On November 2, 2023 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that seven abstracts have been accepted for presentation at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Vertex Pharmaceuticals, NOV 2, 2023, View Source [SID1234636822]). This includes two presentations of updated clinical data describing additional patients with longer follow-up duration from pivotal Phase 3 trials investigating exagamglogene autotemcel’s (exa-cel) potential as a one-time functional cure for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

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Abstract #1052 entitled "Exagamglogene Autotemcel for Severe Sickle Cell Disease" will be an oral presentation on Monday, December 11 at 4:45 p.m. PST.
Abstract #1053 entitled "Exagamglogene Autotemcel for Transfusion-Dependent Βeta-Thalassemia" will be an oral presentation on Monday, December 11 at 5:00 p.m. PST.
Five additional abstracts have been accepted, including two on the improvements in patients’ quality of life following treatment with exa-cel.

Abstract #4997 entitled "Improvements In Health-Related Quality of Life After Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta Thalassemia" will be a poster presentation on Monday, December 11 from 6:00-8:00 p.m. PST.
Abstract #4999 entitled "Improvements In Health-Related Quality of Life After Exagamglogene Autotemcel in Patients With Severe Sickle Cell Disease" will be a poster presentation on Monday, December 11 from 6:00-8:00 p.m. PST.
Abstract #3674 entitled "VOC-free Status Among Patients with Sickle Cell Disease Following Allogeneic Hematopoietic Stem Cell Transplant: A Cohort Study of Medicaid Enrollees" will be a poster presentation on Sunday, December 10 from 6:00-8:00 p.m. PST.
Abstract #3678 entitled "The Impact of Recent Vaso-Occlusive Crisis on Health-Related Quality of Life in Adults with Sickle Cell Disease" will be a poster presentation on Sunday, December 10 from 6:00-8:00 p.m. PST.
Abstract #3900 entitled "Estimating Sickle Cell Disease Prevalence by State: A Model Using US-born and Foreign-born State-specific Population Data" will be a poster presentation on Monday, December 11 from 6:00-8:00 p.m. PST.
The accepted abstracts are available online on the ASH (Free ASH Whitepaper) website.

About exagamglogene autotemcel (exa-cel)

Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited cell therapy that is being evaluated for patients with SCD or TDT, in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exa-cel has the potential to reduce or eliminate painful and debilitating VOCs for patients with SCD and alleviate transfusion requirements for patients with TDT. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021 and presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in 2023.