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References
1.Enlaza Therapeutics . Enlaza Therapeutics Announces Strategic Collaboration with Vertex Pharmaceuticals to Develop War-Lock Drug Conjugates and T-Cell Engagers for Improved Conditioning and Certain Autoimmune Diseases . September 2, 2025 . Available from: https://enlazatx.com/press_release/enlaza-therapeutics-announces-strategic-collaboration-with-vertex-pharmaceuticals-to-develop-war-lock-drug-conjugates-and-t-cell-engagers-for-improved-conditioning-and-certain-autoimmune-diseases/ [Last accessed: September 3 , 2025 ].
2.U.S. Food and Drug Administration . FDA Approves Required Labeling Changes for Increased Risk of Hematologic Malignancy Following Treatment with Skysona (elivaldogene autotemcel) . August 7, 2025 . Available from: https://www.fda.gov/vaccines-blood-biologics/fda-approves-required-labeling-changes-increased-risk-hematologic-malignancy-following-treatment [Last accessed: August 30 , 2025 ].
3.Bluebird Bio . Prescribing Information for SKYSONA™ (elivaldogene autotemcel) . August 2025 . Available from: https://www.bluebirdbio.com/-/media/bluebirdbio/Corporate%20COM/Files/SKYSONA/SKYSONA_prescribing_information.pdf [Last accessed: August 30 , 2025 ].
4.U.S. Securities and Exchange Commission . Form D: Notice of Exempt Offering of Securities completed by Kriya Therapeutics, Filer ID No. 0001811209, and signed by Shankar Ramaswamy . August 15, 2025 . Available from: https://www.sec.gov/Archives/edgar/data/1811209/000181120925000002/xslFormDX01/primary_doc.xml [Last accessed: August 29 , 2025 ].
5.
Yamada
K
,
Hariharan
VN
,
Caiazzi
J
, et al. Enhancing siRNA efficacy in vivo with extended nucleic acid backbones . Nature Biotechnology . August 1, 2024 . Available from: https://www.nature.com/articles/s41587-024-02336-7 [Last accessed: August 27 , 2025 ].
6.Hongene Biotech Corp . Hongene to supply exNA oligonucleotide technology under new licensing deal . August 26, 2025 . Available from: https://www.hongene.com/en/news/hongene-to-supply-exna-oligonucleotide-technology-under-new-licensing-deal[Last accessed: August 27 , 2025 ].
7.Andelyn Biosciences . Andelyn Biosciences and Amplo Biotechnology Partner to Manufacture Novel Gene Therapies for Diseases Affecting the Neuromuscular Junction . August 21, 2025 . Available from: https://www.andelynbio.com/news-media/andelyn-biosciences-and-amplo-biotechnology-partner-to-manufacture-novel-gene-therapies [Last accessed: August 29 , 2025 ].
8.Berlin.de . Senate gives 45 million euros for Mühlendammbrücke and gene therapy . August 20, 2025 . Available from: https://www.berlin.de/en/news/9849097-5559700-senate-gives-45-million-euros-for-muehle.en.html [Last accessed: September 5 , 2025 ].
