Differential Usage of Transcription Factor Binding Sites to Boost Synthetic Promoter Activity 2025 ASGCT Belamarich, C. et al Poster Presentation
Novel Rationally Designed Promoters Surpass CAG in Mouse and Human Models 2025 ASGCT Letko, A. et al Poster Presentation
Preclinical Efficacy and Potency Assay Development of An Optimized AAV-hUPF1 Gene Therapy for Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) 2025 ASGCT Bastiaans, J. et al Poster Presentation
An Ultra-Low Dose of a Localized CNS Gene Therapy for Severe Pediatric Obesity 2025 ASGCT Jin, C. et al Poster Presentation
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study The Lancet 2025; 405: 648-57. M. Michaelides et al
Evolution of a High Performing, Modular Platform for AAV Manufacturing 2024 ESGCT Gunn, R. et al Poster Presentation
AAV-based evaluation of novel in silico promoters to drive expression in rod photoreceptors 2024 ESGCT Naeem, A. et al Poster Presentation
Identification of highly potent and tissue-specific promoters with massively parallel screening 2024 ESGCT Jin, C. et al Poster Presentation
AAV-mediated gene therapy attenuates loss of vision in a mouse model of Bardet- Biedl-Syndrome 10 2024 ESGCT Klaska. et al Poster Presentation
Novel Splicing Driven Gene Regulation Platform for Precise Dose Responsive Control of Gene and Cell Therapies 2024 ESGCT Guo, X. et al Poster Presentation
A CNS-targeted gene therapy for the treatment of severe pediatric obesity SfN Neuroscience 2024 Jin, C. et al Poster Presentation
Controlled, Riboswitch-Regulated Gene and Cell Therapy Targeting HER2+ Tumors 2024 ASGCT Advancing CGT for Cancer, Guo et al. Poster presentation
Novel Splicing Driven Gene Regulation Platform for Precise Dose Responsive Control of Gene and Cell Therapies 2024 BioCentury Grand Rounds Guo et al. Poster Presentation
Riboswitch-regulated chimeric antigen receptor (RiboCAR) enhances CAR-T cell anti-cancer efficacy 2023 ESGCT. Guo et al. Poster Presentation
Riboswitch-regulated chimeric antigen receptor (RiboCAR) enhances CAR-T cell anti-cancer efficacy 2023 ASGCT Spotlight on IO . Guo et al. Poster Presentation
Riboswitch-regulated chimeric antigen receptor (RiboCAR) enhances T cell activity 2023 ASGCT. Guo et al. Poster Presentation
AAV-mediated riboswitch-controlled delivery of anti-HER2 antibody suppresses HER2-positive tumorigenesis 2023 ASGCT. Wang et al. Poster Presentation
AAV-mediated, small molecule-riboswitch-controlled delivery of growth hormone rescues growth in GH-deficient B. Little mice 2023 ASGCT. Guo et al. Poster Presentation
Titratable and reversible control of CAR-T cell receptor and activity by riboswitch via oral small molecule 2022 ESGCT. Guo et al. Poster Presentation
Novel riboswitches regulate AAV-delivered transgene expression in mammals via oral small molecule inducers 2022 ESGCT. Guo et al. Poster Presentation
Riboswitch-controlled delivery of therapeutic antibodies for gene therapy 2022 ESGCT. Guo et al. Poster Presentation
Riboswitch-controlled delivery of therapeutic hormones for gene therapy 2022 ESGCT. Guo et al. Poster Presentation
Novel riboswitches regulate AAV delivered transgene expression in mammals via small molecule inducers. ESGCT 2021. Guo et al. Poster Presentation.
Gene therapy for Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) Preclinical efficacy of AAV-hUPF1 optimized for clinical translation with improved vector genome and novel CNS capsid 2023 ESGCT. Nguyen-Vu, B. et al. Oral Presentation
Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction – The AQUAx Study 2024 AAOM. Brennan, M. et al. Oral Presentation
Results of a phase 1 open-label, dose escalation study of gene therapy with AAV2-hAQP1 as treatment for radiation-induced xerostomia and parotid gland hypofunction 2023 ESGCT. M. Brennan et al. Poster Presentation
Synthetic neuronal promoters that surpass synapsin in the central nervous system 2023 ESGCT. N. Fabela et al. Poster Presentation
fDISCO evaluation of AAV-mediated gene expression upon different routes of administration 2023 ESGCT. B. Han et al. Poster Presentation
Development of alternatives to Triton X-100 cell lysis for AAV2, AAV5 and AAV8 primary recovery 2023 ESGCT. E. de Heras et al. Poster Presentation
Potency assay cell line development for ocular gene therapy vectors 2023 ESGCT. G. Girona et al. Poster Presentation
Multiple in vitro differentiated skeletal muscle models for screening of synthetic muscle promotors for gene therapy 2023 ASGCT. Lee et al. Poster Presentation
Development of in vitro neuronal cytotoxicity models for neurodegenerative disease gene therapy research 2023 ASGCT. Lettko et al. Poster Presentation
Development of rationally designed CAG-based promoters for use in gene therapy 2023 ASGCT. Liu et al. Poster Presentation
Identification of novel inflammation-inducible promoters using a hybrid-barcoded SURE library 2023 ASGCT. Kassiani et al. Poster Presentation
AAV-based evaluation of novel in silico-based promotors to drive expression in rod photoreceptors 2023 ARVO. Klaska et al. Poster Presentation
