WORLDSymposium™ 2026 Preliminary Program* on Lysosomal Diseases
On Monday afternoon, WORLDSymposium presents an exciting program, Patient Voice 2026: Why Does Publish or Perish Apply to Patient Advocate Groups? The goal of this 1-hour CE-session is to identify why it is important to have industry, clinician and advocates involved; the importance of peer-reviewed published data; describe several different approaches to publishing; and illustrate examples of the results and impact of publications.
Following the Patient Voice session, the 4th Annual Speed Mentoring Event will offer a once-in-a-lifetime opportunity for attendees to meet with a wide variety of leaders and icons from the lysosomal and rare disease space. The evening concludes by celebrating 22 Years of WORLDSymposium, with the 4th Annual “Be the Catalyst” event and Catalyst Award presentation. Open to all WORLDSymposium participants, this event provides opportunities to participate in all of the fun scheduled group photos, reconnect with colleagues, make new connections, establish new relationships, welcome new attendees, and celebrate the achievements of past and present WORLDSymposium Award Recipients.
| 15:00 | The Patient Voice 2026 CE Session Accredited provider: Medical Education Resources (MER) | Why Does Publish or Perish Apply to Patient Advocate Groups? This activity is supported, in part, by educational grants from Amgen Inc., JCR Pharmaceuticals, and Ultragenyx Pharmaceutical Inc. |
| 16:30 | Speed Mentoring Event | Scheduled Mentoring Appointments and Complimentary Headshots |
| 16:30 | Industry Expert Theater Sponsored by NS Pharma | Panel Discussion: Challenges and new opportunities in MPS II |
| 17:45 | Be the Catalyst Event | 3rd Annual Catalyst Award presented to Professor Michael H. Gelb |
After the presentation of the Innovation Award, the formal scientific sessions of WORLDSymposium 2026 officially began with presentations on laboratory research for lysosomal disease. Presentations during the Basic Science sessions are designed to improve our understanding or prediction of the phenomena involved in lysosomal pathology at a molecular, cellular, and animal model level in order to forwardly think about diagnosis and treatment of lysosomal conditions. These Basic Science sessions are always innovative and present the latest findings in the field. Download the WORLDSymposium 2026 program (PDF 200KB).
Basic Science
Co-Chairs: Lalitha Belur, Michael Przybilla, Dan Tagle
| 06:45 | Non-CE Satellite Symposium Sponsored by Amicus Therapeutics | Transforming Ambiguity to Action for Personalized Fabry Care |
| 06:45 | Non-CE Satellite Symposium Sponsored by Takeda Pharmaceutical Company Limited | The maturation of evidence: what time teaches us about MPS II |
| 08:00 | Chester B. Whitley University of Minnesota Minneapolis, MN, United States | Welcome and Announcements Presentation of 2026 Roscoe O. Brady Award to Peter Marks |
| Peter Marks Eli Lilly and Company Washington DC, United States | Roscoe O. Brady Award Presentation Accelerating the Global Pace of Progress for Rare Diseases | |
| 08:30 | Elizabeth Braunlin University of Minnesota Minneapolis, MN, United States | Creation and characterization of a large animal model of MPS IVA |
| Angela Gritti Università Vita-Salute San Raffaele Milano, Italy | Therapeutic benefits of hematopoietic stem cell gene therapy using optimized bicistronic lentiviral vectors in mouse models of GM2 gangliosidosis | |
| Ewa A. Ziolkowska Washington University St. Louis St. Louis, MO, United States | Neuropathological alterations in TPP1-deficient cynomolgus macaques recapitulate key features of human CLN2 disease | |
| Martyna Kasprzyk University of Edinburgh Edinburgh, United Kingdom | Production and characterisation of a functional recombinant human palmitoyl-protein thioesterase 1 in K. phaffiifor the treatment of CLN1 | |
| Moderated Q&A | Braunlin, Gritti, Ziolkowska, Kasprzyk | |
| 09:30 | Shiny Nair Yale University New Haven, CT, United States | Spatially resolved mechanisms of liver carcinogenesis in Gaucher disease implicate lipid-driven immunosuppressive niches |
| Xiangli Zhao Yale University New Haven, CT, United States | A progranulin derivative blocks the C5a/C5aR1 signaling and mitigates pathology in Gaucher disease | |
