Publications

  1. Home
  2. Gene Therapy

Hemophilia

Adult lifetime cost of hemophilia B management in the U.S.: Payer and societal perspectives from a decision analytic model

Etranacogene Dezaparvovec (AMT-061 Phase 2b): Normal/Near Normal FIX Activity and Bleed Cessation in Hemophilia B

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

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-Existing Anti-AAV5 NABs 

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

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

Huntington's disease

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Secreted therapeutics: Monitoring durability of microRNA-based gene therapies in the central nervous system 

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

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

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in HD Mice 

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

Potent and Sustained Huntingtin Lowering via AAV5 Encoding miRNA Preserves Striatal Volume & Cognitive Function in a Humanized Mouse Model of HD

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes 

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

Technology

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

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