uniQure N.V. (QURE): Business Model Canvas [Dec-2025 Updated]

You're looking at a gene therapy developer balancing a commercialized win with a high-stakes pipeline, and understanding the mechanics behind that balancing act is key to valuing the stock. Honestly, the business model for this firm is fascinating: it's a hybrid where they rely on CSL Behring's network for Hemgenix revenue while pouring resources into their own platform, highlighted by the promising AMT-130 for Huntington's, which demonstrated a 75% slowing of disease progression. With $694.2 million in cash at the end of Q3 2025, the structure is clearly set up to fund that R&D grind, but the details of how they capture value across all their assets are what really matter. Dive into the full Business Model Canvas below to see the nine essential components driving their strategy.

uniQure N.V. (QURE) - Canvas Business Model: Key Partnerships

You're looking at the core alliances that provide uniQure N.V. with both commercial reach and crucial financial stability as of late 2025. These aren't just names on a slide; they represent tangible cash flows and operational support.

The arrangement with CSL Behring for global commercialization of HEMGENIX (etranacogene dezaparvovec-drbl) for Hemophilia B remains a cornerstone. While uniQure sold a portion of its royalty stream in May 2023 to HealthCare Royalty and Sagard Healthcare for up to $400 million cash (with $375 million upfront), it retains significant upside. uniQure is still eligible for contractual milestones totaling up to $1.5 billion under the original CSL Behring licensing agreement, which includes a $100 million milestone for the first U.S. product sale. Post-divestiture of the manufacturing site, revenue from contract manufacturing of HEMGENIX for CSL Behring is now recorded net of cost within other expenses, showing nil revenue for the three months ended September 30, 2025.

Financially, the September 2025 non-dilutive senior secured term loan facility with Hercules Capital is a big deal for near-term flexibility. This $175 million facility refinances existing debt and provides access to capital for the potential AMT-130 launch, anticipated in the United States in 2026. The deal extends the maturity of $50 million in prior debt to October 2030 and lowers the interest rate on that portion from 11.95% to 9.70%. An additional $125 million is available in tranches contingent on regulatory and financial milestones.

For manufacturing, uniQure strategically divested its commercial viral vector facility in Lexington, Massachusetts, to Genezen in July 2024. The total consideration was $25 million, split between preferred stock ($12.5 million) and a convertible note ($12.5 million). This move was designed to significantly reduce uniQure's annual cash burn by approximately $40 million. Genezen now serves as the manufacturer for HEMGENIX commercial supply for CSL Behring and provides development and manufacturing services for uniQure's pipeline programs, effectively making Genezen a preferred customer and supplier.

The collaboration with Bristol Myers Squibb (BMS) for cardiovascular disease gene therapy research, which originated in 2015 with a $50 million upfront payment, has concluded. BMS terminated the agreement, which quietly expired in February 2023. uniQure did not owe any funds to BMS upon termination, though it did receive a $4.4 million payment in late 2020 when BMS designated one target for IND-enabling studies. It's a past partnership, but it shows the historical structure of their business development efforts.

The relationship with Academic research institutions and clinical trial centers is vital for advancing the proprietary pipeline, especially AMT-130 for Huntington's disease. The company presented pivotal topline three-year data for AMT-130 in September 2025, which demonstrated a statistically significant slowing of disease progression. Further clinical updates are scheduled for the first half of 2026 for other candidates like AMT-260 (refractory mesial temporal lobe epilepsy) and AMT-191 (Fabry disease).

Here's a quick look at the financial scaffolding provided by these key relationships as of late 2025:

The ongoing clinical validation relies heavily on external sites:

• Presented topline, three-year data for AMT-130 in September 2025.
• Expects to present additional clinical data from AMT-260 and AMT-191 studies in the first half of 2026.

uniQure N.V. (QURE) - Canvas Business Model: Key Activities

You're mapping out uniQure N.V.'s operational focus as of late 2025, and the numbers tell a clear story about where the resources are going. The core activities revolve around advancing the pipeline, managing existing partnerships, and navigating the tricky regulatory landscape for their lead asset.

Research and development (R&D) of AAV-based gene therapies.

Research and development spending shows a consistent, high level of investment across the pipeline programs. For the three months ended September 30, 2025, Research and development expenses totaled $34.4 million, an increase from $30.6 million during the same period in 2024. This increase was driven by a $10.1 million rise in direct research and development expenses. For comparison, R&D expenses were $35.4 million for the three months ended June 30, 2025, and $36.1 million for the three months ended March 31, 2025. Honestly, that September quarter spend reflects significant focus on the BLA preparation.

Executing and managing global clinical trials (e.g., AMT-130 for Huntington's).

The pivotal Phase I/II study for AMT-130 has generated compelling efficacy data as of the June 30, 2025, data cutoff. The activity here is centered on demonstrating long-term effect and managing the patient cohorts.

Here's the quick math on the topline 36-month efficacy results for high-dose AMT-130:

The trial involved several cohorts:

• European open-label Phase 1b/2 study enrolled 13 patients (6 low dose; 7 high dose).
• A third cohort enrolled an additional 12 patients across U.S. and EU sites, randomized to both doses with immunosuppression.
• A fourth U.S. based cohort is currently enrolling up to 6 patients with lower striatal volumes.
• Overall, uniQure announced results after 29 patients had completed up to 36 months of the Phase I/II clinical trial.

Manufacturing and supplying Hemgenix drug substance to CSL Behring.

This activity has seen a structural change following the divestment of the Lexington facility in July 2024. Post-divestment, revenue from contract manufacturing is recorded net of cost within other expenses, and cost of contract manufacturing revenues are nil or significantly reduced.

The financial impact on revenue from contract manufacturing of HEMGENIX for CSL Behring shows a trend:

• For the three months ended September 30, 2025, Cost of contract manufacturing revenues were nil, compared to $0.8 million in Q3 2024.
• For the three months ended June 30, 2025, revenue decreased by $2.1 million compared to Q2 2024.
• For the three months ended March 31, 2025, revenue decreased by $4.0 million compared to Q1 2024.

Navigating complex regulatory pathways with the FDA and EMA.

Regulatory navigation is a critical, high-stakes activity. AMT-130 previously received two key designations:

• Breakthrough Therapy designation granted by the FDA in April 2025.
• Regenerative Medicines Advanced Therapy (RMAT) designation granted in May 2024.

However, a recent pre-BLA meeting in late 2025 shifted the landscape. The FDA feedback suggested that Phase I/II data versus an external control may no longer be adequate as primary evidence for a BLA submission, making the timing of a U.S. submission uncertain. The company plans urgent discussions with the FDA to find a path forward, having previously planned a BLA submission in the first quarter of 2026.

Preparing for the potential commercial launch of AMT-130 in the U.S.

Even with regulatory uncertainty, uniQure N.V. is allocating resources for a potential launch. This is visible in Selling, General and Administrative (SG&A) expenses.

For the three months ended September 30, 2025, SG&A expenses were $19.4 million. This was a $7.8 million increase from Q3 2024, which included a $4.9 million increase in professional fees. Specifically, $3.0 million of those fees were incurred to support the preparation of potential commercialization of AMT-130 in the United States. The company's cash position as of September 30, 2025, stood at $649.2 million, which management stated supports operations into 2029.

Finance: draft 13-week cash view by Friday.

uniQure N.V. (QURE) - Canvas Business Model: Key Resources

You're assessing the core assets uniQure N.V. (QURE) is relying on right now to drive its next phase of value creation. These aren't just line items; they are the foundation of their potential first-in-class gene therapy launch. Here's a breakdown of the tangible and intangible resources as of late 2025.

Financial Strength is definitely a primary resource, especially following recent capital raises. As of September 30, 2025, uniQure N.V. held $694.2 million in cash, cash equivalents, and current investment securities. This figure reflects the net proceeds of approximately $323.7 million raised from an upsized public follow-on offering completed in September 2025. This fortified balance sheet is explicitly stated to support operations into 2029. That runway is a critical resource for navigating the regulatory uncertainty surrounding AMT-130.

The clinical and scientific data represent perhaps the most valuable non-cash resource. The pivotal Phase I/II study for AMT-130 in Huntington's disease yielded highly encouraging results at the 36-month mark for the high-dose cohort. This data set is the cornerstone of their near-term strategy.

Here's a quick look at the key financial and clinical metrics underpinning these resources:

uniQure N.V.'s proprietary technology platform is a deep, structural resource. They develop their gene therapies using an innovative and modular technology platform, which allows them to use the same set of components-like capsids and promoters-across different disease targets. This modularity speeds up development for new candidates.

The delivery system itself is a key differentiator. The platform leverages a best-in-class Adeno-Associated Virus (AAV)-based vector delivery system. Specifically, uniQure N.V. uses the AAV5 variant, for which they hold exclusive, worldwide rights for therapeutic products delivered to the brain or the liver. This exclusivity on a proven vector serotype is a significant barrier to entry for competitors in those specific tissue targets.

The Intellectual Property (IP) portfolio backs up the platform's exclusivity and pipeline depth. This IP covers multiple gene therapy candidates across their pipeline, which includes programs for refractory temporal lobe epilepsy (AMT-260), Fabry disease (AMT-191), and ALS (AMT-162), in addition to Huntington's disease. For instance, their IP position in hemophilia B was strengthened by acquiring a patent family covering the hyperactive Padua variant of Factor IX (FIX-Padua) for use in gene therapy.

The specialized personnel are the human capital that brings these assets to life. This resource includes:

• Specialized scientific and clinical development personnel.
• Expertise in AAV manufacturing using insect cells and baculoviruses.
• Leadership with experience in advancing gene therapies through pivotal studies.
• Personnel supporting the preparation for a potential commercialization of AMT-130 in the United States, evidenced by a $3.0 million increase in professional fees in Q3 2025 for this support.

Finance: draft 13-week cash view by Friday.

uniQure N.V. (QURE) - Canvas Business Model: Value Propositions

uniQure N.V. focuses on delivering single-administration, potentially curative gene therapies for severe, rare diseases.

The core value proposition is centered on the clinical success of AMT-130 in Huntington's disease (HD), which represents the first time a drug has been shown to alter the course of HD in people in a clinical trial.

The pipeline is built around addressing high unmet medical needs in neurological and liver disorders with potentially one-time treatments:

• Huntington's disease (AMT-130)
• Refractory temporal lobe epilepsy (AMT-260)
• Fabry disease (AMT-191)
• Amyotrophic Lateral Sclerosis - SOD1 (AMT-162)

The market opportunity for the lead candidate, AMT-130, in Huntington's disease is estimated at $4.27B.

The value proposition is heavily supported by the long-term therapeutic benefit demonstrated from a one-time treatment, as shown by the 36-month follow-up data for AMT-130.

The pivotal Phase I/II study for AMT-130 met its primary endpoint, demonstrating statistically significant slowing of disease progression at 36 months compared to an external control group.

The company is planning a Biologics License Application (BLA) submission for AMT-130 in the first quarter of 2026.

Financially, uniQure N.V. reported cash, cash equivalents, and current investment securities of $694.2 million as of September 30, 2025, which is expected to fund operations into 2029.

The company raised approximately $323.7 million in net proceeds from an upsized public follow-on offering in September 2025.

Research and development expenses for the three months ended September 30, 2025, were $34.4 million, which included $6.6 million related to the preparation for the AMT-130 BLA submission.

The value proposition is the delivery of a potentially first-in-class, one-time treatment for a devastating disorder, underpinned by a strong balance sheet to support near-term commercialization planning.

uniQure N.V. (QURE) - Canvas Business Model: Customer Relationships

High-touch, specialized support for ultra-rare disease patient communities is centered around the development pipeline for conditions like Huntington\'s disease (HD), mesial temporal lobe epilepsy (MTLE), and Fabry disease.

For the lead candidate, AMT-130 in HD, topline 36-month efficacy data presented in September 2025 showed a statistically significant 75% slowing in disease progression measured by the composite Unified Huntington\'s Disease Rating Scale (cUHDRS). Also, a fourth cohort evaluating high-dose AMT-130, consisting of six patients, was fully recruited in October 2025.

Direct engagement with key opinion leaders and clinical investigators is being formalized as uniQure N.V. transitions toward potential commercialization.

• The appointment of Kylie O\'Keefe as Chief Customer and Strategy Officer in June 2025 signals a focus on commercial execution and medical affairs leadership.
• Selling, general and administrative (SG&A) expenses for the three months ended September 30, 2025, were $19.4 million, which included $3.0 million incurred to support the preparation of a potential commercialization of AMT-130 in the United States.

Collaboration with patient advocacy groups for disease awareness is a core component of engaging the rare disease community.

uniQure N.V. maintains connections with various advocacy groups across its target indications, including:

• Global Genes.
• EveryLife Foundation.
• National Organization for Rare Disorders.
• Fabry Support and Information Group.
• The Coalition for Hemophilia B.

Managed access and reimbursement support for high-cost therapies is being planned, leveraging executive experience in this area.

The Chief Customer and Strategy Officer has a proven track record directing the development and execution of reimbursement strategies, including payer engagement and health economic assessments.

The company's cash position as of September 30, 2025, stood at $694.2 million, providing a strong balance sheet to fund these pre-commercialization relationship-building activities, with expectations to fund operations into 2029.

Here's a quick look at the patient-facing pipeline focus areas as of late 2025:

uniQure N.V. (QURE) - Canvas Business Model: Channels

You're looking at how uniQure N.V. gets its value proposition-transformative gene therapies-into the hands of patients and prescribers as of late 2025. For their approved product, HEMGENIX, the channel is entirely outsourced to a major partner, CSL Behring, which handles the global commercialization.

CSL Behring's established global commercial and distribution network (Hemgenix)

The channel for HEMGENIX is defined by the licensing agreement with CSL Behring. uniQure N.V. focuses on manufacturing, while CSL Behring manages the entire commercial and distribution network globally. This split means uniQure N.V. relies on CSL Behring's established infrastructure to reach the market.

Here's a look at the financial structure tied to this channel arrangement:

uniQure N.V. is responsible for the global manufacturing of HEMGENIX at its licensed facility in Lexington, Massachusetts. Still, the actual commercial sales channel is CSL Behring's domain.

Specialized gene therapy treatment centers and hospitals

For its pipeline candidates like AMT-130, the channel will require highly specialized infrastructure. Gene therapies are not dispensed through a standard pharmacy; they are administered directly into the patient, often requiring specific surgical or infusion capabilities.

• The commercialization plan for AMT-130, once approved, will likely mirror the model used for other complex, one-time treatments.
• This necessitates establishing a limited network of certified treatment centers capable of handling the product and procedure.
• The company expects its current cash reserves of $694.2 million as of September 30, 2025, to fund operations, including the planned U.S. launch of AMT-130, into the second half of 2029.

Direct regulatory submissions (BLA) to agencies like the FDA

For its proprietary pipeline, uniQure N.V. manages the regulatory channel directly. This involves preparing and submitting the necessary applications to health authorities.

For AMT-130 in Huntington's disease, key channel milestones related to regulatory approval included:

• Alignment with the FDA on the statistical analysis plan and CMC (Chemistry, Manufacturing, and Controls) requirements to support a planned BLA submission in the first quarter of 2026.
• Planning a pre-BLA meeting with the FDA during Q4 2025.
• The company issued a regulatory update on AMT-130 on December 4, 2025.

Scientific publications and medical conferences for data dissemination

Disseminating clinical data is a critical channel for building scientific credibility and supporting future regulatory and commercial efforts. This is how uniQure N.V. communicates progress to the medical community.

Key data dissemination events planned or executed in 2025 included:

• Presenting topline three-year data from the AMT-130 Phase I/II study in September 2025.
• The data presentation in Q3 2025 was expected to include follow-up data on all patients treated in the first two cohorts, totaling three years of follow-up data on 24 treated patients.
• Initial data from the AMT-191 (Fabry disease) study was expected in the second half of 2025.

Finance: draft 13-week cash view by Friday.

uniQure N.V. (QURE) - Canvas Business Model: Customer Segments

You're looking at the specific patient populations uniQure N.V. (QURE) is targeting with its pipeline as of late 2025. This isn't about the overall market size, but the specific groups whose severe, unmet medical needs drive the current strategy.

The customer segments are defined by the indication for each of uniQure N.V.'s key investigational or recently approved assets. For the approved Hemophilia B therapy, the customer segment is defined by the patient population eligible for that specific treatment, which is partnered with CSL Behring.

The focus on these distinct, rare disease populations dictates the clinical trial design and partnership strategy.

The Hemophilia B segment is already commercialized via the partner, CSL Behring, following the November 2022 FDA approval of HEMGENIX. The clinical data supporting this segment showed a 97% reduction in mean Factor IX consumption at 18 months post-treatment.

For Huntington's disease with AMT-130, the customer segment is highly specific: early-stage patients where disease modification is most likely to show a measurable effect on the composite Unified Huntington's Disease Rating Scale (cUHDRS). The high-dose cohort demonstrated a statistically significant 75% slowing of disease progression at 36 months compared to the external control group of n=940.

The AMT-260 program targets patients with refractory MTLE who have failed standard treatments. The Phase I/IIa trial is small, planning for 12 patients across two cohorts. The early signal from the first patient, who previously had about seven seizures a month, showed a 92% reduction.

The Fabry disease segment (AMT-191) is focused on adult males who are on, but not optimally responding to, bi-weekly Enzyme Replacement Therapy (ERT). The initial data from four patients in Cohort A showed $\alpha$-Gal A activity increases ranging from 27- to 208-fold above the mean normal level of 3.57 nmol.

For SOD1-ALS (AMT-162), the patient pool is extremely small, with SOD1 mutations accounting for about 2% of the estimated 170,000 global ALS cases. The Phase I/II trial is designed for only about 12 patients total.

• Patients with Hemophilia B are served via the approved HEMGENIX product.
• Patients with early manifest Huntington's disease are the focus for AMT-130, with 29 treated in the pivotal study to date.
• Refractory MTLE patients are being enrolled in the GenTLE trial, aiming for 12 total participants.
• Fabry disease patients are being recruited into a study that has dosed 7 patients as of late 2025.
• SOD1-ALS patients are part of the EPISOD1 trial, which has three dose-escalating cohorts.

The company's cash position as of September 30, 2025, was $694.2 million, which uniQure N.V. expects will be sufficient to fund operations into 2029, supporting the development across these distinct customer segments.

uniQure N.V. (QURE) - Canvas Business Model: Cost Structure

You're looking at the core expenses driving uniQure N.V.'s operations as they push AMT-130 toward potential commercialization. The cost structure is heavily weighted toward R&D, which is typical for a late-stage gene therapy company, but you're also seeing significant spending shift toward commercial readiness.

The largest single operating cost category is Research and Development. For the three months ended September 30, 2025, uniQure N.V. reported Research and Development expenses totaling $34.4 million. This was an increase from $30.6 million during the same period in 2024.

This R&D spend directly reflects the intense focus on regulatory milestones. A major driver of the increase was $6.6 million specifically tied to the preparation for the Biologics License Application (BLA) submission for AMT-130. You're also seeing costs associated with clinical trial execution and navigating the regulatory path, especially following the recent pre-BLA meeting with the FDA, which introduced some uncertainty regarding the required primary evidence.

Selling, General, and Administrative (SG&A) expenses saw a substantial jump, reaching $19.4 million for the third quarter of 2025, up from $11.6 million in Q3 2024. This rise is largely due to increased professional fees and employee-related costs. Specifically, $3.0 million of these professional fees were incurred to support the preparation of a potential commercialization of AMT-130 in the United States.

Regarding manufacturing, the cost structure has changed following a divestiture. Cost of contract manufacturing revenues was nil for the three months ended September 30, 2025, compared to $0.8 million in Q3 2024. This reflects the divestment of the Lexington facility in July 2024. Quality control and AAV vector production costs are now likely being managed differently or are embedded elsewhere, but the direct contract manufacturing revenue offset is gone.

Debt servicing is another key cost component. While I don't have the exact interest expense for Q3 2025, we can look at the recent refinancing and prior period data. For the three months ended March 31, 2025, the interest expense specifically related to the Hercules Capital debt facility was $1.8 million. This is down from $3.7 million in Q1 2024, partly due to a $50.0 million repayment in July 2024. The September 2025 refinancing with Hercules Capital extended the maturity of that $50 million debt to October 2030 and lowered the interest rate to a current rate of 9.70%, an improvement from the previous 11.95%.

Here's a quick view of the major reported operating expenses for Q3 2025:

The spending profile shows a clear prioritization of advancing the pipeline while simultaneously building the infrastructure for a potential launch. You'll want to watch how R&D spend shifts now that the BLA submission timeline is uncertain, and how much of the SG&A increase is sustained post-launch.

Other notable expenses impacting the bottom line during the period include:

• Interest expense - Royalty Financing Agreement: $13.3 million for the three months ended March 31, 2025
• Non-operating items, net expense: $20.9 million for Q3 2025
• Net Loss for Q3 2025: $80.5 million

Finance: review the Q4 2025 budget against the new Hercules Capital interest rate of 9.70% for the refinanced $50 million tranche by next Tuesday.

uniQure N.V. (QURE) - Canvas Business Model: Revenue Streams

You're looking at how uniQure N.V. generates its top-line income, which, as a clinical-stage company, is heavily weighted toward non-sales revenue sources as of late 2025. The core of the operational revenue comes from partnerships, not product sales yet, so every milestone and license fee is critical for funding the pipeline.

The Trailing Twelve Month (TTM) revenue as of September 30, 2025, stood at $15.75 million. This figure reflects the non-sales revenue streams recognized over the preceding four quarters. For the third quarter of 2025 specifically, total revenue was $3.7 million.

Here's a breakdown of the key components contributing to uniQure N.V.'s revenue:

• License and collaboration revenues, totaling $3.7 million in Q3 2025.
• Milestone payments from strategic partners like CSL Behring.
• Royalties on net sales of Hemgenix (commercialized by CSL Behring).
• Proceeds from financing activities, like the $323.7 million net from the Q3 2025 public offering.

The Q3 2025 revenue of $3.7 million saw an increase of $1.4 million year-over-year, driven by a $1.5 million increase in license revenues, partially offset by a $0.1 million decrease in collaboration revenues.

The revenue streams related to the CSL Behring partnership for HEMGENIX are structured to provide both upfront/early payments and ongoing performance-based income:

It's important to note that the specific royalty receipts and any milestone payments received from CSL Behring in Q3 2025 are embedded within the reported license revenues, as the financial reporting for Q3 2025 only itemized the aggregate license and collaboration revenue. The company also fortified its balance sheet through non-operating cash flow events, which are separate from core revenue but vital for operations. Specifically, uniQure N.V. raised approximately $323.7 million in net proceeds from an upsized public follow-on offering in September 2025. That cash infusion is intended to fund commercialization readiness and pipeline development. Finance: draft 13-week cash view by Friday.