Back to search
Adeno-Associated Virus (AAV)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Adeno-Associated Virus (AAV) trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Adeno-Associated Virus (AAV) trials may be worth asking aboutNorth America
Europe
Asia-Pacific
Rest of World
Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric…
Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult part…
The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to t…
This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult p…
This is a multi-center, randomized, double-masked, active-comparator-controlled, Phase 3 study in a broad participant population (treatment-naïve and treatment-…
This Phase 1/2a, open-label, non-randomized study is designed to evaluate the safety and tolerability of intramuscular AAV9-Follistatin gene therapy administere…
This is a first-in-human clinical trial to test whether a protein administered into the brain continuously by gene therapy, Brain-Derived Neurotrophic Factor (B…
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor…