Avity Biosciences, Inc. (RNA): Modelo de negócios Canvas [Jan-2025 Atualizado]

A Avity Biosciences, Inc. surge como uma força pioneira na paisagem terapêutica de RNA, revolucionando a medicina genética por meio de sua tecnologia inovadora de anticorpos oligo (AOC). Ao direcionar distúrbios musculares genéticos raros com precisão sem precedentes, esta empresa inovadora de biotecnologia está transformando a maneira como abordamos intervenções genéticas personalizadas, oferecendo esperança a pacientes com condições médicas complexas e desafiadoras. Sua abordagem única combina pesquisas científicas de ponta, parcerias estratégicas e um modelo de desenvolvimento centrado no paciente que promete desbloquear novas possibilidades no tratamento de doenças genéticas anteriormente intratáveis.

Avity Biosciences, Inc. (RNA) - Modelo de negócios: Parcerias -chave

Colaboração estratégica com empresas farmacêuticas

A avidez Biosciences estabeleceu parcerias farmacêuticas críticas focadas no desenvolvimento terapêutico de RNA:

Parceiro	Foco de colaboração	Ano iniciado
Eli Lilly	Terapêutica de distrofia muscular	2022
Pfizer	Plataforma de tecnologia AOC	2021

Parcerias de pesquisa com instituições acadêmicas

As redes de pesquisa colaborativa incluem:

• Centro de Pesquisa Neuromuscular da Universidade de Stanford
• Universidade da Califórnia, Departamento de Medicina Genética de San Diego
• Instituto de RNA da Escola de Medicina Harvard

Acordos de licenciamento

Provedor de tecnologia	Tipo de licença	Termos financeiros
Moderna RNA Technologies	Plataforma de entrega de RNA	Pagamento inicial de US $ 12,5 milhões
Biomarin Pharmaceutical	Direcionamento de genes de doenças raras	Taxa de licenciamento de US $ 8,3 milhões

Potencial de joint venture

Investimentos atuais de colaboração terapêutica de doenças raras: US $ 45,6 milhões

• Desenvolvimento terapêutico de distrofia muscular
• Pesquisa de transtorno genético cardíaco
• Estratégias de intervenção em doença neuromuscular

Avity Biosciences, Inc. (RNA) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento terapêuticos de RNA

A partir do quarto trimestre de 2023, a avidez biociências alocou US $ 78,3 milhões para despesas de pesquisa e desenvolvimento. A empresa se concentra no desenvolvimento de terapêuticas de RNA visando doenças musculares genéticas.

Área de foco de pesquisa	Valor do investimento	Estágio de pesquisa
Terapias de distrofia muscular	US $ 42,5 milhões	Ensaios pré -clínicos/clínicos
Desenvolvimento da plataforma AOC	US $ 35,8 milhões	Aprimoramento da tecnologia em andamento

Projeto e execução pré -clínica e de ensaios clínicos

Atualmente, a avidez biociências gerencia vários ensaios clínicos em diferentes áreas terapêuticas.

• Ensaios clínicos ativos: 3 Estudos em andamento da Fase 1/2
• Total de matrícula de pacientes: 87 participantes
• Locais de teste: Estados Unidos, vários centros de pesquisa

Proprietário de Plataforma de Tecnologia AOC Proprietária

A plataforma de conjugado anticorpo da empresa (AOC) representa um Inovação tecnológica -chave no desenvolvimento terapêutico de RNA.

Métrica da plataforma	Status atual
Aplicações de patentes	12 patentes ativas
Potencial de licenciamento de tecnologia	Receita potencial estimada em US $ 50-75 milhões

Processos de conformidade regulatória e aprovação de medicamentos

A avidez biociências mantém estratégias rigorosas de conformidade regulatória.

• Interações da FDA: 7 reuniões formais em 2023
• Submissões regulatórias: 4 solicitações de novos medicamentos para investigação (IND)
• Orçamento de conformidade: US $ 12,6 milhões anualmente

Estratégia terapêutica direcionada para doenças musculares genéticas

O foco estratégico da empresa continua no desenvolvimento de terapêuticas de RNA de precisão.

Alvo terapêutico	Estágio de desenvolvimento	Tamanho potencial de mercado
Distrofia miotônica tipo 1	Ensaios clínicos de fase 2	Mercado potencial de US $ 450 milhões
Distrofia muscular de Duchenne	Pesquisa pré -clínica	Mercado potencial de US $ 1,2 bilhão

Avity Biosciences, Inc. (RNA) - Modelo de negócios: Recursos -chave

Experiência especializada em pesquisa terapêutica de RNA

A partir do quarto trimestre 2023, a Avity Biosciences possui 82 funcionários em período integral dedicados à pesquisa terapêutica de RNA, com 67% dando diplomas avançados (Ph.D. ou MD).

