Rallybio is a clinical-stage biotechnology company dedicated to developing transformative therapies for patients with rare diseases. Since its founding in 2020, the company has built a diversified pipeline of therapeutic candidates that leverage protein replacement, antibody-based modalities, and novel small molecules. Rallybio focuses on areas of high unmet medical need, including complement-mediated disorders, lysosomal storage diseases and genetic cardiometabolic conditions.
Rallybio’s lead programs encompass multiple stages of clinical development. Among these, a complement inhibitor targeting paroxysmal nocturnal hemoglobinuria and related indications is in late-stage study, while enzyme replacement and substrate reduction approaches are advancing for select mucopolysaccharidoses. The company also pursues earlier-stage candidates for alpha-1 antitrypsin deficiency and other genetic disorders, drawing on partnerships with academic institutions and technology platforms to accelerate discovery and development.
Headquartered in Waltham, Massachusetts, Rallybio conducts clinical trials across the United States and Europe, collaborating with a global network of investigative sites. The company maintains strategic alliances for manufacturing and process development to support its pipeline, and leverages real-world evidence initiatives and patient advocacy groups to inform trial design and ensure patient-centric approaches.
Rallybio’s leadership team combines deep industry experience and scientific expertise. Chief Executive Officer Thomas W. Lynch brings over two decades of biopharma leadership, including senior roles in research and development. The broader executive team and board of directors feature seasoned professionals with backgrounds in commercial strategy, regulatory affairs and rare disease drug development, all united in Rallybio’s mission to bring novel therapies to patients with limited or no treatment options.
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