ECUSA Hosts Inspiring Seminar on Gene Therapy for Liver Inherited Diseases

Washington DC

On November 20, 2024, ECUSA Washington, D.C., in collaboration with the Cultural Office of the Embassy of Spain, welcomed Dr. Gloria González-Aseguinolaza for an engaging seminar at the Spanish Cultural Center.

Dr. González-Aseguinolaza, director of the Rare Disease Gene Therapy Program at CIMA Universidad de Navarra, and Chief Scientific Officer at Vivet Therapeutics SL, shared her an inspiring presentation on the potential of gene therapy to treat inherited liver diseases.

Her talk, «Gene Therapy for Liver Inherited Diseases,» provided fascinating insights into the development of AAV-based therapies for inherited conditions like Wilson disease, highlighting the latest advancements in gene therapy, including the creation of recombinant AAVs optimized for gene delivery and the potential of therapies like VTX-801 to reduce copper accumulation in the liver and throughout the body. Dr. González-Aseguinolaza also shared promising preclinical results showing how these therapies could restore liver functionality and improve survival rates in animal models. that multiple leading centers in the U.S. and Europe are currently participating in the GATEWAY Phase 1/2 trial, which is evaluating the safety, tolerability, and potential efficacy of VTX-801 in adult patients with Wilson disease.

The event was a resounding success, hosted by Naroa Gimenez-Camino on behalf of ECUSA Washington DC. She expressed heartfelt gratitude to the Cultural Office of the Embassy of Spain for their unwavering support.

ECUSA Washington DC remains committed to promoting knowledge exchange within the scientific community and will continue to bring together leading experts to share innovations that can transform healthcare and improve lives. Stay tuned for more enriching events from ECUSA!

dc@ecusa.es

FOSTERING GRADS​

Fostering Grads es un programa de mentorazgo enmarcado dentro de la plataforma de Career Development & Advisory (CDA) de la sociedad sin ánimo de lucro Españoles Científicos en USA (ECUSA).

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