| Name | Title | Contact Details |
|---|
Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases. We are headquartered in the UK and we also have operations in Germany and the US. Freeline was founded in 2015 and builds upon the pioneering work by our Clinical and Scientific Advisor and Director, Professor Amit Nathwani, Professor of Haematology at UCL. With our liver-based investigational gene therapy technology, we aim to improve the lives of people with chronic systemic diseases and realise the potential of gene therapy treatments.
ReCode Therapeutics is a biopharmaceutical company developing precision medicines for pulmonary diseases with significant unmet medical need.
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
Nohla Therapeutics is a cellular therapy company driven to provide leading off the shelf therapies for patients with critical diseases. Nohla’s first product is a universal donor cell therapy targeting hematopoietic recovery and chemotherapy induced neutropenia, and is being extended to treat a range of other medical indications. This and other products in the pipeline are based on a unique umbilical cord blood expansion platform developed at the Fred Hutchinson Cancer Research Center
Calgary Scientific is a Calgary, AB-based company in the Healthcare, Pharmaceuticals, and Biotech sector.