Why Quality Matters in AAV Gene Therapy Development
An overview of why product characterization, analytical methods, manufacturing consistency, and potency assessment matter in AAV gene therapy development.
New Book on Gene Therapy in Neurodegenerative Disorders Published by Elsevier
A new Elsevier book edited by Dr. Vahid Mansouri and Dr. Maryam Bemanalizadeh explores gene therapy approaches for neurodegenerative and neuromuscular disorders, from delivery technologies to clinical translation.
Newborn Screening Awareness Month: Early Detection, Earlier Intervention, Better Outcomes
Newborn screening offers a critical opportunity to identify rare genetic diseases early and enable timely interventions, including gene therapy for conditions such as SMA.
Exir Gene Pharmed Research Presented at the 2026 ASGCT Annual Meeting
Three abstracts involving members of the Exir Gene Pharmed team were presented at the 2026 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Boston.
New Publication: Long-Term Safety and Efficacy of AAV9-SMN1 Gene Therapy for SMA
A long-term preclinical study by members of our scientific team evaluated the safety and sustained transgene expression of systemic AAV9-mediated SMN1 gene delivery in neonatal mice.
Exir Gene Pharmed Research Presented at the 28th ASGCT Annual Meeting
Research by members of the Exir Gene Pharmed team was presented at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans in May 2025.