Great — below is a concise, source-backed market reference for the Haemophilia Gene Therapy Market, organized exactly by your headings and finishing with company references + public values. I ran the latest filings/news so the regulatory and commercial items are current (sources cited after the key paragraphs).

Read complete report at: https://www.thebrainyinsights.com/report/haemophilia-gene-therapy-market-13217

Market sizing (short summary)

• Public market estimates vary because some firms treat the segment narrowly (only approved gene-therapies) while others include pipeline/value-based pricing and adjacent services. Examples:  estimates ~USD 2.6B in 2024 and fast growth to 2035;  and others report smaller base numbers for 2023–2024 but project high CAGRs (20%+ in some niche estimates). Use a range: ~USD 0.4–2.6 billion (2023–2024 base) with forecasts showing multi-billion values by 2030 depending on uptake. 

Recent Development

• First wave of approvals and commercial launches for haemophilia gene therapies has begun: CSL’s HEMGENIX® (etranacogene dezaparvovec) and Pfizer’s BEQVEZ™ (fidanacogene elaparvovec) for hemophilia B are now approved in major markets; BioMarin’s ROCTAVIAN® (valoctocogene roxaparvovec) has regulatory milestones/approvals for hemophilia A in major regions and ongoing post-authorization commitments. These approvals have moved the market from “promise” to early commercialization and real-world rollout. 

Drivers

1. One-time durable therapies that can substantially reduce bleeding rates and the lifetime cost/burden of prophylactic protein replacement — high clinical value and patient interest. 

2. High list prices for single-dose gene therapies (pricing in the multi-million USD range) makes the market economically significant even at modest patient penetration. 

3. Growing infrastructure for advanced biologic manufacturing, pay-for-performance/value contracting pilots and payer willingness to discuss novel payment models. 

Restraints

• Limited eligible patient pool (rare disease prevalence + exclusion criteria such as pre-existing neutralizing antibodies to AAV vectors).

• Uptake friction: complexity of delivery (specialised centers), long-term durability / safety uncertainty, and payer negotiation hurdles.

• Regulatory & safety scrutiny (long post-marketing follow-up obligations and careful benefit/risk assessments). 

Regional segmentation analysis

• North America: leader in early uptake (largest spend, early reimbursement conversations, manufacturer launches).

• Europe: mixed — EMA approvals/recommendations exist for some products but national reimbursement and adoption vary (some conditional/managed entry). 

• Asia-Pacific: high growth potential (large patient pools in China/India) but slower commercial rollout because of pricing and infrastructure; some regional regulatory approvals/paths under discussion. 

Emerging Trends

• Multiple approved hemophilia-B gene therapies competing on durability, safety, and pricing (Hemgenix, Beqvez, others in clinic). 

• Caution / reshaping of pipelines after some program setbacks and commercial realities (partnering changes / terminations have occurred).

• Greater focus on payer models (outcomes-based contracts, annuity payments) and center-of-excellence certification for administration and follow-up. 

Top Use Cases

1. Severe / moderate haemophilia B — primary current commercial indication for approved therapies (one-time FIX production). 

2. Hemophilia A gene therapies (future / conditional approvals) — large addressable population if durable and safe (BioMarin’s ROCTAVIAN is the leading hemophilia-A program entering real-world use). 

Major Challenges

• Durability & safety follow-up (long-term surveillance required for potential declines in factor levels or late adverse events).

• Payer access & pricing controversy (single-dose multi-million USD prices create political and payer pushback). 

• Operational readiness (specialist centers, viral vector supply, manufacturing scale). 

Attractive Opportunities

• High-value, one-time treatments mean commercial upside for manufacturers that can navigate payers and demonstrate durable benefit.

• Regional roll-outs and manufacturing localisation (CMO contracts and capacity expansion to serve Europe/APAC). 

• New entrants and second-generation vectors that can address current limitations (antibody prevalence, durability).

Key factors of market expansion

• Regulatory approvals and label expansions for both hemophilia A and B. 

• Payer reimbursement models that enable access (outcomes-based/annuity) and willingness to fund single high-cost products.

• Demonstrated long-term durability and real-world safety to convince physicians, payers and patients. 

Company references (major players) — company + public value(s) / market facts

Below are the leading companies most commonly cited in market/clinical reports, with the most relevant public figures (revenues, approvals, program updates). Use them as directional benchmarks; for modeling or procurement please confirm with company filings.

1. CSL / CSL Behring — HEMGENIX® (etranacogene dezaparvovec) — first approved hemophilia B gene therapy (FDA approval: Nov 22, 2022; Swissmedic authorisation noted in Jan 2024). CSL Group operating revenue ~USD 14.8 billion (FY2024 total operating revenue). Hemgenix has been a foundational commercial entry in hemophilia-B gene therapy. 

2. Pfizer, Inc. — BEQVEZ™ (fidanacogene elaparvovec) — FDA approval April 25, 2024 (second hemophilia-B gene therapy approved in US); Pfizer full-year 2024 revenues ≈ USD 63.6 billion (company reporting). Note: some commercial adjustments/strategic changes have occurred in 2024–2025 for Pfizer’s gene-therapy efforts. 

3. BioMarin Pharmaceutical — ROCTAVIAN® / valoctocogene roxaparvovec (hemophilia A program) — regulatory milestones/approvals and ongoing post-marketing commitments; BioMarin total revenues ~USD 2.9 billion (2024) and actively reporting Roctavian durability data and regulatory updates. 

4. uniQure / partners — clinical-stage leader in hemophilia B gene therapy (AMT-061 program / ongoing studies) and manufacturing/capacity activities; uniQure reported 2024 revenue ≈ USD 27.1 million (2024 results). 

5. Sangamo Therapeutics — earlier partnerships (e.g., with Pfizer) and pipeline activity; experienced partner termination / program setbacks (Pfizer ended its hemophilia A pact late-2024), and Sangamo reported 2024 revenues ≈ USD 57.8 million. These program changes materially affect the timing of some A-program launches.

6. Other notable players / niche specialists: companies involved in vector manufacturing, CMOs (contract manufacturers), and academic spinouts — these include specialized vector CMOs and clinical centres of excellence that enable commercial rollout (many listed in market reports and filings).

Quick interpretation / practical notes

• The market is commercially nascent but high-value: only a handful of commercial gene therapies for haemophilia are approved so far, but each product’s per-patient price and the need for specialized care make the addressable commercial market significant even at low penetration. 

• Estimates vary widely across market research firms — always check the provider’s definition (hemophilia A vs B, inclusion of radio-pharmaceuticals, inclusion of services/diagnostics).

If you’d like, I can now (pick one) and I’ll build it immediately:

1. a company × metric spreadsheet (columns: company, most recent annual revenue, approved gene therapy (brand), approval date(s), list price (if public), program stage for other candidates, notes on market penetration), or

2. Reconcile market-size estimates from 3 chosen reports (methodology comparison + recommended figure for a conservative vs aggressive business case), or

3. pull region × patient-penetration scenarios (low/medium/high uptake) and model potential revenue to 2030.

Pick 1 / 2 / 3 and I’ll produce it in this reply.