Eva Segura Gensler

Designed and Developed Autologous Gene Therapy Created and assessed erythroid-specific lentiviral vectors for autologous gene therapy targeting alpha thalassemia major, achieving restoration of therapeutic α-globin expression and hemoglobin levels in patient-derived hematopoietic stem and progenitor cells.

Presented at International and National Conferences

2024: Thalassemia Western Consortium Meeting (remote); Molecular Biology Institute Retreat (UCLA); TransAtlantic Gene Therapy Retreat (MD); American Society of Gene and Cell Therapy (ASGCT) (MD); 21st Stem Cell Symposium (UCLA);

2023: 4th International Conference Stem Cell (Italy); ASGCT (CA); 2nd Annual Ginsburg Symposium (UCLA); 20th Stem Cell Symposium (UCLA); International Alpha Thalassemia Major Quarterly Meeting (remote);

2022: 11th Cooley's anemia Symposium (NY)

Attended only: American Society of Hematology (2022 - 2024); ASGCT (2021)

Authored Grant Writing and Fellowship and Funding Acquisition leading to $ 4 millions ensuring the creation of project to its pre-clinical development.

Awarded grants: NIH-R01, CIRM-DISC2, CIRM-TRAN1

Fellowships: - Broad Stem Cell Research Center Training Fellowship (Rose Hills Foundation), 2022,2023

Authored and Prepared Clinical and Regulatory Processes. Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) requests. Generated pre-IND enabling preclinical data to establish safety and efficacy. Authored TRAN1 grant covering Lentiviral Vector Manufactoring (CMC) with GMP-compliant procedure and Good-Laboratory Practice (GLP) Toxocology Studies and pharmacology studies. Authored INTERACT package and met with FDA. Currently working on developing clinical protocol for alpha thalassemia.

First Author Publications

Development of Lentiviral Vectors for Hematopoietic Stem Cell Gene Therapy for the Correction of Alpha Thalassemia (on going manuscript)

Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials (doi.org/10.3390/v15030713)