Milan, ITALY - 8 May 2025 - Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, today announces an upcoming oral presentation showcasing critical preclinical insights into dosing for first-in-human in vivo liver-directed immune shielded lentiviral vector (ISLV) gene therapy for methylmalonic acidemia (MMA) at the American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting to be held May 13-17, 2025 in New Orleans.
Presentation details:
Oral presentation title: Definition of a Minimal Therapeutic Dose of In Vivo Liver-Directed Lentiviral Gene Therapy for Methylmalonic Acidemia
Session title: Gene and Cell Therapy for Metabolic Diseases
Date: Tuesday, May 13, 2025
Presenter: Dr. Elena Barbon, Research Scientist at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in collaboration with Genespire
Genespire is developing a novel proprietary class of lentiviral vectors, the Immune Shielded Lentiviral Vectors (ISLVs), with lead candidate GENE202 nearing clinical development for the treatment of methylmalonic acidemia (MMA), a devastating genetic disorder impairing the metabolism of certain amino acids and fats.
Enquiries:
| Genespire | Tel: +39 02 80896651 info@genespire.com (mailto:info@genespire.com) |
| ICR Healthcare | Tel: +44 (0) 20 3709 5700 |
| Amber Fennell/Ashley Tapp/Jonathan Edwards | Genespire@icrhealthcare.com (mailto:Genespire@icrhealthcare.com) |
About Genespire
Genespire is a biotechnology company, developing off-the-shelf gene therapies based on immune shielded lentiviral vectors.
