Tenaya Therapeutics, Inc. (TNYA): Modelo de Negocio Canvas [Actualizado en Ene-2025]

En el reino de vanguardia de la terapéutica cardíaca genética, Tenaya Therapeutics surge como un faro de esperanza, pionero en soluciones de medicina de precisión transformadora que prometen revolucionar el tratamiento de enfermedades cardíacas complejas. Al aprovechar las tecnologías avanzadas de ingeniería genética y las colaboraciones estratégicas con líderes de la industria como Roche, esta innovadora compañía de biotecnología está desarrollando terapias innovadoras que se dirigen a mutaciones genéticas específicas, ofreciendo enfoques de tratamiento personalizados para pacientes con condiciones genéticas cardíacas previamente no para que no sean las condiciones genéticas cardíacas. Su lienzo de modelo de negocio integral revela una estrategia sofisticada preparada para remodelar la medicina cardiovascular a través de intervenciones dirigidas y científicamente rigurosas que podrían desbloquear nuevas vías para la curación.

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: asociaciones clave

Colaboración estratégica con Roche para la investigación genética de enfermedades cardíacas

En enero de 2023, Tenaya Therapeutics anunció una colaboración estratégica con Roche centrada en el desarrollo de terapias genéticas de enfermedades cardíacas. Los detalles clave de la asociación incluyen:

Aspecto de asociación	Detalles específicos
Enfoque de colaboración	Investigación de enfermedades cardíacas genéticas y desarrollo terapéutico
Términos financieros	$ 65 millones de pago por adelantado de Roche a Tenaya
Pagos potenciales de hitos	Hasta $ 700 millones basados ​​en logros de investigación y desarrollo

Asociación con centros médicos académicos para ensayos clínicos

Tenaya Therapeutics ha establecido asociaciones de ensayos clínicos con múltiples centros médicos académicos:

• Facultad de Medicina de la Universidad de Stanford
• Universidad de California, San Francisco
• Escuela de Medicina de Harvard
• Centro médico de la Universidad Johns Hopkins

Colaboración con instituciones de investigación farmacéutica

Institución de investigación	Enfoque de colaboración	Año establecido
Asociación Americana del Corazón	Investigación genética cardiovascular	2022
Institutos Nacionales de Salud	Desarrollo terapéutico de la enfermedad genética	2021

Posibles asociaciones con laboratorios de pruebas genéticas

Tenaya está explorando asociaciones con laboratorios de pruebas genéticas para avanzar en los enfoques de medicina de precisión:

• Invitae Corporation
• Salud genómica
• 23 y

Valor potencial de asociación total: Aproximadamente $ 765 millones en colaboraciones actuales y potenciales a partir de 2024.

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: actividades clave

Investigación y desarrollo de terapias genéticas de precisión

Tenaya Therapeutics se centra en desarrollar terapias genéticas de precisión con una inversión total de I + D de $ 83.4 millones a partir del año fiscal 2023. La compañía mantiene 3 plataformas de investigación de terapia genética distintas dirigidas a enfermedades genéticas cardíacas.

Área de enfoque de I + D	Inversión ($ m)	Etapa de investigación
Terapias cardíacas genéticas	42.6	Preclínico/fase 1
Tecnologías de edición de genes	25.8	Fase de descubrimiento
Orientación molecular	15.0	Investigación traslacional

Realización de ensayos clínicos avanzados para los tratamientos de enfermedades cardíacas

Las actividades de ensayos clínicos abarcan múltiples estudios continuos con gastos actuales de $ 47.2 millones en 2023. La compañía tiene 2 programas primarios de etapa clínica en desarrollo.

• TDN-001: ensayo clínico de miocardiopatía genética
• TDN-002: Estudio de intervención genética de insuficiencia cardíaca

Desarrollo de productos de terapia génica

Tenaya mantiene 3 tuberías distintas de desarrollo de productos de terapia génica con costos totales de desarrollo de productos de $ 62.7 millones en 2023.

Tubería de productos	Etapa de desarrollo	Inversión proyectada
Terapia génica cardíaca	Preclínico	$ 28.3M
Plataforma de intervención genética	Descubrimiento	$ 19.5M
Terapia de orientación molecular	Investigación	$ 14.9m

Investigación molecular y celular

La investigación se centra en las condiciones cardíacas genéticas con un equipo de investigación dedicado de 42 científicos y un presupuesto de investigación anual de $ 35.6 millones.

Medicina traslacional e innovación terapéutica

La compañía invirtió $ 22.1 millones en investigación de medicina traslacional, dirigida a 4 mecanismos específicos de enfermedad genética cardíaca en 2023.

• Identificación de mutación genética
• Técnicas de reprogramación celular
• Orientación molecular avanzada
• Desarrollo terapéutico de precisión

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: recursos clave

Tecnologías avanzadas de ingeniería genética

Therapeutics de Tenaya se ha desarrollado 3 tecnologías genéticas patentadas dirigido a la enfermedad cardíaca:

• Plataforma de terapia génica para enfermedades cardíacas genéticas
• Plataforma de terapia celular para regeneración cardíaca
• Plataforma de molécula pequeña para trastornos cardíacos genéticos

Plataformas de terapia génica patentadas

Plataforma	Tipo de tecnología	Condición objetivo
TNYA-001	Terapia génica AAV	Trastornos del músculo cardíaco genético
TNYA-002	Regeneración celular	Insuficiencia cardiaca
TNYA-003	Intervención de molécula pequeña	Mutaciones genéticas cardíacas

Equipo de investigación y desarrollo especializado

Composición del personal de investigación:

• Personal total de I + D: 68 empleados
• Investigadores de doctorado: 42
• Especialistas en ingeniería genética: 26

Cartera de propiedades intelectuales

Categoría de IP	Número de patentes	Estado de patente
Tecnologías de terapia génica	12	Otorgada
Métodos de regeneración celular	8	Pendiente
Técnicas de intervención molecular	6	Archivado

Instalaciones de laboratorio e investigación de vanguardia

Detalles de la infraestructura de investigación:

• Espacio total de la instalación de investigación: 35,000 pies cuadrados
• Ubicación: South San Francisco, California
• Equipo de secuenciación genética avanzada: 7 plataformas
• Inversión anual de I + D: $ 48.3 millones (año fiscal 2023)

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocio: propuestas de valor

Terapias genéticas de precisión innovadora para enfermedades cardíacas

Tenaya Therapeutics se centra en el desarrollo de terapias genéticas dirigidas a condiciones cardíacas específicas con los siguientes programas terapéuticos clave:

Programa	Condición objetivo	Etapa de desarrollo
TDN-1	Miocardiopatía dilatada genética	Preclínico
TDN-2	Miocardiopatía hipertrófica	De investigación
TDN-3	Insuficiencia cardíaca genética	Etapa de investigación

Enfoques de tratamiento personalizados para afecciones cardíacas genéticas

El enfoque de precisión de Tenaya implica intervenciones genéticas específicas con plataformas tecnológicas específicas:

• Modulación de terapia génica
• Edición de genes de precisión
• Estrategias de orientación molecular

Potencial para abordar las necesidades médicas no satisfechas en los trastornos cardíacos genéticos

Categoría de desorden	Población de pacientes estimada	Limitaciones de tratamiento actuales
Miocardiopatía dilatada	1 en 2.500 individuos	Terapias genéticas específicas limitadas
Miocardiopatía hipertrófica	1 de cada 500 personas	Manejo sintomático solamente

Soluciones terapéuticas avanzadas dirigidas a mutaciones genéticas específicas

Las plataformas tecnológicas de Tenaya permiten estrategias precisas de intervención genética:

• Edición de genes basada en CRISPR
• Enfoques terapéuticos de ARN
• Mecanismos de corrección molecular

Potencial prometedor para tratamientos genéticos cardíacos transformadores

La inversión financiera en investigación y desarrollo demuestra el compromiso con las terapias genéticas innovadoras:

Año fiscal	Gasto de I + D	Solicitudes de patentes
2022	$ 42.3 millones	7 nuevas aplicaciones
2023	$ 56.7 millones	12 nuevas aplicaciones

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: relaciones con los clientes

Compromiso directo con profesionales médicos

Tenaya Therapeutics mantiene la participación directa a través de canales de comunicación médica específicas.

Método de compromiso	Frecuencia	Público objetivo
Presentaciones de conferencias científicas	4-6 por año	Cardiólogos, genetistas
Seminarios médicos especializados	3-4 por trimestre	Médicos de investigación
Actualizaciones de investigación personalizadas	Trimestral	Líderes de opinión clave

Programas de apoyo y educación del paciente

Tenaya proporciona mecanismos integrales de soporte centrados en el paciente.

• Recursos de asesoramiento genético
• Portal de información del paciente en línea
• Talleres de concientización sobre enfermedades
• Guía de tratamiento individualizado

Comunicación científica y transparencia

Se prioriza la comunicación transparente de los resultados de la investigación.

Canal de comunicación	Frecuencia de publicación	Plataforma
Publicaciones de revistas revisadas por pares	2-3 por año	Naturaleza, Cell, Jama
Informes de transparencia de datos de investigación	Biannual	Sitio web de la empresa

Interacciones de los participantes del ensayo clínico

Enfoque estructurado para administrar las relaciones de los participantes de los ensayos clínicos.

• Protocolos de consentimiento informado
• Monitoreo de salud regular
• Comunicación integral de seguimiento
• Mecanismos de protección de la privacidad del paciente

Colaboración de investigación continua con la comunidad médica

Colaboración activa con instituciones de investigación y centros médicos.

Tipo de colaboración	Número de asociaciones activas	Enfoque de investigación
Asociaciones de investigación académica	7 colaboraciones activas	Trastornos genéticos cardíacos
Redes de investigación clínica	12 Instituciones participantes	Iniciativas de medicina de precisión

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: canales

Publicaciones directas de investigación médica

Tenaya Therapeutics publica investigación en revistas revisadas por pares como:

Nombre del diario	Frecuencia de publicación	Factor de impacto
Biotecnología de la naturaleza	Mensual	41.4
Celúla	Quincenal	38.6

Conferencias y simposios científicos

Detalles de participación de la conferencia clave:

• Sesiones científicas de la American Heart Association
• Conferencia de tecnologías de investigación cardiovascular
• Sociedad Europea de Cardiología Congreso

Redes de la industria farmacéutica

Plataformas de redes y métricas de compromiso:

Plataforma de redes	Conexiones anuales	Valor de colaboración potencial
Convención BiO International	16,000 asistentes	$ 45 millones posibles asociaciones
Conferencia de atención médica de JP Morgan	9,000 asistentes	$ 30 millones de inversiones potenciales

Plataformas de investigación médica en línea

Canales de compromiso de investigación digital:

• Investigador
• PubMed Central
• Ciencia directa

Alcance del profesional médico dirigido

Decuestros de estrategia de divulgación:

Método de divulgación	Alcance anual	Tasa de compromiso
Campañas de correo electrónico directas	5.200 cardiólogos	12.3%
Serie de seminarios web	3.800 profesionales médicos	8.7%

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: segmentos de clientes

Pacientes de enfermedad genética cardíaca

Población total de pacientes direccionables para enfermedades cardíacas genéticas: 1.5 millones en los Estados Unidos

Segmento de paciente	Población estimada	Necesidad de tratamiento potencial
Miocardiopatía hipertrófica	200,000	Alto
Miocardiopatía dilatada	500,000	Medio
Síndromes de arritmia hereditarios	100,000	Alto

Cardiólogos y especialistas genéticos

Número de cardiólogos en ejercicio en los Estados Unidos: 33,700

• Subespecialistas de cardiología genética: 1.200
• Volumen de pruebas genéticas anuales: 250,000 pruebas genéticas cardíacas

Instituciones de investigación médica académica

Número de instituciones de investigación que se centran en la genética cardíaca: 87

Tipo de institución	Número de instituciones	Presupuesto de investigación anual
Universidades de investigación de primer nivel	22	$ 500 millones - $ 1.2 mil millones
Centros de investigación médica	45	$ 100 millones - $ 400 millones
Centros de investigación cardíacos especializados	20	$ 50 millones - $ 200 millones

Organizaciones de investigación farmacéutica

Gasto total de I + D farmacéutica en terapias genéticas: $ 15.2 mil millones en 2023

• Número de compañías farmacéuticas que invierten en terapias cardíacas genéticas: 42
• Inversión anual promedio por empresa: $ 362 millones

Sistemas de atención médica centrados en las terapias genéticas

Número de sistemas de salud con programas de terapia genética dedicados: 63

Tipo de sistema de salud	Número de sistemas	Inversión de terapia genética
Grandes centros médicos académicos	28	$ 75 millones - $ 250 millones
Redes de salud regionales	35	$ 20 millones - $ 100 millones

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Según el informe financiero de tercer trimestre de Therapeutics de Tenaya, los gastos de investigación y desarrollo fueron de $ 31.4 millones para el trimestre que finalizó el 30 de septiembre de 2023.

Categoría de gastos	Cantidad (tercer trimestre 2023)
Costos de personal	$ 12.6 millones
Suministros de laboratorio	$ 8.2 millones
Contratos de investigación externos	$ 6.9 millones
Costos de la instalación	$ 3.7 millones

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para el año 2023 totalizaron aproximadamente $ 45.2 millones.

• Pruebas de fase 1: $ 15.6 millones
• Pruebas de fase 2: $ 22.8 millones
• Cumplimiento regulatorio: $ 6.8 millones

Protección de propiedad intelectual

Tenaya Therapeutics gastó $ 2.3 millones en protección de propiedad intelectual en 2023.

Categoría de protección de IP	Costo
Presentación de patentes	$ 1.4 millones
Honorarios legales	$ 0.9 millones

Inversiones avanzadas de tecnología y equipos

Las inversiones en tecnología y equipos para 2023 alcanzaron $ 7.5 millones.

• Equipo de investigación de terapia génica: $ 4.2 millones
• Infraestructura de biología computacional: $ 2.1 millones
• Instrumentación de laboratorio especializada: $ 1.2 millones

Reclutamiento y retención de talento especializado

Los gastos totales relacionados con el talento en 2023 fueron de $ 18.6 millones.

