Rocket Pharmaceuticals, Inc. (RCKT): Business Model Canvas [Dec-2025 Updated]

As a former portfolio head, I see Rocket Pharmaceuticals, Inc. standing at a classic biotech crossroads: a focused, high-potential pivot toward AAV cardiovascular gene therapies, backed by a $222.8 million cash cushion as of September 30, 2025, but still pre-revenue with Q3 2025 R&D spending at $34.1 million. The entire near-term thesis hinges on the KRESLADI™ regulatory decision coming in early 2026, which could unlock a Priority Review Voucher and future product sales. I've mapped out their entire strategic engine-from their specialized treatment center channels to their cost structure-so you can clearly see the operational levers they are pulling to reach this make-or-break moment. Keep reading to see the full Business Model Canvas.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Key Partnerships

You're looking at the structure of Rocket Pharmaceuticals, Inc. (RCKT)'s collaborations as they pivot hard into the cardiovascular space. The partnerships here aren't just nice-to-haves; they are absolutely essential for managing complex, late-stage gene therapy trials and manufacturing.

Clinical Research Organizations (CROs) for global trial execution.

While specific CRO names aren't public record for every trial, the execution of global studies, like the pivotal Phase II trial for RP-A501 in Danon disease, necessitates deep reliance on external clinical expertise. The FDA lifting the clinical hold on this trial in August 2025 signaled that the protocols, likely managed in partnership with a CRO, met regulatory standards following the required root cause analysis. The resumption of dosing for additional patients is anticipated in the first half of 2026, a timeline heavily dependent on the CRO's ability to rapidly re-activate sites and manage patient flow globally.

Academic medical centers for patient enrollment and clinical expertise.

Securing patient enrollment for ultra-rare disorders requires embedding trials within specialized centers. For instance, the natural history study of Danon disease, published in the Journal of the American Heart Association in March 2025, points to established relationships with leading cardiology institutions. Similarly, the ongoing Phase 1 trial for RP-A601 in PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) relies on academic centers experienced in managing ventricular arrhythmias. The market opportunity for PKP2-ACM is internally estimated to affect approximately 50,000 people in the U.S. and Europe, meaning the partnership network must be robust to capture this patient population.

Specialized raw material and vector supply vendors for cGMP manufacturing.

Rocket Pharmaceuticals, Inc. has made significant investments to control a critical part of this chain internally. They operate a world-class R&D and manufacturing facility in Cranbury, N.J., with approximately one-half of the space scaled for Adeno-Associated Virus (AAV) Current Good Manufacturing Practice (cGMP) production. This internal capacity is designed to support clinical development and potential commercialization, giving them control over supply, cost, and quality for their AAV cardiovascular programs. However, specialized raw materials and upstream vector components still require established vendor relationships to ensure the continuous supply needed for producing batches, such as those supporting the RP-A701 IND clearance received in June 2025.

Potential external partners to offload non-core assets like Fanconi Anemia.

The strategic shift in July 2025, which included a workforce reduction of approximately 30%, directly impacted the Fanconi Anemia (FA) program (RP-L102). Rocket voluntarily withdrew the Biologics License Application (BLA) with the FDA on October 3, 2025, and the European Marketing Authorization Application (MAA) in July 2025. Management explicitly stated they will no longer invest internal resources but will consider partnering with another group to take RP-L102 forward. This is a clear action to preserve capital, as evidenced by the Q3 2025 cash position of $222.8 million, expected to last into the second quarter of 2027. Offloading this asset via partnership de-risks the balance sheet from future development costs.

Regulatory bodies (FDA, EMA) as critical stakeholders in the approval process.

The FDA and EMA are the ultimate gatekeepers. The relationship is dynamic, involving both collaboration and stringent review. Key milestones in late 2025 illustrate this:

• The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601 for PKP2-ACM.
• The FDA accepted the resubmission of the BLA for KRESLADI™ (LAD-I), setting a PDUFA target action date of March 28, 2026.
• The FDA lifted the clinical hold on the Danon disease trial in August 2025.

