Explore the Agenda
8:00 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Evaluating Preclinical Models to Better Predict Clinical Outcomes in Muscular Disorders
9:00 am Discussing the Appropriateness of 2D, 3D, & in vivo Models to Build a Robust Preclinical Data Package in Rare Disease Programs
- Exploring the use of animal models within a program’s means to ensure preclinical work that is predictive into the clinic
- Comparing the advantages and disadvantages of 2D and 3D models for long term rare disease studies in terms of dosing and translatability
- Establishing the importance of a robust preclinical data package and how to achieve this in early strategic development
10:00 am Mitigating Clinical Risk with Strategic Preclinical Design for DMD Exon Skipping Therapeutics
- Applying AOC platform for exon skipping in DMD as therapeutic approach
- Creating successful and translatable preclinical package for Duchenne’s
10:30 am Muscular Innovation Exchange
Join the discussion most relevant to your work and gain insights from experts facing shared translational and clinical challenges. Move between tables split by therapeutic area throughout the session to connect with peers, and gain insight through meaningful industry connections.
11:00 am Morning Break
Comparing Therapeutic Modalities to Develop Meaningful Patient Outcomes & Better Address Muscle Disease Pathology
11:30 am Targeting Muscle Function in DM1 With Small Molecules to Expand Treatment Options Beyond Gene Therapy
- Highlighting advances in RNA targeting small molecules to demonstrate the benefit of functional improvements achieved through non-genetic mechanisms
- Exploring emerging small molecules designed to enhance therapeutic benefit and broaden therapeutic development options
12:00 pm Comparing Antibodies & Small Molecules to Best Address Immunogenicity, Biodistribution & Efficacy in Drug Development
- Comparing the molecular and pharmacological properties of antibodies and small molecules to understand how size, affinity, valency and molecular architecture influence target engagement and therapeutic performance
- Exploring how modality choice impacts biodistribution, tissue penetration, dosing and productive intracellular delivery, while considering modality-specific challenges including immunogenicity and off-target exposure
- Using preclinical and clinical examples to define where antibodies and small molecules offer distinct advantages, providing a practical framework for selecting the right targeting modality for the target, tissue and therapeutic payload
12:30 pm Lunch Break & Networking
Tailoring Drug Development to Prioritize Symptomatic Burden & Deliver Meaningful Benefits for Patients
1:30 pm From Dystrophin to Function: A Patient-Centered Approach to Drug Development in Becker Muscular Dystrophy
- Moving beyond protein restoration towards meaningful functional improvement for patients
- Understanding why muscle function is what matters most to patients
- Exploring the clinical development of Sevasemten to reveal the lessons learned for future development of other muscular dystrophies
- Establishing the role of small molecules within an increasingly diversified treatment landscape
2:00 pm Roundtable Discussion: Considering Non-Muscular Products to Uncover Novel Targets & Address Previously Unmet Patient Needs
- Discussing lesser addressed symptoms for muscular disorders to improve patient quality of life beyond enhancing muscle function
- Examining how biomarkers used in cardiovascular, inflammatory, GI and neurodegenerative diseases can be adapted for muscular disorders to broaden biomarker discovery opportunities and tailor patient characterization
- Investigating successful target identification strategies from other therapeutic areas to uncover shared pathways and reveal new intervention points for muscular disorders
2:30 pm Afternoon Break
Decoding Patient Heterogeneity in Muscular Dystrophy: Biomarkers, Disease Progression, & Clinical Outcomes
3:15 pm Refining Patient Pool Selection to Overcome Patient Heterogeneity Challenges & Improve Clinical Success
- Leveraging genetic, molecular and disease-specific biomarkers to distinguish patient subgroups within Duchenne, Becker, FSHD, DM1 and LGMD and strengthen the interpretation of clinical outcomes
- Characterizing differences in ambulatory status, muscle degeneration patterns and rates of disease progression across patients
3:45 pm Anticipating Heterogeneity in Rate of Dystrophy to Prepare for Endpoint Variation & Optimize Trial Design
- Characterizing differences in disease progression trajectories across patient populations to improve endpoint selection
- Designing adaptive and risk-adjusted clinical trial strategies that account for heterogeneous decline patterns and maximize the likelihood of detecting treatment effects