| Ying Sun Cincinnati Children’s Hospital Medical Center Cincinnati, OH, United States | Brain delivery of long-acting enzymes via SapC-DOPS nanocarrier for neuronopathic Gaucher disease | |
| Pau Sarlé Vallés Universitat Autònoma de Barcelona Bellaterra, Spain | Next-generation enzyme replacement strategies for Gaucher disease *2026 Young Investigator Award Recipient | |
| Moderated Q&A | Nair, Zhao, Sun, Sarlé Vallés | |
| 10:30 | Break | |
| 11:00 | Matthias Dierick University of Edinburgh Edinburgh, United Kingdom | Engineering low immunogenic enzyme replacement therapies for Fabry disease *2026 Young Investigator Award Recipient |
| Stephanie Tannous University of Veterinary Medicine Hannover Hannover, Germany | Lipid-dependent uptake of pegunigalsidase alfa across cell types: Implications for multiorgan treatment | |
| David Dmitrivich Smerkous University of Washington Seattle, WA, United States | A novel highly sensitive machine learning model for automated measurement of kidney peritubular capillary endothelial cell globotriaosylceramide accumulation | |
| Jessica Doxey Duke University Durham, NC, United States | The important role of CRIM analysis by western blot in the setting of novel GAA variants | |
| Moderated Q&A | Dierick, Tannous, Smerkous, Doxey | |
| 12:00 | Break and Satellite Symposia | |
| 12:15 | Non-CE Satellite Symposium Sponsored by Sanofi | Evolving Landscape of LDs in Era of Newborn Screening: Monitoring and Treatment Initiation |
| 12:15 | Non-CE Satellite Symposium Sponsored by Takeda Pharmaceutical Company Limited | Optimizing lifelong therapy in Fabry disease: navigating complexity and maximizing impact |
| 13:30 | Maria Athanasopoulos McMaster University Hamilton, ON, Canada | Decoding NEU1 – a molecular switch linking sialidosis, hepatic receptor regulation, and atherosclerosis *2026 Young Investigator Award Recipient |
| Mahin Hossain National Human Genome Research Institute, National Institutes of Health Bethesda, MD, United States | Glial cell dysfunction and neurodegeneration in a novel knock-in mouse model of lysosomal free sialic acid storage disorder *2026 Young Investigator Award Recipient | |
| Jordi Diaz-Manera Newcastle University Newcastle Upon Tyne, United Kingdom | Cartography of transcriptomic changes in muscle biopsies of patients with late-onset Pompe disease | |
| Shih-Chang Hsueh Columbia University New York, NY, United States | Novel cyclodextrins enhance potency of neutralization and clearance of endogenous psychosine in cellular and in vivo models of Krabbe disease | |
| Moderated Q&A | Athanasopoulos, Hossain, Diaz-Manera, Hsueh | |
| 14:30 | Katia Alileche Universite Paul Sabatier Toulouse, France | Investigating microglial heterogeneity in a mouse model of mucopolysaccharidosis type IIIB |
| Richard Steet Greenwood Genetic Center Greenwood, SC, United States | MPS I missense variants that cause a gain of glycosylation effect are amenable to treatment with novel glycosylation inhibitors | |
| Ibrar Siddique University of California, Los Angeles Los Angeles, CA, United States | Blood-based biomarkers for mucopolysaccharidosis type I and III | |
| Keerthana Iyer University of Pennsylvania Philadelphia, PA, United States | Comparative proteomic analysis of saliva, urine, and serum in mucopolysaccharidosis type I patients | |
| Moderated Q&A | Alileche, Steet, Siddique, Iyer | |
| 15:30 | Basic Science Poster Session | Exhibits Open |
| 15:45 | Industry Expert Theater Sponsored by Amicus Therapeutics | |
| 17:45 | Non-CE Satellite Symposium Sponsored by Chiesi Global Rare Diseases | Closing the Loop: From Clinical Management to Patient Experience with Elfabrio® (pegunigalsidase alfa-ivxj) |
| 17:45 | Non-CE Satellite Symposium Sponsored by Azafaros B.V. | The value of conducting an 18-month placebo-controlled study in Rare Diseases: GM1, GM2 and NPC; the NAVIGATE experience |
After the presentation of the 2026 Young Investigator Awards and the Patient Advocate Leader (PAL) awards, the entirety of the research presentations on Wednesday are dedicated to the Translational Research category. In 2026, many of the presentations will be dedicated to research topics in gene therapy, including innovations occurring in genetic therapeutic approaches in translation from laboratory to the clinic. Download the WORLDSymposium 2026 program (PDF 200KB).
