Publications

Hero publications

Huntington’s disease

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

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

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  • 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

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

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  • AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

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

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

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

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

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

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Hemophilia

  • Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

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  • Evidence of a disability paradox in patient-reported outcomes in haemophilia

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  • Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

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

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  • Health care resource utilization and cost burden of hemophilia B in the United States

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  • Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

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

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

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

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

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

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Technology

  • PhP.B Enhanced Adeno-Associated Virus Mediated-Expression Following Systemic Delivery or Direct Brain Administration

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  • Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery

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

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

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

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

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

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