Dyne Therapeutics (DYN) investor relations material

Platform and technology overview

• Developed a targeted delivery platform using a monovalent Fab to engage transferrin receptor 1, enabling broad and safe distribution of genetic medicines for neuromuscular diseases.

• Designed to avoid anemia and cross-linking issues seen with monoclonal antibodies, allowing higher dosing and CNS delivery.

• Platform validated in DMD and DM1, showing functional improvement from baseline and placebo across multiple measures.

Competitive landscape and clinical differentiation

• Differentiates from competitors by using antisense oligonucleotides targeting the nucleus, unlike siRNA-based approaches acting in the cytoplasm.

• Achieved higher levels of splicing correction and functional improvement at higher doses without safety limitations.

• Emphasizes the importance of sufficient nuclear delivery and splicing correction, measured by CASI analytics.

Clinical data and regulatory strategy

• Presented data showing 25% splicing correction and broad functional improvement at 6.8 mg/kg dose in DM1.

• Upcoming data from a 12-month, 25-patient cohort will include functional measures, CNS subscales, and vHOT, compared to natural history.

• Pursuing accelerated approval with vHOT as an intermediate endpoint, while phase III will use Five Times Sit-to-Stand as the primary, clinically meaningful endpoint.