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RebornGene is a clinical-stage biotechnology company specializing in the development of innovative gene therapies for rare genetic disorders and intractable diseases. The company leverages a proprietary AAV vector discovery and engineering platform designed to optimize tissue-specific targeting and reduce immunogenicity. Its lead program, RG001, is an AAV-based gene therapy currently in Phase 1/2 clinical trials for the treatment of Leber hereditary optic neuropathy (LHON) caused by ND4 mutations. RebornGene's broader pipeline focuses on neuromuscular and ophthalmic indications, including programs for Duchenne muscular dystrophy and spinal muscular atrophy. Founded by industry veterans with extensive experience in gene therapy manufacturing and development, the company is headquartered in Hangzhou, China.
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