Conference Day One: Wednesday, March 27
7:00 am Registration & Coffee
7:50 am Chair’s Opening Remarks
ACCELERATING THE DEVELOPMENT OF RARE GENE THERAPIES
8:00 am Gene Therapy’s Promise: Current Outlook and Future Directions for the Field
Synopsis
- Assessing the short- and long- term impact of recent headwinds and tailwinds facing the gene therapy field
- In the context of a growing mass of programs entering the clinic, how do we ensure these therapies reach rare disease communities?
- Addressing what the next steps look like for fostering a collaborative, sustainable, and commercially viable gene therapy landscape
8:30 am What to Look Forward to for Gene Therapy in 2024
Synopsis
- Market trends and expectations for Gene Therapies & Rare Diseases in 2024
- Predictions on Potential Approvals in 2024
- Innovative Trends & Technologies
- Evolution of CMC and manufacturing
9:00 am Establishing a Robust Framework to Accommodate for the Increased Number of Clinical Stage Products
Synopsis
- Benchmarking progress made with Operation Warp Speed for Rare Diseases
- Accelerated approval: clarifying its use for gene therapies and advice for programs submitting for it
- Outlining the key ‘call to actions’ needed for the rare gene therapy field to advance
9:30 am Panel Discussion: So You Have an Accelerated Approval: What Now?
Synopsis
- Outlining challenges in the post-market studies to provide confirmatory data of a clinical benefit
- Implications for drug developers if confirmatory trials are negative
- Exploring the challenge of these therapies being deemed “unproven” or “experimental” by private insurers
- Understanding how to operationalize and source data being requested by the FDA
10:00 am Morning Refreshments & Speed Networking
Synopsis
Our speed networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond.
MANUFACTURING
Addressing Key Upstream & Downstream Vector Production Challenges
11:00 am Levelling Up Upstream Manufacturing to Improve Batch Productivity & Consistency
Synopsis
- Strategies for reducing the risk of contamination in cell culture
- Why is there a need to shift from adherent- to suspension-based cultures?
- Learnings from scaling up a suspension-based cell line production process
11:30 am Evolution of Downstream Manufacturing Process Design for Productivity, Product Quality and Process Consistency
Synopsis
- Moving towards scalable and commercial manufacturing – ready purification processes
- Process design for increased productivity, high product quality, and reduced manufacturing cost
- Speed to clinic considerations
- Process characterization and control strategy definition to enable robust critical quality attributes (CQAs) control for commercialization
12:00 pm Case Studies of Resolving AAV Aggregation & Removing Empty Vectors
Synopsis
- Case study one: aggregation occurring prior to the polishing chromatography step during final UFDF formulation and how this was resolved
- Case study two: novel purification to remove empty capsids – impurities potentially inducing an adverse immune response
- Outlining Fronter’s novel purification platform, removing empty capsids to <1% in the final drug substance
CLINICAL
Clinical Trial Design Nuances in Rare Diseases
11:00 am Rare Disease Trials: To Placebo or Not to Placebo?
Synopsis
- Key considerations when considering incorporating a placebo control
- Outlining the implications to the sponsor, regulators, and patients
- When is a placebo considered unethical or unfeasible?
- How are global regulators’ attitudes on placebo control arms evolving?
