Sarepta Therapeutics Business Model Canvas

Sarepta collaborates with major global pharmaceutical firms to bring its approved treatments to markets beyond its core territories. These partnerships are crucial for expanding access and maximizing the reach of their innovative therapies.

A prime example is Sarepta's agreement with F. Hoffman-La Roche Ltd. concerning ELEVIDYS. This collaboration encompasses not only the commercial supply of the drug but also generates royalty income from sales and triggers milestone payments. For instance, Sarepta received a $63.5 million milestone payment following the approval of ELEVIDYS in Japan, highlighting the financial benefits of these strategic alliances.

Sarepta's key partnerships span academic institutions, other biopharma companies, and contract manufacturers, all crucial for advancing its pipeline and production. Collaborations with academic centers like Duke University and the Broad Institute fuel innovation in gene therapy and RNA technologies. Strategic alliances with companies such as Arrowhead Pharmaceuticals, involving significant financial commitments like Sarepta's $500 million deal, grant access to novel platforms and assets.

Furthermore, partnerships with global pharmaceutical giants, exemplified by the F. Hoffman-La Roche Ltd. agreement for ELEVIDYS, are vital for market expansion and revenue generation, as seen with the $63.5 million milestone payment received after ELEVIDYS' approval in Japan. The company also relies on contract manufacturing organizations (CMOs) to produce its complex genetic medicines, a necessity reflected in its manufacturing costs reported in early 2024.

Engagement with patient advocacy groups, particularly those focused on Duchenne muscular dystrophy like Parent Project Muscular Dystrophy, is instrumental in understanding patient needs and facilitating therapy access.

Sarepta Therapeutics' commercialization efforts focus on bringing its approved gene therapies and RNA-targeted therapies to patients. This involves robust marketing and sales teams engaging directly with healthcare providers who prescribe these treatments. The company's portfolio includes key products like ELEVIDYS, EXONDYS 51, VYONDYS 53, and AMONDYS 45, all targeting Duchenne muscular dystrophy (DMD).

Establishing efficient distribution networks is crucial for patient access. Sarepta works to ensure these complex therapies reach the appropriate treatment centers. Navigating the reimbursement landscape is also a significant activity, aiming to secure coverage for these high-value treatments, which directly impacts sales volume and revenue generation.

In 2024, Sarepta reported significant growth, with net product revenues reaching $1.2 billion for the full year, a substantial increase driven by the expanding use of its DMD therapies. ELEVIDYS, in particular, saw strong uptake following its expanded indication, contributing significantly to this revenue growth and demonstrating the effectiveness of their commercialization strategy.

Sarepta’s key activities encompass the entire lifecycle of its gene therapy products, from initial research and development to commercialization and ongoing regulatory compliance. This includes significant investment in clinical trials, manufacturing scale-up, and market access strategies to ensure patient access to its therapies.

The company's commitment to innovation is evident in its robust pipeline and its direct involvement in manufacturing its complex genetic medicines. Sarepta’s strategic focus on intellectual property protection is also a crucial activity, ensuring its groundbreaking technologies remain secure.

In 2024, Sarepta reported net product revenues of $1.2 billion, with ELEVIDYS showing strong growth following an expanded indication. This financial performance highlights the success of its commercialization and market access efforts for its Duchenne muscular dystrophy (DMD) treatments.

Sarepta's scientific and medical teams are critical. These highly specialized groups include researchers, clinical development experts, and regulatory professionals. Their profound knowledge of rare genetic diseases and advanced therapies fuels Sarepta's pipeline and drug development success.

Sarepta's key resources are its proprietary technology platforms, including RNA-targeted approaches and AAV gene therapy delivery systems, which are fundamental to its innovative genetic medicines. The company's intellectual property portfolio is a critical asset, safeguarding its therapies and manufacturing technologies, ensuring market exclusivity for its treatments like those for Duchenne muscular dystrophy. Furthermore, Sarepta possesses a strong team of scientific and medical experts, alongside significant financial capital, including approximately $1.5 billion in cash and investments as of December 31, 2024, to drive its research, development, and commercialization efforts.

Sarepta Therapeutics' cutting-edge therapeutic platforms are central to its business model. They utilize a diverse array of advanced technologies, including RNA-targeted therapies, gene therapy, and gene editing. This multi-pronged strategy aims to address a wide spectrum of genetic mutations and diseases, offering a versatile pipeline of innovative treatments.

This commitment to multiple technology platforms is crucial for tackling the complexity of genetic disorders. For instance, their RNA-targeted therapies, like those used for Duchenne muscular dystrophy, have shown significant progress, with products like Exondys 51 and Amondys 45 generating substantial revenue. In 2023, Sarepta reported approximately $1.1 billion in net product revenue, largely driven by these RNA-based therapies, underscoring the commercial viability of their platform approach.

Sarepta's value proposition centers on delivering life-changing therapies for devastating rare genetic diseases, primarily Duchenne muscular dystrophy (DMD). They offer hope and tangible improvements in quality of life for patients with limited or no other treatment options.

The company's success is rooted in its pioneering scientific approach and its ability to translate complex genetic science into approved, commercially viable treatments. This has led to significant market impact, as evidenced by their strong revenue generation.

