AP003
Arg-TGA (Arginine to TGA) Liver Stop Codon Disease (e.g., Methylmalonic Acidemia, Phenylketonuria, Organic Acidemias, Urea Cycle Disorders)
Key Facts
About Alltrna
Alltrna, a Flagship Pioneering company, is unlocking the therapeutic potential of transfer RNA (tRNA) to create a new paradigm in genetic medicine. Its platform aims to treat thousands of diseases with a single, universal tRNA medicine by correcting shared premature termination codon (PTC) mutations. The company has demonstrated preclinical proof-of-concept for its first development candidate, AP003, and is building a robust pipeline for liver and other stop codon diseases. Backed by significant venture capital, Alltrna is led by an experienced team focused on translating its novel platform into transformative therapies.
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