Alex Sapir

Mimecast Hubspot WP Engine IoT Data Analytics Remote AI

gene regulation neuromuscular diseases rare genetic diseases cardiovascular diseases biotechnology: pharmaceutical preparations health care biotechnology research biopharmaceutical gene expression rare diseases genetically defined diseases drug targets therapeutic focus clinical trials patient-centered small molecules sickle cell disease facioscapulohumeral muscular dystrophy fshd beta-thalassemia modified gene expression therapeutic development regulatory approval biotechnology clinical development precision medicine patient advocacy drug discovery investigational therapies muscle weakness genetic counseling clinical research compassionate use orphan drugs genetic disorders patient outcomes fda approval muscle degeneration treatment access biomarker evaluation symptomatic treatment fatal diseases hemoglobinopathies biopharma pipeline patient engagement safety & efficacy disease biology patient-driven development clinical innovations pathophysiology community outreach healthcare advancements scientific leadership corporate governance investor relations clinical-stage biopharmaceutical small molecule therapies gene mis-expression disease-modifying therapies patient-centered approach proprietary technology genomic screening losmapimod ftx-6058 facioscapulohumeral muscular dystrophy (fshd) sickle cell disease (scd) neuromuscular disorders hematologic disorders patient-derived models biomarker-guided discovery collaboration with sanofi innovative research high unmet medical need healthcare providers genetic counselors pharmaceutical partnerships rare disease community fulcrumseek™ genetic medicine therapeutic pipeline health wellness & fitness hospital & health care