These interactions dictate trial design, data requirements, and ultimate market access. The company's General and Administrative (G&A) expenses for the third quarter ending September 30, 2025, were $18.4 million, which included a decrease of $6.6 million in commercial preparation-related expenses, reflecting a streamlining of efforts toward the most immediate regulatory targets like KRESLADI™.

Here's a look at the financial context supporting these strategic relationships as of the end of Q3 2025:

Financial Metric	Amount / Date	Context
Cash Position (as of 9/30/2025)	$222.8 million	Funds operations runway into Q2 2027, excluding potential PRV proceeds.
R&D Expense (Q3 2025)	$34.1 million	Direct spend supporting ongoing clinical trials with academic centers and regulatory submissions.
G&A Expense (Q3 2025)	$18.4 million	Reflects reduced commercial prep costs of $6.6 million year-over-year post-restructuring.
KRESLADI™ PDUFA Date	March 28, 2026	Key near-term regulatory milestone with the FDA for the LAD-I therapy.
RP-L102 BLA Withdrawal	October 3, 2025	Finalized decision to seek external partnership for the Fanconi Anemia asset.

Finance: review Q4 2025 projected spend against the $222.8 million cash balance by next Tuesday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Key Activities

You're managing a portfolio where clinical setbacks directly impact runway, so understanding Rocket Pharmaceuticals, Inc.'s current operational focus is key. Their Key Activities are now sharply defined around their AAV cardiovascular platform and pushing KRESLADI™ through the final regulatory hurdle. Here's the quick math on what they are actively doing as of late 2025.

Research and development of AAV-based cardiovascular gene therapies.

Rocket Pharmaceuticals, Inc. is intensely focused on its adeno-associated virus (AAV) cardiovascular platform, which targets genetically defined causes of cardiomyopathies affecting more than 100,000 patients in the U.S. and EU. This focus came after a strategic reorganization that saw the deprioritization of the Fanconi Anemia (RP-L102) and Pyruvate Kinase Deficiency (RP-L301) programs. The R&D spend reflects this concentration; for the three months ended June 30, 2025, Research and development expenses were $42.7 million. The company is advancing RP-A601 for PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), which received Regenerative Medicine Advanced Therapy (RMAT) designation in July 2025, and RP-A701 for BAG3-associated dilated cardiomyopathy (BAG3-DCM), which is preparing to enter the clinic.

Executing pivotal Phase 2 trials for Danon disease (RP-A501).

The pivotal Phase 2 trial for RP-A501 in Danon disease remains a critical, albeit complicated, activity. The company is actively engaging with the U.S. Food and Drug Administration (FDA) to resolve a clinical hold placed on May 23, 2025, following a Serious Adverse Event (SAE) that resulted in a patient death. Honestly, that setback definitely changed the near-term timeline. The trial is designed as a global, single-arm, multi-center study, and here are the specifics of the execution:

Trial Parameter	Detail/Value
Investigational Therapy	RP-A501 (AAV9 gene therapy)
Indication	Danon disease
Total Patients in Pivotal Trial	12 patients
Pediatric Safety Run-in (n=)	2
Dose Level Assessed	6.7 x 1013 GC/kg
Clinical Hold Status (as of late 2025)	Active, working with FDA to resolve

The company cannot provide guidance on the trial completion timing while the hold is in place.

Manufacturing gene therapy vectors at the Cranbury, N.J. cGMP facility.

Internal manufacturing control is a core activity, utilizing the state-of-the-art facility in Cranbury, New Jersey. This site was built to support the production of their lentivirus (LV) and AAV gene therapies. The facility spans approximately 103,720 ft², with about half of that space dedicated to AAV Current Good Manufacturing Practice (cGMP) production. This internal capacity is instrumental for controlling the supply, cost, and quality of clinical drug product for their pipeline programs.

Advancing regulatory activities for KRESLADI™ (severe LAD-I) toward March 2026 PDUFA.

A major current activity is advancing KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I). The FDA accepted the resubmission of the Biologics License Application (BLA) in October 2025. This acceptance established a Prescription Drug User Fee Act (PDUFA) action date of March 28, 2026. The BLA is supported by data from the global Phase 1/2 study (NCT03812263), which demonstrated compelling results:

• 100% overall survival rate at 1 year post-treatment.
• Survival maintained through the entire duration of follow-up.
• KRESLADI was deemed well tolerated with no treatment-related serious adverse events.
• Substantial decrease in the incidence of significant infections.

If approved, Rocket Pharmaceuticals, Inc. is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), which has been noted to sell for $150 million or more.

Strategic corporate reorganization and workforce reduction of approximately 30%.

To fortify the path to sustained value creation and conserve cash, Rocket Pharmaceuticals, Inc. executed a significant strategic corporate reorganization in July 2025. This involved a workforce reduction of approximately 30% across all functions. The company ended 2024 with 299 full-time employees, and the layoffs affected about 80 people, with expected restructuring costs of $3.5 million primarily for severance payments. This action, coupled with other cost-saving measures, is projected to reduce 12-month operating expenses by nearly 25%. The goal of this activity was clear: extend the operational runway using existing resources into the second quarter of 2027. For context, as of June 30, 2025, the cash position was $271.5 million, though the Q3 2025 report showed cash and equivalents had fallen to $75.9 million as of September 30, 2025.

Reorganization Metric	Value/Target
Workforce Reduction Percentage	30%
Expected 12-Month OpEx Reduction	Nearly 25%
Projected Cash Runway Extension	Into Q2 2027
Severance Cost Estimate	$3.5 million
Employees Affected (Approx.)	80

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Key Resources

You're looking at the core assets Rocket Pharmaceuticals, Inc. (RCKT) relies on to drive its late-stage development and potential commercialization. These aren't just line items; they are the engines for their gene therapy ambitions.

Financial Capital and Runway

The immediate financial strength is a key resource, giving the company time to hit critical regulatory and clinical targets. As of September 30, 2025, Rocket Pharmaceuticals, Inc. reported cash, cash equivalents, and investments totaling exactly $222.8 million. This capital base is crucial for funding ongoing operations, especially as they push their lead candidates through late-stage trials.

Here's a quick look at how that cash position relates to their operational burn and runway, based on their Q3 2025 reporting:

Metric	Value as of September 30, 2025	Context
Cash, Cash Equivalents, and Investments	$222.8 million	Primary operating capital.
Expected Operational Runway	Into the second quarter of 2027	Excluding potential Priority Review Voucher proceeds.
R&D Expenses (3 Months Ended 9/30/2025)	$34.1 million	Reflects disciplined resource allocation.
General & Administrative (G&A) Expenses (3 Months Ended 9/30/2025)	$18.4 million	Decreased from $27.1 million in Q3 2024.

This runway into Q2 2027 is defintely a significant asset, especially with the KRESLADI™ PDUFA date approaching in March 2026.

Intellectual Property and Technology Platforms

The scientific foundation of Rocket Pharmaceuticals, Inc. rests on its proprietary technology. This IP is what allows them to design therapies for specific, rare genetic disorders.

• Proprietary AAV (adeno-associated viral) vector gene therapy platforms, which are central to their cardiovascular pipeline focus.
• Proprietary lentiviral (LV) vector-based platforms, which are part of their broader genetic therapy approach.

Physical and Manufacturing Assets

Controlling the manufacturing process is a massive advantage in gene therapy, reducing reliance on external, often capacity-constrained, third parties. Rocket Pharmaceuticals, Inc. maintains this control through its dedicated facility.

