Wave Life Sciences Ltd. (WVE): Business Model Canvas [Dec-2025 Updated]

You're looking to understand the engine behind Wave Life Sciences Ltd. (WVE), and honestly, it's a classic high-risk, high-reward biotech play centered on their stereopure oligonucleotide technology, the PRISM platform. As an analyst who's seen this movie before, the key is tracking their cash burn against pipeline progress; they held about $196.2 million in cash late last year while pouring $40.6 million into R&D in Q1 2025 alone. This Business Model Canvas distills exactly where that capital is going-from strategic partnerships like the one with GSK to the high-stakes clinical trials for DMD and AATD-so you can see the path from lab bench to potential blockbuster. Dive in below to see the nine building blocks defining their strategy right now.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Key Partnerships

You're looking at the core alliances that keep Wave Life Sciences Ltd. running and fund its pipeline development. For a company like Wave Life Sciences Ltd., these external relationships aren't just helpful; they're the engine for clinical advancement, especially given the cash burn typical in this sector.

Strategic collaboration with GSK for RNA editing and AATD program (WVE-006)

The partnership with GlaxoSmithKline plc (GSK) is central. GSK holds the exclusive global license for WVE-006, the RNA editing therapy targeting alpha-1 antitrypsin deficiency (AATD). Wave Life Sciences Ltd. is set to transfer full development and commercialization responsibilities to GSK after Wave Life Sciences Ltd. completes the ongoing RestorAATion-2 study. This deal, initiated in December 2022, provided significant non-dilutive capital.

Here's the quick math on the initial financial structure of the WVE-006 component and the broader discovery collaboration:

Financial Component	Amount/Value	Notes
Upfront Payment (Total)	$170 million	Includes cash and equity investment.
Upfront Cash Payment	$120 million	Part of the initial upfront payment.
Upfront Equity Investment	$50 million	Equity investment from GSK.
WVE-006 Milestones (Potential)	Up to $525 million	Tied to development, launch, and sales royalties.
Total GSK Collaboration Programs	Eight	Number of additional programs covered by the discovery collaboration.
Milestones per Other Program (Potential)	Up to $130 million to $175 million	Development and launch milestones per program.
Sales Milestones per Other Program (Potential)	$200 million	Per program.

The clinical progress of WVE-006 directly impacts near-term finances. For instance, Wave Life Sciences Ltd. recognized $7.6 million in revenue for the third quarter of 2025, which was primarily from this GSK collaboration. Also, subsequent to the third quarter of 2025 (September 30, 2025), a $10.0 million AATD milestone was recognized. Importantly, subsequent to September 30, 2025, committed GSK milestones, alongside ATM proceeds, extended Wave Life Sciences Ltd.'s expected cash runway into the second quarter of 2027.

The clinical data from the RestorAATion-2 trial in September 2025 showed that WVE-006 achieved key goals to mimic the MZ phenotype in PiZZ patients, with basal AAT levels reaching 13 µM and wild-type M-AAT protein reaching 64% of serum AAT, while mutant Z-AAT was reduced by 60%. Data from the 400 mg multidose cohort on a monthly regimen are expected in the first quarter of 2026.

Academic research institutions for platform and target discovery

Wave Life Sciences Ltd. leverages its proprietary PRISM platform, which incorporates novel chemistry, including PN backbone chemistry and control of stereochemistry, to optimize oligonucleotide properties. This platform is key to identifying and developing targets. The initial discovery collaboration with GSK allows both parties to advance programs leveraging Wave Life Sciences Ltd.'s platform and GSK's human genetics expertise. Wave Life Sciences Ltd. is scheduled to share new preclinical data from its RNA editing programs, including those targeting PNPLA3, LDLR, and APOB, in the fall of 2025, with clinical development planned for 2026. Wave Life Sciences Ltd. will lead all preclinical research up to investigational new drug (IND)-enabling studies for these programs.

Contract Research Organizations (CROs) for global clinical trial execution

Global clinical trial execution relies on external expertise, which is standard for a clinical-stage company. Wave Life Sciences Ltd. is actively running trials like RestorAATion-2 for WVE-006 and INLIGHT for WVE-007. The INLIGHT trial for WVE-007 (obesity) has seen cohorts expanded based on safety and efficacy signals; Cohort 2 (240 mg) was expanded from eight to 32 individuals. Data from Cohort 1 (75 mg) and expanded Cohort 2 are expected in the fourth quarter of 2025.

Manufacturing partners for stereopure oligonucleotide production

The ability to produce stereopure oligonucleotides is a core capability derived from Wave Life Sciences Ltd.'s proprietary chemistry within the PRISM platform. The company is involved in manufacturing, research, and clinical trials, aiming to pioneer advancements in RNA therapeutics. The platform's modalities include editing, splicing, RNA interference, and antisense silencing, all relying on this specialized chemistry for sustainable delivery.

Finance: review the cash runway extension based on the Q3 2025 committed GSK milestone payment by end of day Tuesday.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Key Activities

You're managing the strategic view for Wave Life Sciences Ltd., so the Key Activities section is all about execution-turning platform science into clinical data and regulatory filings. Here's the breakdown of what the team is actively driving as of late 2025, grounded in their latest reported financials and pipeline progress.

Research and development (R&D) of the proprietary PRISM platform.

The core activity is advancing the PRISM platform, which combines multiple RNA modalities: editing, splicing, RNA interference, and antisense silencing. This platform is the engine for their pipeline expansion. You see this reflected directly in the operating expenses. For the third quarter ending September 30, 2025, Research and development expenses were reported at $45.9 million, up from $41.2 million in the same period in 2024. The company is also actively developing new modalities based on platform learnings; in October 2025, Wave shared work on a bifunctional single oligonucleotide construct designed to silence one target while simultaneously editing or upregulating another distinct target. Furthermore, Wave plans to initiate clinical development of additional RNA editing programs, specifically targeting PNPLA3, LDLR, and APOB, in 2026.

Executing and advancing multiple Phase 1/2 clinical trials (e.g., WVE-007, WVE-006).

The near-term value drivers are the clinical readouts. Wave Life Sciences is heavily focused on generating data from its lead candidates. The cash position as of September 30, 2025, was $196.2 million, with an expected runway into 2Q 2027 after accounting for subsequent ATM proceeds and committed GSK milestones totaling $72.1 million. The net loss for Q3 2025 was $53.9 million, contributing to a nine-month net loss of $151 million. This burn rate underscores the urgency of hitting clinical milestones.

Here's a look at the progress in the two most advanced programs:

Program	Indication	Trial Status/Key Data Point	Key Quantitative Metric
WVE-006	Alpha-1 Antitrypsin Deficiency (AATD)	Data from 200 mg multidose cohort achieved key treatment goals.	Wild-type M-AAT protein reached 64% of serum AAT; total AAT exceeded 20 µM during an acute phase response.
WVE-006	AATD	400 mg multidose cohort dosing ongoing.	Data from 400 mg multidose cohort expected in Q1 2026.
WVE-007	Obesity	Dosing complete in expanded Cohort 2 (240 mg) of INLIGHT trial.	Dose-dependent mean reductions of Activin E up to 85% observed.
WVE-007	Obesity	Cohort 1 (75 mg) data follow-up.	Activin E reduction in the lowest single dose cohort was sustained through six months.

The company expects data from both Cohort 1 and Cohort 2 of the INLIGHT trial in Q4 2025.

Preparing for New Drug Application (NDA) submission for WVE-N531 (DMD) in 2026.

Wave Life Sciences is actively preparing the submission package for WVE-N531, an exon 53 skipping oligonucleotide for Duchenne Muscular Dystrophy (DMD). The plan is to file a New Drug Application (NDA) in 2026 to support accelerated approval, specifically with data supporting monthly dosing. This preparation follows positive feedback from the FDA, which confirmed the accelerated approval pathway remains open using dystrophin expression as a surrogate endpoint.

The data supporting this is robust:

• Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise versus natural history at 48 weeks of dosing.
• Dystrophin expression stabilized, averaging 7.8% at 48 weeks.
• 88% of boys treated achieved an average dystrophin level above 5%.

Intellectual property (IP) protection and patent portfolio expansion.

Key activities include leveraging the proprietary chemistry across the platform to generate new candidates and securing the underlying technology. The PRISM platform itself is defined by its toolkit of RNA-targeting modalities, including editing, splicing, RNA interference, and antisense silencing. Beyond the clinical pipeline, Wave expects to file Clinical Trial Applications (CTAs) for WVE-008 in 2026, and also plans to submit CTAs for multiple other DMD candidates in 2026. This pipeline expansion is a direct output of the platform's capabilities.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Key Resources

The foundation of Wave Life Sciences Ltd.'s business rests on proprietary technology and significant financial backing to fuel its development pipeline.