Translational Research
Co-Chairs: Tierra Bobo, PJ Brooks, Francyne Kubaski
| 06:45 | Non-CE Satellite Symposium Sponsored by Astellas Pharma Inc | From Diagnosis to Care: Transforming rare diseases with gene therapies and advanced technologies |
| 06:45 | CE Satellite Symposium Accredited provider: AffinityCE Jointly Provided by: AffinityCE and Lysosomal & Rare Disorders Research & Treatment Center, Inc (LDRTC) | Shared Mechanisms and Clinical Implications of Secondary GSL Deposition in Lysosomal Disorders |
| 08:00 | Chester B. Whitley University of Minnesota Minneapolis, MN, United States | Welcome and Announcements Presentation of 2026 Patient Advocate Leader (PAL) Award to Bob Stevens and 2026 Young Investigator Awards Presentation |
| 08:30 | Françoise Piguet Paris Brain Institute Paris, France | Novel intravenous AAV gene therapy for mucopolysaccharidosis type IIIA and IIIB in mouse and canine model of the pathology – towards clinical translation |
| Rafael A. Badell-Grau University of California, San Diego La Jolla, CA, United States | Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC | |
| Sampurna Saikia University of Delaware Newark, DE, United States | The complementary strength of the AAV9 gene therapy in hematopoietic stem cells transplanted into MPS IVA mice *2026 Young Investigator Award Recipient | |
| Jillian Gallagher University of Massachusetts Worcester, MA, United States | Testing a dual AAV gene therapy vector construct to treat sialidosis and galactosialidosis using small and large animal models | |
| Moderated Q&A | Piguet, Badell-Grau, Saikia, Gallagher | |
| 09:30 | Udayanga Wanninayake Saint Louis University St. Louis, MO, United States | Enhanced CIMPR binding and cellular uptake of HP-GALNS (M161): A next-generation enzyme replacement therapy for Morquio syndrome type A *2026 Young Investigator Award Recipient |
| Jose Victor Alvarez Gonzalez Instituto de Investigación Sanitaria de Santiago de Compostela Santiago de Compostela, Spain | Advances in the administration of ERT (oral pathway) improving the biodistribution of enzymes *2026 Young Investigator Award Recipient | |
| Chia-Feng Yang Taipei Veterans General Hospital Taipei, Taiwan | Long-term outcomes of very early treated infantile-onset Pompe disease with the improvement after 24-month switching to avalglucosidase alfa: Real-world experiences based on Taiwan nationwide newborn screening program | |
| Allan Feng Stanford University Stanford, CA, United States | Therapeutic efficacy of a novel glucocerebrosidase variant in a new preclinical model of neuronopathic Gaucher disease | |
| Moderated Q&A | Wanninayake, Alvarez Gonzalez, Yang, Feng | |
| 10:30 | Break and Exhibits | |
| 11:00 | Shelly Goomber Duke University Durham, NC, United States | Functional profiling-based evaluation of GAA VUS in Pompe disease using a transient expression system upgraded for capacity and robustness |
| Xuntian Jiang Washington University St. Louis St. Louis, MO, United States | Diagnostic and therapeutic applications of the glycan biomarker H3N2b in GM1 gangliosidosis | |
| Melissa Greco Virginia Tech Roanoke, VA, United States | Infantile Krabbe disease presenting with intermediate psychosine (2-10 nmol/L) values in dried bloodspots *2026 Young Investigator Award Recipient | |
