Upstream Bio, traded on NASDAQ under the ticker UPB, is a clinical-stage biotechnology company focused on developing in vivo base editing therapies for monogenic diseases. The company’s proprietary platform leverages adenine base editing to precisely convert targeted adenine–thymine base pairs to guanine–cytosine without introducing double-strand breaks, aiming to correct disease-causing mutations at the DNA level. This approach is designed to deliver single-dose treatments that can durably address the underlying genetic root of rare disorders.
Upstream Bio’s pipeline includes multiple preclinical and early clinical programs targeting central nervous system, liver, and muscle diseases with significant unmet medical need. Lead programs include therapies for GM2 gangliosidosis (Tay–Sachs and Sandhoff diseases) and Friedreich’s ataxia, with additional discovery efforts directed toward other rare inherited disorders. The company employs a modular vector design to optimize tissue targeting and base editor expression, supported by in-house capabilities for vector engineering, preclinical modeling, and translational development.
Founded in 2021 as a spin-out from leading gene-editing research, Upstream Bio is headquartered in Cambridge, Massachusetts, with research and manufacturing collaborations across North America and Europe. The company has established strategic partnerships with academic institutions and contract development and manufacturing organizations to accelerate the advancement of its pipeline. Through these alliances, Upstream Bio is advancing both platform innovation and program-specific milestones toward clinical proof of concept.
Upstream Bio is led by a senior executive team with extensive experience in biotechnology and gene therapy development. The leadership group combines expertise in molecular biology, clinical development, regulatory affairs, and biomanufacturing. By harnessing the precision of base editing technology and a focused strategic approach, Upstream Bio aims to deliver transformative treatments that address the genetic basis of rare diseases and improve patient outcomes worldwide.
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