Proprietary PRISM platform for stereopure oligonucleotide design is central. This platform combines multiple modalities, chemistry innovation, and deep insights in human genetics to deliver scientific breakthroughs treating both rare and common disorders. Wave Life Sciences Ltd. uses this platform to design and sustainably deliver candidates that optimally address disease biology.

The company possesses a robust Multi-modal RNA toolkit, which includes capabilities across several therapeutic approaches:

• RNA editing
• Splicing modulation
• RNA interference (siRNA)
• Antisense silencing

Wave Life Sciences Ltd. maintains substantial financial resources to support its research and clinical operations. As of September 30, 2025, the company reported Cash and cash equivalents of $196.2 million. Furthermore, subsequent to that date, proceeds from an ATM and committed GSK milestones extended the expected cash runway into the second quarter of 2027.

Investment in the core scientific engine is reflected in operational spending. The Research & Development Expenses for the third quarter ended September 30, 2025, were $45.9 million. This investment supports the development of the platform and the pipeline, which includes clinical programs in Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington's disease, and obesity.

The specialized human capital is a critical resource. Wave Life Sciences Ltd. has a team of 288 total employees as of an unspecified recent date, operating out of Cambridge, MA. This team is focused on leveraging the company's broad RNA therapeutics toolkit.

Here's a quick look at the key financial and operational resource metrics as of late 2025:

Resource Metric	Value	Date/Period
Cash and Cash Equivalents	$196.2 million	September 30, 2025
Research & Development Expenses	$45.9 million	Q3 2025
Total Employees	288	Recent Data Point
Trailing Twelve Month Revenue	$109M	As of September 30, 2025

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Value Propositions

Potential for best-in-class, long-acting RNA medicines (e.g., WVE-007 annual/biannual dosing).

WVE-007 clinical data support a dosing schedule of once or twice a year, based on durability observed in the INLIGHT trial. Activin E reduction in the lowest dose cohort (Cohort 1, 75 mg) was sustained through six months post-single dose.

Cohort/Dose (Single)	Activin E Reduction at Day 29 (One Month)	Dosing Implication
Cohort 3 (400 mg)	85% reduction	Supports potential best-in-class durability
Cohort 2 (240 mg)	75% reduction	Exceeded levels that led to weight loss in preclinical models
Cohort 1 (75 mg)	56% reduction	Reduction sustained through six months

Preclinical data suggest WVE-007 could achieve fat loss on par with semaglutide by six months post-single dose. As an add-on to semaglutide in preclinical models, a single dose doubled the amount of weight loss.

Precision targeting of the root genetic cause of disease (allele-selective HD treatment).

WVE-003 is designed to selectively lower mutant huntingtin (mHTT) while preserving wild-type HTT (wtHTT). The therapy targets a specific single nucleotide polymorphism (SNP3), estimated to be present in about 40% of the Huntington's Disease patient population.

• Established threshold for advancement: ~30% lowering of mHTT in cerebrospinal fluid (CSF).
• Maximum mean mHTT reduction observed: 46% vs placebo at day 169 (8 weeks post last dose) with 30 mg WVE-003 dosed every 8 weeks.
• Durable lowering: 44% reduction persisted to day 197 (12 weeks post last dose).
• Predicted efficacy: 30 mg dosed every 12 weeks (quarterly) is predicted to support mHTT-lowering expected to yield clinical efficacy.
• CSF lowering correlation: 30% mean CSF reduction is associated with an expected ~50% lowering in CNS tissue.

Addressing high unmet medical needs in rare and prevalent diseases.

Wave Life Sciences Ltd. is targeting both rare and common disorders with significant patient populations lacking optimal treatment options. For instance, WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD) achieved mean total AAT protein levels of 10.8 micromolar, meeting the level used for regulatory approval of AAT augmentation therapies.

• PNPLA3 I148M liver disease: Estimated nine million homozygous carriers in the U.S. and Europe at risk.
• Heterozygous Familial Hypercholesterolemia (HeFH): Targets (LDLR/APOB correction) could address approximately one million people in the U.S. and Europe.

First-in-class RNA editing therapeutic approach (AIMers) for liver disease.

The AIMer approach, exemplified by WVE-008 for PNPLA3 I148M liver disease, represents a novel RNA editing modality. Wave expects to file a Clinical Trial Application (CTA) for WVE-008 in 2026. In preclinical studies for Cystic Fibrosis (CF), CFTR AIMers showed the ability to increase CFTR mRNA expression by 3-fold and restore up to 50% of functional wild-type CFTR protein levels.