| Troy Lund University of Minnesota Minneapolis, MN, United States | GAG endogenous non-reducing ends as a new biomarker in CSF and plasma for Hurler syndrome | |
| Moderated Q&A | Goomber, Jiang, Greco, Lund | |
| 12:00 | Break, Exhibits and Satellite Symposia | |
| 12:15 | CE Satellite Symposium Accredited provider: Medical Education Resources (MER) Jointly Provided by MER and Saterdalen & Associates LLC Supported by an independent educational grant from Takeda Pharmaceuticals U.S.A., Inc. | Leveraging Imaging Strategies for Bone Disease in Gaucher Disease: A Case Study Deep Dive |
| 12:15 | Non-CE Satellite Symposium Sponsored by Amicus Therapeutics | Optimizing late-onset Pompe disease (LOPD) monitoring in an era of multiple treatment options |
| 13:30 | Anna-Maria Wiesinger Paracelsus Medical University Salzburg Salzburg, Austria | A decision analysis framework for individualized immunomodulatory therapy in MPS: Early clinical insights |
| Chester B. Whitley University of Minnesota Minneapolis, MN, United States | Long-term outcome of Hurler syndrome following bone marrow transplantation | |
| Ashwin Roy University of Birmingham Birmingham, United Kingdom | Early atrial remodeling: A driver of arrhythmia in Fabry disease | |
| Salvatore Recupero San Raffaele Scientific Institute Milan, Italy | Gallbladder abnormalities in metachromatic leukodystrophy: Preliminary analysis in patients treated with atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) and untreated patients | |
| Moderated Q&A | Wiesinger, Whitley, Roy, Recupero | |
| 14:30 | Tae Un Han National Institutes of Health Bethesda, MD, United States | Development of a novel systemic AAV gene therapy for neuronopathic Gaucher disease |
| Grace R. Kick Washington University St. Louis St. Louis, MO, United States | Efficacy of AAV-mediated gene therapy in a sheep model of CLN1 disease | |
| Alex Liu Huang Boston Children’s Hospital Boston, MA, United States | Self-amplifying mRNA enhances transamniotic fetal mRNA delivery *2026 Young Investigator Award Recipient | |
| Surendra Raj Sharma University of North Carolina Chapel Hill Chapel Hill, NC, United States | Rapid transient antibody depletion using genetically engineered IgG-degrading enzyme allows efficient rAAV9 gene delivery in an α-AAV9-Ab+ rabbit model | |
| Moderated Q&A | Han, Kick, Huang, Sharma | |
| 15:30 | Translational Research Poster Session | Exhibit Hall |
| 15:45 | Industry Expert Theater Sponsored by Sanofi | |
| 17:45 | Non-CE Satellite Symposium Sponsored by Sanofi | Biomarkers in Gaucher Disease: A Critical Dialogue on Patient Care Evolution Through Clinical Cases |
| 17:45 | Non-CE Satellite Symposium Sponsored by Chiesi Global Rare Diseases | Rational Design Meets Real-World Relevance: Pegunigalsidase Alfa in the Treatment of Fabry Disease |
Thursday begins with a special Catalyst Award keynote address from Dr. Michael H. Gelb: “Biochemical newborn screening for all treatable lysosomal diseases.” Following Dr. Gelb’s address, the presentations shift to Clinical Applications, including abstracts on Clinical Trials for Registration. Abstracts presented in this category had a US FDA Investigational New Drug (IND) application for a phase I-III clinical trial or hold an EMA Investigational Medicinal Product Dossier (IMPD) or equivalent. Clinical Outcomes abstracts will also be presented. Download the WORLDSymposium 2026 program (PDF 200KB).
