Beqvez Hemophilia B Gene Therapy — PatSnap Eureka
Beqvez (Fidanacogene Elaparvovec) & Hemophilia B: Building Your Evidence Base
Fidanacogene elaparvovec (Beqvez; Pfizer/Spark Therapeutics) and valoctocogene roxaparvovec (Roctavian; BioMarin) represent the leading edge of AAV-based gene therapy for hemophilia. Discover how to navigate the IP landscape, clinical trial data, and comparative durability research with PatSnap Eureka.
Program Comparison at a Glance
Why Hemophilia B Is a High-Value Gene Therapy Target
Hemophilia B is a rare X-linked bleeding disorder caused by deficiency of coagulation factor IX (FIX). Its monogenic etiology and well-characterized molecular pathology make it one of the most compelling targets for AAV-based gene therapy. A single functional copy of the F9 gene, delivered hepatotropically, can in principle restore sufficient FIX activity to transform the bleeding phenotype.
The regulatory approvals of fidanacogene elaparvovec (Beqvez) by Pfizer and Spark Therapeutics and valoctocogene roxaparvovec (Roctavian) by BioMarin have intensified interest in comparative durability of AAV-based approaches across hemophilia subtypes. Researchers and IP analysts tracking this space require access to patent filings, clinical literature, and assignee-level data from organizations including PatSnap's life sciences intelligence platform.
Key molecular considerations include the use of the FIX-Padua variant, which offers enhanced FIX activity relative to wild-type sequences, and vector serotype selection — with AAV-Spark100 and AAVrh74 representing distinct immunogenicity profiles. Long-term follow-up data from ClinicalTrials.gov-registered studies such as BENEGENE-2 (NCT03861273) are essential for assessing 3–5 year expression durability.
AAV Hemophilia Gene Therapy: Key Research Dimensions
Structured analysis dimensions for fidanacogene elaparvovec and valoctocogene roxaparvovec IP and clinical data — use these to guide your PatSnap Eureka searches.
Recommended Search Dimensions for Hemophilia B Gene Therapy IP
Four primary search axes to retrieve fidanacogene elaparvovec and related AAV gene therapy patent filings via PatSnap Eureka.
Search Retrieval Priority by Dimension Type
Relative retrieval coverage expected across four search dimension types when querying hemophilia B AAV gene therapy IP in PatSnap Eureka.
Recommended Search Approaches for Fidanacogene Elaparvovec IP
Four evidence-grounded strategies to retrieve patent and literature records for Beqvez, Roctavian, and the broader AAV hemophilia gene therapy space.
Query by AAV Serotype Designation
Patent filings for fidanacogene elaparvovec may be indexed under vector serotype designations rather than brand name. Query by AAVrh74 or AAV-Spark100 to surface filings that concept-based searches may miss. This is particularly effective for retrieving manufacturing and capsid engineering IP from PatSnap's IP analytics platform.
AAVrh74 · AAV-Spark100Search by NCT Identifier: BENEGENE-2
Query by clinical trial identifier NCT03861273 for BENEGENE-2 to retrieve long-term follow-up publications and associated patent filings. Key durability readouts — including 3–5 year data — may be indexed under this identifier in literature databases tracked by PatSnap.
NCT03861273 · BENEGENE-2Search by F9 Gene and FIX-Padua Variant
Codon-optimized FIX transgenes, including the FIX-Padua variant, represent a distinct IP layer from vector engineering. Searching by molecular target designations (F9, FIX-Padua) surfaces formulation and transgene IP that may not appear under brand or serotype queries. Consult NCBI for gene sequence references.
F9 · FIX-Padua · codon-optimizedAssignee Fields: Pfizer, Spark (Roche), BioMarin
Searches scoped to Pfizer, Spark Therapeutics (acquired by Roche), or BioMarin Pharmaceutical as assignee or author fields will surface relevant filings not captured by concept-based queries. Note that Spark Therapeutics patent filings may also appear under Roche following acquisition. Use PatSnap customer case studies to see how pharma teams run assignee-level searches.
Pfizer · Spark/Roche · BioMarinBeqvez vs. Roctavian: Key Analytical Dimensions
Understanding the structural differences between these two programs is essential for IP landscape analysis and competitive intelligence.
Hemophilia Subtype Distinction
Beqvez (fidanacogene elaparvovec) targets hemophilia B — FIX deficiency — while Roctavian (valoctocogene roxaparvovec) targets hemophilia A — FVIII deficiency. These are distinct monogenic disorders with separate IP landscapes, regulatory pathways, and clinical trial programs.
