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Founded in 2011, PANTHERx Rare is a specialty pharmacy solely focused on rare and orphan diseases, distributing several orphan products and providing access and support services to those needing them most. Known as The Rare Disease Pharmacy®, PANTHERx transforms lives by delivering therapeutic breakthroughs, clinical excellence, personalized support, and a range of access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is the seven-time winner of the prestigious MMIT Patient Choice Award, including the 2024 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. #ReinventingSpecialty #RevolutionizingPharmacy #RedefiningCare #DefiningRarePharmacy #TheRareDiseasePharmacy
Panacos Pharmaceuticals, Inc. is a Watertown, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Pillar Biosciences aims to "Make precision medicine the first option for every patient" by developing and manufacturing targeted next-generation sequencing (NGS)-based assays and software for today`s high-throughput specialty NGS laboratories. Through our SLIMamp technology (a multiplex overlapping PCR chemistry) Pillar offers a streamlined, robust and economical workflow with high mapping and on-target metrics. In conjunction with the SLIMamp assay technology, Pillar Biosciences has an informatics pipeline called the Pillar Variant Analysis Toolkit (PiVAT) that is not only highly accurate, enabling somatic variant calls down to 1% allele frequency without use of Unique Identifiers (UIDs), but also fast and efficient, returning valuable sample data quickly. For higher-sensitivity applications such as cell-free DNA analyses, Pillar Biosciences has developed a dedicated assay technology for cell-free DNA that uses UID methodology, to work with the PiVAT pipeline to drive sensitivity down to 0.1%-0.2%. Current as of May 25, 2021.
AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Noramco, Inc. is a Wilmington, DE-based company in the Healthcare, Pharmaceuticals, and Biotech sector.