Conference Day Two
Wednesday June 18, 2025
8:25 am Chair’s Opening Remarks
Spearheading Target Validation to Demonstrate Safety & Efficacy for a Novel Target Class
8:30 am An End-to-End Discovery & Validation Platform for Dark Genome Targets
Synopsis
- Enhancing discovery of dark genome targets by overcoming characterization challenges for low expression targets
- Developing optimized assays and tools to detect and quantify dark genome-derived transcripts and peptides
- Identifying optimal modality with deep iterative screening
9:00 am Advancing LINE-1 Reverse Transcriptase Inhibitors in Autoimmune & Neurodegenerative Diseases
Synopsis
- Repeat elements like LINE-1 make up most of our DNA, and their expression is silenced in healthy cells
- When aberrantly expressed in disease, LINE-1 reverse transcriptase (RT) drives interferon signaling and “sterile inflammation”
- LINE-1 RT inhibitors inhibit tissue interferon signaling and inflammation. They target large patient populations and have the potential to be non-immune suppressive therapies
9:30 am Dark Antigens® – Novel Tumor-Specific Targets for Cancer Immunotherapy
Synopsis
- Mining the dark genome to uncover regions encoding putative Dark Antigens®
- Integrating large-scale, multiomic data to identify and validate cancer-specific Dark Antigens®
- Characterizing Dark Antigens® with broad therapeutic potential
10:00 am Morning Refreshment Break & Networking
The Untrodden Path: Paving a Smooth Road to the Clinic to Ensure Translatable Success
11:00 am Translating Discovery of Novel LncRNA Targets into Clinical Programs
Synopsis
- Discovering high-value LncRNA targets to reprogram diseased cell states
- Understanding underlying LncRNA and target biology to curate preclinical and clinical strategy
- Building smooth drug development strategy and uncovering robust biomarkers for smooth recruitment and progression
11:30 am Bringing Dark Genome-Targeted Personalized Cancer Vaccines to the Clinic
Synopsis
- A new class of cancer vaccines based on an off-the-shelf antigen-presenting cell line (PDC*line)
- Harnessing the PDC*line Pharma platform to develop effective personalized cancer vaccines (PDC*neo)
- Unlocking the dark genome: discovering novel neoantigen targets for cancer immunotherapy
12:00 pm Round Table: Equipping Smooth Translation & Maneuvering a Novel Regulatory Path to Drive Efficient Progression
Synopsis
- Discussing the shared challenges of preclinical models translatability and approaching regulatory authorities about novel non-coding therapeutics
- Evaluating applicable models and computational systems to demonstrate translatability
- Reviewing preclinical requirements and regulatory expectations to understand required data and build robust IND packages
- Brainstorming potential regulatory pushback to determine early preparations to address challenges in advance
1:00 pm Lunch Break & Networking
Advancing Tools & Technologies to Mine Deeper into the Dark Genome
2:00 pm Unravelling Disease Complexity at the Isoform Frontier
Synopsis
- Bringing single-cell and spatial resolution to 3rd generation -omics approaches
- Challenges and opportunities in bioinformatics and machine learning with these new data types
- Applications across cancer and chronic disease
2:30 pm A Robust Computational Approach to Unraveling the Role of Highly Repetitive Elements in Human Health & Disease
Synopsis
- Highly repetitive elements comprise > 50% of the human genome, while canonical protein-coding genes comprise only ~2%
- Naive approaches to the quantification of the expression of these elements fail to address their unique challenges, resulting in estimates that obscure their association with disease
- ROME’s custom ML algorithms and curated databases enable highly accurate locus level quantification of expression and activity of these repetitive elements, supporting their relevance in disease
3:00 pm Harnessing Immunopeptidomics Techniques to Uncover Hidden Peptides
Synopsis
- Conducting immunopeptidomics experiments to understand the interplay between genome, transcriptome and proteome
- Enhancing computational tools to analyze large datasets generated by immunopeptidomics