| Name | Title | Contact Details |
|---|---|---|
Philip Lee |
Co-Founder and Chief Technology Officer | Profile |
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.
Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe #geneticdiseases by precisely upregulating protein expression. We develop antisense oligonucleotide medicines that increase gene expression to treat genetic epilepsies and other severe monogenic diseases, including genetic conditions affecting the central nervous system, eye, liver and kidney. At Stoke, we believe our proprietary technology platform, Targeted Augmentation of Nuclear Gene Output, or #TANGO, will allow us to deliver disease-modifying therapies in a highly precise, durable and controlled manner. We have identified approximately 2,900 genetic diseases which we believe may be amenable to TANGO. At Stoke, we are united around a common goal: to improve the lives of people living with severe genetic diseases. Stoke is located in Bedford, MA with offices in Cambridge, MA.
PCI Pharma Services is a global leader in Contract Development and Manufacturing Organization (CDMO) services, offering integrated solutions for drug development, manufacturing, and packaging. The company focuses on innovation and patient-centric approaches, supporting pharmaceutical and biopharmaceutical companies at various stages of drug development and commercialization. With 30 Good Manufacturing Practice (GMP) facilities across seven countries, PCI employs over 4,300 people dedicated to delivering life-changing therapies. The company provides a comprehensive range of services, including drug development and manufacturing for various dosage forms, advanced drug delivery systems, and complex packaging solutions. PCI also manages storage, distribution, and logistics to support the pharmaceutical supply chain and offers clinical trial supply chain management. Committed to growth, PCI has made significant investments, including a $100 million expansion in New England to enhance its global capacity and capabilities.
GENFIT is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative therapeutic and diagnostic solutions in metabolic and liver related diseases where there are considerable unmet medical needs, corresponding to a lack of approved treatments. GENFIT is a leader in the field of nuclear receptor-based drug discovery with a rich history and strong scientific heritage spanning almost two decades. Its most advanced drug candidate, elafibranor, is currently evaluated in pivotal Phase 3 clinical trial (“RESOLVE-IT”) as a potential treatment for nonalcoholic steatohepatitis, or NASH. NASH is considered by regulatory authorities as a medical emergency because of its potentially severe consequences, although often asymptomatic until late stages, and because its prevalence is on the rise. Elafibranor has also obtained positive preliminary results in a Phase 2 clinical trial in primary biliary cholangitis (PBC), a severe chronic liver disease. As part of GENFIT’s comprehensive approach to clinical management of NASH patients, the Company is also developing a new, non-invasive and easy-to-access blood-based in vitro diagnostic, or IVD, test to identify patients with NASH who may be appropriate candidates for drug therapy. With facilities in Lille and Paris, France, and Cambridge, MA, USA, the Company has approximately 170 employees. GENFIT is a public company listed on the Nasdaq Global Select Market and in compartment B of Euronext’s regulated market in Paris.
Human BioSciences is a Gaithersburg, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.