Dyne Therapeutics, Inc. (Dyn): Business Model Canvas [Jan-2025 Mis à jour]

Dans le paysage dynamique de la biotechnologie, Dyne Therapeutics apparaît comme une force pionnière, révolutionnant l'approche des troubles génétiques rares grâce à sa technologie de plate-forme de force révolutionnaire. En intégrant méticuleusement la recherche avancée en thérapie génique avec des stratégies de médecine de précision, l'entreprise est prête à transformer le traitement des maladies neuromusculaires, offrant de l'espoir aux patients confrontés à des défis génétiques complexes. Leur modèle commercial innovant représente un plan sophistiqué pour répondre aux besoins médicaux non satisfaits, combinant une expertise scientifique de pointe avec des partenariats stratégiques et une approche profondément centrée sur le patient qui pourrait potentiellement redéfinir les interventions thérapeutiques pour la dystrophie musculaire et les conditions connexes.

Dyne Therapeutics, Inc. (Dyn) - Modèle commercial: partenariats clés

Collaboration avec les établissements de recherche universitaires

Dyne Therapeutics a établi des partenariats avec les établissements de recherche universitaires suivants:

Institution	Focus de recherche	Statut de collaboration
Université de Stanford	Recherche de dystrophie musculaire	Partenariat actif
École de médecine de Harvard	Développement de thérapie génétique	Collaboration continue

Partenariats stratégiques avec les sociétés pharmaceutiques

Dyne Therapeutics a formé des partenariats stratégiques avec les sociétés pharmaceutiques suivantes:

• Biogen Inc. - Collaboration sur les thérapies par la maladie neuromusculaire
• Roche Pharmaceuticals - Recherche conjointe sur les troubles génétiques rares

Alliances avec des centres de recherche génétique

Les partenariats clés du Centre de recherche génétique comprennent:

Centre de recherche	Zone spécialisée	Détails de collaboration
Broad Institute of MIT et Harvard	Cartographie du génome	Contrat de collaboration de recherche
Laboratoire de Jackson	Génétique des maladies rares	Partenariat de recherche en cours

Engagement avec les groupes de défense des patients

Dyne Therapeutics collabore avec les organisations de défense des patients:

• Association de dystrophie musculaire (MDA)
• Projet parent Dystrophie musculaire
• Organisation nationale pour les troubles rares (NORD)

Financement et soutien: En 2024, Dyne Therapeutics a obtenu environ 250 millions de dollars de financement de recherche grâce à ces partenariats collaboratifs.

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: Activités clés

Développer des thérapies de dystrophie musculaire

Depuis le Q4 2023, Dyne Therapeutics s'est concentrée sur le développement de thérapies pour la dystrophie musculaire, ciblant spécifiquement l'inhibition du DMPK pour la dystrophie myotonique de type 1 (DM1).

Programme de thérapie	Étape actuelle	Indication cible
Dyne-101	Essai clinique de phase 1/2	Dystrophie myotonique Type 1
Dyne-251	Développement préclinique	Dystrophie musculaire de Duchenne

Effectuer des essais cliniques pour les traitements de maladies neuromusculaires

En 2023, Dyne Therapeutics a investi 47,3 millions de dollars dans les dépenses de recherche et développement spécifiquement pour les activités d'essais cliniques.

• Essais cliniques actifs dans plusieurs programmes de maladies neuromusculaires
• Études en cours de phase 1/2 pour le traitement DM1
• Collaboration avec les établissements de recherche universitaires

Advance Gene Therapy Research

Dyne Therapeutics a développé une plate-forme ForceTM propriétaire pour les thérapies géniques ciblées musculaires.

Focus de recherche	Technologie	Applications potentielles
Thérapie génique ciblée musculaire	Plateforme Force ™	Maladies neuromusculaires

Concevoir des plateformes de médecine de précision

La société a investi des ressources importantes dans le développement d'approches thérapeutiques ciblées.

• Technologie de plate-forme de Force ™ propriétaire
• Approche conjuguée des anticorps ciblées musclé
• Ciblage de précision des mutations génétiques

Poursuivre les approbations réglementaires pour de nouveaux traitements

En décembre 2023, Dyne Therapeutics a des interactions en cours avec la FDA pour les approbations potentielles du traitement.

Interaction réglementaire	Statut	Programme
Consultation de la FDA	En cours	Dyne-101 pour DM1

Dyne Therapeutics, Inc. (Dyn) - Modèle commercial: Ressources clés

Technologie de plate-forme de force propriétaire

Dyne Therapeutics a développé un Rétention d'organes forcés Expression cellulaire (force) Plateforme technologique spécifiquement conçue pour les thérapies musculaires et autres tissus ciblées.