The company possesses an in-house, dedicated cGMP (current Good Manufacturing Practice) manufacturing and R&D facility located in New Jersey. This asset supports the production of AAV batches for clinical trials and future commercial supply, which is a key component of their integrated model.

Human Capital and Leadership

Executing complex late-stage trials and preparing for a potential commercial launch requires specific expertise. Rocket Pharmaceuticals, Inc. has bolstered its team with key appointments throughout 2025 to manage this transition.

Specialized talent includes recent high-level appointments:

• The new Chief Operating Officer, Christopher Stevens.
• The new Chief Medical Officer, Dr. Syed Rizvi.
• The addition of Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer.

Regulatory and Commercial Assets

Regulatory designations translate directly into potential future value and market advantage. These assets de-risk the path to market for specific products.

A critical asset is the Rare Pediatric Disease Designation for KRESLADI™ (for severe LAD-I). This designation carries the potential for a Priority Review Voucher (PRV) upon FDA approval, which is a highly valuable, transferable asset.

Furthermore, the pipeline programs carry their own strategic designations:

• RP-A601 for PKP2-ACM has received FDA RMAT designation (Regenerative Medicine Advanced Therapy).
• RP-A701 for BAG3-DCM has received FDA Fast Track designation.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Value Propositions

Potential one-time curative genetic therapies for devastating rare diseases.

Rocket Pharmaceuticals, Inc. is advancing therapies targeting conditions with high unmet medical need, evidenced by its late-stage pipeline focus.

Addressing ultra-rare, life-threatening disorders with high unmet medical need.

• Severe Leukocyte Adhesion Deficiency-I (LAD-I) is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant.
• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) affects approximately 50,000 adults and children in the U.S. and Europe.
• The cardiovascular programs collectively target over 100,000 patients across the U.S. and EU.

KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe LAD-I offers a potential alternative to bone marrow transplant.

Clinical data from the global Phase 1/2 study for KRESLADI™ demonstrated 100% overall survival at 12 months post-infusion for all enrolled patients. The Prescription Drug User Fee Act (PDUFA) target action date for the Biologics License Application (BLA) resubmission is set for March 28, 2026. Approval would make Rocket Pharmaceuticals eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). The therapy was well tolerated with no treatment-related serious adverse events.

Focused AAV platform for cardiovascular diseases (Danon, PKP2-ACM, BAG3-DCM).

The company has sharpened its strategic focus on its adeno-associated virus (AAV) cardiovascular gene therapy portfolio. The cash, cash equivalents, and investments as of September 30, 2025, totaled $222.8 million, expected to fund operations into the second quarter of 2027. This focus was supported by a strategic reorganization that included a workforce reduction of approximately 30%, expected to lower 12-month operating expenses by around 25%.

Program (Indication)	Platform/Vector	Latest Status (Late 2025)	Key Efficacy/Status Metric
KRESLADI™ (Severe LAD-I)	Lentiviral (LV)	BLA Resubmission Accepted	100% overall survival at 12 months post-infusion.
RP-A501 (Danon Disease)	AAV	Clinical Hold lifted in August 2025	Dosing for Phase 2 pivotal trial to resume in 1H 2026.
RP-A601 (PKP2-ACM)	AAV	Received RMAT Designation	All 3 adult patients showed increased PKP2 protein expression.
RP-A701 (BAG3-DCM)	AAV	IND Clearance Received	Preparing for first-in-human evaluation.

Regenerative Medicine Advanced Therapy (RMAT) designation for RP-A601.

The U.S. Food and Drug Administration (FDA) granted RMAT designation to RP-A601 for PKP2-ACM. This designation was supported by Phase 1 trial data where all three adult patients treated with a single dose showed improved heart structure and function. Specific data points included:

• Increased PKP2 protein expression, with 110% & 398% increase in 2 patients with low baseline levels.
• 34-41 point increases in KCCQ-12 scores.
• NYHA class improvement from II to I.

