Investors & Newsroom:

Publications

Pipeline/Products 

Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic

Gene Therapy with Adeno-Associated Virus Vector 5-Human Factor IX in Adults with Hemophilia B

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia-B

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntington for Development of Gene Therapy for Huntington’s Disease

AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

Vector Development

Optimization of viral protein ratios for production of rAAV serotype 5 in the baculovirus system

Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5ch and AAV1

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

Insect Cell-Based Recombinant Adeno-Associated Virus Production: Molecular Process Optimization

Prevalence of Serum IgG and Neutralizing Factors Against AAV Types: 1,2,5,6,8, and 9 in Healthy Population

Phase I Open Label Liver-Directed Gene Therapy Clinical Trial for Acute Intermittent Porphyria

Glybera (alipogene tiparvovec)

From mutation identification to therapy: Discovery and origins of the first approved gene therapy in the Western World