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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.
Regenacy Pharmaceuticals is developing a novel, disease-modifying approach to treating peripheral neuropathies that goes beyond pain and symptom management to restore peripheral nerve function.
Maze Therapeutics is translating novel genetic insights into lifesaving medicines, through an approach that integrates human genetics and functional genomics.
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel`s pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.