Design Therapeutics (DSGN) Jefferies London Healthcare Conference 2024 summary

Strategic vision and platform differentiation

• Pioneering small molecule genomic medicines that modulate gene expression for monogenic diseases.

• Genomic medicine platform aims to surpass gene editing and gene therapy in efficacy and safety.

• Four lead programs target Friedreich ataxia, Fuchs endothelial corneal dystrophy, Huntington's disease, and myotonic dystrophy type 1.

• GeneTAC molecules offer broad tissue distribution and act without altering the genome.

• Platform designed for allele-selective targeting and modulation of disease-causing mutations.

Pipeline progress and clinical milestones

• Friedreich ataxia program to enter clinic in H1 2025 with new DT216P2 formulation; patient trials to follow later in 2025.

• Fuchs endothelial corneal dystrophy program in phase 1 with healthy volunteer data expected in H1 next year.

• Huntington's and myotonic dystrophy programs advancing toward development candidate declaration.

• Two of four pipeline programs expected in clinic next year.

• Cash runway extends into 2029, supporting clinical proof of concept for up to four programs.

Scientific and clinical insights

• FA GeneTAC molecules restore frataxin expression in patient cells without affecting healthy cells.

• New DT216P2 formulation resolves prior injection site issues and achieves sustained exposure in preclinical models.

• Consistent potency observed across FA patients regardless of genotype or repeat length.

• FECD program targets toxic RNA foci in corneal cells, with DT168 showing nanomolar potency and formulated as an eye drop.

• Observational studies underway to refine endpoints and patient selection for future interventional trials.