Dyne Therapeutics
Dyne Therapeutics is a pioneering biotech company focused on developing life-transforming therapies for genetically driven neuromuscular diseases. Utilizing their FORCE™ platform, they aim to deliver targeted therapeutics to muscle and the central nervous system to stop or reverse disease progression. The company is committed to engaging with the community, advancing clinical trials, and delivering innovative treatments for serious muscle diseases such as DM1, DMD, and FSHD. They are actively involved in clinical trials for these conditions and are building a robust portfolio of investigational therapeutics.
Industries
Nr. of Employees
medium (51-250)
Dyne Therapeutics
Cambridge, Massachusetts, United States, North America
Products
TfR1-targeted siRNA conjugate for FSHD (preclinical program)
A transferrin receptor 1 (TfR1)-binding antibody-fragment conjugated to siRNA targeting DUX4 mRNA, demonstrated preclinical DUX4 suppression and functional benefit in vitro and in proprietary in vivo models.
TfR1-targeted antisense oligonucleotide (ASO) conjugate for DM1 (Phase 1/2)
An antisense oligonucleotide conjugated to a TfR1-binding antibody fragment being evaluated in a Phase 1/2 global trial to reduce mutant DMPK RNA, restore normal splicing and assess safety, PK/PD and clinical endpoints.
TfR1-targeted PMO exon-skipping conjugate for DMD (Phase 1/2)
A phosphorodiamidate morpholino oligomer (PMO) conjugated to a TfR1-binding antibody fragment designed to promote exon skipping (e.g., exon 51) and increase dystrophin production; evaluated in a Phase 1/2 randomized, placebo-controlled study assessing safety and functional outcomes.
Preclinical discovery programs for additional neuromuscular indications
Platform-enabled preclinical programs targeting multiple neuromuscular diseases with candidate selection driven by disease-model data and translational endpoint alignment.
TfR1-targeted siRNA conjugate for FSHD (preclinical program)
A transferrin receptor 1 (TfR1)-binding antibody-fragment conjugated to siRNA targeting DUX4 mRNA, demonstrated preclinical DUX4 suppression and functional benefit in vitro and in proprietary in vivo models.
TfR1-targeted antisense oligonucleotide (ASO) conjugate for DM1 (Phase 1/2)
An antisense oligonucleotide conjugated to a TfR1-binding antibody fragment being evaluated in a Phase 1/2 global trial to reduce mutant DMPK RNA, restore normal splicing and assess safety, PK/PD and clinical endpoints.
TfR1-targeted PMO exon-skipping conjugate for DMD (Phase 1/2)
A phosphorodiamidate morpholino oligomer (PMO) conjugated to a TfR1-binding antibody fragment designed to promote exon skipping (e.g., exon 51) and increase dystrophin production; evaluated in a Phase 1/2 randomized, placebo-controlled study assessing safety and functional outcomes.
Preclinical discovery programs for additional neuromuscular indications
Platform-enabled preclinical programs targeting multiple neuromuscular diseases with candidate selection driven by disease-model data and translational endpoint alignment.
Services
Clinical trial sponsorship and operations for early-phase rare-disease studies
Sponsorship and operational execution of global Phase 1/2 randomized, placebo-controlled clinical trials with MAD and registrational expansion cohorts for neuromuscular diseases.
Patient engagement and participant support services
Design and provision of participant support informed by community advisory input, including travel assistance, reimbursement and accommodations to reduce participation burden.
Regulatory strategy and submission support
Development of regulatory strategies and preparation of regulatory submissions (IND/CTA and later-stage filings) including pursuit of orphan, fast-track and rare pediatric designations.
Academic licensing and collaboration management
Identification, negotiation and integration of in-licensed academic technologies and partnerships to expand therapeutic programs and target novel genetic mechanisms.
Clinical trial sponsorship and operations for early-phase rare-disease studies
Sponsorship and operational execution of global Phase 1/2 randomized, placebo-controlled clinical trials with MAD and registrational expansion cohorts for neuromuscular diseases.
Patient engagement and participant support services
Design and provision of participant support informed by community advisory input, including travel assistance, reimbursement and accommodations to reduce participation burden.
Regulatory strategy and submission support
Development of regulatory strategies and preparation of regulatory submissions (IND/CTA and later-stage filings) including pursuit of orphan, fast-track and rare pediatric designations.
Academic licensing and collaboration management
Identification, negotiation and integration of in-licensed academic technologies and partnerships to expand therapeutic programs and target novel genetic mechanisms.
Expertise Areas
- Targeted oligonucleotide therapeutics development
- Receptor-mediated delivery to muscle and CNS
- Clinical trial design and execution for rare neuromuscular diseases
- Biomarker development and translational analytics (molecular and imaging)
Key Technologies
- Transferrin receptor 1 (TfR1)-targeted delivery using antibody fragments
- Antisense oligonucleotides (ASO)
- Small interfering RNA (siRNA)
- Phosphorodiamidate morpholino oligomers (PMO)