Conference Day One
Wednesday, December 4
7.30 Check-In & Coffee
8.20 Chair’s Opening Remarks
Plenary Session: Clinical Highlights from 2024
8.30 Making the Leap to Phase 3 Studies: Overcoming Clinical Development & Operational Challenges
Synopsis
• Unpacking the treatment site selection and education process
• Finding and working effectively with the right CRO partners
• Key considerations for Phase 3 study trial design and operations
9.00 Session Reserved for Aldevron
9.30 Leveraging EBT-101 Preclinical & Early Clinical Development for New In Vivo Multiplex Editing Programs
Synopsis
• EBT-101 preclinical on- and off-target assessments
• Clinical study highlights of EBT-101, the first CSRISR-based therapy by systemic AAV delivery
• Platforms repurposed and unique challenges for new programs
10.00 Panel Discussion: Ensuring Diversity, Equity & Inclusivity in Gene Editing Clinical Trials
Synopsis
• Outlining current barriers to disadvantaged patients participating in clinical trials
• Exploring the need to quantify off-target effects in the context of genetic heterogeneity
• Advice for trial sponsors to implement FDA guidance on trial diversity
• Practical steps for drug developers to expand their reach beyond centers of excellence and into communities
10.30 Morning Refreshments & Speed Networking
Innovation & Early Development Track
Translating to the Clinic Track
Spearheading Large DNA Fragment Insertion
Advancing Assay Development and Safety
Monitoring Methods
11.30 Programmable Genomic Integration
• Advanced cell engineering applications
• Efficient replacement of endogenous genes in NHP
• Novel methods of PGI
John Finn, Chief Scientific Officer, Tome Biosciences
11.30 Leveraging Duplex Sequencing for Highly Sensitive
Off-Target Assessment
• Used Duplex Sequencing to overcome the sensitivity limit of
off-target detection with NGS
• This Hybridization-Capture based approach allows for the
identification of rare Chromosomal translocations
• Discuss the characteristics and performance of the PsCas9
variant in comparison to SpCas9
Barrett Nuttall, Director, AstraZeneca
12.15 Highly Efficient Integrase Technology for Site-specific
Genomic Integration of Large DNA Payload
• Generation of highly efficient integrases by directed evolution
• Site-specific genomic integration of von Willebrand Factor
(15.7 kb therapeutic DNA cargo)
• Bringing next-gen genome engineering technology to
enhance gene editing applications, advance cell & gene
therapies and regenerative medicine; and accelerate
cell line development timelines for biologics production,
enabling pandemic preparedness and rapid response to new pathogens
Omid Harandi, Chief Scientific Officer, Komo Biosciences
12.00 Talk Details to be Announced Shortly
Speaker TBC: CRISPR QC
12.30 Clinical Safety Monitoring Strategies for Gene Editing
Therapies
• Preclinical work to identify and characterize the risk of gene
editing at on- and off-target loci
• FDA guidance on safety monitoring for human gene therapy
products incorporating human genome editing
• Detection of on- and off-target genome editing events in the
clinical setting
Priya Chockalingam, Vice President, Head of Clinical
Bioanalytics & Translational Sciences, Beam Therapeutics
1.00 Lunch: Hosted by Edilytics
Innovation & Early Development Track
Translating to the Clinic Track
Optimizing Guide Molecule Design
A 101 in Preclinical Development
2.00 Prime Editing pegRNA: Optimizing Guides
• Understanding the demand for pegRNA optimization to
improve editing efficiencies
• Presenting optimization methods conducted and resulting
increases in in vivo editing potency and accuracy data
• Analytical techniques for quantitative assessments of highly
modified guides
Andrew Anzalone, Director, Head of Prime Editing Platform,
Prime Medicine
2.00 AFNT-212: A Non-Viral Gene-Edited TCR-T Cell Therapy
• Targeted integration of large transgene for developing T cell
therapies targeting KRAS G12D-expressing solid cancers
• Preclinical development of gene edited T cell therapies
• Robust activity of AFNT-212 therapy in preclinical models
Ankit Gupta, Senior Director, Head of Gene Editing,
Affini-T Therapeutics
2.30 Talk Details to be Announced Shortly
Bryant Chica, Scientist II, Analytical Chemistry & Structural
Biology, Editas Medicine
2.30 Roundtable Discussion: Outlining a Path from
Preclinical Models to the Clinic
Roundtables include a larger focus on group discussion. A
moderator will introduce the session topic to attendees with a
presentation, before they split into groups to work through a
series of topic areas. At the end, all groups report back on their
discussions, and findings are collated.
This roundtable will cover:
• The key challenges working with, and translating between,
cell-based models, including induced pluripotent stem cells
(iPSCs), rodents and large animal models
• Strategy when encountering genetic heterogeneity in animal
models
• The translatability of phenotypic correction in animal models
to the clinical setting
Vivian Choi, Head of Liver, Metabolic, Lung, Eye & Ear
Disease Unit, Prime Medicine
3.00 Afternoon Refreshments
Plenary Session: Intellectual Property Protection, Patent Disputes & Technology Licensing
3.45 Establishing ‘Freedom to Operate’ as a Start-Up Gene Editing Biotech
Synopsis
• Understanding the thorny patent landscape and how this complicates freedom to operate
• Sharing practical advice for de-risking against potential patent infringement
• Considerations for building internal legal expertise and/or outsourcing to partners
4.15 Panel Discussion: Licensing Deals for Gene Editing Therapeutic & Delivery Technologies
Synopsis
• Recapping the Broad vs CVC CRISPR patent dispute, and implications for startups in the field
• Understanding the challenges associated with licensing gene editing IP for development and commercialization
• Considerations for exclusive versus non-exclusive deals
• Outlining reasonable licensing deal structures to monetize your technology