Amets Biotechnology
Developing curative gene therapies for rare pediatric diseases.
Our mission: To cure children with PFIC2.
Our Science
The Science
A Disease Without Answers — Until Now
The Problem
PFIC2 is a rare, degenerative pediatric liver disease — and 90% of rare diseases have no cure. Current standards of care fail 66% of patients, often leading to liver failure or cancer before age 5. Available therapy involves daily medications that address symptoms, not the cause.
The Solution: AMB-01
AMB-01 is a safe, single-dose gene therapy designed to be curative. It targets the genetic root cause by delivering a corrected version of the defective gene directly into affected liver cells — eliminating the need for lifelong symptomatic treatment.
Daily Symptomatic Treatment
Ongoing medications, repeated interventions, disease progression — and no path to cure.
AMB-01: One-Time Curative Dose
A single administration targeting the genetic cause — designed for lifelong benefit.
Validation & Roadmap
Built on Rigorous Science and Strategic Milestones
Proof-of-Concept
Successfully cured PFIC2 in juvenile mice during preclinical testing — a critical scientific milestone demonstrating AMB-01's therapeutic potential.
Secured IP
Patent granted covering the core therapeutic DNA sequence, establishing a strong and defensible intellectual property position.
Strategic Partners
Backed by specialized expertise from Vivet Therapeutics, Amplo Biotechnology and Cima Universidad de Navarra.
Roadmap
Development plan
Clear roadmap with strategic milestones
Leadership
Decades of Specialized Expertise
The Amets founders bring over 95 combined years of focused experience in gene therapy, metabolic disease, and biotech development — uniquely positioned to deliver AMB-01 from bench to clinic.
Laia Trigueros Motos, PhD
Chief Executive Officer
25+ years in metabolic disease research. Driving the strategic vision and operational execution of Amets.
Gloria Gonzalez-Aseguinolaza, PhD
Chief Scientific Officer
30+ years in gene therapy. Co-founder of Vivet Therapeutics — a defining voice in the field.
Marina Ruiz de Galarreta, PhD
Chief Operating Officer
15+ years in hepatic disease research, overseeing research operations and translational strategy.
Nicholas Weber, PhD
Chief Business Development Officer
25+ years in research incluing 10+ in biotech innovation, leading partnerships and commercial strategy.
Clinical Advisor: Dr. Lorenzo D'Antiga
Key Opinion Leader and leading pediatric hepatologist at Ospedale di Bergamo, bringing critical clinical trial and rare disease expertise to the team.
Strategic Partners
Backed by leading innovators in gene therapy and biotech development.
Partner With Us
Join Us in Changing the Future of Pediatric Rare Diseases
We are actively seeking investors and strategic partners who share our commitment to delivering curative therapies to children who have no other options. Let's build something that matters.
Contact Us
Laia Trigueros Motos, PhD — CEO
Investor Deck
Download our full pitch deck for a deep dive into the science, market opportunity, and financial roadmap.
Connect
Follow our progress and engage with the Amets team on LinkedIn for the latest updates and milestones.