BioTech IP Review

Publication Number: EP4488288A3
Filing Date: July 10, 2018 (original priority), but published April 16, 2025 in Europe
Publication Date: April 16, 2025
Applicant: Uniqure IP B.V. (European patent application)
Status: Pending publication in EU jurisdictions

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What This Patent Covers

This published European patent application describes methods, agents, and compositions for AAV‑based gene therapy in humans, reflecting core technology used in uniQure’s clinical programs such as AMT‑130 for Huntington’s disease:

AAV Gene Therapy Methods: The application discloses means and methods for delivering therapeutic genetic material using adeno‑associated virus (AAV) vectors to treat human diseases. AAV vectors are widely used for gene delivery due to their relatively low immunogenicity and ability to mediate long‑term gene expression.

Therapeutic Constructs: It covers AAV constructs designed to introduce specific nucleic acids — e.g., therapeutic transgenes or silencing RNAs — into target cells, which is the basis of uniQure’s miQURE™ gene‑silencing platforms and disease‑modifying therapy candidates like AMT‑130.

Methods of Use: The patent encompasses methods of administering these AAV gene therapy agents to patients, potentially including routes of administration and conditions treated. While the publication itself is broad, its claims provide foundational IP protection for AAV‑based therapies across multiple indications.

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Why This Patent Is Important

1) Core Platform Protection
This patent secures foundational intellectual property on AAV gene therapy methods — the backbone of uniQure’s entire therapeutic pipeline, including AMT‑130 (for Huntington’s) and other gene therapy candidates. Patents covering the delivery mechanism are often more valuable than single‑use inventions because they can protect platform technologies used across multiple products.

2) Supports High‑Value Therapeutic Programs
AAV‑based gene therapies command premium pricing in rare diseases (e.g., hemophilia, neurological disorders). uniQure’s AMT‑130 program, for example, has shown promising mid‑stage clinical results and received Breakthrough Therapy designation, underscoring its potential blockbuster value if approved.

3) Broad Applicability
Beyond Huntington’s, the technology could be applied to other genetic and neurodegenerative diseases in uniQure’s pipeline (e.g., AMT‑260 for temporal lobe epilepsy, AMT‑162 for SOD1‑ALS, AMT‑191 for Fabry disease). Protection of AAV construction and delivery supports multiple product lifecycles.

4) Strategic IP Position
While individual patents directly tied to specific products (like AMT‑130) are valuable, patents on the means of safe and efficient AAV gene delivery create a defensible IP moat around uniQure’s core gene therapy approach.

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UniQure also owns or licenses patent families specifically directed to AMT‑130 and similar constructs, including issued patents covering miRNA expression cassettes designed to lower huntingtin protein using AAV vectors. These patents strengthen protection on the therapeutic agent itself and its implementation, beyond the delivery platform.

Note: Many key patents covering both delivery technologies and product‑specific inventions (e.g., those covering the RNA constructs of AMT‑130) were originally issued years earlier (e.g., U.S. Patents 10,174,321 and EP 3237618) and are being actively maintained with pending applications and continuations to extend protection into the mid‑2030s.