Categoría de gastos de talento	Cantidad
Compensación	$ 14.3 millones
Reclutamiento	$ 2.5 millones
Capacitación y desarrollo	$ 1.8 millones

Tenaya Therapeutics, Inc. (TNYA) - Modelo de negocios: flujos de ingresos

Venta potencial de productos terapéuticos futuros

A partir del cuarto trimestre de 2023, Tenaya Therapeutics aún no ha generado ingresos comerciales de productos. Los candidatos a la terapia génica principal de la compañía para enfermedades cardíacas se encuentran en etapas de desarrollo clínico.

Acuerdos de colaboración de investigación

Socio de colaboración	Tipo de acuerdo	Valor potencial
Vértices farmacéuticos	Colaboración de investigación	$ 65 millones de pago por adelantado en 2022

Licencias de tecnologías de terapia genética

Cartera de propiedades intelectuales:

• 3 plataformas de tecnología de terapia genética central
• Múltiples aplicaciones de patentes en terapia génica cardiovascular

Pagos potenciales de hitos de asociaciones farmacéuticas

Programa	Pagos potenciales de hitos	Etapa de desarrollo
Colaboración de vértices	Hasta $ 700 millones en posibles pagos de hitos	Desarrollo preclínico/clínico

Subvimiento de financiación y apoyo de investigación

Financiación total de la subvención recibida: $ 12.3 millones de NIH y otras instituciones de investigación a partir de 2023.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Value Propositions

Potentially curative, one-time treatment for genetic heart diseases.

Tenaya Therapeutics, Inc. is advancing gene therapies designed to offer a single-administration solution for inherited cardiomyopathies, aiming to halt or reverse disease progression. As of the third quarter of 2025, the company reported a net loss of $20.3 million, with Research & Development Expenses at $15.4 million for the quarter, reflecting investment in these pipeline assets. The cash, cash equivalents, and investments stood at $56.3 million as of September 30, 2025, which management believes, along with potential loan availability, funds operations into the second half of 2026.

The core value proposition is rooted in the potential for durable, disease-modifying effects from a single dose, contrasting with chronic management strategies. The company's market capitalization was reported at $221.5 million in early December 2025.

Program	Indication	Trial Phase/Status (Late 2025)	Key Clinical Data Point
TN-201	MYBPC3-associated HCM	Phase 1b/2a MyPEAK-1 (Dose Expansion)	LVPWT reduced 21-39% at week 52 in Cohort 1
TN-401	PKP2-associated ARVC	Phase 1 RIDGE-1 (Cohort 2 Dosing Complete)	Safety/biopsy data from Cohort 1 expected by year-end 2025
TN-301	HFpEF	Preclinical/Early Stage	Small molecule HDAC6 inhibitor

Addressing the underlying cause of MYBPC3-HCM and PKP2-ARVC.

For MYBPC3-HCM, the value proposition is correcting the genetic defect causing the disease. The MyPEAK-1 trial has dosed a total of seven patients to date, with the study designed to recruit up to 30 patients. Data presented in November 2025 showed that all Cohort 1 patients achieved NYHA Class I by one year post-treatment.

For PKP2-ARVC, the therapy aims to restore the structural integrity of heart muscle cells by delivering a functional PKP2 gene. The RIDGE-1 trial is assessing a one-time intravenous infusion of TN-401.

• The MyClimb natural history study for pediatric MYBPC3-associated HCM enrolled over 200 individuals across 29 clinical sites worldwide.
• In a study of MYBPC3-associated HCM patients, nearly 95% would be below the 1:80 neutralizing antibody (NAb) titer threshold for the AAV9 vector used in TN-201.
• For TN-401, a seroprevalence study in PKP2-associated ARVC indicated 84% of volunteers had NAb titers of less than 1:20 against AAV9.
• The PKP2 mutation is found in about 45% of people diagnosed with genetic ARVC in the U.S.

Therapies targeting rare diseases with high unmet medical need.

The gene therapies target rare, progressive, and life-threatening conditions where current treatment options are largely supportive rather than curative. The scale of the need is defined by the prevalence of the underlying genetic variants.

Condition	Pathogenic Variant Prevalence (Per 1,000,000)	Disease Status
ARVC (Pathogenic Variants)	Approximately 1,730 (1 in 578)	Fewer than 200,000 people affected in the U.S.
HCM (Pathogenic Variants)	Approximately 6,711 (1 in 149)	MYBPC3 variants account for 20 of 32 individuals in one HCM G+ cohort.

Small molecule (TN-301) for more prevalent heart failure (HFpEF).

