- In April 2023, Ultragenyx Pharmaceutical Inc., a company focused on rare and ultra-rare diseases, announced positive interim data from its ongoing clinical trial of an investigational nucleoside therapy for Thymidine Kinase-2 Deficiency
- In March 2023, Stealth BioTherapeutics, a clinical-stage biotechnology company, initiated a new Phase 2 study for its investigational mitochondrial therapy in patients with TK2d, expanding its clinical program and demonstrating continued commitment to addressing this rare genetic disorder
- In March 2023, Rocket Pharmaceuticals, Inc., a leading gene therapy company, presented preclinical data for its investigational gene therapy for TK2d at a major rare disease conference, highlighting promising results in animal models
- In February 2023, a collaboration between a leading academic research institution and BridgeBio Pharma, Inc., a company focused on genetic diseases, received new grant funding to accelerate preclinical research into novel therapeutic targets for TK2d
- In January 2023, Alexion Pharmaceuticals, Inc. (AstraZeneca) announced the expansion of its rare disease diagnostic support program to include broader genetic screening for mitochondrial disorders, which could lead to earlier identification of TK2d patients
