Explore the Agenda
8:00 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Overcoming the Complexities of Clinical Trial Design: Derisking Development for Rare Muscular Disorders to Improve the Likelihood of Success
9:00 am Pursuing Accelerated Approval in Rare Diseases: Leveraging Novel Biomarkers & Endpoints to Support Regulatory Decision Making
- Evaluating the use of validated biomarkers and surrogate endpoints as earlymindicators of treatment benefit to generate rapid evidence for regulatory bodies
- Exploring regulatory expectations for novel endpoint qualification in rare muscular disorders
- Learning from successful accelerated approval case studies to identify best practices and increase the likelihood of regulatory success
9:30 am Derisking Drug Development with a Disease-Focused Approach to Gain Investor Confidence for the Evolving Muscular Disorder Landscape
- How Solve FSHD are positioned as strategic investors to provide early funding, scientific guidance, and credibility that advance pipelines for disease-focused progress
- Building and validating key drug development tools, including prognostic biomarkers, molecular biomarkers, and clinically meaningful outcome measures, to reduce uncertainty and strengthen development pathways
- Demonstrating how foundation-led partnerships can bridge critical funding and knowledge gaps, creating a stronger path from discovery through clinical development for muscular disorder therapies
10:00 am Roundtable Discussion: Statistically Significant vs Clinically Meaningful: Discussing the Shortfalls & Future Direction of Clinical Endpoints for Muscular Disorders
- Examining the disconnect between statistical significance and meaningful patient benefit in muscular disorder trials to deliver more tangible improvements for patients
- Assessing emerging functional, digital and patient-centered endpoints alongside conventional measures to focus on treatment effects which are relevant to disease burden
- Defining a clinically meaningful change for patients, clinicians and regulators to collaboratively guide endpoint selection and increase confidence in development outcomes
10:30 am Morning Break & Speed Networking
Join our speed networking session tailored for muscular dystrophy professionals, like yourselves, to connect with fellow industry peers to facilitate rapid yet meaningful exchanges of insights and expertise. Elevate your networking experience during this session designed for impactful connections within the space.
Novel Biomarkers & Endpoints to Strengthen Trial Design & Reduce Late-Stage Failure Risk
11:30 am Using Modeling & Simulation in Duchenne to Build More Efficient, Informative Clinical Trials & Reduce Development Uncertainty
- Characterizing disease progression in Duchenne using longitudinal natural history and clinical trial data to better understand variability, trajectories, and relationships across clinically meaningful outcomes
- Using disease progression models and clinical trial simulation to inform decisions around endpoint selection, eligibility criteria, study duration, sample size, and other key trial design assumptions
- Applying model-informed drug development approaches to reduce development uncertainty, support more evidence-based decision-making, and strengthen regulatory interactions
12:00 pm Delivering RNA Therapeutics for FSHD: Innovations in DUX4 Silencing & Biomarkers for Clinical Development
- Exploring the use of RNA therapeutics for the treatment of FSHD to deliver efficacious patient outcomes
- Uncovering novel DUX4 silencing technologies and biomarkers to tackle challenges with patient heterogeneity and asymmetric disease progression
- Strengthening end point selection to reduce trial uncertainty and increase confidence in development decisions
12:30 pm Lunch Break & Networking
Advancing Targeted Muscle Delivery to Enhance Efficacy & Minimize Toxicity
1:30 pm Improving Targeting with Antibody & AAV-Mediated Delivery of Nucleic Acid Drugs for Muscular Disorders to Reduce Off Target Effects & Enhance Efficacy
- Utilizing AAV delivery systems to improve targeting of therapeutic payload to relevant tissues with efficacy
- Evaluating antibody-mediated delivery to overcome biodistribution challenges and reduce toxicity
- Enhancing targeting to improve therapeutic index of nucleic acid drugs and support safer and more efficacious clinical outcomes
2:00 pm Engineering Novel Targeted Muscle Delivery Technologies to Enhance Oligonucleotide Delivery & Biodistribution
- Moving towards delivery platforms that enhance payload delivery to increase efficient drug delivery
- Addressing poor specificity and the consequences of long-term drug accumulation
- Exploring how we can achieve more accurate delivery and trafficking characteristics to the muscle for DM1 therapies
2:30 pm Afternoon Break & Poster Session
Take this opportunity to showcase your latest research and innovations with your peers and understand the strategies of your fellow muscular dystrophy experts.
Reviewing the Emerging Cross-Modality Landscape for Muscular Disorder Drug Development
3:15 pm Exploring the Lessons Learnt from Gene Therapies to Optimize the Efficacy & Delivery of Next-Generation Therapeutics
- Examining clinical and regulatory findings from early gene therapy programs to understand where the field is moving to advance safety, patient selection and efficacy
- Assessing how historical limitations are being addressed to improve therapeutic performance and enable the development of safer and more effective treatments
- Translating real-world and clinical trial insights into future modality development strategies to guide better
3:45 pm Panel Discussion: Evaluating the Strengths & Limitations of Genetic, Stem Cell, Small Molecule & RNA-Based Muscle Disease Therapies
- Examining the use of different modalities to address the underlying drivers of muscle degeneration and guide development decisions
- Debating the practical trade-offs between administration, manufacturing and patient accessibility to understand the implications of each modality
- Sharing cross-modality learnings from clinical and translational programsto identify common barriers to pipeline advancement