7:00 am Breakfast & Registration
7:50 am Chair’s Opening Remarks
- Chris Mason CSO , AVROBIO
Highlighting Current Gene Therapy Progress – Where Are We Now?
8:00 am Understanding the Current Global Gene Therapy Landscape: What Can we Expect in 2022?
- Janet Lambert CEO , ARM
- Exploring the current clinical trial landscape and products gearing up for approval
- Where are the key focus areas to continue the trend of rapid growth in gene therapy?
- Establishing the outlook for 2022: are we where we thought we would be this time last year?
8:30 am Virtual: Rare Disease Gene Therapy Development: Where Has It Been and Where Is It Going?
- Peter Marks Director, CBER , FDA
- Comparing progress in rare disorders in vivo with developments seen in CAR-T and ex vivo cell modified gene therapies
- Where are the current bottlenecks for gene therapy approvals?
- Addressing FDA’s outlook for gene therapy in 2022
9:00 am A Patient-Centric Approach to the Development of Advanced Therapies
- Anshul Mangal President, Precision ADVANCE & Project Farma
- Phil Cyr SVP, Precision Value & Health / Precision ADVANCE
- Kyle Bryant Spokesperson, FARA (Friedreich’s Ataxia Research Alliance)
- Ensuring your organization is patient-centric not only delivers more relevant and impactful patient outcomes, but it also can increase speed, efficiency, and productivity in the development advanced medicines. How do we ensure that patients are always top-of-mind during drug development? How has having a patient-focused mindset made a difference in finding cures for rare genetic orders?
- Hear from Kyle Bryant, Director of rideATAXIA at FARA (Friedreich’s Ataxia Research Alliance), on his perspective as a patient living with a degenerative neuro-muscular disorder and the impact the gene therapy community is making for patients and their families
- Learn how a patient-focused mindset can optimized speed-to-market for the development of gene therapies
9:30 am Industry Leaders Panel: Accelerating Gene Therapies for Rare Disease Patients – Where are the Bottlenecks & How Can we Overcome Them?
- Chris Mason CSO , AVROBIO
- Federico Mingozzi CSO , Spark Therapeutics
- Eric Crombez Chief Medical Officer, Ultragenyx
- Sheila Mikhail Chief Executive Officer & Co-Founder, AskBio
Gene therapy continues to make significant progress with no signs of slowing down, but the past year has also flagged distinct development challenges that need to be overcome. This panel discussion will bring together experts from pioneering gene therapy companies leading the way to pinpoint these obstacles and explore how we can address them to bring gene therapies to patients more quickly, safely, and effectively:
- How are manufacturing processes evolving to meet regulatory benchmarks?
- Adapting to the novelty of gene therapies with respect to regulatory, pricing, market access, and patient needs
- Accelerating gene therapy programs while maintaining safety and quality standards
- Leading by example: how the leading companies are paving the way for the future of gene therapy
10:00 am Structured Networking & Morning Break
Our structured 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.
1:00 pm Lunch Briefing Hosted by Cytiva
- Yasser Kehail Single Use & Enterprise Business Development Leader, Cytiva
Leveraging Lessons from the COVID-19 Pandemic to Accelerate the Development and Production of Adeno-Associated Virus (AAV)-Based Gene Therapies
While there are currently over 1400 clinical cell and gene therapy programs, trial recruitment, raw material supply chains, and low-yield processes are just some of the multiple challenges facing the industry. The recent pandemic revealed that with the proper framework, a drug product can be developed, clinically tested, and produced for hundreds of millions of patients in less than a year.
- What lessons can we apply from this effort for AAV development and production?
- How can we rethink regulatory filing strategy?
- And why does the cell and gene therapy arena so urgently need these learnings?
3:30 pm Afternoon Refreshments & Networking
Developing the Next Generation of Gene Therapies
4:15 pm AAV Manufacturing Process Development Using PATfix HPLC Analytics
- Ales Strancar Managing Director, BIA Separations, Part of Sartorius Group
- When the expression of the AAV results in less than 10% full capsid it is quite impossible to reach better than 90% full capsid in the final product. It is therefore mandatory to optimise the expression to result in better empty/full ratio
- This can be realised by using at-line HPLC to allow for analysis of the full and empty capsids ratio in the bioreactor filtrate from the transfection to the harvest
- The residual empty capsids can be removed by polishing purification step using different anion exchange and multimode columns
4:45 pm Machine-Guided Design of Optimized AAV Vectors
- Eric Kelsic CEO , Dyno Therapeutics
- Designing new AAV capsids can be greatly accelerated using AI methods
- Optimizing capsids for specific disease indications and administration routes
- Working towards higher specificity, lower overall dose, and improved safety
5:15 pm Azzur Cleanrooms on Demand™ – A Paradigm Shift in Early Phase Biopharmaceutical Manufacturing, Resulting in Acceleration of Critical Therapies
- Ravi Samavedam Chief Innovation Officer (CINO), Azzur Group
- Current industry challenges around the transition from R&D through early-phase clinical manufacturing
- Discussion of the history of Azzur Cleanrooms on Demand™ and introduction of value proposition of the hybrid model vs. traditional “build or broker’’ models for early- to mid-phase innovators
- Case studies from 2018 to present of acceleration of clinical timelines resulting from Azzur Cleanrooms on Demand™ service offerings
5:45 pm Investigating the Promise of Anellovectors to Address Tropism, Immunogenicity & Redosing in Gene Therapy
- Simon Delagrave SVP, Ring Therapeutics
- Sequencing of human anelloviruses reveals a vast genetic diversity with potential implications for gene therapy, such as:
- Generating more targeted gene delivery vectors
- Enabling re-dosing and improving the therapeutic index of gene therapies
- An update will be provided on Ring Therapeutics’ efforts in developing Anellovectors
6:15 pm Chair’s Closing Remarks
- Chris Mason CSO , AVROBIO
6:30 pm Poster Session & Drinks Reception Hosted by Precision ADVANCE