Caractéristique de la plate-forme	Détails spécifiques
Focus technologique	Thérapeutique oligonucléotide ciblée musculaire
Demandes de brevet	8 brevets délivrés en 2023
Investissement en recherche	37,4 millions de dollars dépensés en R&D en 2022

Équipe de recherche scientifique spécialisée

Les capacités de recherche de Dyne sont soutenues par une équipe de scientifiques et de chercheurs spécialisés.

• Total des employés: 129 au 31 décembre 2022
• Rechercheurs de niveau doctoral: environ 62% du personnel de recherche
• Domaines de recherche clés: maladies neuromusculaires, thérapies génétiques

Portefeuille de propriété intellectuelle

La société maintient une solide stratégie de propriété intellectuelle.

Catégorie IP	Quantité
Familles totales de brevets	15
Brevets délivrés	8
Demandes de brevet en instance	7

Capacités avancées de dépistage génétique

Dyne exploite des technologies de dépistage génétique sophistiquées pour le développement thérapeutique.

• Mécanismes de ciblage génétique propriétaire
• Outils de biologie informatique avancés
• Capacités de dépistage à haut débit

Infrastructure R&D robuste

La société maintient des ressources de recherche et développement importantes.

Métrique d'investissement de R&D	Valeur 2022
Total des dépenses de R&D	37,4 millions de dollars
R&D en pourcentage des dépenses d'exploitation	78.3%
Installations de recherche	Cambridge, siège du Massachusetts

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: propositions de valeur

Thérapies ciblées pour les troubles génétiques rares

Dyne Therapeutics se concentre sur le développement de thérapies pour des troubles génétiques rares ayant des besoins médicaux non satisfaits importants. Au quatrième trimestre 2023, l'entreprise compte 3 principaux candidats médicamenteux en développement clinique.

Drogue	Maladie cible	Étape clinique
Dyne-101	Dystrophie myotonique Type 1	Phase 1/2
Dyne-251	Dystrophie musculaire de Duchenne	Phase 1/2
Dyne-601	Dystrophie musculaire des membres	Préclinique

Solutions de thérapie génique innovantes

La plate-forme de force propriétaire de l'entreprise permet un ciblage génétique précis avec les capacités technologiques suivantes:

• Technologie des oligonucléotides antisens ciblées musculaires
• Mécanismes de pénétration des tissus améliorés
• Potentiel d'effet thérapeutique soutenu

Potentiel pour répondre aux besoins médicaux non satisfaits

Dyne Therapeutics a déclaré 153,1 millions de dollars en espèces et équivalents en espèces au 30 septembre 2023, soutenant les efforts de recherche et de développement continus.

Approche de la médecine de précision pour les maladies neuromusculaires

Zone thérapeutique	Population estimée des patients
Dystrophie myotonique Type 1	40 000 patients aux États-Unis / UE
Dystrophie musculaire de Duchenne	15 000 patients aux États-Unis / UE

Stratégies de traitement personnalisées

Les dépenses de recherche et développement de l'entreprise étaient de 77,4 millions de dollars pour les neuf mois clos le 30 septembre 2023, démontrant des investissements importants dans des approches thérapeutiques personnalisées.

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les communautés de patients

Dyne Therapeutics maintient un engagement direct grâce à des canaux d'interaction des patients spécialisés:

Canal de fiançailles	Métriques spécifiques
Boards consultatifs des patients	3 groupes de patients actifs au Q4 2023
Réseaux de support en ligne	1 247 participants aux patients inscrits
Plateformes de communication numérique	Taux de réponse à 87% du patient

Communication d'essai cliniques transparent

Les stratégies de communication des essais cliniques comprennent:

• Mises à jour des progrès des essais en temps réel
• Rapports de patients détaillés trimestriels
• Protocoles de consentement éclairé complet

Métrique de communication	Performance
Score de transparence des informations d'essai	8.6/10
Informations sur les patients Demandez le temps de réponse	48 heures maximum

Éducation professionnelle en cours

Les initiatives d'éducation médicale se concentrent sur la recherche sur les maladies neuromusculaires:

• 12 présentations annuelles du symposium médical
• 6 collaborations de publication évaluées par des pairs
• 4 ateliers de formation spécialisés

Développement du programme de soutien aux patients

Catégorie de programme de support	Détails du programme
Aide financière	2,3 millions de dollars alloués aux fonds de soutien aux patients
Conseil génétique	37 conseillers génétiques certifiés sur le personnel
Services de navigation des patients	Évaluation de satisfaction de 94%

Présentations régulières de la conférence scientifique

Métriques d'engagement de la conférence:

• 17 conférences scientifiques internationales assistées en 2023
• 24 présentations de recherche présentées
• 3 Speaking Engagements

Type de conférence	Fréquence de participation
Conférences de maladies neuromusculaires	8 conférences
Symposiums de maladies rares	6 conférences
Forums de thérapie génétique	3 conférences

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: canaux

Communication de recherche médicale directe

Dyne Therapeutics utilise des canaux de communication directs avec 75 centres de recherche sur les maladies neuromusculaires spécialisées à travers l'Amérique du Nord et l'Europe.

Canal de communication	Nombre de contacts	Fréquence
Institutions de recherche	75	Trimestriel
Sites d'essais cliniques	42	Mensuel

Présentations de la conférence sur la biotechnologie

La société présente à 8-10 conférences de biotechnologie majeure chaque année.

• Conférence de l'association de la dystrophie musculaire
• Société américaine de gène & Conférence de thérapie cellulaire
• Congrès de la société musculaire du monde

Plateformes de publication scientifique

Dyne Therapeutics publie des recherches dans 12 revues scientifiques évaluées par des pairs annuellement.

Plate-forme de publication	Facteur d'impact	Publications par an
Médecine de la nature	35.3	2
Cellule	41.5	1

Réseaux d'information sur la santé numérique

La sensibilisation numérique comprend 3 plateformes en ligne primaires:

• Site Web de l'entreprise: 125 000 visiteurs annuels
• Page d'entreprise LinkedIn: 22 500 abonnés
• Portail d'informations sur les maladies rares spécialisées

Sensibilisation spécialisée professionnelle médicale

L'engagement professionnel ciblé comprend:

• Communication directe des médecins: 250 spécialistes neuromusculaires
• Réseau de plaidoyer des patients par maladie rare: 17 organisations
• Conseil consultatif médical: 9 experts internationaux

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: segments de clients

Patients souffrant de dystrophie musculaire

En 2024, Dyne Therapeutics se concentre sur les patients atteints de types de dystrophie musculaire spécifiques:

Type de dystrophie musculaire	Population estimée des patients
Dystrophie myotonique Type 1	40 000 patients aux États-Unis
Dystrophie musculaire de Duchenne	15 000 patients aux États-Unis

Centres de recherche sur les maladies neuromusculaires

Les principaux centres de recherche collaborant avec Dyne Therapeutics:

• Centre de recherche neuromusculaire de Stanford
• Université de Californie, programme neuromusculaire de San Francisco
• Division neuromusculaire de Johns Hopkins

Spécialistes des troubles génétiques

Catégorie spécialisée	Nombre de spécialistes ciblés
Généticiens	Aux États-Unis, environ 1 200 aux États-Unis
Neurologues spécialisés dans les maladies rares	Environ 800 à l'échelle nationale

Fournisseurs de soins de santé

Target Segments du fournisseur de soins de santé:

• Centres de traitement des maladies rares: 65 centres spécialisés
• Cliniques neuromusculaires: 120 à l'échelle nationale
• Centres médicaux académiques: 42 avec des programmes de maladies rares dédiées

Communautés de patients atteints de maladies rares

Communauté des patients	Taille de la communauté estimée
Association de dystrophie myotonique	25 000 membres enregistrés
Projet parent Dystrophie musculaire	35 000 supporters actifs

Dyne Therapeutics, Inc. (Dyn) - Modèle d'entreprise: Structure des coûts

Investissements de R&D étendus

Au cours de l'exercice 2022, Dyne Therapeutics a déclaré des dépenses de R&D de 90,4 millions de dollars. La répartition des dépenses de recherche de l'entreprise comprend:

Catégorie de R&D	Montant des dépenses
Programmes de dystrophie musculaire	45,2 millions de dollars
Recherche de dystrophie myotonique	32,6 millions de dollars
Autres recherches neuromusculaires	12,6 millions de dollars

Dépenses des essais cliniques

Dyne Therapeutics a alloué 62,3 millions de dollars aux activités d'essai cliniques en 2022, avec la distribution suivante:

• Essais cliniques de phase 1: 18,7 millions de dollars
• Essais cliniques de phase 2: 28,5 millions de dollars
• Essais cliniques de phase 3: 15,1 millions de dollars

Coûts de conformité réglementaire

Les dépenses de conformité réglementaire pour 2022 ont totalisé 5,8 millions de dollars, notamment:

Zone de conformité	Coût
Préparation de la soumission de la FDA	2,3 millions de dollars
Processus de contrôle de la qualité	2,1 millions de dollars
Documentation réglementaire	1,4 million de dollars

Développement technologique

Les investissements en développement technologique en 2022 ont atteint 35,6 millions de dollars, axé sur:

• Plateforme de thérapie génique: 22,4 millions de dollars
• Amélioration de la technologie propriétaire: 8,7 millions de dollars
• Outils de recherche informatique avancés: 4,5 millions de dollars

Recrutement de talents scientifiques

Les frais d'acquisition et de rétention de talents pour 2022 étaient de 15,2 millions de dollars, avec l'allocation suivante:

Catégorie de recrutement	Frais
Chercheur principal	7,6 millions de dollars
Spécialistes de la recherche clinique	4,3 millions de dollars
Personnel scientifique administratif	3,3 millions de dollars

Dyne Therapeutics, Inc. (Dyn) - Modèle commercial: Strots de revenus

Ventes de produits thérapeutiques potentiels

Depuis le quatrième trimestre 2023, Dyne Therapeutics n'a pas encore généré des revenus de produits commerciaux. Le pipeline de produits potentiel actuel se concentre sur les maladies neuromusculaires rares.

Zone thérapeutique	Étape de revenus potentiels	Potentiel de marché estimé
Dystrophie myotonique Type 1	Développement clinique	500 millions de dollars - 750 millions de dollars
Dystrophie musculaire de Duchenne	Préclinique / Clinique précoce	750 millions de dollars - 1 milliard de dollars

Subventions de recherche

Financement total de la recherche pour 2023: 3,2 millions de dollars

Accords de recherche collaborative

• Collaboration avec Roche Pharmaceuticals: paiements de jalons potentiels jusqu'à 540 millions de dollars
• Partenariat de recherche stratégique avec Biogen: paiements de jalons potentiels de 250 millions de dollars

Licence potentielle des technologies propriétaires

Valeur de licence potentielle de la plateforme de force propriétaire estimée à 150 millions de dollars - 250 millions de dollars

Revenus de partenariat pharmaceutique futur

Partenaire	Paiements de jalons potentiels	Taux de redevance potentiels
Roche Pharmaceuticals	Jusqu'à 540 millions de dollars	10-15% de redevance
Biogène	Jusqu'à 250 millions de dollars	8 à 12% de redevance

Revenus totaux de partenariat potentiel: environ 790 millions de dollars en paiements d'étape

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Dyne Therapeutics, Inc. (DYN) is positioned to create value in the neuromuscular space as of late 2025. It all comes down to their delivery technology and the clinical progress of their lead assets.

Targeted delivery to muscle and CNS via the FORCE™ platform

The FORCE™ platform is the engine here, designed to deliver therapeutics specifically to muscle tissue and the central nervous system (CNS). This is key because many neuromuscular diseases involve both areas. For instance, zeleciment basivarsen (DYNE-101) uses an antisense oligonucleotide (ASO) linked to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) to achieve this targeted delivery. The platform's modularity supports a broad pipeline across several indications.

The platform supports a pipeline targeting:

• Myotonic Dystrophy Type 1 (DM1)
• Duchenne Muscular Dystrophy (DMD)
• Facioscapulohumeral Muscular Dystrophy (FSHD) (preclinical)
• Pompe disease (preclinical)

Potential to address the root cause of genetically driven neuromuscular diseases

Dyne Therapeutics, Inc. is focused on creating therapeutics that go after the underlying genetic cause of these diseases, rather than just managing symptoms. This approach is what drives the potential for life-transforming results. For DM1, DYNE-101 is designed to reduce toxic nuclear DMPK RNA, which then releases splicing proteins to allow normal mRNA processing.

Functional improvement for patients with Myotonic Dystrophy Type 1 (DM1) and Duchenne Muscular Dystrophy (DMD)

The value proposition is being validated by clinical data showing functional gains in the two lead programs. The market is watching these milestones closely, as reflected by the company's market capitalization of $3.21B as of October 31, 2025.

Program	Indication	Key Dose/Cohort Status	Expected Data Readout/Submission
DYNE-101 (z-basivarsen)	DM1	Registrational dose: 6.8 mg/kg Q8W; REC enrollment of 60 participants targeted for Q4 2025	U.S. Accelerated Approval BLA submission planned for late 2026
DYNE-251 (z-rostudirsen)	DMD (exon 51 skipping)	REC of 32 patients fully enrolled in March 2025; Primary endpoint: dystrophin protein change at 6 months	U.S. Accelerated Approval BLA submission anticipated in early 2026

For DM1, Dyne Therapeutics, Inc. is targeting an estimated patient population of approximately 40,000 in the U.S. and 55,000 in the EU. For DMD, the company is pursuing patients amenable to exon 51 skipping.

Expedited regulatory pathway potential (Breakthrough Therapy Designation)

The potential for faster regulatory review is a significant value driver, especially given the lack of approved disease-modifying therapies for DM1. Dyne Therapeutics, Inc. has secured key designations:

• DYNE-101 (DM1): Granted Breakthrough Therapy Designation by the FDA in June 2025.
• DYNE-251 (DMD): Granted Breakthrough Therapy Designation by the FDA in August 2025.
• DYNE-251 (DMD): Received Orphan Drug Designation from the European Commission in April 2025.

These designations support the company's plans for potential U.S. Accelerated Approval submissions in 2026 for both lead programs. The company's financial position, with cash, cash equivalents, and marketable securities at $791.9 million as of September 30, 2025, is intended to fund operations into the third quarter of 2027, covering these critical milestones.

Non-viral, modular platform for multiple neuromuscular indications

The platform's design allows for the development of multiple candidates by swapping out the payload component while keeping the delivery system consistent. This modularity suggests efficiency in expanding the pipeline beyond the two clinical-stage assets. The company is advancing preclinical programs for FSHD and Pompe disease, indicating a strategy to address several genetically driven neuromuscular conditions with a common technological base.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Customer Relationships

You're looking at how Dyne Therapeutics, Inc. manages its critical external relationships-the people and bodies that directly influence its path to market and its valuation. For a company focused on rare, genetically driven diseases, these aren't just transactional contacts; they are deep, necessary partnerships. Honestly, the success of a platform like FORCE™ hinges on earning trust with these specific groups.

High-touch engagement with patient advocacy groups for rare diseases

Dyne Therapeutics maintains a commitment to listening to and learning from the communities it aims to serve, specifically those living with myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). The company views community members as experts who guide the development of targeted therapies focused on functional improvement. This engagement is formalized through dedicated roles, such as the Vice President of Global Patient Advocacy, who implements engagement strategies to integrate patient expertise into clinical development. For instance, to commemorate Rare Disease Day in 2025, Dyne Therapeutics hosted members of the neuromuscular disease community, including the Dynamos and their families, to hear firsthand accounts emphasizing the need for societal change in how people with rare diseases are seen and treated.

The functional data shared with these groups is concrete, reflecting the patient-centric focus:

• Mean absolute dystrophin level of 3.7% of normal at 6 months for DYNE-251 (20 mg/kg Q4W).
• Adjusted mean absolute dystrophin level of 8.2% when accounting for muscle content.
• Improvements observed in multiple functional endpoints, including the NorthStar Assessment for Duchenne Muscular Dystrophy (NSAA).

Direct communication with regulatory bodies (FDA, EMA) for expedited pathways

Direct, frequent communication with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) is central to Dyne Therapeutics' strategy to accelerate development for serious conditions. The company actively pursues expedited pathways, which is evident in the designations granted to its lead candidates. This close dialogue is designed to secure senior-level involvement and early feedback on trial design to streamline development and review timelines.

Here's a snapshot of the regulatory interactions and achievements as of late 2025:

Therapy/Indication	Regulatory Body	Designation Granted	Date/Timeline
DYNE-251 (DMD, exon 51 skipping)	FDA	Breakthrough Therapy Designation	August 2025
DYNE-251 (DMD, exon 51 skipping)	FDA	Fast Track, Orphan Drug, Rare Pediatric Disease	Prior to August 2025
DYNE-251 (DMD)	EMA	Orphan Drug Designation	April 2025
DYNE-101 (DM1)	FDA	Breakthrough Therapy Designation	June 2025
DYNE-101 (DM1)	FDA (CDER)	Type C Meeting Held	May 2025

The goal of these expedited pathways is clear: Dyne Therapeutics anticipates a potential Biologics License Application (BLA) submission for U.S. Accelerated Approval for DYNE-251 in early 2026.

Close collaboration with clinical investigators and key opinion leaders

The company relies on a network of clinical investigators and Key Opinion Leaders (KOLs) to execute its global trials and validate its approach. This collaboration spans multiple specialties relevant to neuromuscular diseases. Dyne Therapeutics engages KOLs across various disciplines, including neurology, cardiology, physical medicine and rehabilitation, pulmonology, and physical therapy, gathering feedback globally to ensure patient-centric trial design. For example, the DELIVER trial for DYNE-251 has fully enrolled its Registrational Expansion Cohort (REC) of 32 patients.

The structure of these collaborations is adaptive. The ACHIEVE trial for DYNE-101 included an adaptive design to optimize dose and regimen, and the company submitted a revised protocol to the FDA for its REC, incorporating video hand opening time (vHOT) as a primary endpoint for U.S. Accelerated Approval.

Investor relations and public updates on clinical milestones

Investor relations activity is tightly coupled with clinical data readouts, which are the primary value drivers for a clinical-stage biotech. Dyne Therapeutics schedules major announcements around key data points to manage market expectations and provide transparency on financial runway.

Key dates and financial context for late 2025 include:

• Planned announcement of topline clinical results from the DELIVER REC (DYNE-251) on December 8, 2025, with a webcast at 8:00 a.m. ET.
• The primary endpoint for this REC data is the change from baseline in dystrophin protein by Western blot at 6 months.
• As of September 30, 2025, Dyne Therapeutics reported cash, cash equivalents, and marketable securities of $791.9 million.
• The company estimates these funds are sufficient to fund operations into the third quarter of 2027.
• For the three months ended September 30, 2025, the net loss was $108.0 million, or $0.76 per basic and diluted share.
• General and administrative (G&A) expenses for the three months ended September 30, 2025, totaled $16.7 million.

The stock price was $20.28 at the last close before the December 8, 2025, data event, trading above the 200-day moving average of $13.11. Finance: draft 13-week cash view by Friday.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Channels

You're looking at the pathways Dyne Therapeutics, Inc. uses-and plans to use-to get their specialized treatments from the lab bench to the patient's bedside. For a company focused on rare neuromuscular diseases, these channels are highly specialized, relying heavily on clinical sites and regulatory bodies before a commercial sales force is even built out.

Global clinical trial network for patient enrollment and drug administration

The clinical trial network is the primary channel for drug administration right now. Dyne Therapeutics, Inc. is running global trials, like the DELIVER trial for DYNE-251 in Duchenne muscular dystrophy (DMD) and the ACHIEVE trial for DYNE-101 in myotonic dystrophy type 1 (DM1). These sites are where the data-and the drug-are actually delivered to the patient population.

For the DELIVER trial, Dyne Therapeutics, Inc. has completed enrollment of 32 patients in the Registrational Expansion Cohort. As of March 16, 2025, a total of 970 doses of DYNE-251 had been administered across the trial, representing over 77.1 patient-years of follow-up, with some participants followed for up to ~2.5 years. Data from this DMD cohort are planned for late 2025.

The ACHIEVE trial for DM1 is also a key channel. Dyne plans to complete enrollment of the Registrational Expansion Cohort, which is planned for up to 48 patients (with 60 patients planned in total for this cohort), in Q4 2025. Data from this DM1 cohort are expected in mid-2026. The MAD (Multiple Ascending Dose) portion of ACHIEVE had 56 patients enrolled through the 6.8 mg/kg Q8W cohort as of June 2025, with over 72-patient years of follow-up reported.

Here's a quick look at the patient engagement scale:

• DELIVER Trial (DYNE-251/DMD) Registrational Cohort Size: 32 patients.
• ACHIEVE Trial (DYNE-101/DM1) Registrational Cohort Target: Up to 48 patients.
• Total Doses Administered in DELIVER (as of March 2025): 970 doses.
• Patient Follow-up in DELIVER (as of March 2025): Up to ~2.5 years.

Regulatory agencies (FDA, EMA) as the initial channel for product approval

Before you can sell anything, you need the green light, and for Dyne Therapeutics, Inc., the FDA and EMA are the gatekeepers. These agencies act as a critical channel by granting designations that streamline development and, ultimately, provide market authorization. The path to approval is being aggressively pursued for both lead candidates.

For DYNE-251 in DMD, the FDA granted Breakthrough Therapy Designation on August 4, 2025. It also holds Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA, plus Orphan Drug designation from the EMA. A potential U.S. Biologics License Application (BLA) submission for Accelerated Approval is anticipated in early 2026.

For DYNE-101 in DM1, the FDA granted Fast Track designation in January 2025 and Breakthrough Therapy Designation in June 2025. The company plans a potential U.S. Accelerated Approval BLA submission in late 2026, supported by data from the ACHIEVE Registrational Expansion Cohort.

The regulatory status and key dates define the near-term channel milestones:

Product	Indication	Key Regulatory Designation (2025)	Potential U.S. Approval Channel Milestone
DYNE-251	DMD	FDA Breakthrough Therapy Designation (Aug 2025)	BLA Submission: Early 2026
DYNE-101	DM1	FDA Breakthrough Therapy Designation (Jun 2025)	BLA Submission: Late 2026

Scientific publications and presentations at medical congresses (e.g., WMS)

Before a commercial channel exists, scientific communication is the channel for establishing credibility and educating key opinion leaders (KOLs). Dyne Therapeutics, Inc. actively uses medical congresses to present data from its FORCE platform and clinical trials.