This designation enables more intensive FDA guidance and an expedited review process for the therapy, which marks Rocket Pharmaceuticals, Inc.'s fifth RMAT designation.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Relationships

You're building a commercial foundation for gene therapies, which means the relationship with the treating centers and the medical community is everything. For Rocket Pharmaceuticals, Inc., this is a high-touch, highly specialized engagement model, especially given the complexity of administering these one-time treatments.

High-touch, direct engagement with specialized treatment centers and physicians.

The focus is on centers capable of handling complex gene therapy administration. This requires deep scientific exchange, not just sales calls. For instance, the PKP2-ACM program, targeting about 50,000 people in the U.S. and Europe, necessitates working with specialized cardiology centers experienced in inherited heart disease. The collective cardiovascular portfolio targets over 100,000 patients across the U.S. and EU. Engagement is driven by clinical milestones; for example, the FDA lifting the clinical hold on the RP-A501 Danon disease study in August 2025 immediately intensified coordination with those treating centers to resume dosing of additional patients in the first half of 2026. The specific dosing protocol for the next three patients involves a recalibrated dose of 3.8 × 10¹³ GC/kg with a modified immunomodulatory regimen, which requires precise physician training and oversight.

Close collaboration with patient advocacy groups for rare disease communities.

Rocket Pharmaceuticals, Inc. views patients and families as its North Star, which translates directly into deep ties with advocacy groups. A key example is the company's longstanding collaboration with the Danon Foundation, where their commitment to research, advocacy, and patient support is integral to advancing the field. This partnership structure helps Rocket understand the patient journey, which is crucial when dealing with devastating rare disorders.

• Patients and families are the guiding force behind the mission.
• The company cultivates trusted partnerships with advocacy organizations.

Direct communication with regulatory agencies (FDA, EMA) for complex submissions.

Regulatory dialogue is constant and critical, especially given the late-stage nature of several assets. The relationship with the FDA has been highly active through 2025. You saw the FDA accept the Biologics License Application (BLA) resubmission for KRESLADI™ in October 2025, which has a Prescription Drug User Fee Act (PDUFA) date set for March 28, 2026. Furthermore, the company secured a Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA for RP-A601 (PKP2-ACM) in July 2025, following positive Phase 1 data. The ability to align with the FDA quickly to lift the clinical hold on the RP-A501 Danon disease trial in under three months in August 2025 demonstrates a working, if intense, relationship with health authorities.

Dedicated medical affairs and commercial teams for patient support and access.

As Rocket Pharmaceuticals, Inc. moves toward potential commercialization, the structure of its customer-facing teams has been refined. Sarbani Chaudhuri joined as Chief Commercial & Medical Affairs Officer in April 2025, bringing experience from Johnson & Johnson, AstraZeneca, and Pfizer, specifically in rare cardiac and hematology diseases. However, the company made a strategic pivot in mid-2025, reducing its workforce by approximately 30% to align with pipeline prioritization, which impacts the scale of these dedicated teams. This cost-saving measure is reflected in the financial reporting.

Metric/Expense Category	Q3 2025 Amount (USD)	Comparison Period	Change
General and Administrative (G&A) Expenses	$18.4 million	Q3 2024	Decrease of $8.7 million
Commercial Preparation-Related Expenses (Included in G&A)	Not specified	Q3 2024	Decrease of $6.6 million
Total Employees	299 (As of late 2025)	N/A	Workforce reduced by approx. 30% in 2025

The decrease of $6.6 million in commercial preparation-related expenses within G&A expenses for the third quarter of 2025, compared to the third quarter of 2024, shows the immediate financial impact of the strategic realignment away from broader commercial readiness for paused programs.

Personalized patient support programs for complex gene therapy administration.

For the Danon disease program, the Phase 2 pivotal trial is designed around a small, highly monitored group. The trial evaluates efficacy and safety in 12 patients, which includes a pediatric run-in cohort of n=2. This small number underscores the personalized, intensive support required for each patient receiving an investigational gene therapy like RP-A501. The company's overall cash position as of September 30, 2025, was $222.8 million, which is being managed to fund operations into the second quarter of 2027, ensuring resources are available for these critical, high-touch patient interactions.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Channels

You're looking at the distribution and communication channels for Rocket Pharmaceuticals, Inc. (RCKT) as they transition from late-stage development to potential commercialization for their rare disease gene therapies in late 2025. Given the nature of these treatments, the channels are highly specialized, focusing on centers of excellence and regulatory milestones.

Highly specialized gene therapy treatment centers (ATCs) for administration

For gene therapies targeting ultra-rare disorders like severe Leukocyte Adhesion Deficiency-I (LAD-I) with KRESLADI™, the channel is inherently limited to specialized centers capable of administering complex cell and gene therapies. While a specific number of Authorized Treatment Centers (ATCs) isn't publicly quantified as of late 2025, the strategy is clearly focused on high-acuity sites. The market opportunity for their cardiovascular pipeline alone-Danon disease, PKP2-ACM, and BAG3-DCM-represents over 100,000 patients in the U.S. and EU, suggesting a targeted network of transplant or specialized cardiology centers will be necessary for future launch. The company's internal focus on AAV cardiovascular programs reinforces this need for specialized infusion sites.

Direct sales and medical affairs teams targeting key opinion leaders (KOLs)

Rocket Pharmaceuticals, Inc. is still pre-revenue as of the third quarter ending September 30, 2025, reporting $0.0 million in revenue. Therefore, the team build-out is focused on medical affairs and commercial preparation rather than broad sales execution. The company made key leadership additions in 2025 to bolster this, including appointing Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer in April 2025. Commercial preparation-related expenses were noted at $1.5 million for the three months ended March 31, 2025. The channel strategy involves engaging Key Opinion Leaders (KOLs) within the specific rare disease communities, such as the estimated 50,000 PKP2-ACM affected individuals in the U.S. and Europe.

Regulatory submission pathways (BLA/MAA) to the FDA and European Medicines Agency

Regulatory milestones are the primary channel driver for a pre-commercial company. Rocket Pharmaceuticals, Inc. has seen significant shifts in this area in late 2025, prioritizing programs with clearer pathways.

Program/Therapy	Regulatory Action/Status (as of late 2025)	Agency
KRESLADI™ (LAD-I)	FDA acceptance of BLA resubmission in October 2025. Complete BLA submission anticipated before the end of 2025.	FDA
KRESLADI™ (LAD-I)	PDUFA date anticipated in March 2026.	FDA
RP-L102 (Fanconi Anemia)	Voluntarily withdrew U.S. BLA on October 3, 2025, based on strategic considerations.	FDA
RP-L102 (Fanconi Anemia)	Withdrew Marketing Authorization Application (MAA) in July 2025.	European Medicines Agency (EMA)

The company is focusing resources on its AAV cardiovascular programs, which have clear biomarker opportunities for accelerated approval.

Clinical trial sites for patient identification and initial treatment access

Clinical trial sites serve as the initial, most critical access points for patients, especially for therapies addressing conditions like Danon disease, which affects an estimated 15,000 to 30,000 people in the US and Europe. The channel is defined by the active studies:

• Phase 2 pivotal study for RP-A501 (Danon disease) is ongoing following the lifting of a clinical hold.
• Phase 1 trial start-up activities are underway for RP-A701 (BAG3-DCM).
• Initial data from the Phase 1 study of RP-A601 (PKP2-ACM) was expected in May 2025.

The company has experienced a 30% workforce reduction to align resources with these late-stage cardiovascular programs.

Scientific publications and conferences to disseminate clinical data

Dissemination of clinical data through peer-reviewed channels and scientific presentations is a key channel for establishing credibility and informing the medical community. Rocket Pharmaceuticals, Inc. actively participated in major industry events in 2025 to present its pipeline progress.

• Presented updates at the Morgan Stanley 23rd Annual Global Healthcare Conference in September 2025.
• Presented at the 8th Annual Evercore Healthcare Conference in December 2025.
• Corporate Presentation and specific data updates (RP-A601, RP-A501) were shared in November 2025.

The company's experience from treating over 15 cardiac patients informs its late-stage AAV programs.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Customer Segments

You're looking at the patient populations Rocket Pharmaceuticals, Inc. (RCKT) is targeting with its gene therapy pipeline as of late 2025. The focus is clearly on ultra-rare, genetically defined diseases, especially in the cardiovascular space, which dictates resource allocation.

The core customer segments are defined by specific, devastating genetic disorders requiring one-time, potentially curative treatments. The company has strategically prioritized its AAV cardiovascular platform, which includes three major cardiomyopathy programs, alongside its LV hematology program for severe LAD-I.

Here's a breakdown of the addressable patient populations based on the latest internal estimates and prevalence data:

Disease Indication	Therapy Program	Estimated Prevalence (U.S. and Europe)	Market Significance
PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM)	RP-A601 (AAV)	Approximately 50,000 people	Largest market opportunity in disclosed pipeline programs
Danon disease	RP-A501 (AAV)	15,000 to 30,000 patients	Late-stage clinical program
PKP2-ACM and BAG3-DCM (Combined with Danon Disease)	RP-A601 (AAV) and RP-A701 (AAV)	More than 100,000 patients	Collectively target major genetically defined cardiomyopathies
BAG3-associated dilated cardiomyopathy (BAG3-DCM)	RP-A701 (AAV)	Included in the >100,000 total for the three cardiomyopathies	Preclinical/IND accepted status

The other critical segment involves patients with severe LAD-I, for whom KRESLADI™ is seeking approval. This is a severe pediatric genetic disorder causing life-threatening infections.

• Pediatric and adult patients with severe Leukocyte Adhesion Deficiency-I (LAD-I) targeting a March 28, 2026 PDUFA date for KRESLADI™.
• Ultra-rare disease populations, definitely defined by specific genetic mutations, as the entire portfolio is focused on monogenic disorders.

The financial reality of supporting these segments as of the third quarter of 2025 shows significant investment in R&D:

• Q3 2025 Net Loss was $50.3 million.
• Total Operating Expense for Q3 2025 was $52.2 million.
• Research and Development Expenses for Q3 2025 accounted for $34.1 million.
• Cash, cash equivalents, and investments stood at $75.9 million at the end of Q3 2025.
• The company expects current resources to fund operations into the second quarter of 2027.

The cash position at the end of Q1 2025 was $318.2 million.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Cost Structure

You're looking at the cost side of the ledger for Rocket Pharmaceuticals, Inc. (RCKT) as of late 2025. For a late-stage biotech focused on gene therapy, the cost structure is heavily weighted toward the science and the path to the clinic and market. It's all about burning cash to create future value, so every dollar spent on R&D and trials is a strategic decision.

Here's a quick look at the headline operating expenses from the third quarter ending September 30, 2025:

Expense Category	Q3 2025 Amount
Research and Development (R&D) Expenses	$34.1 million
General and Administrative (G&A) Expenses	$18.4 million
Total Operating Expenses (R&D + G&A)	$52.5 million

The primary cost driver, as you'd expect, is Research and Development. For the three months ended September 30, 2025, Rocket Pharmaceuticals reported R&D expenses of $34.1 million. This figure reflects a disciplined approach to resource allocation following the company's organizational realignment earlier in the year.

General and Administrative costs also factor in significantly, though they saw a reduction year-over-year. G&A expenses totaled $18.4 million for the third quarter of 2025, down from $27.1 million in the same period of 2024. This decrease was largely due to lower commercial preparation-related expenses, which dropped by $6.6 million, plus a $1.5 million reduction in non-cash stock-based compensation expense.