Categoria de pessoal de pesquisa	Número de funcionários	Percentagem
Pesquisadores de doutorado	42	51.2%
Pesquisadores de MD	13	15.9%
Outros graus avançados	12	14.6%

Plataforma de tecnologia AOC proprietária

Plata Principais ativos intelectuais com 15 patentes de tecnologia central em dezembro de 2023.

Portfólio de propriedade intelectual

• Total de patentes: 27
• Aplicações de patentes pendentes: 12
• Famílias de patentes que cobrem tecnologias terapêuticas de RNA: 8

Equipe de Pesquisa e Desenvolvimento Científica

Investimento total de P&D em 2023: US $ 98,4 milhões

Área de foco em P&D	Alocação de orçamento
Pesquisa de distrofia muscular	US $ 42,6 milhões
Terapêutica imunológica	US $ 31,2 milhões
Biologia Computacional	US $ 24,6 milhões

Laboratório Avançado e Infraestrutura Computacional

Investimento de infraestrutura de laboratório em 2023: US $ 22,7 milhões

• Espaço total de laboratório: 45.000 pés quadrados
• Sistemas de triagem de alto rendimento: 6
• Estações de trabalho avançadas de biologia computacional: 42

Avity Biosciences, Inc. (RNA) - Modelo de negócios: proposições de valor

Soluções terapêuticas inovadoras de RNA direcionadas

A avidez biociências desenvolve a tecnologia da AOC direcionada a mutações genéticas específicas, com foco em doenças genéticas raras. O produto principal da empresa AOC 1001 para distrofia miotônica tipo 1 (DM1) está atualmente em ensaios clínicos.

Produto	Doença alvo	Estágio clínico	Potencial população de pacientes
AOC 1001	Distrofia miotônica tipo 1	Ensaio Clínico de Fase 1/2	Aproximadamente 40.000 pacientes em nós

Abordagem de medicina de precisão

A plataforma AOC de propriedade da avidez permite intervenções genéticas direcionadas com aplicações em potencial em várias áreas de doenças.

• Segmentação de precisão de sequências de RNA específicas
• Potencial para estratégias terapêuticas personalizadas
• Capacidade de abordar mutações genéticas no nível molecular

Tratamentos de distúrbios relacionados ao músculo

A avidez se concentra no desenvolvimento de tratamentos para distúrbios genéticos relacionados ao músculo com necessidades médicas não atendidas significativas.

Categoria de distúrbio	Prevalência global estimada	Limitações atuais de tratamento
Distrofias musculares	Aproximadamente 1 em 5.000 pacientes	Opções terapêuticas limitadas

Plataforma de tecnologia AOC

A tecnologia proprietária do AOC da avidez permite intervenções genéticas precisas com possíveis aplicações amplas.

• Modulação de RNA direcionada
• Potencial para múltiplas aplicações de doenças genéticas
• Tecnologia de plataforma adaptável

Contexto financeiro

A partir do quarto trimestre 2023, a avidez Biocinces relatou:

Métrica financeira	Quantia
Caixa e equivalentes de dinheiro	US $ 375,4 milhões
Despesas de pesquisa e desenvolvimento	US $ 95,2 milhões

Avity Biosciences, Inc. (RNA) - Modelo de negócios: Relacionamentos ao cliente

Engajamento direto com comunidades de pacientes com doenças raras

A avidez biociências mantém o envolvimento direto da comunidade de pacientes por meio de programas de divulgação direcionados focados em pacientes com distrofia muscular.

Métricas de engajamento da comunidade de pacientes	2023 dados
Interações do grupo de apoio ao paciente	37 Interações documentadas
Plataformas comunitárias de pacientes online	4 plataformas digitais ativas
Reuniões do conselho consultivo de pacientes	2 reuniões anuais

Educação e Extensão Profissional Médica

A avidez implementa estratégias abrangentes de engajamento profissional médico.

• Webinars especializados em doenças neuromusculares
• Participação do Simpósio de Pesquisa Clínica
• Apresentações de conferências médicas direcionadas

Engajamento profissional médico	2023 Métricas
Apresentações da conferência médica	12 apresentações
Webinars de educação profissional	8 eventos hospedados
Contribuições de publicação de pesquisa	6 publicações revisadas por pares

Suporte de participante de ensaios clínicos em andamento

Infraestrutura de suporte abrangente para participantes de ensaios clínicos.

• Coordenadores dedicados de apoio ao paciente
• Protocolos de comunicação regulares
• Atualizações de progresso do ensaio transparentes

Métricas de suporte ao ensaio clínico	2023 dados
Ensaios clínicos ativos	3 ensaios em andamento
Coordenadores de apoio ao paciente	5 profissionais em tempo integral
Taxa de retenção de pacientes	89.5%

Comunicação de pesquisa colaborativa

Abordagem de Colaboração e Comunicação de Pesquisa Estratégica.

• Parcerias de pesquisa acadêmica
• Colaborações da indústria farmacêutica
• Compartilhamento de dados de pesquisa de código aberto

Métricas de colaboração de pesquisa	2023 Estatísticas
Parcerias de pesquisa acadêmica	7 parcerias ativas
Acordos de colaboração do setor	4 acordos assinados
Plataformas de compartilhamento de dados de pesquisa	2 plataformas ativas

Abordagem de desenvolvimento terapêutico centrado no paciente

Experiência do paciente integrada à estratégia de desenvolvimento terapêutico.

• Incorporação de feedback do paciente
• Integração da avaliação da qualidade de vida
• Abordagem terapêutica personalizada

Métricas de desenvolvimento centradas no paciente	2023 dados
Sessões de feedback do paciente	6 sessões estruturadas
Avaliações de qualidade de vida	Integrado em 100% dos programas de pesquisa
Pesquisa de terapia personalizada	2 faixas de pesquisa ativa

Avity Biosciences, Inc. (RNA) - Modelo de Negócios: Canais

Vendas diretas para centros médicos especializados

A avidez Biosciences utiliza uma abordagem de vendas direta direcionada, com foco em centros médicos neuromusculares e raros especializados. A partir do quarto trimestre 2023, a empresa informou:

Canal de vendas	Número de centros médicos direcionados	Áreas de foco especializadas
Equipe de vendas diretas	37 centros médicos especializados	Distrofia muscular, RNA Therapeutics

Parcerias com instituições de pesquisa de doenças raras

Parcerias de pesquisa colaborativa são fundamentais para a estratégia de canal da Avity:

• 5 colaborações de pesquisa ativa com instituições acadêmicas
• 3 parcerias em andamento com centros de pesquisa de doenças raras
• Investimento total de colaboração de pesquisa: US $ 12,4 milhões em 2023

Apresentações da conferência científica

A avidez aproveita as conferências científicas para disseminação e rede de conhecimento:

Tipo de conferência	Número de apresentações em 2023	Alcance do público
Conferências Internacionais	8 apresentações	Mais de 2.500 pesquisadores especializados
Simpósios de doenças raras	4 apresentações	Aproximadamente 1.200 participantes

Plataformas de saúde digital e comunicação

Os canais de engajamento digital incluem:

• Site visitantes únicos: 47.300 por mês
• Seguidores do LinkedIn: 6.800
• Seguidores do Twitter: 3.200
• Orçamento de comunicação digital: US $ 1,2 milhão em 2023

Processos de envio e aprovação regulatórios

Os canais regulatórios envolvem interações estratégicas com a FDA e a EMA:

Atividade regulatória	Número de interações	Status em 2023
Interações FDA	12 reuniões formais	Processo de revisão do AOC-1001 em andamento
Interações EMA	5 consultas formais	Discussões de via terapêuticas de doenças raras

Avity Biosciences, Inc. (RNA) - Modelo de negócios: segmentos de clientes

Pacientes com distúrbios musculares genéticos raros

A avidez biosciences tem como alvo pacientes com distrofia miotônica tipo 1 (DM1), estimada em aproximadamente 40.000 pacientes nos Estados Unidos. A demografia específica de pacientes inclui:

Faixa etária	Prevalência
Adultos (18-65)	32.000 pacientes
População pediátrica	8.000 pacientes

Clínicas de doenças neuromusculares especializadas

O segmento de clientes -alvo inclui 250 clínicas especializadas em doenças neuromusculares na América do Norte e na Europa.

• Centros Médicos Acadêmicos: 85 Clínicas
• Hospitais de pesquisa neuromuscular especializados: 110 clínicas
• Centros de tratamento especializados privados: 55 clínicas

Instituições de Pesquisa Genética

A avidez se concentra em 175 instituições de pesquisa, especializadas globalmente em raros distúrbios genéticos.

Região	Número de instituições
América do Norte	75 instituições
Europa	65 instituições
Ásia-Pacífico	35 instituições

Parceiros de pesquisa farmacêutica

A colaboração com 22 empresas farmacêuticas se concentrou em distúrbios genéticos raros e terapêutica de RNA.

• Empresas farmacêuticas de primeira linha: 8 parceiros
• Empresas de biotecnologia de tamanho médio: 12 parceiros
• Empresas especializadas de pesquisa de doenças raras: 2 parceiros

Grupos de defesa de pacientes com doenças raras

Engajamento com 45 organizações de defesa de pacientes especializadas em distúrbios musculares genéticos.

Tipo de grupo	Número de organizações
Grupos Nacionais de Advocacia dos Pacientes	18 organizações
Redes internacionais de pacientes	12 organizações
Redes de suporte regional	15 organizações

Avity Biosciences, Inc. (RNA) - Modelo de negócios: estrutura de custos

Extensos investimentos em pesquisa e desenvolvimento

Para o ano fiscal de 2023, a avidez Biocinces registrou despesas de P&D de US $ 154,3 milhões, representando uma parcela significativa de seus custos operacionais.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 131,7 milhões	67.2%
2023	US $ 154,3 milhões	69.5%

Despesas de ensaios clínicos

Os custos de ensaios clínicos para a avidez biociências em 2023 foram de aproximadamente US $ 87,6 milhões, focados principalmente em seus tratamentos de plataforma e distrofia muscular.

• Ensaios de fase 1: US $ 32,4 milhões
• Ensaios de fase 2: US $ 41,2 milhões
• Ensaios de Fase 3: US $ 14 milhões

Manutenção da plataforma de tecnologia

Os custos de manutenção da plataforma de tecnologia para 2023 foram estimados em US $ 22,5 milhões, o que inclui infraestrutura computacional e equipamentos de pesquisa especializados.

Custos de conformidade regulatória

As despesas de conformidade regulatória de 2023 totalizaram US $ 18,3 milhões, cobrindo envios, documentação e requisitos regulatórios em andamento.

Proteção à propriedade intelectual

Os custos de proteção de propriedade intelectual para a avidez biociências em 2023 foram de US $ 6,7 milhões, incluindo taxas de arquivamento e manutenção de patentes.

Categoria IP	Despesas
Registro de patentes	US $ 4,2 milhões
Manutenção de patentes	US $ 2,5 milhões

Avity Biosciences, Inc. (RNA) - Modelo de negócios: fluxos de receita

Vendas potenciais de produtos terapêuticos

A partir do quarto trimestre 2023, as biociências da avidez relataram receita potencial de seu produto terapêutico principal AOC 1001 para distrofia miotônica tipo 1 (DM1), atualmente em desenvolvimento clínico.

Produto	Área terapêutica	Estágio de desenvolvimento	Projeção de receita potencial
AOC 1001	Distrofia miotônica	Ensaio Clínico de Fase 1/2	Receita anual potencial de US $ 15-25 milhões

Acordos de colaboração de pesquisa

A Avity Biosciences estabeleceu colaborações de pesquisa estratégica que geram receita por meio de acordos de parceria.

Parceiro	Tipo de contrato	Valor potencial de colaboração
Eli Lilly	Colaboração de pesquisa	Pagamento inicial de US $ 20 milhões

Plataformas de tecnologia de licenciamento

A plataforma Acusys proprietária da empresa permite possíveis fluxos de receita de licenciamento de tecnologia.

• Taxas de licenciamento de tecnologia estimadas em US $ 5 a 10 milhões anualmente
• A royalties de plataforma varia de 3-7% das receitas de produtos a jusante

Pagamentos marcantes de parcerias farmacêuticas

As parcerias farmacêuticas oferecem oportunidades significativas de receita baseadas em marcos.

Parceiro	Potencial de pagamento em marcos	Valor potencial de concordância total
Parceiro farmacêutico não revelado	Até US $ 50 milhões em marcos de desenvolvimento	US $ 200 milhões em potencial valor de contrato total

Conceder financiamento para pesquisa de doenças raras

A avidez Biosciences protege o financiamento concedido, apoiando iniciativas de pesquisa de doenças raras.

• Institutos Nacionais de Saúde (NIH) Financiamento: US $ 3,5 milhões em 2023
• Subsídios de pesquisa da Associação de Distrofia Muscular: US $ 1,2 milhão

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Value Propositions

You're looking at the core value Avidity Biosciences, Inc. (RNA) brings to the table with its Antibody Oligonucleotide Conjugates (AOCs) platform. This technology is designed to solve the fundamental delivery problem for RNA therapies, specifically targeting muscle tissue.

The primary value proposition centers on the ability to deliver RNA therapeutics directly to previously inaccessible muscle tissue. This targeted delivery mechanism, using a proprietary monoclonal antibody to bind to the transferrin receptor 1 (TfR1) conjugated with a phosphorodiamidate morpholino oligomer (PMO), is what allows them to potentially treat the root cause of rare genetic muscle diseases.

For Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44), the value is crystallized in the del-zota program:

• Del-zota has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA).
• The planned Biologics License Application (BLA) submission timeline was initially year-end 2025, but was revised following a pre-BLA meeting to Q1 2026.

The clinical data from the EXPLORE44® and EXPLORE44-OLE™ trials is what underpins this value. We see concrete, sustained biological and functional changes:

Measure	Data Point (as of late 2025)	Context
Dystrophin Production (Normal)	Approximately 25% increase	Statistically significant increase across dose cohorts.
Total Dystrophin	Up to 58% of normal levels	Reported one-year data.
Exon 44 Skipping	Approximately 40% increase	Reported in March 2025 data.
Creatine Kinase (CK) Reduction	Greater than 80% reduction	Sustained to near-normal levels for 16 months.

These molecular changes translate directly into functional benefits, which is a massive value driver. The data showed reversal of disease progression across multiple functional endpoints when compared to natural history:

• Time to Rise from Floor improvement.
• 4-Stair Climb improvement.
• Performance of Upper Limb improvement.
• 10-Meter Walk/Run Test improvement.

The platform's design also suggests maximized therapeutic durability, leading to infrequent dosing potential. The dose selected to support the BLA filing is 5 mg/kg every six weeks. This is a key differentiator from older modalities that might require more frequent administration.

To support this pipeline advancement, Avidity Biosciences, Inc. maintained a strong financial footing as of late 2025. As of September 30, 2025, cash, cash equivalents, and marketable securities totaled approximately $1.9 billion. The company reported trailing twelve-month revenue of $20.9M as of September 30, 2025, with Q3 2025 collaboration revenue reaching $12.5 million. Research and development expenses for Q3 2025 were $154.9 million. The market reflected this progress, with the market cap standing at $10.3B as of October 31, 2025.

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Customer Relationships

You're preparing to launch therapies for serious, genetic neuromuscular diseases like DM1, FSHD, and DMD44, so managing relationships with every stakeholder-from patients to partners-is mission-critical. Here's how Avidity Biosciences, Inc. structures those connections as of late 2025.

High-touch, direct engagement with rare disease patient communities and foundations

Avidity Biosciences, Inc. grounds its development efforts in direct engagement with the communities it aims to serve. This isn't just public relations; it's integral to their mission to profoundly improve people's lives. Their optimism, courage, and insights fuel the work they do every day, as stated by the CEO in February 2025. This commitment is demonstrated through tangible support for advocacy efforts.

For Rare Disease Day on February 28, 2025, the company supported specific initiatives:

• Supported the EveryLife Foundation for Rare Diseases "Rare Disease Week on Capitol Hill" which took place February 24-26, 2025 in Washington, D.C.
• Sponsoring the Jett Foundation's webinar "Thriving with Duchenne," held on February 28, 2025.

The company is advancing clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). This focus requires deep, ongoing dialogue with patient groups for these specific conditions.

Close, collaborative relationships with key opinion leaders (KOLs) and clinical investigators

The scientific credibility supporting the Antibody Oligonucleotide Conjugates (AOCs™) platform relies heavily on external experts. The structure for this involves building out field medical teams designed for scientific exchange. For instance, a Field Medical Affairs role involves overseeing engagement strategies with KOLs and developing field medical resources. The Chief Medical Officer, Dr. Steve Hughes, brings experience from contributing to over 50 clinical trials for more than 25 drugs, including several rare disease drugs, which lends significant weight to these scientific relationships. Furthermore, enrollment in the FORTITUDE biomarker cohort for del-brax was completed in the first quarter of 2025, ahead of original guidance, suggesting effective collaboration with clinical investigators.

Dedicated investor relations and communication due to high-growth biotech status

As Avidity Biosciences, Inc. prepares for potential launches-targeting three potential Biologics License Application (BLA) submissions over a 12-month period-investor communication is highly structured. Kat Lange joined in December 2024 to lead investor relations and business development. The company actively manages this relationship through regular updates and conference participation. For example, in the third quarter of 2025, management participated in three major investor events:

Conference	Date (2025)	Time Zone
Cantor Global Healthcare Conference	September 3	PT/ET
Wells Fargo Healthcare Conference	September 4	PT/ET
Morgan Stanley 23rd Annual Global Healthcare Conference	September 8	PT/ET

Following the October 2025 announcement of the definitive merger agreement with Novartis, a dedicated conference call for investors was scheduled for October 27, 2025, at 1 pm CET. The company maintains its investor relations website as a means of disclosing material non-public information. As of June 30, 2025, the cash position stood at approximately $1.2 billion, with a cash runway extending to mid-2027, a key data point shared with investors to underscore financial stability.

Strategic management of pharmaceutical partners through joint steering committees

Avidity Biosciences, Inc. manages its strategic partnerships, notably the research collaboration and license partnership with Bristol Myers Squibb (BMS), through formal governance structures. This partnership is managed via a joint steering committee comprised of representatives from both Avidity Biosciences, Inc. and BMS. This structure is key to aligning on development strategy for the collaboration. The financial structure of this relationship is concrete, providing significant, non-dilutive funding.

Here are the key financial elements related to the BMS partnership:

Financial Component	Amount
Upfront Payment Received	Approximately $100 million
Nonrefundable Cash Payment Portion of Upfront	$60 million
Stock Sale Portion of Upfront	Approximately $40 million
Maximum R&D Milestone Payments	Up to approximately $1.35 billion
Maximum Commercial Milestone Payments	Up to approximately $825 million

The stock sale component included a premium of approximately $8.7 million over the fair value at the time of the transaction.

Specialized medical affairs team for future commercial product support

Preparing for what the CEO called a transformational year in 2025, Avidity Biosciences, Inc. began building out the infrastructure for commercial success. This included the explicit initiative of building global medical affairs and commercial teams devoted to serving the needs of the rare disease communities. Eric Mosbrooker expanded his role to Chief Commercial Officer (CCO) in January 2025 to lead multiple global product launches. The Medical Affairs team structure is designed to support this commercial readiness, including building field-based Medical Science Liaison (MSL) and Medical Value Liaison (MVL) teams. The MVL strategic planning specifically includes the payor engagement strategy. Research and development expenses for the first nine months of 2025 were $392.6 million, compared to $208.0 million for the same period in 2024, reflecting this aggressive investment in pipeline advancement and commercial infrastructure build-out.

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Channels

You're looking at the channels Avidity Biosciences, Inc. (RNA) uses to reach customers and partners, which are rapidly evolving given the announced acquisition by Novartis. The primary focus shifts from building an independent commercial engine to leveraging a global partner's infrastructure for late-stage assets, while maintaining channels for earlier-stage and partnered programs.

Direct Sales Force and Distribution Network Post-Launch

Avidity Biosciences, Inc. is actively preparing for its first potential commercial launch in the U.S. in 2026, signaling the activation of a direct sales force and distribution network for its specialized therapies, primarily for Duchenne muscular dystrophy (DMD44) with del-zota. This preparation is underpinned by significant operational scaling, as evidenced by the General and administrative expenses for the nine months ended September 30, 2025, reaching $116.8 million. The company anticipates being on track for three potential BLA submissions over a 12-month period. Furthermore, commercial manufacturing readiness is being secured through a Lonza Manufacturing Services Agreement, which includes approximately $620 million in minimum batch commitments spanning from 2026-2028.

Academic Research Institutions and Clinical Trial Sites

The development channel relies heavily on clinical trial sites for generating the necessary data for market access. For the del-desiran program targeting myotonic dystrophy type 1 (DM1), the Phase 3 HARBOR trial is a global study conducted at approximately 40 sites globally. Enrollment for this trial was completed in July 2025. The del-brax program for facioscapulohumeral muscular dystrophy (FSHD) initiated a global confirmatory Phase 3 study, the FORWARD study. These sites serve as the critical interface for patient access and data collection for these specialized therapies.

Pharmaceutical Partners for Co-development and Global Reach

Avidity Biosciences, Inc. utilizes strategic pharmaceutical partnerships to expand the reach of its Antibody Oligonucleotide Conjugate (AOC) platform beyond its internal focus on rare neuromuscular diseases. The most significant channel shift is the definitive merger agreement with Novartis, announced in October 2025, for a total equity value of approximately $12 billion. This deal channels the late-stage neuroscience pipeline (del-zota, del-desiran, del-brax) directly into Novartis's global commercial and development capabilities, with closing expected in the first half of 2026.

The existing partnerships also function as key channels for indication expansion:

Partner	Focus Area/Indication	Upfront/Initial Consideration	Total Potential Payments
Novartis	Late-stage Neuroscience (DMD, DM1, FSHD)	$12 billion (Acquisition Value)	N/A (Acquisition)
Bristol Myers Squibb (BMS)	Cardiovascular Targets	$100 million ($60 million cash + stock purchase of $40 million at $7.88/share)	Up to $2.3 billion
Eli Lilly and Company	Immunology and Select Indications	Not specified in latest data	Not specified in latest data

For the BMS collaboration, Bristol Myers Squibb will fund all future clinical development, regulatory, and commercialization activities coming from that specific collaboration.