TN-301 is a small molecule HDAC6 inhibitor being developed for Heart Failure with preserved Ejection Fraction (HFpEF), a more prevalent condition than the rare cardiomyopathies in the pipeline. This program diversifies the value proposition toward a larger patient population, though it is in earlier development stages as of late 2025. The company's Q3 2025 operating expenses totaled $20.9 million, demonstrating cost control while advancing this and other pipeline assets.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Customer Relationships

You're hiring before product-market fit, so the relationship with the scientific and investment communities is everything right now. Tenaya Therapeutics, Inc. (TNYA) focuses its customer relationship strategy on deep engagement with the specialized groups that validate its science and fund its pipeline.

High-touch, direct engagement with clinical investigators and key opinion leaders

Direct scientific exchange is paramount for a clinical-stage company. Tenaya Therapeutics, Inc. actively engages investigators through trial execution and scientific presentations. For instance, interim clinical data from the MYPEAK™-1 Phase 1b/2a trial of TN-201 for MYBPC3-associated Hypertrophic Cardiomyopathy (HCM) was presented at the American Heart Association Scientific Sessions in November 2025. This trial, a multi-center, open-label, dose-escalation study, had dosed a total of seven patients with TN-201 across two dose levels as of November 10, 2025. Also, the company hosted a Virtual Key Opinion Leader event on August 19, 2025, focused on 'Measuring Protein Expression in Cardiac Gene Therapy,' featuring experts like Michael Previs, Ph. That kind of focused event helps shape the perception of the science among the most influential clinicians.

The RIDGE™-1 trial for TN-401 also required close investigator management, with Cohort 2 dosing completed as of the Q3 2025 update. The company's visibility is amplified by presenting at major medical meetings; for example, late-breaker data on MyPEAK-1 was featured at the ACC Annual Scientific Session in March 2025.

Patient advocacy groups for support and natural history studies (MyClimb, RIDGE)

Tenaya Therapeutics, Inc. treats patients with rare genetic cardiomyopathies, making advocacy groups crucial partners for understanding disease burden and trial recruitment. The company runs two significant non-interventional natural history studies to characterize the patient populations they aim to treat. These studies provide the real-world context needed to design better trials and manage expectations with regulators and investors alike.

Here's a quick look at the scale of engagement in these patient-centric studies as of mid-to-late 2025:

Study Name	Indication	Patient Count (Approx.)	Site Count (Approx.)	Key Data Event (2025)
MyClimb™	Pediatric MYBPC3-associated HCM	More than 200	29 worldwide	Interim Data at ESC Congress
RIDGE™	Adult PKP2-associated ARVC	191 (as of April 2025 cut-off)	18 (U.S., UK, Europe)	Interim Data at HRS Meeting

The MyClimb study, initiated in 2021, characterized disease burden in patients under eighteen, where 93% of participants had the nonobstructive HCM phenotype, an area with no approved treatments. For the RIDGE study, data showed a high burden of disease, with more than 80% of participants experiencing $\ge \mathbf{500}$ premature ventricular contractions (PVCs) per day.

Investor relations and conference presentations to manage market expectations

Managing market expectations is a constant relationship Tenaya Therapeutics, Inc. maintains with its shareholders. The company provided a business update alongside its Third Quarter 2025 Financial Results on November 10, 2025. Financially, the Q3 2025 Net Loss narrowed to $20.3 million, an improvement from $25.6 million a year prior. Cash and cash equivalents stood at $56.3 million as of September 30, 2025, supported by net proceeds of approximately $48.8 million from a March 2025 follow-on offering. The management team held a conference call that day at 8:00 a.m. ET/5:00 a.m. PT to discuss the latest clinical data and financial standing.

Investor engagement is structured around key milestones. Beyond the earnings call, Tenaya Therapeutics, Inc. participated in several investor conferences:

• H.C. Wainwright Genetic Medicines Virtual Conference on October 14, 2025.
• Morgan Stanley 23rd Annual Global Healthcare Conference on September 9, 2025.

These events are used to communicate progress, such as the positive Data Safety Monitoring Board (DSMB) recommendations for both TN-201 and TN-401 programs, which allowed for dose escalation and expansion cohorts.

Regulatory body interactions (FDA, EMA) for expedited pathways (Fast Track, Orphan Drug)

Regulatory interactions define the path to market for orphan and rare disease therapies. Tenaya Therapeutics, Inc. has secured significant designations for its lead candidates, which streamlines development and provides market exclusivity incentives. The relationship with the FDA and EMA is characterized by these positive regulatory acknowledgments.

Key regulatory statuses as of late 2025 include:

• TN-401 (for PKP2-associated ARVC): Received Orphan Drug and Fast Track Designations from the FDA, plus orphan medicinal product designation from the European Commission.
• TN-201 (for MYBPC3-associated HCM): Received Fast Track, Orphan Drug and Rare Pediatric Drug Designations from the FDA, and orphan medicinal product designation from the European Commission.

However, these relationships also involve managing challenges; for example, the FDA placed the MyPEAK-1 trial for TN-201 on a clinical hold to standardize monitoring and immunosuppression protocol activities, a situation the company is actively working to resolve.

Finance: draft 13-week cash view by Friday.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Channels

You're looking at how Tenaya Therapeutics, Inc. gets its investigational therapies and corporate story out to the world-from clinical sites to the capital markets. This isn't about selling widgets; it's about reaching highly specialized physicians, researchers, and investors, so the channels are very targeted.

Specialized cardiac and genetic disease clinical centers worldwide

The core channel for clinical validation is the network of specialized centers running the trials. Tenaya Therapeutics, Inc. uses a global footprint to enroll patients in its gene therapy programs for genetic cardiomyopathies. This is how they generate the safety and efficacy data needed for regulatory approval.

For the MyPEAK-1 trial of TN-201 (for MYBPC3-associated HCM), the trial is multi-center, and the company has been advancing through dose cohorts. For the RIDGE-1 trial of TN-401 (for PKP2-associated ARVC), the structure is also global multicenter.

Here's a look at the scale of their natural history studies, which feed into trial design and site identification:

Study/Trial Program	Patient Population/Focus	Enrollment/Site Count (as of late 2025)	Geographic Reach Mentioned
MyClimb (Natural History)	Pediatric MYBPC3-associated HCM	More than 200 participants across 29 clinical sites worldwide	Worldwide
RIDGE (Natural History)	PKP2-associated ARVC	More than 100 participants across 18 clinical sites (as of Jan 2025)	U.S., UK, and EU
RIDGE-1 (Clinical Trial)	PKP2-associated ARVC (TN-401)	Cohort 1 enrollment completed (3 patients at 3E13 vg/kg dose)	Global multicenter

The company was planning to activate its first ex-U.S. RIDGE-1 clinical site in the first half of 2025.

Scientific and medical conferences (ACC, ASGCT) for data dissemination

Disseminating clinical and preclinical data is crucial for establishing scientific credibility. Tenaya Therapeutics, Inc. prioritizes high-impact medical meetings to present their findings directly to key opinion leaders and the broader scientific community. This is where they showcase the results from trials like MyPEAK-1 and RIDGE-1.

Key conference engagements in 2025 included:

• Presenting new TN-201 clinical data at the American Heart Association (AHA) 2025 Scientific Sessions in New Orleans, Louisiana (November 7-10, 2025).
• Presenting data at the American College of Cardiology's (ACC) Annual Scientific Session in Chicago, IL (March 29-31, 2025).
• Presenting a poster detailing the RIDGE-1 trial design at the European Society of Gene and Cell Therapy (ESGCT 2025) Annual Congress in October 2025.
• Presenting interim data from the MyClimb study at the European Society of Cardiology Congress in August 2025.

For investor awareness, the CEO participated in the Morgan Stanley 23rd Annual Global Healthcare Conference on September 9, 2025, and the H.C. Wainwright Genetic Medicines Virtual Conference on October 14, 2025.

Direct communication with patient advocacy organizations

Direct engagement with patient advocacy groups is a primary channel for gathering insights into the patient journey and driving awareness for genetic testing and precision medicines. Tenaya Therapeutics, Inc. states they partner with leading heart disease patient organizations to better understand community needs and inform drug development efforts.

The company's patient advocacy team connects with patients, caregivers, and advocates to drive awareness of the unmet needs in conditions like MYBPC3-associated HCM and PKP2-associated ARVC. However, for their investigational products, clinical trial participation remains the main pathway for access; for instance, the Expanded Access Policy for TN-201 is specifically limited to pediatric patients with confirmed MYBPC3-associated HCM.

Investor presentations and press releases for capital markets

Keeping the capital markets informed is vital for a clinical-stage company funding its pipeline. This channel relies heavily on formal financial reporting and strategic presentations to analysts and investors. You need to know the cash position to gauge operational runway.

Key financial metrics reported as of the end of Q3 2025 (September 30, 2025) include:

Financial Metric	Amount (Q3 2025 or As of Sept 30, 2025)
Cash, Cash Equivalents, and Investments	$56.3 million
Net Loss (Q3 2025)	$20.3 million
R&D Expenses (Q3 2025)	$15.4 million
G&A Expenses (Q3 2025)	$5.6 million
Total Operating Expenses (Q3 2025)	$20.9 million
Shares Outstanding (as of Nov 4, 2025)	166,505,885

Capital raising efforts are also part of this channel; Tenaya Therapeutics, Inc. completed a March 2025 follow-on offering that brought in net proceeds of approximately $48.8 million. Furthermore, an SVB loan agreement offers the right to draw $10.0 million through December 31, 2025. As of November 10, 2025, the Market Cap stood at $226.45 million, with Institutional Ownership at 22.21%. The company stated that existing resources, potential loan availability, and warrant exercises will fund operations for at least twelve months following the filing.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Customer Segments

You're looking at the core groups Tenaya Therapeutics, Inc. (TNYA) targets with its gene therapy pipeline, which is still pre-revenue as of late 2025. These segments are defined by the specific, rare genetic heart conditions they are trying to address, and the financial ecosystem that supports their development.

