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Serapha Bio
seraphabio.comLocations
San Diego, CA, USA
About
Serapha Bio is a clinical-stage genetic medicines company developing a proprietary base editing therapy, SERP-01, for patients with alpha-1 antitrypsin deficiency (AATD), the leading genetic cause of serious, progressive lung and liver disease. SERP-01 is in clinical development for the treatment of AATD patients with the PiZZ genotype, the most severe genetic mutation responsible for >90% of AATD cases. SERP-01 consists of a lipid nanoparticle formulation containing messenger RNA encoding Serapha’s proprietary adenine base editor together with a guide RNA. The therapy is designed to enable precise correction of the disease-causing mutation in PiZZ AATD. Correcting PiZZ, the most common gene variant associated with severe AATD, could address a large population in the U.S. and Europe.
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