Conference Day Two
Thursday, December 5
7.45 Check-In & Coffee
8.35 Chair’s Opening Remarks
Plenary Session: Academic Pioneers
8.45 Therapeutic Gene Editing for Cardiovascular and Metabolic Diseases: From the Leading Cause of Death to N-Of-1 Disorders
9.15 Engineering and Evolution of Hyperactive, CRISPR-Associated Transposases (VIRTUAL)
9.45 Morning Refreshments
Innovation & Early Development Track
Translating to the Clinic Track
Progressing Epigenome Editors into the Clinic
Navigating a Complicated Regulatory Environemnt
10.30 Programmable Gene Activation, Silencing &
Multiplexing for Precise Gene Regulation
• Exploring the basics of epigenetic editing to regulate gene
expression
• Presenting the latest efficacy and safety data from recent in
vivo studies
• Outlining a roadmap towards demonstrating therapeutic
Shane Wright, Associate Scientist II, Chroma Medicine
10.30 Regulatory Strategies to Enable the Clinical Trial
Applications of Genome Editing Products
• Pre-clinical assessment of safety, efficacy, and dose supporting FIH
• CMC requirements and analytical testing
• Safety assessment, including off-target toxicity
• Clinical study considerations
Jim Wang, Vice President Regulatory Affairs, Genetic Medicine, Regeneron Pharmaceuticals
11.00 Epigenome editing with CRIPSR-GNDM® as a treatment platform for unmet medical conditions
• Lead program (MDL-101) demonstrating sufficient and durable gene upregulation in mouse disease models and NHPs for the treatment of LAMA2-CMD
• Regulatory-aligned path to a first-in-human trial with optimized biomanufacturing process
• Up/down gene regulation technology combined with tissue-tropic AAV capsids for non-hepatic, neuromuscular, cardiovascular, and CNS disorders
Tetsuya Yamagata, Chief Scientific Officer, Modalis Therapeutics
11.00 Compact Nucleases: Outlining a Roadmap Towards
Clinical Evaluation
• Understanding the features of compact nucleases and their expanded delivery opportunities
• Confirming efficacy and pharmacology with NHP data
• Detailining IND-enabling studies to support clinical development
Gabor Veres, Senior Vice President, Translational Research,
Mammoth Biosciences
11.30 CRISPR Epigenetic Modification to Treat Hepatitis B
• An overview of the mechanism-of-action of TEMPO, and the
intricacies of HBV as a disease target
• Efficacy and safety data from proxy target non-human primate
studies
• Key safety and regulatory considerations ahead of commencing a first-in-human trial
Britta Jones, Senior Scientist, Tune Therapeutics
12.00 Lunch
Innovation & Early Development Track
Translating to the Clinic Track
Validating the Next Generation of Delivery Vehicles
Driving Novel Nucleases Towards the Clinic
1.00 Novel Gene Editing Therapeutics Enabled by ARRDC1-
Mediated Microvesicles
• Engineered ARMMs permit transient delivery of CRISPR-Cas
RNPs to retina and LSECs in NHP
• Engineered ARMMs are very well tolerated in NHP
• Differentiated delivery of genome editors by ARMMs supports
therapeutic strategies for ocular and hematology indications
Joseph Nabhan, Chief Scientific Officer, Vesigen Therapeutics
1.00 Solving the Challenges of Editing and In Vivo Delivery
• The unique attributes of X-Editor nucleases
• Challenges facing progressing these technologies into precise
gene editing therapeutics, and strategies we’re deploying to overcome them
• New data from their application in therapeutic programs
Brett Staahl, Co-Founder & Vice President, External Innovation, Scribe Therapeutics
1.30 In Vivo Application of Exosome-Based Genome Editing
Technology
• A focused look at the delivery mechanism behind exosomebased
delivery
• Understanding the capabilities for precise delivery of gene
editing technologies
• Outlining preclinical results to date
Dave Carter, Head of Research, Evox Therapeutics
1.30 Disruption of Aberrant Splicing by Gene Editing with
a Type V CRISPR-Cas Enzyme as a Potential Treatment for
ALS
• TDP43 pathology is a hallmark of many neurodegenerative
conditions, including ALS. A consequence of this pathology is
aberrant splicing of multiple neuronal genes
• Mis-splicing of STMN2 in the absence of TDP43 leads
to incorporation of a cryptic exon and production of an
unproductive transcript
• Removal of the cryptic splice site by gene editing results in
restoration of the full-length STMN2 transcript in cultured
human motor neurons and transgenic mice
• Editing also restored neurite outgrowth in motor neurons with
significantly reduced TDP43
John Murphy, Chief Scientific Officer, Arbor Biotechnologies