Conference Day One
7:45 am Morning Coffee and Registration
8:50 am Chair’s Opening Remarks
Highlighting Success Stories to Re-energize your Neurological Gene Therapy Development
9:00 am Utilizing Novel Delivery & Route of Administration to Bypass the Blood Brain Barrier: A Case Study on an Approved Direct Brain Delivery Gene Therapy
Synopsis
- Weighing up patient burden and therapeutic effect of bypassing the blood brain barrier with direct brain delivery for gene therapies
- Challenges of conducting complex surgical delivery procedures in the clinic
- Outlining opportunities and limitations of direct brain delivery for gene therapy
9:30 am Announcing a New, Minimally Invasive Endovascular Approach to the Cerebellopontine Cistern Enables Broad CNS Biodistribution of AAV9
10:00 am Morning Refreshments & Speed Networking
Synopsis
This session is an opportunity to connect with peers working in development of gene therapies for neurological disorders and hold in-depth conversations with some of the brightest minds to establish meaningful and lasting relationships.
Preclinical Stream
Advancing Gene Therapy Modelling Systems for Nonclinical Safety & Efficacy Studies in the CNS
11:30 am Designing Preclinical Gene Therapy Models for Neurological Disease Indications
Synopsis
- Underlining challenges in preclinical development when working with neurological disorders
- Selecting the right animal model systems and route of administration for nonclinical testing
- Progressing the neurological disorders field by developing a more standardized approaches
12:00 pm Overviewing The use of Human iPSC Derived Neuronal Models to Enable Preclinical Drug Discovery in Neurodegenerative diseases for Gene Therapy
Synopsis
- Understanding the value of transcriptomic characterization of models
- Weighing pros and cons of 2D versus 3D neuronal cultures
- On neurophysiology endpoints: methods, reproducibility, and throughput
Clinical Stream
10:59 am Investigating More Translational Gene Therapy Biomarkers & Endpoints for CNS Indications for Improved Clinical Monitoring
11:30 am Developing a Novel ATXN2-Targeting AAV Gene Therapy Treatment for Amyotrophic Lateral Sclerosis
Synopsis
- Using human genetics, bioinformatics, and functional screens to identify a potent miRNA payload and an inert stuffer sequence
- Demonstrating preclinical efficacy in disease models of ALS
- Evaluating pharmacology, biodistribution in nonhuman primates in preparation for IND-enabling studies
12:00 pm Identifying More Translatable Gene Therapy Biomarkers for CNS Indications
Synopsis
- Understanding limitations of current standard biomarkers
- Preclinical analysis of biomarkers and identifying translatable selections
- Challenges when progressing to a human brain
12:30 pm Lunch
Identifying Targets for Neurological Gene Therapy Drug Development & Influence on Capsid Selection
1:30 pm Encouraging Results in Young Children With MPSIIIA Following Intraparenchymal AAV Gene Therapy
Synopsis
- Delving into viral vector developments including the use of antibodies for facilitative transportation across the blood brain barrier
- Going beyond AAVs for neurological gene therapies
- Aiding in your viral vector selection with a greater understanding of choices
2:00 pm Selecting Gene Therapy Capsid & Promoter to Overcome Toxicity Due to Transgene Overexpression
Synopsis
- Outlining how this will help you to gain expression in specific cell types to your disease target
- Presenting the case of SMN toxicity due to overexpression in sensory neurons
- Comparing SMN expression in the nervous system of mice using different promoters
- Combining capsid and transcriptional targeting for the selective expression of transgenes in spinal motor neurons
2:30 pm Roundtable Discussion: The Potential of Non-Viral Vectors to Successfully Traverse the Blood Brain Barrier From Gene Therapy Development
Synopsis
- Outlining development of non-viral vectors for CNS targeting
- Describing the mechanisms of action of non-viral vectors to benefit Neurological treatments
- Surveying the horizon of development for non-viral vector development and which vector should you choose
Optimizing Translation of Non-Clinical Data to Set Up for Successful Gene Therapy Clinical Trials
1:30 pm Confronting Challenges with Clinical Study Design for Gene Therapy CNS Treatments & Allowing Early Intervention
Synopsis
- Discussing challenges in neurological gene therapy clinical trial design
- Understanding difficulties in early identification for clinical treatment
- Identifying biomarkers and tools for improved clinical design
2:00 pm Designing more Translatable Preclinical Rodent Models for Rare & Ultra Rare Diseases
Synopsis
- Outlining challenges in nonclinical development of rare and ultra-rare disorders
- Scaling up NHP data to humans
- Identifying your dosage regimen and allowing for flexibility in your clinical trial design
2:30 pm Translation of Gene Therapy Brain Biomarkers from Nonclinical Science to Clinical Trials: A Case Study in Canavan Disease
Synopsis
- MRI and MRS evaluations of candidate biomarkers
- Preclinical assessments in a murine model
- Clinical evidence by magnetic resonance spectroscopy in humans by standardized spectrum analyses
- Potential uses in clinical trials to support regulatory activities
3:00 pm Afternoon Refreshments & Poster Session
Synopsis
The scientific poster session will serve as the perfect opportunity to showcase your recent work to your peers and allow you to learn and share insights in a relaxed atmosphere.
Discovering Different Gene Therapy Delivery Modalities for Improved Safety, Efficacy & Distribution Across the Blood Brain Barrier
4:00 pm Considering Patient Burden & Safety Associated with More Invasive Delivery Techniques to Bypass the Blood Brain Barrier
Synopsis
- Weighing up the patient burden when considering routes of administration for neurological disorders
- Considering your capsid when choosing your delivery technique
- Factoring in physicians: Can complex surgical delivery methods be used widespread?
4:30 pm Session Hosted by ClearPoint Neuro
5:00 pm Panel Discussion: Addressing Safety Concerns Associated with Viral & Nonviral Neurological Gene Therapies
Synopsis
- Considering safety and immunogenicity challenges associated with delivering viral gene therapies to the CNS
- Looking in to dose related toxicity when using a higher dose to ensure high efficacy
- Debating whether nonviral vectors pose more or less of a safety concern when delivering to the CNS than viral vectors
5:45 pm End of Conference Day One
