GRIDS 2026 Symposium

Program Agenda

Advancing Therapeutic Development and Clinical Trials
in Lysosomal & Rare Disorders

Tentative program — subject to change. Times shown are draft and may be adjusted.

Program format: Each presentation is 20 minutes, with an open Q&A involving all session speakers at the end of each session. Coffee breaks are 15 minutes and lunch is 60 minutes. The Regulatory Keynote Panel runs 90 minutes total (introductory talk + three panelist perspectives + moderated Q&A).

Day 1
Trial Design, Endpoints, Biomarkers & Safety
8:00 AM Check-In · 9:00 AM – 5:05 PM
🍳 Breakfast & Check-In · 8:00 AM
9:00 AM · Welcome, Faculty Introductions & Program Overview
Introductory Talk 9:05 AM

Thirty Years of Progress and Unfinished Business in Rare Disease Clinical Trials: What Lysosomal Diseases Have Taught Us — and What We Still Get Wrong

Session 1 Regulatory Keynote Panel 9:40 – 10:55 AM

What It Takes to Get It Approved: Navigating FDA Expectations in Lysosomal and Rare Disease Clinical Development

  • 9:40 AMFrom Discovery to Approval: The complexities in translating scientific discoveries into approved therapies
  • 9:55 AMCurrent regulatory expectations, endpoint acceptability, and interpretation of safety signals; what sponsors miss in FDA submissions
  • 10:10 AMSponsor / Investigator Perspective: 30 years navigating FDA from NIH, academia, and industry — what works and what does not across Fabry, Gaucher, NPC, and leukodystrophy programs
  • 10:25 AMFDA Perspective: Regulatory considerations in rare disease drug development, followed by moderated panel Q&A
☕ Coffee Break · 10:55 AM
Session 2

Designing Trials That Succeed in Rare Diseases

11:05 AM – 12:50 PM
  • 11:05 AMTrial architecture for small, heterogeneous populations — lessons from NPC, sphingolipidoses, and rare neurodegenerative LSDs
  • 11:25 AMAdaptive, pragmatic, and decision-focused trial designs
  • 11:45 AMBayesian and adaptive methodologies that are acceptable to regulators
  • 12:10 PMUse of composite endpoints in Gaucher disease and other rare disorders
  • 12:30 PMSession Q&A — Open discussion with all session speakers
🍽 Lunch Break · 12:50 PM
Session 3

Developing Clinical Endpoints That Matter to Patients

1:30 – 3:00 PM

Measuring meaningful benefit beyond biochemical correction

  • 1:30 PMAppropriate use of natural history data as external controls: when it works and when it fails
  • 1:50 PMEndpoint selection in pediatric-onset lysosomal disorders and prediction of adult outcomes
  • 2:10 PMPatient-centered outcomes and trial burden in lysosomal disorders
  • 2:30 PMSession Q&A — Open discussion with all session speakers
☕ Coffee Break · 3:00 PM
Session 4

Biomarkers and Long-Term Monitoring in Clinical Trials

3:15 – 5:05 PM

Biomarkers as decision-support tools; distinguishing disease progression from treatment-related risk

  • 3:15 PMPharmacodynamic vs. disease-burden biomarkers: Fabry disease as the model case
  • 3:35 PMRenal biomarker harmonization and longitudinal interpretation
  • 3:55 PMBiomarkers for patient selection and enrichment in NPC trials: oxysterols, NfL, and cholestane-triol
  • 4:15 PMInterpreting biomarkers in treated and late-treated patients: when numbers mislead
  • 4:35 PMSession Q&A — Open discussion with all session speakers
5:05 PM · End of Day 1
Day 2
Therapeutic Modalities & Clinical Implementation
9:00 AM – 1:30 PM
Session 5

Small Molecules, Chaperones & Substrate Reduction: Clinical Reality

9:00 – 10:00 AM

Partial responders, CNS vs. peripheral benefit, and durability

  • 9:00 AMThe clinical trial endpoint journey, and lessons learned on how to design a trial for approval for small molecules
  • 9:20 AMLessons from GBA1-associated Parkinson disease trials
  • 9:40 AMNPC as the case study: NALL, the NPCCSS endpoint journey, and lessons from a CRL to first FDA approval
Session 6

Enzyme, Biologic & Gene Therapies: Clinical Execution

10:00 – 11:45 AM

Immunogenicity, residual disease, and real-world effectiveness

  • 10:00 AMResidual disease in treated Fabry patients and implications for future trials
  • 10:20 AMNeuronopathic lysosomal disorders in the treated era: biomarkers, neurocognition, and residual disease
  • 10:40 AMChallenges in global clinical trial execution for sphingolipidoses
  • 11:00 AMSession Q&A — Open discussion with all session speakers
☕ Coffee Break · 11:30 AM
Session 7

Gene Therapy: Clinical Trial & Post-Trial Reality

11:45 AM – 1:30 PM

Clinical management after dosing

  • 11:45 AMDurability of response and loss of efficacy following gene therapy
  • 12:05 PMWhen stabilization equals success: endpoints in neurodegenerative lysosomal disorders
  • 12:25 PMPost-trial monitoring and real-world implementation following gene therapy
  • 12:45 PMImmune risk stratification and patient eligibility criteria for gene therapy trials
  • 1:05 PMSession Q&A — Open discussion with all session speakers
  • 1:25 PMClosing Remarks & Adjournment
1:30 PM · End of Program
Attendee Information
Accommodations & Travel
Shuttle Service

Complimentary Airport Shuttle

LDRTC provides a complimentary shuttle from Orlando International Airport (MCO) to the venue, the Hilton Melbourne Beach Oceanfront.

Please send your request and flight information to Uyensa Dinh (Beese) at Ldrtc2018@gmail.com.