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
Patents
Muscle targeting complexes and uses thereof for treating dystrophinopathies
US-12329824-B1
View DetailsMuscle targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of use thereof to induce exon skipping of exon 44 of dystrophin in a subject
US-12329825-B1
View DetailsMethod of using an anti-transferrin receptor antibody to deliver an oligonucleotide to a subject having facioscapulohumeral muscular dystrophy
US-12325753-B2
View DetailsComplexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US-12319743-B2
View DetailsMuscle targeting complexes and uses thereof for treating myotonic dystrophy
US-12280122-B2
View DetailsMuscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of use thereof to target dystrophin and to treat Duchenne muscular dystrophy
US-12263225-B2
View Details
Muscle targeting complexes and uses thereof for treating dystrophinopathies
US-12329824-B1
View DetailsMuscle targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of use thereof to induce exon skipping of exon 44 of dystrophin in a subject
US-12329825-B1
View DetailsMethod of using an anti-transferrin receptor antibody to deliver an oligonucleotide to a subject having facioscapulohumeral muscular dystrophy
US-12325753-B2
View DetailsComplexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US-12319743-B2
View DetailsMuscle targeting complexes and uses thereof for treating myotonic dystrophy
US-12280122-B2
View DetailsMuscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of use thereof to target dystrophin and to treat Duchenne muscular dystrophy
US-12263225-B2
View DetailsProducts
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
Sponsorship and operational execution of global Phase 1/2 randomized, placebo-controlled clinical trials with MAD and registrational expansion cohorts for neuromuscular diseases.
Design and provision of participant support informed by community advisory input, including travel assistance, reimbursement and accommodations to reduce participation burden.
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.
Identification, negotiation and integration of in-licensed academic technologies and partnerships to expand therapeutic programs and target novel genetic mechanisms.
Sponsorship and operational execution of global Phase 1/2 randomized, placebo-controlled clinical trials with MAD and registrational expansion cohorts for neuromuscular diseases.
Design and provision of participant support informed by community advisory input, including travel assistance, reimbursement and accommodations to reduce participation burden.
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.
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)
News & Updates
Dyne Therapeutics announced it will present new preclinical data at the FSHD Society International Research Congress.
Dyne announced the acceleration of its FSHD programs through exclusive licensing of DUX4-targeting technologies and the appointment of Dr. Jeffrey Statland to its Scientific Advisory Board. The licensed technology targets the genetic basis of FSHD, developed by UMONS in Belgium, and complements Dyne’s FORCE platform.
Dyne Therapeutics announced it will present new preclinical data at the FSHD Society International Research Congress.
Dyne announced the acceleration of its FSHD programs through exclusive licensing of DUX4-targeting technologies and the appointment of Dr. Jeffrey Statland to its Scientific Advisory Board. The licensed technology targets the genetic basis of FSHD, developed by UMONS in Belgium, and complements Dyne’s FORCE platform.