Patients with rare, monogenic cardiomyopathies (MYBPC3-HCM, PKP2-ARVC)

This is the primary, most critical segment for Tenaya Therapeutics, Inc. (TNYA) right now, as their lead candidates, TN-201 and TN-401, target specific genetic mutations. For MYBPC3-associated hypertrophic cardiomyopathy (HCM), the target population is estimated to be roughly 20% of the entire U.S. HCM patient base, which itself is estimated at 120,000 patients in the United States. The company's natural history study, MyClimb^TM, enrolled more than 200 pediatric patients diagnosed before age 18 across 29 clinical sites worldwide. Furthermore, a September 2025 seroprevalence study found that nearly 95% of these MYBPC3-associated HCM patients would be below the Nab titers of 1:80 threshold required for the TN-201 gene therapy trial. For PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), the prevalence is estimated at more than 70,000 people in the U.S. alone, with PKP2 mutations accounting for approximately 40 percent of the overall ARVC population. The RIDGE natural history study for this indication enrolled more than 175 patients across about 20 clinical sites in the U.S., UK, France, Germany, Italy and Sweden.

Here's a quick look at the patient pool sizes and study scopes:

Indication	Target Gene Therapy	Estimated U.S. Population Size	Natural History Study Enrollment (Approx.)
MYBPC3-HCM	TN-201	~24,000 (20% of 120,000)	Over 200 (MyClimb)
PKP2-ARVC	TN-401	Over 70,000	Over 175 (RIDGE)

Cardiologists and specialized heart failure treatment centers

These are the key prescribers and gatekeepers for clinical trial participation and future product adoption. They are the ones who manage the patients with these rare conditions. The clinical trial infrastructure itself shows the reach into these centers. For instance, the MyClimb study for TN-201 utilized 29 clinical sites worldwide, and the RIDGE study for TN-401 involved approximately 20 clinical sites across multiple countries. These centers are where the data on TN-201's improvements-like reductions in left ventricular posterior wall thickness (LVPWT) by 21-39% at week 52 in Cohort 1 patients-are generated and validated. It's all about building trust with the specialists who see the unmet need daily.

Payers and government health systems (future commercialization)

While Tenaya Therapeutics, Inc. (TNYA) is pre-revenue, payers-both private insurers and government systems like Medicare/Medicaid-become a critical segment upon regulatory approval. Their focus will be on the cost-effectiveness of a one-time curative therapy versus decades of standard-of-care management for chronic, progressive diseases. The company's Q3 2025 net loss was $20.3 million, reflecting the high cost of development that payers will eventually need to justify. The CIRM grant recognized the public health importance, providing $1.5 million of an $8.0 million award for the TN-401 program, which signals early validation from a public health funding body.

Investors and institutional funds focused on biotech and gene therapy

This segment provides the necessary capital to fund the expensive clinical development. As of September 30, 2025, Tenaya Therapeutics, Inc. (TNYA) held $56.3 million in cash and cash equivalents, with total assets at $105.0 million. The company completed a March 2025 follow-on offering that brought in net proceeds of approximately $48.8 million. Management stated that current resources, plus an available $10.0 million tranche from an SVB loan agreement (with an additional $20.0 million discretionary option), should fund operations for at least twelve months following the Q3 2025 filing. The stock trades on NASDAQ GS: TNYA, and the consensus target price as of late 2025 was $10.14 based on 7 estimates. The book value P/B ratio stood at 2.7, slightly below the peer average of 2.9. You've got to keep these folks happy with clinical milestones.

• Shares outstanding were 166,505,885 as of November 4, 2025.
• The nine-month net loss through Q3 2025 was $70.4 million.
• R&D expenses for Q3 2025 were $15.4 million.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Cost Structure

You're looking at the major cash outlays for Tenaya Therapeutics, Inc. as they push their gene therapies through critical clinical stages. For a company at this stage, the cost structure is almost entirely driven by research, development, and keeping the lights on while waiting for data readouts.

The Research & Development (R&D) expenses are the single largest component, reflecting the cost of advancing the TN-201 and TN-401 programs. For the first half of 2025 (H1 2025), the total R&D spend hit $38.5 million. This total is derived from the reported quarterly figures: $21.1 million in the first quarter (Q1 2025) and $17.4 million in the second quarter (Q2 2025).

