Gene Therapies
Gene therapies offer curative potential across a broad spectrum — from inherited to acquired diseases, from rare syndromes to common indications. Omega manages gene therapy programs from vector trial support to immunogenicity monitoring, from patient selection to long-term safety follow-up.
Vector Platforms and Beyond
Each gene therapy modality has its own scientific and operational requirements. AAV-based therapies demand immunogenicity and dose-limiting toxicity management; lentiviral therapies require insertional mutagenesis surveillance; mRNA-based therapies call attention to repeat dosing and cold chain logistics.
Omega provides the laboratory, genetic, and clinical operations infrastructure to meet each of these requirements through an integrated model.
Our Gene Therapy Capabilities
Vector Trial Support
Protocol design, regulatory affairs, and operations management for clinical trials of AAV (adeno-associated virus), lentiviral, and adenoviral vectors, and mRNA-based therapies.
Preclinical Biodistribution
Vector biodistribution, organ-specific expression, and toxicity studies at the GLP-compliant animal facility, combined with qPCR-based vector quantification.
Immunogenicity Monitoring
Anti-drug antibody (ADA) and neutralizing antibody (NAb) kinetics are monitored with standardized assays at the HelixLab central laboratory, supporting immunosuppression strategies.
Patient Selection & Screening
Genotypic confirmation, carrier screening, variant interpretation, and eligibility assessment with the Omega Genetics infrastructure; patient finding strategies in rare disease populations.
Hemophilia Gene Therapy Experience
Experience with PK/PD sampling, factor activity monitoring, and inhibitor screening in gene therapy trials for inherited bleeding disorders associated with the F8 and F9 genes.
Long-Term Safety Follow-Up
Annual follow-up visits per regulatory requirements, longitudinal biomarker sampling, and long-term sample storage at the Omega Biorepository.