Sarepta also focuses on enabling patient access through robust support programs, addressing the complexities of treatment administration and reimbursement to ensure patients can benefit from their innovative medicines.

Sarepta Therapeutics actively partners with and supports patient advocacy organizations, recognizing their crucial role in understanding the needs of the rare disease community. These collaborations help Sarepta gather vital patient insights, ensuring its research and development efforts are aligned with what matters most to patients and their families. For instance, in 2024, Sarepta continued its deep engagement with groups like the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD), co-hosting educational webinars and participating in awareness campaigns.

Sarepta's customer relationships are built on deep empathy and direct support for patients and their families navigating rare diseases. The company actively engages with patient advocacy groups, such as the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD), to understand and address their needs, as seen in their collaborative educational initiatives throughout 2024. This commitment extends to providing resources that simplify treatment access and adherence, underscoring a patient-centric approach.

Sarepta Therapeutics relies on a worldwide network of clinical trial sites to find patients, run studies, and gather crucial data for its pipeline products. These sites are vital for early patient engagement and the generation of evidence needed for regulatory submissions and eventual market launch.

In 2024, Sarepta continued to expand its global clinical footprint, working with hundreds of specialized sites across North America, Europe, and other key regions. This extensive network ensures efficient patient recruitment, particularly for rare genetic diseases, and facilitates the collection of high-quality data, a cornerstone for advancing therapies through development.

Sarepta's channels encompass specialized distribution for gene therapies, direct engagement with healthcare providers via a dedicated sales force, and a global network of clinical trial sites for patient identification and data collection.

Digital platforms like the corporate website and investor relations portal are crucial for broad information dissemination, while scientific conferences and peer-reviewed publications serve to validate findings and engage key opinion leaders.

In 2024, Sarepta's Elevidys commercialization relied on these robust networks, with significant investment in customer-facing teams and an expanded global clinical footprint.

The company's presence at conferences like ASGCT and publication in journals such as The Lancet underscore their commitment to scientific validation and market adoption.

Caregivers and families are pivotal in the treatment journey for Duchenne muscular dystrophy (DMD) and other neuromuscular conditions. They are the primary navigators of healthcare, influencing treatment decisions and actively participating in patient care. Sarepta’s engagement with this segment is crucial for successful patient outcomes.

In 2024, the focus on patient support and education for families remains paramount. Sarepta Therapeutics continues to invest in programs that provide resources and information, acknowledging that families are key advocates. This segment’s understanding and acceptance of therapies directly impact adherence and overall patient well-being.

Sarepta's core customers are patients diagnosed with Duchenne muscular dystrophy (DMD), particularly those with specific genetic mutations treatable by their exon-skipping therapies. The approval of ELEVIDYS in 2023 expanded this to include both ambulatory and non-ambulatory DMD patients, significantly broadening the addressable market. By 2024, Sarepta continued to prioritize this patient group, focusing on access and support for these individuals and their families.

Beyond DMD, Sarepta is actively targeting patients with Limb-Girdle Muscular Dystrophies (LGMDs), a diverse group of rare genetic disorders. As their LGMD pipeline advances, these patients represent a crucial and growing segment for the company. This strategic expansion reflects Sarepta's commitment to addressing multiple rare neuromuscular diseases.

Selling, General, and Administrative (SG&A) expenses are a major component of Sarepta Therapeutics' cost structure. These costs directly fuel the commercialization of their therapies, encompassing everything from sales force compensation and marketing initiatives to crucial patient education programs and the general administrative overhead required to run the company. For the second quarter of 2025, Sarepta reported SG&A expenses totaling $137.9 million, underscoring the significant investment in bringing their innovative treatments to market and maintaining essential corporate functions.

Sarepta’s cost structure is heavily weighted towards Research and Development (R&D) and manufacturing. For the first quarter of 2025, R&D expenses were $749.2 million, while cost of revenue, reflecting manufacturing, was $478.5 million in 2023. These figures highlight the significant investment in developing and producing its complex gene and RNA therapies.

Selling, General, and Administrative (SG&A) expenses are also substantial, reaching $137.9 million in the second quarter of 2025, supporting commercialization and corporate functions. Protecting its intellectual property, which is crucial for market exclusivity, adds further costs through patent filings, prosecution, and potential litigation.

Clinical trial operations and regulatory affairs represent another major cost center, with R&D expenses in 2023 totaling $766.3 million, much of which funds these critical activities. These expenses cover patient recruitment, site management, data analysis, and regulatory submissions.

Sarepta Therapeutics generates revenue through contract manufacturing within certain collaboration agreements. This involves producing and supplying commercial quantities of its therapies to global partners, adding a valuable revenue stream that highlights its manufacturing capabilities.

Sarepta Therapeutics' revenue streams are primarily driven by net sales of its Duchenne muscular dystrophy (DMD) therapies, ELEVIDYS and its RNA-based PMO franchise. The company reported $1.79 billion in total net product revenue for 2024, with ELEVIDYS contributing $820.8 million. Beyond product sales, Sarepta generates income through strategic collaborations, licensing agreements, and option payments, which provide upfront capital and validate its research pipeline.