Alliance members

BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions that it seeks to treat, helping to better the lives of those struggling with rare genetic disease. BioMarin discoveries have led to eight first or best-in-class commercial treatments and a pipeline of multiple product candidates applying the same science-driven, patient-forward approach to a broader group of genetic disorders.

Chiesi USA, Inc. is headquartered in Cary, North Carolina and an affiliate of Chiesi Farmaceutici S.p.A., a family-owned and R&D focused global pharmaceutical company based in Parma, Italy. Chiesi USA is a specialty pharmaceutical company committed to the development and commercialization of products for settings that include hospital, rare diseases, and adjacent specialty care.

GeneDx combines unmatched clinical expertise, advanced technology, and the power of GeneDx Infinity™, the world’s largest rare disease genomic dataset. This foundation powers GeneDx’s ExomeDx™ and GenomeDx™ tests — ranked #1 by expert geneticists and granted FDA Breakthrough Device Designation — enabling clinicians to deliver precise, fast, and actionable diagnoses. With over 25 years of innovation, more than 4,800 genetic diseases diagnosed, and over 1,000 scientific publications, GeneDx is building the genomic intelligence network that’s shaping the future of precision medicine: for patients, providers, and partners alike.

Revvity provides health science solutions, technologies, expertise and services that deliver complete workflows from discovery to development, and diagnosis to cure. Revvity is revolutionizing what’s possible in healthcare, with specialized focus areas in translational multi-omics technologies, biomarker identification, imaging, prediction, screening, detection and diagnosis, informatics and more. Revvity serves customers across pharmaceutical and biotech, diagnostic labs, academia and governments. It is part of the S&P 500 index and has customers in more than 190 countries.

Stoke Therapeutics is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus areas are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach.