Cambridge Healthtech Institute’s 7th Annual

RNA & DNA Targeting Small Molecule Drugs

Identifying New Pathways, Modalities, and Strategies for Therapeutic Intervention

September 23-24, 2025

There is a lot of interest in finding new ways to modulate RNA and DNA function and to get the desired biological outcome for therapeutic intervention. However, understanding their structure, function, and interactions to find small molecules that offer in vivo specificity, selectivity, and safety has been quite challenging. Cambridge Healthtech Institute's conference on RNA & DNA Targeting Small Molecule Drugs will highlight some of the innovative approaches such as AI/ML, biophysical techniques, and degradation strategies being pursued to modulate these targets. Along with therapeutic use, these small molecule DNA and RNA binders are also being looked at for modification, regulation, and targeted delivery.

Coverage will include, but is not limited to:

Emerging designing and screening strategies (structure-based, fragment-based, and phenotypic screening)
Identifying pathways (transcription, splicing, translation, transcription) for modulation
Determining the structural and physicochemical constraints for RNA/DNA-ligand interactions
Strategies for mechanistic characterization and optimization of small-molecule binders
Targeting protein-RNA/DNA interfaces
Biophysical/phenotypic screening for identifying and quantifying RNA/DNA binding
Utilizing artificial intelligence (AI) and machine learning (ML) for RNA/DNA drug discovery

Topics for Targeting DNA:

Exploration of synthetic lethality, DNA repair, and epigenetic modifications
Modulating DNA Damage Response (DDR) pathways
Next-generation poly-ADP ribose polymerase (PARP) inhibitors
Targeting cell-cycle checkpoints and DNA damage-repair kinases
Targeting DNA G-quadruplexes and telomerases
Affecting CRISPR/Cas9 genome editing using small molecules

Topics for Targeting RNA:

Identifying the right tertiary and quaternary structures for therapeutic intervention
Targeting pre-mRNA, mRNA with splicing modulators
Inhibiting mRNA translation
Strategies for RNA degradation
Targeting RNA G-quadruplexes
Targeting non-coding RNAs (ncRNAs)

The deadline for priority consideration is March 7, 2025.

All proposals are subject to review by session chairpersons and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute’s policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.

Opportunities for Participation: