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References
1.Graphite Bio. Graphite Bio announces voluntary pause of phase 1/2 CEDAR study of nulabeglogene autogedtemcel (nula-cel) for Sickle Cell Disease. 2023 . https://www.businesswire.com/news/home/20230105005859/en/Graphite-Bio-Announces-Voluntary-Pause-of-Phase-12-CEDAR-Study-of-nulabeglogene-autogedtemcel-nula-cel-for-Sickle-Cell-Disease (last accessed
January 14 , 2023
).
2.4D Molecular Therapeutics. 4D Molecular Therapeutics announces updates on clinical pipeline and additional preclinical programs. 2023 . https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4d-molecular-therapeutics-announces-updates-clinical-pipeline (last accessed
January 15 , 2023
).
3.Neurocrine Biosciences and Voyager Therapeutics. Neurocrine Biosciences and Voyager Therapeutics enter strategic collaboration for development and commercialization of Voyager's GBA1 program and other next-generation gene therapies for neurological diseases. 2023 . https://ir.voyagertherapeutics.com/news-releases/news-release-details/neurocrine-biosciences-and-voyager-therapeutics-enter-strategic (last accessed
January 14 , 2023
).
4.BioMarin Pharmaceutical. BioMarin announces stable and durable annualized bleed control for ROCTAVIAN™ in largest phase 3 gene therapy study in adults with severe hemophilia A; 134-participant study met all primary and secondary efficacy endpoints at 3-year analysis. 2023 . https://investors.biomarin.com/2023-01-08-BioMarin-Announces-Stable-and-Durable-Annualized-Bleed-Control-for-ROCTAVIAN-TM-in-Largest-Phase-3-Gene-Therapy-Study-in-Adults-with-Severe-Hemophilia-A-134-Participant-Study-Met-All-Primary-and-Secondary-Efficacy-Endpoints-at-3-Year-Analysis (last accessed
January 14 , 2023
).
5.Krystal Biotech. Krystal Biotech announces FDA's 3-month extension of BLA PDUFA date and regulatory update for B-VEC to treat patients with dystrophic epidermolysis bullosa. 2023 . https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-fdas-3-month-extension-bla-pdufa-date (last accessed
January 13 , 2023
).
6.Bluebird Bio. FDA lifts partial clinical hold for bluebird bio's sickle cell disease (SCD) studies for patients under the age of 18. 2022 . https://investor.bluebirdbio.com/news-releases/news-release-details/fda-lifts-partial-clinical-hold-bluebird-bios-sickle-cell (last accessed
January 14 , 2022
).
