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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
Spruce is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need. Our wholly-owned product candidate, tildacerfont, is a CRF1 receptor antagonist currently in late-stage trials in adult patients with classic congenital adrenal hyperplasia (CAH), with a planned Phase 2 trial in pediatric classic CAH. We aim to advance the treatment paradigm for classic CAH with a well-tolerated, non-steroidal approach designed to offer markedly improved disease control and reduced steroid burden for patients. Tildacerfont may also benefit patients with other disorders characterized by elevated levels of or hyperresponsiveness to adrenocorticotropic hormone (ACTH), a hormone involved in the production of cortisol, including a rare form of polycystic ovary syndrome (PCOS).
Rain Therapeutics is a clinical-stage precision oncology company. Our uncompromised dream is to develop and implement the best precision oncology therapies for patients suffering from cancer. We are rooted in this purpose — extending the quality and length of life for cancer patients where we may be their best hope.
Insitus Biotechnologies is a Albuquerque, NM-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Axcella is designing and developing AXA Candidates, compositions of endogenous metabolic modulators, or EMMs, engineered in distinct ratios, designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Axcella`s AXA Candidates are generated from its proprietary, human-focused AXA Development Platform. Axcella believes its expertise and capabilities in EMMs position it to become a preeminent biotechnology company reprogramming metabolism to address a diverse set of complex diseases and support health. Axcella`s AXA Development Platform has already produced a pipeline of product candidates in programs targeting liver, muscle and blood. Axcella was founded by Flagship Pioneering.