research and development arm of AstraZeneca, for global development and commercialization by PhaseBio. PhaseBio continues to explore opportunities to apply our ELP biopolymer technology to compounds that may benefit from its unique properties. We are privately owned, with headquarters and research laboratories in Malvern, PA.
AngioScore is a Fremont, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
GenPrime is a Spokane, WA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Korro Bio is an RNA editing company focused on the discovery and development of a new class of precision genetic medicines for both rare and common diseases. The company`s proprietary and modular platform, OPERA™ (Oligonucleotide promoted editing of RNA), builds on a deep understanding of ADAR biology, and combines data-driven design, oligo discovery and chemistry with clinically validated delivery vehicles, to achieve highly selective RNA editing. This unique technology enables the development of RNA editing therapeutics that deliver the functional benefits of gene therapy with a reversible, transient, titratable and specific treatment regimen, offering the potential to propel genetic medicine beyond rare genetic diseases into larger patient populations with common diseases. Korro is based in Cambridge, Mass.