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References
1.Cure Rare Disease. To our community: An update on our CRD-TMH-001 clinical trial. 2022 . https://www.cureraredisease.org/blog-posts/to-our-community-an-update-on-our-crd-tmh-001-clinical-trial (last accessed
November 14 , 2022
).
2.
Ungar
L
.
Death in
US gene therapy study sparks search for answers
.
Associated
Press
. 2022. https://apnews.com/article/science-technology-health-business-genetics-79f4a9b76426ec40c367957e3bb9cf4a (last accessed
November 14 , 2022
).
3.
Allen
JK
.
Intellia
Therapeutics
, Inc. (NTLA)
External In Vivo CRISPR Program Updates Could Foreshadow Cautious
FDA
.
Baird
Morning Report
. 2022. https://baird.bluematrix.com/sellside/EmailDocViewer?encrypt=eaa93c66-dfbd-476b-8973-734cd86e4500&mime=pdf&co=Baird&id=replaceme@bluematrix.com&source=mail (last accessed
November 14 , 2022
).
4.Verve Therapeutics. Verve therapeutics provides regulatory update on VERVE-101 investigational new drug application and reports third quarter 2022 financial results. 2022 . https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-provides-regulatory-update-verve-101 (last accessed
November 14 , 2022
).
5.
Patchen
T
. Novartis cuts 275 jobs at Illinois site to consolidate Zolgensma manufacturing in North Carolina. Endpoints News. 2022. https://endpts.com/novartis-cuts-275-jobs-at-illinois-site-to-consolidate-zolgensma-manufacturing-in-north-carolina/ (last accessed
November 14 , 2022
).
6.
The Motley Fool. Sarepta therapeutics (SRPT) Q3 2022 earnings call transcript. 2022. Transcribed November 3, 2022. https://www.fool.com/earnings/call-transcripts/2022/11/03/sarepta-therapeutics-srpt-q3-2022-earnings-call-tr/ (last accessed
November 14 , 2022
).
7.Beam Therapeutics. Beam therapeutics announces portfolio progress and reports third quarter 2022 financial results. 2022 . https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-portfolio-progress-and-reports-third (last accessed
November 14 , 2022
).
8.BioMarin Pharmaceutical. BioMarin announces incremental progress on biologics license application (BLA) review for valoctocogene roxaparvovec AAV gene therapy for adults with severe hemophilia a program. 2022 . https://investors.biomarin.com/2022-11-07-BioMarin-Announces-Incremental-Progress-on-Biologics-License-Application-BLA-Review-for-Valoctocogene-Roxaparvovec-AAV-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A-Program (last accessed
November 14 , 2022
).
9.Danaher. Danaher launches beacon initiative and its first partnership focused on gene therapy innovation. 2022 . https://www.prnewswire.com/news-releases/danaher-launches-beacon-initiative-and-its-first-partnership-focused-on-gene-therapy-innovation-301675012.html (last accessed
November 14 , 2022
).