Gene therapy and the liver
The liver is where many gene and RNA therapies do their work. That is not the same as treating a liver disease. Of the gene and RNA therapies licensed or recommended in the UK that act on the liver, this platform has found none whose licensed use is liver failure, cirrhosis or chronic liver disease.
Etranacogene dezaparvovec holds a conditional marketing authorisation — not a full one — for treating severe and moderately severe haemophilia B, a congenital factor IX deficiency, in adults without a history of factor IX inhibitors. Haemophilia B is an inherited bleeding disorder, not a disease of the liver.
NICE — Chapter 2 Information about etranacogene dezaparvovec, section 2.1
The exact wording of the source
Chapter 2 Information about etranacogene dezaparvovec, section 2.1 · status: current, verified 2026-09-02
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Etranacogene dezaparvovec (Hemgenix, CSL Behring) has a conditional marketing authorisation 'for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors'.
Its licence contraindicates it in patients with known advanced hepatic fibrosis or cirrhosis, and in patients with active acute or uncontrolled chronic infections.
CSL Behring GmbH, via the electronic Medicines Compendium — Section 4.3 Contraindications, second and third bullets
The exact wording of the source
Section 4.3 Contraindications, second and third bullets · status: current, verified 2026-09-02
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- Active infections, either acute or uncontrolled chronic (see section 4.4). - Patients with known advanced hepatic fibrosis, or cirrhosis (see section 4.4).
NICE recommends it with managed access, and only if the conditions in the managed access agreement are followed.
NICE — Chapter 1 Recommendation, section 1.1, first sentence · NICE — Chapter 1 Recommendation, section 1.1, second sentence
The exact wording of the source
Chapter 1 Recommendation, section 1.1, first sentence · status: current, verified 2026-09-02
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Etranacogene dezaparvovec is recommended with managed access as an option for treating moderately severe or severe haemophilia B (congenital factor IX [FIX] deficiency) in adults without anti‑FIX antibodies.
Chapter 1 Recommendation, section 1.1, second sentence · status: current, verified 2026-09-02
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It is only recommended if the conditions in the managed access agreement for etranacogene dezaparvovec are followed.
Before it is given, the licence requires that liver transaminases are assessed and that liver ultrasound and elastography are performed, along with tests for active hepatitis B and C.
CSL Behring GmbH, via the electronic Medicines Compendium — Section 4.4 Special warnings and precautions for use, baseline assessment
The exact wording of the source
Section 4.4 Special warnings and precautions for use, baseline assessment · status: current, verified 2026-09-02
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Prior to the treatment with Hemgenix, patient's liver transaminases should be assessed and liver ultrasound and elastography performed, along with laboratory tests to evaluate for active Hepatitis B and C.
Afterwards, transaminases are closely monitored — for example once weekly for at least three months — and a corticosteroid taper is considered if alanine aminotransferase rises above the upper limit of normal, or to double the patient's baseline. The licence's own heading for this passage is Hepatotoxicity.
CSL Behring GmbH, via the electronic Medicines Compendium — Section 4.4, subheading 'Hepatotoxicity'
The exact wording of the source
Section 4.4, subheading 'Hepatotoxicity' · status: current, verified 2026-09-02
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After Hemgenix administration, transaminases should be closely monitored, e.g. once per week for at least 3 months. A course of corticosteroid taper should be considered in the event of ALT increase to above the upper limit of normal or to double the patient's baseline levels
Because of the risk of malignancy from vector integration, patients undergo regular liver ultrasound screening and are monitored for alpha-fetoprotein elevations, for example annually, for at least five years after administration.
CSL Behring GmbH, via the electronic Medicines Compendium — Section 4.4, subheading 'Risk of malignancy as a result of vector integration'
The exact wording of the source
Section 4.4, subheading 'Risk of malignancy as a result of vector integration' · status: current, verified 2026-09-02
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undergo regular liver ultrasound screenings and are regularly monitored for alpha-fetoprotein (AFP) elevations (e.g. annually) for at least 5 years after Hemgenix administration
In UK law a gene therapy medicinal product is one whose active substance contains or consists of a recombinant nucleic acid administered with a view to regulating, repairing, replacing, adding or deleting a genetic sequence, and whose effect relates directly to that sequence or to its expression product. A vaccine against infectious diseases is not one.
UK Statutory Instruments, via legislation.gov.uk — Regulation 2A(2)(a) · UK Statutory Instruments, via legislation.gov.uk — Regulation 2A(2)(b) and 2A(3)
The exact wording of the source
Regulation 2A(2)(a) · status: current, verified 2026-09-02
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(a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; and
Regulation 2A(2)(b) and 2A(3) · status: current, verified 2026-09-02
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(b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence. ... A vaccine against infectious diseases is not to be treated as a gene therapy medicinal product.
Related
- Cirrhosis — should be avoided in
- Alcohol-related liver disease — related through cirrhosis
- Ascites — related through cirrhosis
- Hepatocellular carcinoma — related through cirrhosis
- Hepatic encephalopathy — related through cirrhosis
- Steatotic liver disease — related through cirrhosis
What we have not established
Relationships involving AAV vectors that we have not evidenced are listed, never drawn — a line would read as a weak relationship, which is a different and false claim.
- AAV vectors → Hepatocyte — not established
Evidence search date: 2026-09-02
Content reviewed and approved
What we have not established here
Known gaps on this concept:
NO CATALOGUE OF LIVER-TARGETED THERAPIES, BY DECISION. Givosiran, patisiran, vutrisiran, eplontersen, inclisiran and lumasiran were all acquired and verified with their exact NICE wording, populations and conditions. None is published as an entry. An adversarial review found the misreading risk highest for acute HEPATIC porphyria, whose name alone a reader with cirrhosis will take as their own condition, and noted that NICE guidance records liver transplant as an option for some people with that disease — which makes the misreading reasonable rather than careless.
THE UNIVERSAL NEGATIVE IS NOT CLAIMED. This page says that of the therapies acting on the liver which this platform examined, none is licensed for liver disease. It does NOT say that no such therapy exists anywhere. No systematic search of that question was recorded, and the difference is stated rather than glossed.
Givosiran raises liver transaminases, and NICE records that elevated transaminases have led to people stopping treatment. That is the single most relevant fact about that drug for this audience and it was surfaced by adversarial review rather than by the sweep. It is not published because the drug is not published.
MHRA confirmed by freedom-of-information response that there is no authorised product called Roctavian in the UK. Verified and unpublished: a page saying which gene therapies are NOT authorised would invite exactly the pipeline reading this phase forbids.
No content on gene or RNA therapies under investigation for liver disease itself — fazirsiran for alpha-1 antitrypsin deficiency, bepirovirsen for chronic hepatitis B, OTC deficiency gene therapy. All are investigational, none is licensed, and naming them would be pipeline language.
NHS genomic medicine services and the National Genomic Test Directory are not covered. NHS genomics largely delivers diagnosis, and this platform will not blur diagnosis into treatment.
No plain-language explanation of how gene therapy works. The plain layer carries two facts and stops.
What has changed here
2026-09-02 — Concept created at plain, clinical, specialist and research resolutions.
Phase 18, the last audience layer. The acceptance criteria required this phase to test an inversion — not 'gene therapy may treat your liver' but 'your liver determines whether you can have gene therapy at all'. The Hemgenix licence evidences it exactly.