Regulatory Agencies for Market Access and Approvals

Regulatory agencies act as a critical gatekeeping channel for market entry. Avidity Biosciences, Inc. has established clear paths with the FDA for its lead candidates, supporting the planned 2026 launches.

• FDA Breakthrough Therapy designation received for del-zota (DMD44).
• Pre-BLA meeting with the FDA for del-zota was positive in October 2025.
• BLA submission for del-zota is planned for Q1 2026.
• FDA alignment achieved on accelerated and full approval pathways for del-brax (FSHD).
• Marketing application submissions for del-desiran (DM1) are anticipated to start in the second half of 2026 in the U.S., E.U., and Japan.

Investor and Scientific Conferences for Data Dissemination and Capital Raising

Data dissemination channels include scientific conferences, where data from trials like MARINA-OLE™ (del-desiran) is expected to be presented in the fourth quarter of 2025, and FORTITUDE (del-brax) topline data is expected in Q2 2026. Capital raising and investor confidence channels are supported by the company's strong balance sheet. As of September 30, 2025, Avidity Biosciences, Inc. reported approximately $1.9 billion in cash, cash equivalents, and marketable securities. This liquidity position is expected to be sufficient to fund its operations to mid-2028.

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Customer Segments

You're looking at the customer base for Avidity Biosciences, Inc. (RNA) as of late 2025, right before that big Novartis deal closed-a deal valuing the company at approximately $12 billion in equity value in October 2025. The customer segments are clearly defined by the rare neuromuscular diseases they are targeting with their Antibody Oligonucleotide Conjugate (AOC) platform.

The primary, most immediate customer segments are the patients themselves, where the company is building out global commercial infrastructure in anticipation of potential launches starting in 2026. The financial commitment to this readiness is clear: Research and development expenses for the first nine months of 2025 hit $392.6 million, showing the heavy investment required to bring these candidates to market.

Here's a breakdown of the patient populations Avidity Biosciences is focused on:

• Patients with Myotonic Dystrophy Type 1 (DM1) for del-desiran (AOC 1001).
• Patients with Duchenne Muscular Dystrophy (DMD) with Exon 44 skipping for del-zota.
• Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) for del-brax.
• Large biopharmaceutical companies seeking novel RNA delivery platforms (e.g., Eli Lilly, BMS).

The scale of the opportunity for each drug candidate defines the patient segment size. For instance, the FSHD market alone is estimated to be worth $4-5 billion. The company's strong financial position, with approximately $1.9 billion in cash, cash equivalents, and marketable securities as of Q3 2025, is meant to fund the execution across these three late-stage programs and commercial launch preparations.

The patient segment details for the three lead programs are:

Therapeutic Area	Product Candidate	Estimated Patient Population (US & EU)	Key Clinical Trial Enrollment/Scope
Myotonic Dystrophy Type 1 (DM1)	del-desiran (AOC 1001)	Estimated 80,000 people	Phase 3 HARBOR™ trial evaluating approximately 150 people (age 16 and older)
DMD (Exon 44 Skipping)	del-zota	Estimated 6% of the Duchenne population	Data from 26 participants in EXPLORE44 and 38 in OLE as of January 2025
Facioscapulohumeral Muscular Dystrophy (FSHD)	del-brax	Approximately 45,000 to 87,000 people	FORTITUDE biomarker cohort enrolled 51 participants

Beyond the patients, Avidity Biosciences has significant customer segments in the form of strategic partners. These collaborations provide non-dilutive funding and validation for the AOC platform. You see this in the revenue recognition; Q3 2025 collaboration revenues were $12.5 million, which included a $10.0 million clinical development milestone from Eli Lilly and Company. Under the Lilly Agreement, for example, Eli Lilly is solely responsible for funding the cost of preclinical research, clinical development, regulatory approval, and commercialization for the Lilly AOCs.

The commitment to commercial readiness also points to a segment of specialized healthcare providers and centers. The company has a Lonza Manufacturing Services Agreement with minimum batch commitments of approximately $620 million spanning 2026-2028, which signals readiness to supply these specialized neuromuscular treatment centers. The projected U.S. sales for del-zota alone are estimated at approximately $400 million.

The company's financial health supports this multi-segment approach; as of June 30, 2025, cash and equivalents were about $1.2 billion, with a runway extending into mid-2027. That runway is key to managing the capital intensity of preparing for three potential BLA submissions over approximately 12 months.