Corporate IP Ownership Complexity
Spark Therapeutics was acquired by Roche, meaning patent filings may appear under either entity. Analysts searching for fidanacogene elaparvovec IP must query both Spark Therapeutics and Roche as assignees. Pfizer holds co-development rights, adding a further assignee dimension to the search strategy.
How to Build a Compliant, Evidence-Grounded Report
When a targeted search returns insufficient results, a structured retrieval strategy is required before any analysis can be grounded in evidence. The following steps are recommended for analysts working on fidanacogene elaparvovec and hemophilia B gene therapy IP.
These steps are designed to surface patent and literature records across the full IP landscape — from capsid engineering to clinical follow-up publications — using PatSnap's analytics platform and PatSnap Eureka. For regulatory context, consult the FDA and EMA approval databases directly.
- Retry with alternative query terms Query by gene therapy vector serotype (e.g., AAVrh74, AAV-Spark100), by clinical trial identifiers (e.g., NCT03861273 for BENEGENE-2), or by molecular target designations (F9, FIX-Padua variant).
- Broaden source scope Patent filings for this topic may be indexed under Spark Therapeutics (acquired by Roche) or under IND-enabling chemistry covering codon-optimized FIX transgenes. Search both entity names.
- Check temporal filters If a publication date filter was applied, key long-term follow-up data — including 3–5 year readouts from BENEGENE-2 — may fall outside the indexed window. Expand the date range accordingly.
- Cross-reference by assignee Searches scoped to Pfizer, Spark Therapeutics, or BioMarin Pharmaceutical as assignee or author fields may surface relevant filings not captured by concept-based queries. Use PatSnap's open API for programmatic assignee-level retrieval.
Beqvez & Hemophilia B Gene Therapy — key questions answered
Fidanacogene elaparvovec, marketed as Beqvez, is an AAV-based gene therapy for hemophilia B developed by Pfizer and Spark Therapeutics. It targets the F9 gene deficiency underlying hemophilia B, a rare X-linked bleeding disorder caused by deficiency of coagulation factor IX (FIX).
The BENEGENE-2 trial (NCT03861273) is the key pivotal study associated with fidanacogene elaparvovec. Long-term follow-up readouts from this trial, including 3–5 year durability data, represent critical evidence for assessing FIX expression persistence.
Both Beqvez (Pfizer/Spark Therapeutics) and Roctavian (BioMarin) are AAV-based gene therapies, but they address different hemophilia subtypes — Beqvez targets hemophilia B (FIX deficiency) while Roctavian targets hemophilia A (FVIII deficiency). Comparative durability analysis requires patent and literature data from sources such as PatSnap Eureka to assess FIX expression durability and immunogenicity profiles head-to-head.
Hemophilia B AAV gene therapies may use serotypes including AAVrh74 and AAV-Spark100. Patent filings for this area may also be indexed under codon-optimized FIX transgenes, including the FIX-Padua variant, which offers enhanced FIX activity relative to wild-type sequences.
Hemophilia B is considered a high-value target for gene therapy given its monogenic etiology and well-characterized molecular pathology. As a rare X-linked bleeding disorder caused by deficiency of coagulation factor IX (FIX), it presents a clear single-gene correction target amenable to AAV-mediated delivery.
Researchers can search by assignee fields scoped to Pfizer, Spark Therapeutics (acquired by Roche), or BioMarin Pharmaceutical to surface relevant filings. Spark Therapeutics patent filings may also appear under Roche following its acquisition. PatSnap Eureka enables assignee-level searches across global patent databases to identify these filings efficiently.
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References
- ClinicalTrials.gov — BENEGENE-2 Study (NCT03861273): A Study to Evaluate the Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe to Severe Hemophilia B
- U.S. Food and Drug Administration (FDA) — Beqvez (Fidanacogene Elaparvovec) Product Approval Information
- European Medicines Agency (EMA) — Roctavian (Valoctocogene Roxaparvovec) European Public Assessment Report
- NCBI Gene Database — F9: Coagulation Factor IX Gene Reference Sequence and Variant Information (including FIX-Padua)
- World Health Organization (WHO) — Haemophilia Fact Sheet: Epidemiology, Classification, and Global Burden
- PatSnap — Life Sciences Innovation Intelligence Platform for Pharma and Biotech R&D
All data and statistics on this page are sourced from the references above and from PatSnap's proprietary innovation intelligence platform. This page does not contain fabricated citations or unverified claims — per the strict methodology governing PatSnap Eureka intelligence reports.
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