These R&D costs inherently cover the heavy lifting of the clinical programs. Specifically, the costs associated with the clinical trial costs for TN-201 (for MYBPC3-associated HCM) and TN-401 (for PKP2-associated ARVC) are embedded here. The progression itself is a cost driver; for instance, the Data Safety Monitoring Boards (DSMBs) endorsed dose escalation and expansion for both programs following safety reviews.

General and administrative (G&A) expenses represent the overhead required to run the business. For Q2 2025, the G&A expense was $6.7 million. This figure was down from $8.2 million in the same period of 2024, partly due to a decrease in stock-based compensation.

The structure of these operating expenses is detailed below, showing the quarterly trend for the first half of 2025 and the subsequent third quarter (Q3 2025) data, which shows continued cost containment efforts:

Metric	Q1 2025 (Approx.)	Q2 2025	Q3 2025
Research & Development Expense	$21.1 million	$17.4 million	$15.4 million
General & Administrative Expense	Not explicitly stated	$6.7 million	$5.6 million
Total Operating Expenses (R&D + G&A)	Not explicitly stated	$24.1 million	$21.0 million

The personnel costs for specialized scientific and clinical teams are a major component within the R&D spend. While a specific dollar amount for personnel alone isn't broken out separately from R&D, we know that non-cash stock-based compensation within R&D for Q2 2025 was $1.9 million. This gives you a sense of the non-cash element of compensation for those specialized teams.

The costs related to manufacturing and process development for AAV vectors are also captured within R&D. The advancement of the trials, such as dosing patients at 6E13 vg/kg for TN-201 and dosing the second cohort for TN-401, necessitates ongoing vector production and quality control, which are significant fixed and variable costs for a gene therapy company.

You can see the operating spend is high, but management is actively managing it. Here's the quick math: the sequential drop in operating expenses from Q2 2025 ($24.1 million) to Q3 2025 ($21.0 million) helped extend the cash runway. The cash position at the end of Q2 2025 was $71.7 million, projected to last into the second half of 2026.

Key cost drivers embedded within the R&D line item include:

• Costs for AAV vector production and characterization.
• Site initiation and patient enrollment fees for clinical sites.
• Biomarker analysis, such as biopsy sample processing for TN-401 DNA/mRNA.
• Salaries and benefits for research scientists and clinical operations staff.

Finance: draft 13-week cash view by Friday.

Tenaya Therapeutics, Inc. (TNYA) - Canvas Business Model: Revenue Streams

As of late 2025, Tenaya Therapeutics, Inc. is firmly in the clinical-stage, meaning its revenue streams are entirely composed of non-operating, financing-related inflows, as the company has not yet reached commercialization for any of its product candidates.

Non-Dilutive Grant Funding

A key non-dilutive source of capital supporting clinical development is grant funding. Tenaya Therapeutics, Inc. secured a significant award from the California Institute for Regenerative Medicine (CIRM) in early 2025. This was an $8.0 million CLIN2 grant announced on February 3, 2025.

• The $8.0 million grant proceeds are earmarked to help fund clinical trial costs for the ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy.
• One record indicates a CIRM award value of $9,017,000 to Tenaya Therapeutics, Inc..

Equity Financing Proceeds

The most substantial recent inflow has been from equity financing, which provides the necessary runway to advance the lead gene therapy candidates, TN-201 and TN-401. In March 2025, Tenaya Therapeutics, Inc. executed an underwritten public offering.

Here's a look at the key figures from that financing event:

Metric	Value
Gross Proceeds (Approximate)	$52.5 million
Net Proceeds (Approximate)	$48.8 million
Units Sold	75 million
Price Per Unit	$0.70
Cash Position as of March 31, 2025	$88.2 million

The net proceeds of approximately $48.8 million from the March 2025 public offering, combined with existing cash, were expected to support planned company operations into the second half of 2026. The offering structure included warrants, which represent potential future equity dilution but are not current revenue.

Future Potential Revenue from Strategic Licensing or Collaboration Agreements

Tenaya Therapeutics, Inc. is pursuing a strategy that involves developing its portfolio of genetic medicines internally. While the current financial statements reflect no realized revenue from this area, the development of clinical candidates like TN-201 and TN-401 creates the potential for future revenue streams through strategic out-licensing or collaboration agreements with larger pharmaceutical partners upon achieving key clinical milestones.

No Product Revenue

Tenaya Therapeutics, Inc. is explicitly a pre-commercial stage company. Consequently, there is no revenue generated from the sale of products. Analyst expectations for the quarter ending September 30, 2025, reflect this reality, projecting no change in quarterly revenue.

• The company is focused on advancing its lead candidates through clinical trials, such as TN-201 in the MyPEAK-1 trial and TN-401 in the RIDGE-1 trial.
• The cash runway extension into the second half of 2026 is based on current cash levels and the assumption of continued operational expenditure without product sales.