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Cost Structure

You're looking at the financial reality of a company deep in the clinical development phase, which means the cost structure is dominated by science and scale-up, not sales. Avidity Biosciences, Inc. is burning capital to advance its Antibody Oligonucleotide Conjugates (AOCs) platform, a necessary step before any potential revenue stream from product sales materializes. This is a capital-intensive business model by design.

Here are the key financial markers that define the current cost outlay for Avidity Biosciences, Inc. as of late 2025:

Cost Metric	Period	Amount (USD)
Research and Development (R&D) Expenses	First Nine Months of 2025	$392.6 million
General and Administrative (G&A) Expenses	Q3 2025	$46.3 million
Projected Net Loss	Full Year 2025	Approximately $-675.4 million

The primary engine driving these substantial expenses is the simultaneous advancement of the pipeline. Honestly, the costs are climbing because the company is executing on its plan to become a commercial entity, which requires significant upfront investment in both the science and the infrastructure to support future launches. The R&D spend is the most visible component of this burn rate.

The major cost drivers feeding into this structure include:

• High Research and Development (R&D) expenses, totaling $392.6 million for the first nine months of 2025.
• Increasing General and Administrative (G&A) costs, hitting $46.3 million in Q3 2025, directly tied to building out the commercial infrastructure.
• Significant manufacturing costs for clinical and commercial supply scale-up; this is reflected in the R&D increase and includes commitments like the Lonza Manufacturing Services Agreement with approximately $620 million in minimum batch commitments scheduled from 2026-2028.
• The expense of running three concurrent late-stage global clinical trials for del-zota (DMD44), del-desiran (DM1), and del-brax (FSHD), each requiring extensive operational oversight and data management.

This heavy operational burn is what results in the capital-intensive model driving a projected net loss of approximately $-675.4 million for the 2025 fiscal year. Finance: draft 13-week cash view by Friday.

Avidity Biosciences, Inc. (RNA) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Avidity Biosciences, Inc. (RNA) as of late 2025, and honestly, the story isn't about selling pills yet; it's all about the value locked up in their Antibody Oligonucleotide Conjugates (AOCs) platform through partnerships.

The bulk of Avidity Biosciences, Inc.'s current income comes from collaboration revenue, which is essentially non-product income from their strategic pharma deals. This revenue is lumpy, driven by hitting specific development milestones or receiving ongoing research funding. For instance, you saw a clear example in the third quarter of 2025, where Avidity Biosciences, Inc. booked a $10.0 million clinical development milestone payment from Eli Lilly and Company under their existing research collaboration and license agreement.

To be fair, these milestone payments are the lifeblood right now, but they aren't the only source. You should also factor in the ongoing research funding and other payments from strategic pharmaceutical partnerships, like the one with Bristol Myers Squibb. The total collaboration revenue for the third quarter ending September 30, 2025, hit $12.5 million, which was a massive jump from the $2.3 million in the same period last year. This shows the platform is actively delivering value to its partners.

Here's a quick look at how that collaboration revenue stacked up recently:

Metric	Amount	Period/Date
Total Trailing Twelve Months (TTM) Revenue	$20.86 million	As of December 2025
Collaboration Revenue (Q3 2025)	$12.5 million	Quarter ended September 30, 2025
Collaboration Revenue (First Nine Months 2025)	$17.9 million	Nine months ended September 30, 2025

The TTM revenue as of December 2025 stands at $20.86 million. That figure is a significant step up from the $10.89 million reported for the full year in 2024. It's defintely clear that the milestone achievements in 2025 are what's driving that top-line growth.

Looking ahead, the real potential for product sales revenue is tied to the three late-stage neuromuscular programs. Avidity Biosciences, Inc. is preparing for what they call three potential successive product launches starting in 2026, following regulatory approval for del-zota, del-desiran, and del-brax. For del-zota specifically, U.S. sales are projected to reach approximately $400 million at peak, assuming approval. You won't see this product sales revenue stream until post-regulatory approval, which is a key near-term catalyst.

Also, you need to track the planned SpinCo. This entity is being set up to hold Avidity Biosciences, Inc.'s early-stage precision cardiology programs. The revenue stream here is less certain but potentially lucrative, involving future licensing revenue or value capture from those early assets once SpinCo is operating independently. The structure suggests a mechanism to potentially monetize those cardiology programs separately from the main neuromuscular focus, which is currently being acquired by Novartis.

Here are the key future revenue drivers you need to keep on your radar:

• Future product sales for del-zota, del-desiran, and del-brax.
• Potential peak annual sales approaching $10 billion across all three lead assets if approved.
• Monetization events from the planned SpinCo's cardiology pipeline.
• Continued milestone and research payments from existing partners like Eli Lilly and Bristol Myers Squibb.

Finance: draft 13-week cash view by Friday.