Clinical Applications
Co-Chairs: Rebecca Ahrens-Nicklas, Roberto Giugliani, Filippo Vairo
| 06:45 | CE Satellite Symposium Accredited provider: AKH Inc., Advancing Knowledge in Healthcare Jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC Supported by an independent educational grant from Amicus Therapeutics, Inc. | Homing in on Holistic Management of Late Onset Pompe Disease: Contemporary Insights on Disease Monitoring, Treatment Selection and Switching, and Shared Decision-Making |
| 06:45 | Non-CE Satellite Symposium Sponsored by Denali Therapeutics | Transforming Patient Care in MPS II |
| 08:00 | Chester B. Whitley University of Minnesota Minneapolis, MN, United States | Welcome and Keynote Speaker Introduction |
| Michael H. Gelb University of Washington Seattle, WA, United States | Special Catalyst Award Keynote Address: Biochemical newborn screening for all treatable lysosomal diseases | |
| 08:30 | John A. Bernat University of Iowa Health Care Iowa City, IA, United States | Isaralgagene civaparvovec (ST-920) shows positive mean annualized eGFR slope in adults with Fabry disease: Topline results from the registrational phase 1/2 STAAR gene therapy study and long-term follow-up study |
| Michael L. West Dalhousie University Halifax, NS, Canada | Comparison of outcomes of the FACTS lentivirus mediated gene therapy trial in Fabry disease with controls from the Canadian Fabry Disease Initiative Registry (CFDR) | |
| Dominique P. Germain University of Versailles – University Paris Saclay Montigny, France | Hidden double hits in Fabry disease: A critical confounder in clinical trials | |
| Peter Nordbeck University Hospital Würzburg Würzburg, Germany | Real-world effectiveness of migalastat versus enzyme replacement therapy in previously treatment-naïve patients with Fabry disease: Analyses of matched populations from the global followME Pathfinders registry | |
| Moderated Q&A | Bernat, West, Germain, Nordbeck | |
| 09:30 | Ida Vanessa D. Schwartz Federal University of Rio Grande do Sul Porto Alegre, Brazil | Two-year follow up of FLT201 AAV gene therapy in adults with type 1 Gaucher disease: Results from GALILEO-1 and GALILEO-2 |
| Deepa Rajan University of Pittsburgh Pittsburgh, PA, United States | Interim results from the PROVIDE Clinical Trial – A phase I/II Study of LY3884961(PR001) an AAV9-based gene therapy for type 2 Gaucher disease | |
| Aimee Donald University of Manchester Manchester, United Kingdom | Lentiviral stem cell gene therapy of neuronopathic Gaucher disease (GD3) achieves prolonged enzyme delivery, substrate reduction and stabilisation of neurologic and somatic disease manifestations | |
| Pramod K. Mistry Yale University New Haven, CT, United States | Beyond ERT/SRT: A neuro-pulmonary-lymphatic spatial-omics atlas redefines pathology and guides immunotherapy in neuronopathic Gaucher disease | |
| Moderated Q&A | Schwartz, Rajan, Donald, Mistry | |
| 10:30 | Break and Exhibits | |
| 11:00 | Joseph Muenzer University of North Carolina Chapel Hill, NC, United States | Phase I/II study of intravenous tividenofusp alfa for mucopolysaccharidosis type II |
| Can Ficicioglu The Children’s Hospital of Philadelphia Philadelphia, PA, United States | Effect of clemidsogene lanparvovec (RGX-121), an investigational gene therapy, on neurodevelopmental outcomes in patients with Hunter syndrome | |
| Simon A. Jones St. Mary’s Hospital Manchester, United Kingdom | Ex-vivo modification of autologous CD34+ HSPCs using a CD11b-directed lentiviral vector encoding ApoEII-tagged human IDS leads to supraphysiological enzyme activity and biochemical correction of neuronopathic MPS II patients | |
| Elizabeth Jalazo University of North Carolina Chapel Hill Chapel Hill, NC, United States | Preliminary results from phase I/II, first-in-human, open-label study of DNL126 in children with mucopolysaccharidosis type IIIA (MPS IIIA) | |
| Moderated Q&A | Muenzer, Ficicioglu, Jones, Jalazo | |
| 12:00 | Break, Exhibits and Satellite Symposia | |
| 12:15 | Non-CE Satellite Symposium Sponsored by Zevra Therapeutics | Charting a Path in Niemann-Pick Disease Type C: Diagnostic Challenges, Therapeutic Innovations, and Real-World Patient Cases |
| 12:15 | Non-CE Satellite Symposium Sponsored by Sanofi | From the Kidney and Beyond: Early, Multi-Organ Impacts in Fabry Disease |
| 13:30 | Brian Bigger University of Edinburgh Edinburgh, United Kingdom | Sustained biochemical correction and improved neurological outcomes at 36-months post hematopoietic stem cell gene therapy for Sanfilippo syndrome |
| Nicole M. Muschol University Medical Center Hamburg-Eppendorf Hamburg, Germany | Long-term administration of tralesinidase alfa enzyme replacement therapy (TA-ERT) results in profound and durable reduction of heparan sulfate (HS) and stabilization of cognitive function and cortical gray matter volume (CGMV) in patients with Sanfilippo syndrome type B (MPS IIIB) | |