The high cost of in-house cGMP manufacturing and quality control is an inherent part of Rocket Pharmaceuticals' structure, especially given their Cranbury, N.J. R&D and manufacturing facility. While specific standalone costs aren't broken out, the R&D expense line reflects these activities. For instance, the R&D spend in Q3 2025 included decreases in manufacturing and development and direct material costs of $3.6 million.

Clinical trial costs for late-stage pivotal studies represent a major, lumpy expense. Rocket Pharmaceuticals is heavily invested in advancing its AAV cardiovascular gene therapy platform, which includes:

• Phase 2 pivotal study for Danon disease (RP-A501).
• Phase 1 study for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) (RP-A601).

The focus on these programs means significant outlay for patient enrollment, monitoring, and data analysis. The Q3 2025 R&D reduction was partly due to a $2.4 million decrease in clinical trial expenses compared to the prior year, showing the impact of cost optimization efforts.

Finally, there are one-time charges related to strategic shifts. Rocket Pharmaceuticals incurred restructuring and severance charges of approximately $3.3 million in 2025 as part of its reorganization plan to narrow pipeline focus and extend its operational runway into the second quarter of 2027. That's a short-term hit to manage the long-term burn rate.

Finance: draft 13-week cash view by Friday.

Rocket Pharmaceuticals, Inc. (RCKT) - Canvas Business Model: Revenue Streams

You're looking at the revenue picture for Rocket Pharmaceuticals, Inc. as of late 2025. Honestly, the current picture is what you'd expect for a company deep in late-stage development, but the near-term catalysts are clear.

Current product revenue is $0.0 million for Q3 2025 (pre-commercial stage). This reflects the reality that Rocket Pharmaceuticals does not yet have commercialized products generating sales, which is consistent with their reported net loss of $50.3 million for that same quarter ending September 30, 2025.

The first significant non-dilutive revenue opportunity is the potential sale of a Priority Review Voucher (PRV) upon KRESLADI™ approval. Analysts estimate this PRV could be valued at over $150 million, which would directly support the company's cash position, currently reported at $222.8 million as of September 30, 2025.

Future product sales of KRESLADI™ (marnetegragene autotemcel; marne-cel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) are tied to a key regulatory date. The Prescription Drug User Fee Act (PDUFA) target action date set by the FDA is March 28, 2026. The data supporting this Biologics License Application (BLA) showed compelling results in the global Phase 1/2 study:

Metric	Result
Overall Survival at 12 Months Post-Infusion	100%
Primary and Secondary Endpoints Met	Yes
Treatment-Related Serious Adverse Events	0

The company has strategically narrowed its focus to maximize the value of its AAV cardiovascular gene therapy portfolio, which represents the next major revenue driver post-KRESLADI™. This focus was reinforced by a workforce reduction of approximately 30% to extend the cash runway into the second quarter of 2027, excluding any PRV proceeds.

This AAV portfolio is centered on rare cardiomyopathies, with specific programs providing future sales potential:

• RP-A501 for Danon Disease (late-stage pivotal Phase 2 trial resuming in 1H 2026).
• RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM) (advancing toward a pivotal Phase 2 study).
• RP-A701 for BAG3-associated dilated cardiomyopathy (DCM) (preparing for first-in-human evaluation).

The market potential for these assets is substantial; for instance, the Danon disease treatment market is projected to grow at a 6.5% CAGR through 2030. The company is betting on this concentrated pipeline for long-term value creation.

Finally, potential upfront or milestone payments from out-licensing non-core assets remain a possible, though secondary, revenue stream. Given the recent strategic reorganization and the decision to focus exclusively on the AAV cardiovascular platform, any non-core asset divestiture would be a deliberate action to further conserve cash and fund the prioritized pipeline.