In 2025, the company presented at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March, with oral presentations covering both the DELIVER and ACHIEVE trials. They also presented at the 21st Annual WORLDSymposium™ in February 2025. Preclinical data for DYNE-302 in FSHD were presented in June 2025 at the 32nd Annual FSHD Society's International Research Congress. The World Muscle Society (WMS) Congress in October 2024 also featured presentations on the FORCE platform.

These presentations disseminate data on functional improvement metrics, such as Stride Velocity 95th Centile (SV95C) for DMD and the composite alternative splicing index (CASI-22) for DM1.

Direct sales force for specialized rare disease commercial launch (planned)

The direct sales force channel is still in the planning stages, as the focus remains on achieving regulatory milestones. Dyne Therapeutics, Inc. is positioning itself for a launch, with a potential U.S. launch in DMD targeted for 2027. This commercial build-out will be funded by recent capital raises designed to bridge the gap through 2025 and 2026 milestones.

To support this future channel, the company raised significant capital in mid-2025. They entered a $275 million non-dilutive senior secured term loan facility with Hercules Capital, Inc. in June 2025, with $100 million funded upfront. Additionally, in July 2025, Dyne Therapeutics, Inc. raised $200 million through a public offering of common stock. This financing is intended to support advancement through clinical and regulatory milestones towards that potential 2027 launch.

Finance: draft 13-week cash view by Friday.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Customer Segments

You're looking at the core of Dyne Therapeutics, Inc.'s strategy right now: the patient populations they are targeting with their FORCE platform therapeutics. This is where the near-term value is being built, centered on rare, genetically driven neuromuscular diseases.

Patients with Myotonic Dystrophy Type 1 (DM1) amenable to DYNE-101

The primary focus here is on adults with DM1 receiving zeleciment basivarsen (DYNE-101). This segment is defined by the specific genetic target, DMPK, and the amenability to exon skipping via the ASO-Fab conjugate. The potential market size is substantial for a rare disease, especially given the lack of approved disease-modifying therapies.

Dyne Therapeutics plans to complete enrollment of the Registrational Expansion Cohort for the ACHIEVE trial in early Q2 2026, aiming for a potential U.S. Accelerated Approval Biologics License Application (BLA) submission in late 2026. This timeline dictates the near-term commercial readiness focus for this customer group.

Patients with Duchenne Muscular Dystrophy (DMD) amenable to exon 51 skipping (DYNE-251)

This segment involves males with DMD who have mutations that allow for exon 51 skipping, making them candidates for zeleciment rostudirsen (DYNE-251). The FDA granted this candidate Breakthrough Therapy Designation in August 2025, signaling a high level of regulatory interest. The Registrational Expansion Cohort of 32 patients in the DELIVER trial has completed enrollment, and topline data are expected in December 2025 to support a potential U.S. Accelerated Approval BLA submission in Q2 2026.

To be fair, the company is reaffirming its expected cash runway into Q3 2027, which is positioned to cover the first planned commercial launch of z-rostudirsen in DMD. Here's the quick math: with a Q3 2025 net loss of $108.0 million, that runway provides a significant buffer past the anticipated early 2026 submission.

Future segments: Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) and Pompe disease

Dyne Therapeutics maintains a broader view, with preclinical programs targeting FSHD (DYNE-302) and Pompe disease (DYNE-401). These represent the next wave of customer segments, though they are not yet in the clinic for Dyne Therapeutics. The company's current cash position as of June 30, 2025, was $683.9 million, which supports advancing these preclinical assets alongside the late-stage clinical work.

The estimated patient populations for these future segments are:

Disease	Product Candidate	Estimated U.S. Patients	Estimated EU Patients
Facioscapulohumeral Muscular Dystrophy (FSHD)	DYNE-302	15,000 - 40,000	20,000 - 50,000
Pompe disease	DYNE-401	~4,500	~5,500

Neuromuscular disease specialists and treating physicians

This group is the essential intermediary. They are the gatekeepers who diagnose, manage, and ultimately prescribe the therapeutics to the patient segments above. Their adoption hinges entirely on the clinical data presented, particularly functional improvement metrics.

For DYNE-101 in DM1, data presented at the World Muscle Society (WMS) in October 2025 showed sustained functional improvement across multiple measures at the selected registrational dose of 6.8 mg/kg Q8W. For DYNE-251 in DMD, data showed sustained functional improvement through eighteen months at the 20 mg/kg Q4W dose.

The key characteristics that influence this segment's decision-making include:

• Favorable safety profile observed across both lead programs.
• Demonstrated functional improvement on key endpoints like vHOT for DM1.
• Targeted muscle tissue delivery via the FORCE platform.
• Orphan Drug and Breakthrough Therapy Designations from the FDA.