| Ozlem Goker-Alpan Lysosomal & Rare Disorders Research & Treatment Center, Inc Fairfax, VA, United States | Safety, tolerability and biological activity of ABX1100, a CD71 centyrin siRNA conjugate targeting GYS1 in late-onset Pompe disease patients | |
| Priya S. Kishnani Duke University Durham, NC, United States | Symptom onset, disease biomarkers, and treatment status in US Pompe disease patients identified by newborn screening | |
| Moderated Q&A | Bigger, Muschol, Goker-Alpan, Kishnani | |
| 14:30 | Roberto Giugliani Federal University of Rio Grande do Sul Porto Alegre, Brazil | Long-term data from a phase II study with oral nizubaglustat for late-infantile/juvenile GM2 and NPC diseases (RAINBOW) |
| Barbara MacFee OPEN Health Parsippany, NJ, United States | Outcomes of patients with rapidly progressive lysosomal acid lipase deficiency treated with sebelipase alfa before hematopoietic stem cell transplantation | |
| Elizabeth Berry-Kravis Rush University Medical Center Chicago, IL, United States | Adrabetadex treatment in individuals with Niemann-Pick disease type C1 re-establishes cholesterol trafficking, resulting in decreased markers of neuronal damage and cell death | |
| Caroline Aimee Hastings UCSF Benioff Children’s Hospital Oakland Oakland, CA, United States | Real-world safety and effectiveness of arimoclomol in patients with NPC: Outcomes from the US early access program (EAP) over a 4-year period | |
| Moderated Q&A | Giugliani, MacFee, Berry-Kravis, Hastings | |
| 15:30 | Clinical Applications Poster Session | Exhibit Hall |
| 15:45 | Industry Expert Theater Sponsored by BioMarin Pharmaceutical | |
| 17:45 | 5th Annual Robert J. Gorlin Symposium CE Session Accredited provider: Medical Education Resources (MER) | Global Access to Newborn Screening: A Call to Action! This activity is supported in part by educational grants from BioMarin Pharmaceutical Inc., Chiesi USA, Inc., Denali Therapeutics, Takeda Pharmaceuticals USA, Inc., and Ultragenyx Pharmaceutical Inc. |
The fourth research day of the meeting begins with the New Treatment Awards. Then, for the seventh year, the Contemporary Forum will allow for presentation of scientific abstracts — Basic, Translational, and Clinical — submitted by industry first-author researchers. Although the first three days of WORLDSymposium are accredited and approved for CE credit, Commercial Interests are not eligible for ACCME accreditation. The Contemporary Forum allows commercial interests to present their work to the WORLDSymposium audience, in this non-CE session, while being held to all the same standards as the ACCME accredited sessions and scored for merit and interest by the same Program Committee.
Toward bringing the most recent research to the platform of WORLDSymposium 2026, after the late-breaking abstract submissions close on December 1, 2025, selected late-breaking abstracts will be identified by the Program Committee as being suitable for platform presentation. In order to provide access to the “hot-off-the-presses” content from these researchers, late-breaking abstracts will be reviewed and scored, and the top-scoring abstracts were selected for presentation during the 2025 meeting. Download the WORLDSymposium 2026 program (PDF 200KB).
Contemporary Forum, Late-breaking Science and the Rapid Fire Competition
Contemporary Forum, Late-breaking Science Co-Chairs:
Marc Patterson, Uma Ramaswami, Cyndi Tifft
| 07:30 | Chester B. Whitley University of Minnesota Minneapolis, MN, United States | Welcome and New Treatment Awards |
| 08:00 | Heather A. Lau Ultragenyx Pharmaceutical Inc Novato, CA, United States | Treatment with UX111 reduced cerebrospinal fluid (CSF) heparan sulfate (HS) exposure and stabilized or improved functioning across dose, age, and stage of MPS IIIA |
| Daniel M. Virga Regeneron Pharmaceuticals Tarrytown, NY, United States | Anti-transferrin receptor 1-targeted AAV9 therapy prevents CNS and visceral pathologies in acid sphingomyelinase deficiency | |
| Marc C. Patterson IntraBio, Inc. Austin, TX, United States | Long-term findings of N-acetyl-L-leucine for Niemann-Pick disease type C | |
| Yanmei Lu Sangamo Therapeutics Richmond, CA, United States | Isaralgagene civaparvovec (ST-920) gene therapy for adults with Fabry disease: Pharmacology and immunogenicity outcomes from the phase 1/2 STAAR study and ongoing long-term follow-up | |
| Moderated Q&A | Lau, Virga, Patterson, Lu | |
| 10:00 | Break | |
| 10:30 | Late-Breaking Abstracts | 4 Speakers To Be Announced (TBA) with Moderated Q&A |
| 11:30 | Break |
Rapid Fire Competition
Back by Popular Demand: The fast-paced and energetic Rapid Fire Competition. Format will be a 5-minute, 5-slide presentation, followed by 5 minutes of live audience Q&A. Speakers will be competing for prizes, and the audience will vote for their favorites.