Promoter Engineering Platform at MeiraGTx 2022 ESGCT. Liu et al. Poster Presentation
AI-driven promoter optimization at MeiraGTx 2022 ESGCT. Liu et al. Poster Presentation
A CNS-targeted gene therapy for the treatment of obesity 2022 ESGCT. Sullivan, et al. Poster Presentation
Development of an in-vitro testing platform for AAV-UPF1 gene therapy to treat ALS 2022 ESGCT. Gordon et al. Poster Presentation
Development of optimized ATP7B gene therapy vectors for the treatment of Wilson’s Disease with increased potency 2022 ESGCT. Sullivan et al. Poster Presentation
Using mechanistic models to design a platform process for the separation of full and empty AAV capsids 2023 ESGCT. J. Senarantne et al. Poster Presentation
Improving the elution step in capture chromatography: a mechanistic approach 2023 ESGCT. A. Molina et al. Poster Presentation
Towards ultra scale-down AAV production in microtiter plates 2023 ASGCT. Wiegmann et al. Poster Presentation
Understanding the factors that influence capsid-column affinity and peak profile in AEX-HPLC to measure empty:full ratio 2023 ASGCT. Whitehead et al. Poster Presentation
Multivariate analysis for increased understanding of MeiraGTx upstream process 2022 ESGCT. Valinhas et al. Poster Presentation
Improving AAV in vitro transducibility for cell-based potency assay development 2022 ESGCT. Georgiadis, et al. Poster Presentation
Use of anion exchange chromatography to provide high empty AAV capsid removal and product yields 2022 ESGCT. Senaratne et al. Poster Presentation
Designing and screening formulations to improve manufacturability and distribution of AAV gene therapies 2022 ESGCT. Senaratne et al. Poster Presentation
Optimization and scale-up of AAV2-AQP1 production using TransIT-VirusGEN 2022 ESGCT. Dzopa et al. Poster Presentation
First-in-Human Gene Therapy Trial of AAV8-hCARp.h CNGB3 in Adults and Children With CNGB3- associated Achromatopsia AJO 2023. M. Michaelides et al.
Humoral immune response to AAV5- RPGR (botaretigene sparoparvovec) gene therapy in RPGR -associated X-linked retinitis pigmentosa 2023 ARVO. Caratzas, E. et al. Oral Presentation
Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP) AAO 2022. M. Michaelides et al. Oral Presentation
AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy for X-Linked Retinitis Pigmentosa Demonstrates Localized Improvements in Static Perimetry ARVO 2022. M. Michaelides et al. Oral Presentation
AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): Human retinal organoid vector efficacy data ARVO 2022. P. Sladen et al. Poster Presentation
A demonstration of cone function plasticity after gene therapy in achromatopsia Brain 2022. M. Farahbakhsh et al.
RetGC-GUCY2D retinal organoid disease model for AAV gene therapy development. ESGCT 2021. A. Naeem, et al. Poster Presentation.
KCNV2 retinal organoid disease model for KCNV2 AAV gene therapy development. ESGCT 2021. S. Ferrara, et al. Poster Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa Reverses Natural Disease Progression. EURETINA 2021. M. Michaelides, et al. Oral Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 12-month Results From a Phase 1/2 Clinical Trial. AAO 2020. M. Michaelides, et al. Oral Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month Results From a Phase 1/2 Clinical Trial. EURETINA 2020. M. Michaelides, et al. Oral Presentation.
AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 6-month Results From a Phase 1/2 Clinical Trial. ASRS 2020. M. Michaelides, et al. Oral Presentation.
Development and efficacy assessment of AAV2/8-hG1.7p.coCNGA3, a CNGA3 gene therapy vector. ARVO 2019. A. Georgiadis, et al. Poster.
Efficacy and safety of AAV2/5-hRKp.RPGR to treat X-linked retinitis pigmentosa. ESGCT 2017. J. Smith, et al. Poster
Pre-clinical toxicology of AAV2/5-OPTIRPE65, an optimised RPE65 gene therapy vector. ESGCT 2017. A. Georgiadis, et al. Poster
Efficacy assessment and pre-clinical toxicology of AAV2/8-hCARp.hCNGB3, a CNGB3 gene therapy vector. ESGCT 2017. J. Telfer, et al. Poster
Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65. Gene Therapy, 2016. A. Georgiadis, et al.
Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa. Gene Therapy, 2015. B. Pawlyk, et al.
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy. Human Molecular Genetics, 2011. L. Carvalho, et al.
UPF1 delivered by novel expression-enhanced promoters protects cultured neurons in a genetic ALS model 2022 ESGCT. Liu et al. Poster Presentation
Gene therapy reduces Parkinson’s disease symptoms by reorganizing functional brain connectivity. Science Translational Medicine, 2018. Martin Niethammer, et al.
Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson’s disease. JCI Insight, 2017. M. Niethammer, et al.
AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomised trial. The Lancet, 2011. P. LeWitt, et al.
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson’s disease. PNAS, 2007. A. Feigin, et al.
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial. The Lancet, 2007. M. Kaplitt, et al.