11:30 am Identifying the Challenges Associated with Conducting Robust External Control Arm Studies in Small, Heterogenous Patient Cohorts
Synopsis
- Understanding the main difficulties in using external databases as high quality external controls, including challenges in obtaining the right informed consent and ensuring frequent data collection in the database
- Exploring the complexities of data scales and sources
- Presenting strategies for overcoming these challenges and building a reliable control arm
12:00 pm Panel Discussion: Unpicking the Complexities of External Control Arms
Synopsis
- Identifying the challenges associated with conducting robust external control arm studies in small, heterogenous patient cohort
- Methodological considerations for using external control arms to credibly demonstrate the effectiveness of a rare gene therapy
- Key lessons learned from clinical trial designs incorporating external controls
- Considerations for using natural history studies as external control arms
REGULATORY
Exploring an Intricate Global Regulatory Landscape
11:00 am Creating a Forward-Looking Framework for Gene Therapies in Canada (Virtual)
Synopsis
- An overview of current Health Canada gene therapy regulatory frameworks
- Addressing commonly observed reasons for regulatory filing rejections and program setbacks
- Ongoing reforms and innovations to accelerate gene therapy development and regulation in Canada
11:30 am Streamline Your Gene Therapy Product Development with the PMDA
Synopsis
- PMDA strategy to facilitate and streamline development of gene therapy products, focusing on consultation services
- Sharing review experience and future perspectives in the 10th anniversary of the new law on regenerative medical products (including gene therapy products) in Japan
12:00 pm Panel Discussion: Harmonizing a Complex Regulatory Environment
Synopsis
- Identifying areas of whitespace for global regulatory convergence
- Reviewing areas of similarities and differences in approaches to streamlining clinical development, including in the use of global expedited programs
- Outlining the collaborative steps needed to encourage regulatory convergence
12:30 pm Lunch & Networking
Navigating Manufacturing Changes During Development
1:30 pm Clarifying the Regulatory Guidance on Manufacturing Process Changes
Synopsis
- Outlining FDA processes for considering manufacturing changes for both investigational and licensed gene therapy products
- Highlighting common downfall areas where drug sponsors fail to demonstrate comparability
- Best practices for drug sponsors to communicate any process changes during drug development and in-market
2:00 pm Efficient Viral Vector Manufacturing with Microcarriers
Synopsis
- Understanding the viral vector manufacturing challenges often faced by drug developers
- Advantages of using microcarriers
- Upstream and downstream considerations for microcarriers cell culture system
Forward-Looking Landscaping & Impact on Clinical Trial Effectiveness
1:30 pm Outlining Progress on NCATS’ AAV Gene Therapy Programs
Synopsis
- Updates from the BGTC portfolio’s eight rare disease clinical trials, and version 1.0 of the BGTC standard operational playbook for clinical development of AAV gene therapies
- Highlighting the Platform Vector Gene Therapy (PaVe-GT) pilot project: it’s scope, goals, and latest updates
- Updates from Phase 2 of The NIH Somatic Cell Genome Editing consortium , including the TARGETTED genome editor delivery challenge
2:00 pm Panel Discussion: Reaching, Enrolling, and Retaining Participants in Rare Disease Gene Therapy Trials
Synopsis
- Multi-stakeholder discussion of friction points in the development lifecycle and how to overcome them: what have we tried, what has been successful and under what circumstances
- Dissection of real-world examples over time and across different patient communities
- Analysis of recent trends and impacts of continuing advances
Defining Regulatory Strategy in an Era of Expedited Programs
1:30 pm Expectations and Considerations for Using the Accelerated Approval Pathway
Synopsis
- Providing an overview of the program and current regulatory attitudes towards its role in the gene therapy field
- Learnings in terms of regulatory strategy from success with accelerated approval
- How to make a compelling case that your gene therapy will be reasonably likely to have a clinical benefit
2:00 pm Panel Discussion: A 101 in Expedited Programs for Gene Therapy
Synopsis
- Outlining the practicalities of gaining FDA expedited designations
- Criteria for designation and best practices with regulatory touchpoints
- Considerations for successful interactions with CBER reviewers
- Common reasons for denial of RMAT applications
2:30 pm Afternoon Refreshments & Scientific Poster Session
ADDRESSING THE COMPLEXITY OF GENE THERAPY INVESTMENT & FUNDING MODELS
3:15 pm Making the Case for Gene Therapy in a Tough Market
Synopsis
- Alternative funding strategies for a tough financing market
- Unusual business models for ultra-rare indications and emerging economies
- Making the commercial proposition for gene therapies more valuable
3:45 pm Revolutionizing Manufacturing Flexibility for Rare Disorders: A Model of Speed and Adaptability
Synopsis
- Critical to accelerating gene therapies for patients with rare diseases is innovative development and manufacturing strategies, and speed-to-market at sustainable cost, and addressing the current complexity and cost of manufacturing processes and infrastructure
- Proposing a new model where the needs of manufacturing infrastructure, processes, and application of expertise are tailored to the specific needs of the pipeline lifecycle – fit-for-purpose, fit-for-phase, and cost-effective infrastructure support and scientific expertise
- We enable the acceleration of advanced therapy development and manufacturing across geographies with optimization of resource utilization and flexibility
4:15 pm Decentralized Development of Gene Therapies for Ultra-Rare Disorders
Synopsis
- Realizing the full value potential for investing into Gene Therapies for Ultra-Rare Disorders
- Strategies and tools for decentralized funding of low-cost CMC platforms for viral vectored Gene Therapies
- Lowering the patient prevalence threshold for financial sustainability through greater industry collaboration
4:45 pm Panel Discussion: What are Investors Looking for in Gene Therapy Companies?
Synopsis
- Discussing the decision-making process for firms and big pharma to invest in gene therapy companies
- Exploring what investors see as the key limitations for investing
- Discovering what drives a high value proposition to investors
- Outlining opportunity areas for investment in the space