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References
1.Intellia Therapeutics . Intellia Therapeutics Announces Third Quarter 2025 Financial Results and Recent Updates . 2025 . Available from: https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-third-quarter-2025-financial [Last accessed: November 7 , 2025
2.uniQure . uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease . 2025 . Available from: https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-provides-regulatory-update-amt-130-huntingtons-disease-0 [Last accessed: November 3 , 2025
3.
Philippidis
A
. StockWatch: StockWatch: UniQure Shares Reach Five-Year High on “Game Changing” Huntington’s Data . Genetic Engineering & Biotechnology News , 2025 https://www.genengnews.com/topics/translational-medicine/stockwatch-uniqure-shares-reach-five-year-high-on-game-changing-huntingtons-data/
4.BioMarin Pharmaceutical . BioMarin Reports Third Quarter 2025 Results and Provides Corporate Update . 2025 . Available from: https://www.biomarin.com/news/press-releases/biomarin-reports-third-quarter-2025-results-and-provides-corporate-update/ [Last accessed: November 2 , 2025
5.
Becker
Z
. BioMarin resigned to ending journey with hemophilia A gene therapy Roctavian, seeks divestiture amid sales struggle . FiercePharma ; 2025 . Available from: https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid [Last accessed: November 2 , 2025
6.4D Molecular Therapeutics . 4DMT Announces Exclusive License Agreement with Otsuka Pharmaceutical Co., Ltd. for Development and Commercialization of 4D-150 in Asia-Pacific . 2025 . Available from: https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-announces-exclusive-license-agreement-otsuka-pharmaceutical [Last accessed: November 1 , 2025
7.AviadoBio and UgeneX . AviadoBio and UgeneX Therapeutics Announce Exclusive Option-License Agreement to Develop Next Generation Optogenetics Gene Therapy to Restore Vision in Patients with Retinal Disease . 2025 . Available from: https://www.businesswire.com/news/home/20251009502471/en/AviadoBio-and-UgeneX-Therapeutics-Announce-Exclusive-Option-License-Agreement-to-Develop-Next-Generation-Optogenetics-Gene-Therapy-to-Restore-Vision-in-Patients-with-Retinal-Disease [Last accessed: November 2 , 2025
8.Adverum Biotechnologies . Lilly to Acquire Adverum Biotechnologies . 2025 . Available from: https://investors.adverum.com/press_releases/news-details/2025/Lilly-to-Acquire-Adverum-Biotechnologies-2025-qGdtYfhh2t/default.aspx [Last accessed: November 1 , 2025
9.
Smith
G
. FDA Clears Way for Faster Personalized Gene Editing Therapy . Bloomberg News ; 2025 . Available from: https://www.bloomberg.com/news/articles/2025-10-31/fda-clears-way-for-faster-personalized-gene-editing-treatments [Last accessed: November 1 , 2025
10.
Ahrens-Nicklas
R
,
Musunuru
K
How to create personalized gene editing platforms: Next steps toward interventional genetics . Am J Hum Genet , 2025 :S0002 ; doi: 10.1016/j.ajhg.2025.10.006