Conference Day Two
8:30 am Morning Coffee & Registration
9:20 am Chair’s Opening Remarks
Diversifying Your Nonclinical Gene Therapy Data Packages for Improved Translatability to the Clinic
9:30 am Describing AAV-Driven Safety Findings (Depatotoxicity & Peripheral Neuropathy) in the Non-Human Primate & Potential Translatability
- Eloise Hudry Associate Director - Cell, Gene Therapy & Preclinical Safety, NIBR
- Outlining the future of preclinical development: Is in vivo modelling on the way out? Presentation of retrospectively analyzed data from preclinical non-human primate (NHP) studies to establish the time course of self-complementary AAV9(scAAV9)-related toxicology findings after systemic or intrathecal administration.
- Investigation of the potential triggers (capsid versus transgene) for such toxicities in the cynomolgus macaque, the pathological mechanisms at play and of the impact of immunomodulatory agents.
- Comparison/contrast between those nonclinical results with clinical findings.
10:00 am Developing a Tissue-Restricted AAV for the Treatment of FA: Safety, Efficacy and IND enabling studies
- Edgar Rodriguez-Lebron Chief Executive Officer & Co-Founder, Lacerta Therapeutics Inc.
- AAV vector encoding frataxin is safe and efficacious across a broad dosing range
- CNS and Cardiac tissues are effectively targeted from rodents to primates
- Clinical strategy to target both CNS and cardiac manifestations of FA
10:30 am Morning Refreshments & Networking
Ensuring Optimal Vector Distribution to the CNS & Undertaking Distribution Studies
11:30 am Translating Vector Biodistribution Studies: Scaling up From a Smaller to Larger Brain
- Greg Stewart Consultant, Alcyone Life Sciences
- Understanding the effects of scaling up from NHP to human brains
- Determining how underlying physiology effects biodistribution in both NHP and human brain
- Solving the complexities of scaling up biodistribution studies
12:00 pm Comparing Biodistribution of AAV9 Vs. PHP.eB in Macaque Models Following Intracerebroventricular (ICV) Route of Administration
- Michal Fortuna Scientist II, NHP Biodistribution Lead, Allen Institute
- Presenting results of head-to-head comparison of two leading viral vectors delivered ICV in Macaca nemestrina
- Revealing brain-wide histological analysis and outlining opportunities and limitations of ICV delivery for CNS gene therapy
- Understanding biodistribution and route of administration studies in Non-human Primate models
Progressing Clinical Gene Therapy Programs & Monitoring Vector Uptake in the Clinic
11:30 am Case Study: Highlighting Progress in a Gene Therapy Clinical Program for ALS
- Sanjay Chandriani Director, Research and Development, Maze Therapeutics
- Outlining the study design for an ALS gene therapy program
- Delving into the successes and learnings from the trial so far
- Future expectations and hopes for progression
12:00 pm Highlighting Recent Clinical Data & Utilization of Novel Biomarkers in Parkinson’s Disease Gene Therapy Trials
- Jennifer Johnston Cofounder & Chief Executive Officer, NysnoBio
- Our ongoing clinical imaging study in preparation for our clinical trial
- Use of novel biomarkers in neurodegenerative (PD) gene therapy trials
12:30 pm Lunch
Addressing Safety & Immunogenicity Concerns Associated with Neurological Gene Therapies
1:30 pm Utilizing Non-Viral Vectors to Allow for Redosing of CNS Gene Therapies
- Torben Moos Professor, Aalborg University
- Improving CNS biodistribution studies in rodent and NHP models
- Utilizing advancements in tools to progress biodistribution
- Advancing analytical methodologies used in biodistribution
2:00 pm Roundtable Discussion: Crossing the Blood Brain Barrier with Novel Viral Vector Technology
- Sarah Jacobo Global Gene Therapy & Vectorology Lead, Takeda Pharmaceutical Co. Ltd.
- Revealing preclinical data on preclinical modelling for an AAV vector
- Understanding safety and efficacy considerations
- Expanding on viral vector delivery to the CNS and considerations for translatability
Confronting Challenges with Gene Therapy Clinical Study Design for CNS Gene Therapies
1:30 pm Identifying Starting Points for Disease Intervention & Gene Therapy Treatment in Neurological Disorders
- Evaluating impacts on vector uptake in the central nervous system
- Understanding metabolic factors effecting vector uptake
- Understanding patient life history and lifestyle in vector uptake
2:00 pm Early Screening & Diagnosis of Neurological Disorders as a Means of Accelerating Gene Therapy Clinical Trials with Earlier Identification of Disease Benefits in Gene Therapy Clinical Trials: A Framework to Assess Disease-Modifying Therapies
- Dorota Gruber Assistant Chief, Pediatric Cardiogenomics, Cohen Children's Medical Center
- Reviewing the status of the NBS for neurological disorders.
- Providing a framework for including neurological disorders in the NBS program. Examining lessons learned from NY NBS Duchenne pilot.
- Reviewing ethical, legal and social implications (ELSI) associated with early screening and diagnosis of late-onset neurological conditions.
2:30 pm Afternoon Refreshments & Networking
Strategizing to Gain Regulatory & Commercial Success for Neurological Gene Therapies in the USA
3:00 pm CMC Considerations: Global Strategies for the AAV Vector for Glycogen Storage Disease Type Ia Treatment
- Jan Panteli Director, Upstream Process Development, Ultragenyx Pharmaceutical Inc
- Acknowledging need for a robust, productive manufacturing process and CMC
- Outlining Downstream Process Improvements
- Unique challenges with Glycogen Storage Disease Type Ia Treatment
3:30 pm Creating a Commercially Successful Product to Treat Rare Disorders
- Julia Taravella Executive Director, Rare Trait Hope Fund
- Exploring strategies and collaborations to ensure success of a therapy for rare and ultra rare disorders.
- Discussing rare disease stakeholders: objectives, goals, accountability, transparency and communications.
- Developing a roadmap for a successful product for ultra-rare disorders.
- Cost breakdown for rare disorder’s gene therapies development – a case study