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References
1.Beam Therapeutics . Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation . 2025 . Available from: https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-positive-initial-data-beam-302-phase [Last accessed:
March 13 , 2025
].
2.Neurotech Pharmaceuticals . Neurotech’s ENCELTO™ (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel ). 2025 . Available from: https://www.neurotechpharmaceuticals.com/wp-content/uploads/Neurotech_Press-Release_BLA_Approval_FINAL.pdf [Last accessed:
March 13 , 2025
].
3.Roche . Finance Report 2024 . 2025 . Available from: https://assets.roche.com/f/176343/x/38d96ed8ec/fb24e.pdf [Last accessed:
March 11 , 2025
].
4.
Liu
A
. Pharma Roche overhauls Spark gene therapy unit, recording $2.4B in full impairment . FiercePharma , 2025 . Available from: https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment [Last accessed:
March 11 , 2025
].
5.Verve Therapeutics . Verve Therapeutics Announces Pipeline Progress and Reports Fourth Quarter and Full Year 2024 Financial Results . 2025 . Available from: https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-pipeline-progress-and-reports-3 [Last accessed:
March 13 , 2025
].
6.Verve Therapeutics . Form 8-K . 2022 . Available from: https://ir.vervetx.com/static-files/ed3ddef7-2c4d-42ab-bcc6-ed3e113c2971 [Last accessed:
March 13 , 2025
].
7.Verve Therapeutics . Form 10-Q Quarterly Report, filed November 5 . 2024 . Available from: https://ir.vervetx.com/static-files/0ea2cb73-ebd9-4cc1-a8a9-71c9d60648d4 [Last accessed:
March 13 , 2025
].
8.Regeneron Pharmaceuticals . Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial . 2025 . Available from: https://investor.regeneron.com/news-releases/news-release-details/latest-db-oto-results-demonstrate-clinically-meaningful-hearing [Last accessed:
March 13 , 2025
].
9.AskBio . AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort . 2025 . Available from: https://www.askbio.com/askbio-advances-gene-therapy-clinical-trial-for-limb-girdle-muscular-dystrophy-type-2i-r9-with-dosing-of-first-participant-in-second-cohort/ [Last accessed:
March 13 , 2025
].
10.
Prosser
D
. Making Gene Therapy Faster and Better With Fuse Vectors . Forbes . 2025 . Available from: https://www.forbes.com/sites/davidprosser/2025/02/25/making-gene-therapy-faster-and-better-with-fuse-vectors/ [Last accessed:
March 13 , 2025
].
11.
Chowdhry
A
. Fuse Vectors: $5.2 Million (Pre-Seed) Raised For Advancing Cell-Free Gene Therapy Technology . Pluse2 . 2025 . Available from: https://pulse2.com/fuse-vectors-5-2-million-pre-seed-raised-for-advancing-cell-free-gene-therapy-technology/ [Last accessed:
March 13 , 2025
].