22nd Orphan Drugs and Rare Diseases Global Congress 2024 Americas
Addressing Scientific and Regulatory Challenges to Advance New Treatments for Rare Diseases
Boston, MA, USA
Thursday 27th - Friday 28th June 2024
Bionical Emas
Bionical Emas is the only Contract Research Organization (CRO) to combine Clinical Development, Clinical Trial Supply (CTS) and Early Access Programs (EAP) bringing life-changing medicines to patients around the world. Their unique integrated business model, with its range of services and capabilities, benefit many of the world’s leading pharma and biotech companies. This distinctive offering enables them to maximize access and generate evidence at every stage of the drug development pathway. Bionical Emas has extensive experience across all phases of clinical research, with a history in rare disease, oncology/heme-oncology and infectious diseases. The company has an established global footprint across the US, EU and Australasia markets and has over 200 employees based around the globe, supporting many thousands of patients. The Bionical Emas vision is to maximize access and generate evidence across the entire clinical development pathway, positively impacting current and future patients.
ThinkGenetic, Inc.
ThinkGenetic is an award-winning digital health company, specializing in finding individuals at risk of rare conditions. The company detects patients for evaluation, treatment, and clinical trials using validated algorithms, AI technology, and the team’s deep experience in genetic and rare diseases. ThinkGenetic is a MIT Solve / Horizon Prize winner and a graduate of the Opportunity Machine / Accelerate South and PULSE@MassChallenge accelerators programs. For more information, visit www.thinkgenetic.com.
PTC Therapeutics
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. Link: www.ptcbio.com