Gene and RNA therapeutics

8 claims in this domain, drawn from 4 sources. A source appears here if any claim citing it carries this domain.

Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B (TA989) National Institute for Health and Care Excellence · 2024-07-24 · Tier 1
Current — checked with the publisher on 2026-09-02 · 2 claims
Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion - Summary of Product Characteristics CSL Behring GmbH, via the electronic Medicines Compendium · 2026-04-21 · Tier 1
Current — checked with the publisher on 2026-09-02 · 4 claims
The Human Medicines Regulations 2012, regulation 2A: definition of advanced therapy medicinal product UK Statutory Instruments, via legislation.gov.uk · 2012 · Tier 1
Current — checked with the publisher on 2026-09-02 · 1 claim
Lumasiran for treating primary hyperoxaluria type 1 (HST25) National Institute for Health and Care Excellence · 2022-11-16 · Tier 1
Current — checked with the publisher on 2026-09-02 · 1 claim