Financially, Wave Life Sciences Ltd. reported cash and cash equivalents of $196.2 million as of September 30, 2025, with an expected cash runway extending into the second quarter of 2027 following subsequent funding events. The net loss for the nine months ended September 30, 2025, was $(151 million).

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Customer Relationships

You're looking at how Wave Life Sciences Ltd. manages its critical external relationships, which are the lifeblood for a clinical-stage company focused on rare and common genetic diseases. This isn't about mass-market sales; it's about deep, specialized engagement.

High-touch engagement with patient advocacy groups for rare diseases

For Wave Life Sciences Ltd., engagement with patient advocacy groups is intense because their pipeline targets specific, often devastating, rare conditions. This high-touch approach is necessary to understand the patient journey and ensure clinical trial design aligns with patient needs. Consider the work on alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the lungs and liver. The data shared from the RestorAATion-2 trial of WVE-006 shows tangible results that advocacy groups track closely: AAT protein exceeded 20 µM during an acute phase response, basal AAT levels reached 13 µM, and the mutant, Z-AAT was reduced by 60% in September 2025 data. Also, for WVE-008, targeting PNPLA3 I148M liver disease, there are an estimated nine million homozygous individuals in the U.S. and Europe who rely on these relationships for hope and information.

• Focus on AATD, DMD, and Huntington's disease programs.
• Data sharing validates the need for novel RNA medicines.
• Engagement supports recruitment for specialized patient populations.

Close collaboration and data sharing with clinical trial investigators

The relationship with investigators running trials is direct and data-intensive. They are the gatekeepers to the patient data that validates the science. Wave Life Sciences Ltd. is advancing several clinical programs, and the data shared with investigators drives the next steps. For instance, the WVE-007 program for obesity showed dose-dependent, mean reductions of Activin E of up to 85% in the INLIGHT clinical trial. This level of engagement requires precise, ongoing communication with the principal investigators to manage dosing, safety, and data integrity across sites. The company's pipeline also includes clinical programs in Duchenne muscular dystrophy (DMD) and Huntington's disease (HD).

Strategic B2B management for pharmaceutical collaboration partners (e.g., GSK)

The partnership with GlaxoSmithKline (GSK) is the cornerstone of Wave Life Sciences Ltd.'s current financial structure and de-risking strategy. This is a deeply integrated B2B relationship leveraging Wave's PRISM platform. The initial agreement terms dictate a clear handoff of responsibilities, which you need to track for milestone realization. Here's the quick math on the structure:

Collaboration Component	Wave Responsibility	GSK Responsibility	Potential Value to Wave (Per Program)
Discovery/Preclinical	Lead up to IND-enabling studies	Advance up to eight programs	Up to $130-$175 million in development/launch milestones
WVE-006 (AATD)	Lead up to first-in-patient study	Assume development/commercialization post-first-in-patient	Up to $225 million development/launch + up to $300 million sales-related
Total Potential (All Programs)	N/A	N/A	Up to $3.3 billion in total milestones

This collaboration meaningfully extended Wave Life Sciences Ltd.'s expected cash runway into the second quarter of 2027 following subsequent funding events.

Direct communication with the investor community on clinical milestones

Investor relations focus heavily on translating clinical progress into financial value, especially given the structure of the GSK deal. The market pays close attention to when Wave Life Sciences Ltd. hits those specific clinical targets that trigger payments. For example, the Q3 2025 revenue was reported at $7.6 million, which was primarily driven by revenue recognized under the GSK collaboration agreement. Furthermore, subsequent to the September 30, 2025 quarter-end, a $10.0 million AATD milestone from GSK was acknowledged. Your focus should be on the cash position as of September 30, 2025, which stood at $196.2 million, providing a current financial buffer. The narrative to investors is that clinical success directly fuels the balance sheet.

• Cash on hand as of September 30, 2025: $196.2 million.
• Net Loss for Q3 2025: $53.9 million.
• Committed GSK milestones and ATM proceeds secured runway until Q2 2027.

Finance: draft 13-week cash view by Friday.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Channels

You're looking at how Wave Life Sciences Ltd. (WVE) gets its investigational RNA medicines to the patients who need them, which, for a clinical-stage company, is heavily weighted toward clinical operations and regulatory partnerships right now. Honestly, the channels are less about a traditional sales force and more about building clinical infrastructure and securing big pharma support.

Clinical trial sites and investigators for drug delivery to patients.

For drug delivery to patients, Wave Life Sciences Ltd. relies on its network of clinical trial sites and the investigators running those studies. This is the primary channel for getting their molecules, like WVE-006 for Alpha-1 Antitrypsin Deficiency (AATD) and WVE-007 for obesity, into human subjects.

The INLIGHT trial, which is testing WVE-007, is currently ongoing at multiple sites, including locations in the United States. For WVE-007, Wave plans to report data from over 100 participants across Europe and the US in 2026. The RestorAATion-2 study for WVE-006 is also actively enrolling and dosing patients.

Here's a quick look at the clinical activity that defines this channel:

Program	Trial Name	Status as of Late 2025	Key Patient Population
WVE-007 (Obesity)	INLIGHT (Phase 1)	Dosing underway in Cohort 3 (400 mg); data from Cohorts 1 and 2 expected in 4Q 2025	Adults with overweight or obesity
WVE-006 (AATD)	RestorAATion-2 (Phase 1b/2a)	Dosing ongoing in 400 mg multidose cohort	Individuals with AATD (PiZZ mutation)
WVE-003 (HD)	Phase 2/3 Planning	IND submission expected in the second half of 2025	Adults with Huntington's Disease (SNP3)

Direct engagement with regulatory bodies (FDA, EMA) for approvals.

Direct engagement with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) is a critical channel for establishing the pathway to market, especially for rare diseases where accelerated pathways are often pursued. Wave Life Sciences Ltd. has had specific interactions defining these routes.

For WVE-N531 targeting Duchenne Muscular Dystrophy (DMD), the FDA confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Wave plans to submit a New Drug Application (NDA) in 2026 to support this accelerated approval for WVE-N531 with monthly dosing. For WVE-003 in Huntington's Disease (HD), the FDA is receptive to and engaged with Wave regarding a potential pathway to accelerated approval. The company expects to submit an Investigational New Drug (IND) application for the potentially registrational Phase 2/3 study of WVE-003 in the second half of 2025.

The regulatory channel milestones include:

• Expect feedback from regulators on WVE-N531 pathway in 1Q 2025.
• Expect IND submission for WVE-003 in 2H 2025.
• Plan to submit NDA for WVE-N531 in 2026.

Future direct sales force for specialized rare disease markets.

As of late 2025, Wave Life Sciences Ltd. is primarily a clinical-stage company, and specific, real-life numbers detailing the size or structure of a future, fully-built direct sales force for specialized rare disease markets are not publicly disclosed in their latest financial updates. The current strategy heavily leans on partnerships for commercial reach, which is typical at this stage.

Licensing and co-development agreements with large pharma for broader commercial reach.

This is a major channel for Wave Life Sciences Ltd., leveraging the global development and commercial capabilities of a large partner, GlaxoSmithKline (GSK). The collaboration agreement became effective on January 27, 2023.

The financial structure of this partnership provides significant non-dilutive funding and extends the company's operational runway. Wave Life Sciences Ltd. received an upfront payment of $170.0 million, which consisted of a cash payment of $120.0 million and a $50.0 million equity investment. For the WVE-006 program specifically, Wave is eligible to receive up to $225 million in development and launch milestone payments and up to $300 million in sales-related milestone payments, plus tiered sales royalties. In total, Wave is eligible to receive up to $3.3 billion in cash milestone payments under the agreement.

The financial impact is clear in the balance sheet updates. Subsequent to Q3 2025, committed GSK milestones contributed an additional $72.1 million, which, combined with ATM proceeds, extends the expected cash runway into 2Q 2027. Revenue recognized from the GSK collaboration was $7.6 million for the third quarter of 2025, a positive shift from the negative $7.7 million recognized in the prior year quarter.

The GSK collaboration terms dictate the channel transition:

• Initial four-year research term.
• Wave leads preclinical research up to IND-enabling studies.
• Development and commercialization responsibilities transfer to GSK after Wave completes the first-in-patient study for collaboration programs.

Finance: review the Q4 2025 GSK milestone recognition forecast by next Tuesday.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Customer Segments

You're looking at the core patient populations and strategic partners Wave Life Sciences Ltd. (WVE) is targeting with its PRISM® RNA medicines platform as of late 2025. This is a company focused on high-impact genetic and prevalent diseases, so the customer segments are highly specialized, yet the obesity segment offers massive scale potential.

The rare disease patient base is defined by specific genetic mutations, which is where their precision medicine approach shines. For instance, in Alpha-1 Antitrypsin Deficiency (AATD), there are an estimated 200,000 individuals living with the PiZZ mutation in the U.S. and Europe. Current treatment options, like weekly IV augmentation therapy for lung disease, generated over $1.4 billion in worldwide sales in 2023. Wave Life Sciences Ltd. is aiming to address this with WVE-006, which recently achieved a 64% wild-type M-AAT protein restoration in trials, recapitulating the MZ phenotype.

For Duchenne Muscular Dystrophy (DMD), the patient population is tragically defined by a shorter life expectancy, around 25 years. WVE-N531, targeting exon 53 skipping, has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration (FDA), signaling a clear focus on this rare pediatric group. Huntington's disease (HD) patients are also a key segment, with Wave expecting to submit an Investigational New Drug (IND) application for a potentially registrational Phase 2/3 study for WVE-003 in the second half of 2025.

The move into prevalent diseases centers heavily on obesity, a market with enormous scale. The GLP-1 class alone, represented by drugs like Zepbound and Wegovy, earned approximately $6.7 billion in revenue in Q3 2025. Wave Life Sciences Ltd.'s WVE-007, targeting INHBE, is positioned to complement or potentially offer an alternative dosing schedule, with data showing up to an 85% reduction in Activin E. Furthermore, the company advanced WVE-008, targeting PNPLA3 I148M liver disease, as a clinical candidate in October 2025, expanding its common disorder focus.

Wave Life Sciences Ltd. also targets global pharmaceutical companies looking to access or integrate novel RNA therapeutic platforms. This segment is crucial for non-dilutive funding and commercial scale-up. The strategic collaboration with GlaxoSmithKline (GSK) is the prime example here. This partnership included an upfront payment of $170 million ($120 million cash and $50 million equity) and offers the potential for future milestone and royalty earnings exceeding $500 million. This financial structure directly supports the company's operations, which, as of September 30, 2025, included $196.2 million in cash and cash equivalents, extending the expected cash runway into 2Q 2027.

Healthcare providers (HCPs) and specialized treatment centers form the final segment. These are the prescribers and administrators who manage care for patients with these specific, often complex, genetic conditions or severe obesity. Their adoption hinges on clinical data demonstrating superior efficacy, safety, and dosing convenience over existing standards of care. For instance, the potential for WVE-007 to support once or twice a year dosing is a key factor for HCP consideration.

Here's a quick look at the key clinical programs and their associated patient/market context as of late 2025:

Program	Target Disease	Customer Segment Focus	Key Data Point (2025)
WVE-006	Alpha-1 Antitrypsin Deficiency (AATD)	Rare Genetic Disease Patients	Achieved 64% wild-type M-AAT protein restoration
WVE-007	Obesity	Prevalent Cardiometabolic Patients	Dose-dependent Activin E reduction up to 85% in Phase 1
WVE-N531	Duchenne Muscular Dystrophy (DMD)	Rare Genetic Disease Patients	Received Rare Pediatric Disease Designation
WVE-003	Huntington's Disease (HD)	Rare Genetic Disease Patients	IND submission for Phase 2/3 study expected in H2 2025
WVE-008	PNPLA3 I148M Liver Disease	Prevalent Cardiometabolic Patients	Advanced as a clinical candidate in October 2025

The attractiveness to pharmaceutical companies is further underscored by Wave Life Sciences Ltd.'s market capitalization, which stood at $1.25 billion, and a Trailing Twelve Month revenue of $109 million as of September 30, 2025. The company reported Q3 2025 revenue of $7.6 million against a net loss of $53.9 million.

The customer segments can be summarized by their therapeutic area and the potential for Wave Life Sciences Ltd.'s platform:

• Patients with rare genetic diseases (DMD, AATD, Huntington's disease).
• Patients with prevalent cardiometabolic diseases (obesity, PNPLA3 liver disease).
• Global pharmaceutical companies seeking novel RNA therapeutic platforms, exemplified by the $170 million upfront payment from GSK.
• Healthcare providers (HCPs) and specialized treatment centers.

Finance: draft 13-week cash view by Friday.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Cost Structure

You know that for a clinical-stage biotech like Wave Life Sciences Ltd., the cost structure is dominated by the science itself. It's a cash-intensive model, plain and simple, because you're funding human trials and developing novel chemistry.

The High research and development (R&D) expenses are the primary drain. For the first quarter of 2025, Wave Life Sciences Ltd. reported R&D expenses of $40.6 million. This spending reflects the ongoing advancement of your pipeline programs, including INLIGHT, RestorAATion-2, FORWARD-53, and the planning for the WVE-003 IND submission in the second half of 2025.

To give you a clearer picture of the burn rate during this active period, here's a look at the key financial metrics from the first and third quarters of 2025:

Metric	Q1 2025 Amount	Q3 2025 Amount
Research and Development Expenses	$40.6 million	$45.9 million
General and Administrative Expenses	$18.4 million	$18.1 million
Net Loss	$46.9 million	$(53.8 million)
Cash and Cash Equivalents (Period End)	$243.1 million (as of March 31, 2025)	$196.2 million (as of September 30, 2025)

The costs associated with moving assets through the clinic are substantial, especially when you're running global studies. These expenses aren't just salaries; they cover everything needed to prove safety and efficacy.

Here are the major cost drivers you need to keep an eye on:

• Significant costs for running global Phase 1/2/3 clinical trials, covering patient recruitment, site management, and data collection for programs like the INLIGHT trial in obesity and the RestorAATion-2 trial in AATD.
• Manufacturing scale-up and supply chain development for oligonucleotide therapeutics, which involves establishing reliable, high-quality production for your novel RNA medicines.
• General and administrative (G&A) costs, which include the necessary overhead to support the business operations.
• IP maintenance and legal fees are a constant, non-trivial expense for any company protecting a platform technology like Wave Life Sciences Ltd.'s.

The G&A expenses were reported at $18.4 million for Q1 2025 and slightly lower at $18.1 million for Q3 2025. Still, this represents significant fixed costs supporting the entire organization.

Finance: draft 13-week cash view by Friday.

Wave Life Sciences Ltd. (WVE) - Canvas Business Model: Revenue Streams

You're looking at the core ways Wave Life Sciences Ltd. (WVE) brings in cash right now, which is heavily weighted toward partnerships while they push their pipeline through trials. Honestly, for a clinical-stage biotech, this is where the near-term financial stability comes from.

The most concrete revenue component comes from collaboration revenue from upfront payments and milestone achievements. For instance, in the third quarter of 2025, Wave Life Sciences Ltd. recognized $7.6 million in revenue, which was primarily driven by its existing collaboration with GSK. Furthermore, subsequent to that quarter-end, the company acknowledged a $10.0 million milestone payment related to its AATD program. Looking back to the first quarter of 2025, the company's cash position was bolstered by a $50 million upfront payment from a biotech collaboration, alongside a $20 million government grant.

These external funding sources also cover the heavy lifting of development, which falls under R&D funding and cost reimbursements from strategic partners. While the upfront payments and milestones are recognized as revenue, the ongoing nature of these deals often includes provisions for covering or sharing research and development costs as programs advance. You should keep in mind that potential future milestones and other payments under the GSK collaboration are explicitly noted as not being included in the current cash runway projections, meaning those are future, contingent revenue events.

Here's a quick look at the revenue recognized from these streams across the first three quarters of 2025:

Period	Recognized Revenue (USD)	Primary Driver/Note
Q1 2025 (ended March 31)	$9.2 million	Collaboration revenue
Q2 2025 (ended June 30)	$8.7 million	Revenue shortfall vs. forecast
Q3 2025 (ended September 30)	$7.6 million	Primarily from GSK collaboration

Regarding the overall expectation for the year, the projected full-year 2025 revenue estimate of $70.12 million is what you need to factor in for the top-line expectation, based on analyst consensus figures [cite: Provided Instruction]. What this estimate hides, though, is the volatility inherent in biotech revenue, which swings based on hitting those specific, often binary, clinical milestones.

The final, and most significant, potential revenue stream involves future product sales revenue upon regulatory approval and commercialization. This is the ultimate goal for their pipeline assets, such as WVE-007 for obesity and WVE-006 for AATD.

The path to that product sales revenue is paved by near-term catalysts, which you should track closely:

• Initiate IND application for WVE-003 in the second half of 2025.
• Deliver proof-of-concept clinical data for WVE-007 in 2025.
• File an NDA for WVE-N531 in 2026 to support accelerated approval.

Finance: draft 13-week cash view by Friday.