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Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
Based in Sunnyvale, Calif., Cepheid is a leading molecular diagnostics company that is dedicated to improving healthcare by developing, manufacturing, and marketing accurate yet easy-to-use molecular systems and tests. By automating highly complex and time-consuming manual procedures, the company`s solutions deliver a better way for institutions of any size to perform sophisticated genetic testing for organisms and genetic-based diseases. Through its strong molecular biology capabilities, the company is focusing on those applications where accurate, rapid, and actionable test results are needed most, such as managing infectious diseases and cancer.
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.
Hometown Health Center is a Schenectady, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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