Rapid Fire Co-Chairs: Amy Gaviglio, Francyne Kubaski, Marc Patterson
| 12:30 | Karolina M. Stepien Salford Royal Hospital Salford, United Kingdom | Evidence of secondary mitochondrial dysfunction in alpha-mannosidosis |
| 12:40 | David Moreno Martínez Cambridge University Hospitals Cambridge, United Kingdom | Neuronal dysfunction beyond lysosomes in Fabry disease: Evidence from iPSC-derived forebrain neurons |
| 12:50 | Betul Celik Nemours Children’s Health Wilmington, DE, United States | Co-transplantation of hematopoietic stem cells and highly purified rapidly expanding clones (REC) of human mesenchymal stem cells rescued the bone pathology of MPS IVA mice |
| 13:00 | Ryunosuke Sanada National Center for Child Health and Development Tokyo, Japan | Development of gene editing technologies to correct a mutation in GNPTAB of mucolipidosis type II/III patients *2026 Young Investigator Award Recipient |
| 13:10 | Maria Ester Bernardo San Raffaele Scientific Institute Milan, Italy | Sustained supraphysiological alpha-L-iduronidase (IDUA) activity, reduction of glycosaminoglycans (GAGs), and clinical benefits at 5 years post-treatment with OTL-203, an autologous hematopoietic stem cell gene therapy (HSC-GT), in patients with mucopolysaccharidosis type I (MPS IH) Hurler syndrome |
| 13:20 | Tahseen Mozaffar University of California, Irvine Orange, CA, United States | 208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers |
| 13:30 | Kathy E. Meyer Sangamo Therapeutics Richmond, CA, United States | A combined fertility, embryofetal development, AAV integration and germline transmission risk study in mice with isaralgagene civaparvovec (ST-920) for Fabry disease |
| 13:40 | Petra Oliva ARCHIMEDlife GmbH Vienna, Austria | Breaking barriers in lysosomal disorder screening: A novel simultaneous LC-MS/MS approach for Tay-Sachs, Sandhoff, and GM1 gangliosidosis diseases |
| 13:50 | Rapid Fire Abstract | 4 Speakers To Be Announced (TBA) |
| 14:00 | Rapid Fire Abstract | |
| 14:10 | Rapid Fire Abstract | |
| 14:20 | Rapid Fire Abstract | |
| 14:30 | Rapid Fire Abstract Competition Voting | Voting and Awards |
| 14:40 | WORLDSymposium 2026 Adjourns |
*This will be a preliminary program only. ALL times and speakers will be subject to change. Be sure to check back weekly as updates are made.
Basic Science, Translational Research, and Clinical Application Sessions are available for CE credits. Contemporary Forum and Late-Breaking Science Sessions are not available for CE credits, however CEUs for GCs may apply.
Attention to All Platform Speakers: It is the policy of WORLDSymposium to publish all abstracts with the list of authors exactly as the abstract was submitted to WORLDSymposium. The first author of the submitted abstract will be listed as the Platform Speaker (presenting author) on the Preliminary Program, Agenda, and Poster List.
All requests for changes to the Platform Speaker (presenting author) will be reviewed by the WORLDSymposium Planning Committee prior to approval. No changes will be made to the Preliminary Program posted online; any changes to the Platform Speaker will be announced from the podium by the Moderators only at the time of the platform presentation. The Preliminary Program will remain published with the original first author, and all communication will continue to go to the first author of the abstract.
The submitting first author will continue to receive all instructions from the automated abstract notification system. Please be sure that the first author is aware they will need to forward any email instructions for PowerPoint preparation, and for advance review of presentations. Meeting the presentation guidelines and submitting presentations for prior review must be done to meet ACCME accreditation requirements.
Reference the Frequently Asked Questions pages for additional information.