Developing gene therapy for children with the rare liver disease PFIC2
PFIC2 is an inherited disorder that impairs bile transport, damaging the liver. Symptoms typically begin in infancy and can include severe itching. The disease often progresses to liver failure before adulthood, and affected children may need a liver transplant.
Amets Biotechnology is developing AMB-01, a single-dose gene therapy for children with PFIC2. The preclinical treatment is designed to deliver a corrected gene to liver cells and address the genetic cause of the disease.
What stands out about Amets Biotechnology
Amets reports positive preclinical AMB-01 results in juvenile PFIC2 mice.
Vivet identifies its preclinical PFIC2 gene-therapy programme VTX-802 as licensed to Amets.
Co-founder and CSO Gloria Gonzalez-Aseguinolaza previously co-founded gene-therapy developer Vivet Therapeutics.
Co-founder and CEO Laia Trigueros Motos holds a PhD and brings metabolic-disease research experience to the PFIC2 programme.
Received a Navarra Government grant under its 2026 scheme for creating and consolidating innovative technology-based companies.
Joint winner of CEIN’s 2025 Aceleradora HEALTH award for innovative health ventures.
Participated in CEIN’s 2025 Aceleradora HEALTH, receiving specialised training, expert mentoring and health-sector ecosystem access.
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