Abstract

Get full access to this article
View all access options for this article.
References
1.
Lek
A
,
Wong
B
,
Keeler
A
, et al. Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-Delivered CRISPR-Transactivator. medRxiv 2023. Available from: https://www.medrxiv.org/content/10.1101/2023.05.16.23289881v1 [Last accessed:
June 14 , 2023
].
2.Verve Therapeutics. Verve Establishes Global Collaboration with Lilly to Advance Verve's In Vivo Gene Editing Program Targeting Lp(a) for the Treatment of Atherosclerotic Cardiovascular Disease . 2023 . Available from: https://ir.vervetx.com/news-releases/news-release-details/verve-establishes-global-collaboration-lilly-advance-verves-vivo [Last accessed:
June 15 , 2023
].
3.
Philippidis
A.
Beacon of Light: Retinal Gene Therapy Developer Launches with $120M. Genetic Engineering & Biotechnology News 2023. Available from: https://www.genengnews.com/gen-edge/beacon-of-light-retinal-gene-therapy-developer-launches-with-120m/ [Last accessed:
June 13 , 2023
].
4.CRISPR Therapeutics and Vertex Pharmaceuticals . FDA Accepts Biologics License Applications for Exagamglogene Autotemcel (Exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. 2023 . Available from: http://ir.crisprtx.com/news-releases/news-release-details/fda-accepts-biologics-license-applications-exagamglogene [Last accessed:
June 13 , 2023
].
5.
Vertex Pharmaceuticals and CR, ISPR Therapeutics. Positive Results from Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress. 2023 . Available from: https://investors.vrtx.com/news-releases/news-release-details/positive-results-pivotal-trials-exa-cel-transfusion-dependent [Last accessed:
June 13 , 2023
].
6.
AAvantgarde Bio AA, Vantgarde Closes €61 Million Series A Financing to Advance Two Therapeutic Programs into the Clinic. 2023 . Available from: https://www.aavantgardebio.com/wp-content/uploads/2023/06/AAVantgarde-SeriesA-English-Updated-Final-06.06.23.pdf [Last accessed:
June 13 , 2023
].
7.Kate Therapeutics . Kate Therapeutics Debuts with $51 Million Series A to Develop Next-Generation Genetic Medicines to Treat Muscle and Heart Diseases. 2023 . Available from: https://www.katetherapeutics.com/news/series-a-financing/ [Last accessed:
June 14 , 2023
].
8.PTC Therapeutics . PTC Therapeutics Announces Strategic Pipeline Prioritization. 2023 . Available from: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-strategic-pipeline-prioritization [Last accessed:
June 14 , 2023
].