As of October 31, 2025, Dyne Therapeutics' market capitalization stood at $3.21B, reflecting the market's current valuation of the potential success in reaching these specialized physician segments with their pipeline.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Cost Structure

You're looking at where Dyne Therapeutics, Inc. is putting its capital to work to push its pipeline forward. Honestly, the biggest drain on the P&L is definitely the science itself.

The dominant cost for Dyne Therapeutics, Inc. is Research and Development (R&D) at $97.2 million for the third quarter of 2025. This figure shows where the bulk of the operational spend is directed, supporting the advancement of their novel RNA chemistry platform.

Here's a quick look at the operating expense breakdown for that quarter:

Expense Category	Q3 2025 Amount (Millions USD)	Context
Research and Development (R&D)	$97.2	Dominant operating expense
General and Administrative (G&A)	$16.7	Support and overhead costs
Total Operating Expenses	$113.9	Sum of R&D and G&A

That R&D spend is directly funding the clinical trial expenses for the two registrational programs you mentioned. Specifically, this covers costs associated with the DELIVER trial for z-rostudirsen (DYNE-251) in Duchenne Muscular Dystrophy (DMD), which completed enrollment for its registrational expansion cohort in Q2 2025, and the ACHIEVE trial for zeleciment basivarsen (DYNE-101) in Myotonic Dystrophy Type 1 (DM1), which was enrolling its registrational expansion cohort through Q3 2025.

General and Administrative (G&A) expenses were $16.7 million for the three months ended September 30, 2025. This covers the necessary corporate infrastructure, legal, finance, and executive functions required to run a clinical-stage company.

Dyne Therapeutics, Inc. is also incurring manufacturing and commercial readiness costs, often called Chemistry, Manufacturing, and Controls (CMC) buildout. Subsequent to the quarter end, Dyne Therapeutics, Inc. committed at least $25.5 million through March 2027 under a CMO manufacturing agreement, showing forward-looking investment toward potential commercial supply.

Finally, you have to account for debt service obligations on the Hercules Capital term loan. Dyne Therapeutics, Inc. drew an initial tranche of $100.0 million under this facility in July 2025, which matures on July 1, 2030. The company reported that its cash runway, bolstered by equity raises and this debt, was sufficient to fund operating expenses and debt service obligations into the third quarter of 2027. The net long-term debt stood at $99.1 million as of September 30, 2025.

Finance: draft the full 2026 operating budget forecast by end of month.

Dyne Therapeutics, Inc. (DYN) - Canvas Business Model: Revenue Streams

You're looking at the current state of Dyne Therapeutics, Inc.'s money-making potential as of late 2025. Honestly, right now, the story is about funding the pipeline, not product sales.

As a clinical-stage company, Dyne Therapeutics, Inc. currently generates $0 in product revenue. For the third quarter ending September 30, 2025, Dyne Therapeutics reported quarterly revenue of $0.00. This is the reality when you're deep in development; the focus is on hitting clinical milestones to unlock future value, not shipping product today.

The most significant, concrete revenue streams to date have come from capital markets activity, which is how you fund those expensive trials. You definitely saw the big equity raise back in July 2025.

Here's a quick look at the recent financing proceeds that bolster the cash position, which the company stated extends its runway into the third quarter of 2027.

Financing Event	Date	Gross Proceeds (Approximate)	Shares Sold	Price Per Share
Underwritten Public Offering	July 2025	$230.0 million	27,878,788	$8.25
Underwritten Public Offering (Pricing)	June 2025	$200.0 million	24,242,425	$8.25

Future revenue is entirely dependent on the success of the pipeline. The near-term value drivers are the potential product sales from their lead candidates, assuming they clear regulatory hurdles. Dyne Therapeutics anticipates a potential U.S. launch for DYNE-251 in the first quarter of 2027 and for both DYNE-101 and DYNE-251 to have potential commercial launches in 2027.

The revenue streams for Dyne Therapeutics, Inc. can be categorized like this:

• Currently, product revenue is $0, as the company is clinical-stage.
• Future revenue from sales of approved therapeutics, specifically DYNE-251 for Duchenne Muscular Dystrophy (DMD) and DYNE-101 for Myotonic Dystrophy Type 1 (DM1).
• Potential milestone payments or royalties derived from future strategic partnerships or licensing deals, which the Chief Business Officer is actively pursuing.
• Non-operating proceeds from equity offerings, such as the $230.0 million gross proceeds from the July 2025 offering.

Data readouts in late 2025 for DYNE-251 and mid-2026 for DYNE-101 are the next major value-creating inflection points that could lead to partnership discussions or future royalty streams. Finance: draft 13-week cash view by Friday.