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References
1.Beam Therapeutics . Beam therapeutics to present data across hematology franchise, including first clinical data for BEAM-101 in sickle cell disease and ESCAPE non-human primate data, at American Society of Hematology (ASH) annual meeting . November 5, 2024 . Available from: https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-present-data-across-hematology-franchise [Last accessed:
November 16 , 2024
].
2.
Philippidis
A
. Patient dies in beam trial of SCD candidate; Company cites conditioning . Genetic Engineering & Biotechnology News . 2024 . Available from: https://www.genengnews.com/topics/genome-editing/patient-dies-in-beam-trial-of-scd-candidate-company-cites-conditioning/ [Last accessed:
November 16 , 2024
].
3.Neurogene . Neurogene reports positive interim efficacy data from first four low-dose pediatric participants in NGN-401 gene therapy clinical trial for rett syndrome . November 11 , 2024 . Available from: https://ir.neurogene.com/news-releases/news-release-details/neurogene-reports-positive-interim-efficacy-data-first-four-low [Last accessed:
November 14 , 2024
].
4.PTC Therapuetics . PTC therapeutics announces FDA approval of AADC deficiency gene therapy . November 13, 2024 . Available from: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-aadc-deficiency-gene [Last accessed:
November 14 , 2024
].
5.Office of New York Governor Kathy Hochul . Governor hochul launches next phase of long island’s nation-leading cell and gene therapy innovation hub: New York biogenesis park . October 22, 2024 . Available from: https://www.governor.ny.gov/news/governor-hochul-launches-next-phase-long-islands-nation-leading-cell-and-gene-therapy [Last accessed:
November 16 , 2024
].
6.
Waldron
J
. Bayer drops Huntington’s gene therapy to focus on ‘most promising assets’ . FierceBiotech , November 12, 2024 . Available from: https://www.fiercebiotech.com/biotech/bayer-drops-huntingtons-gene-therapy-focus-most-promising-assets [Last accessed:
November 14 , 2024
].
7.Ascend Advanced Therapies . Ascend advanced therapies partners with EW healthcare partners to expand United States capacity and capabilities . November 12, 2024 . Available from: /https://www.ascend-adv.com/news-and-insights/news/ascend-advanced-therapies-partners-with-ew-healthcare-partners-to-expand-united-states-capacity-and-capabilities/ [Last accessed:
November 14 , 2024
].
8.Intellia Therapeutics . Intellia announces first clinical evidence from ongoing phase 1 study that Nexiguran Ziclumeran (nex-z), an in vivo CRISPR/Cas9-based gene editing therapy, May favorably impact disease progression in Transthyretin (ATTR) Amyloidosis . November 16, 2024 . Available from: https://ir.intelliatx.com/news-releases/news-release-details/intellia-announces-first-clinical-evidence-ongoing-phase-1-study [Last accessed:
November 16 , 2024
].
