Vertex and CRISPR Therapeutics Present New Data of CTX001 from 22 Patients with TDT and SCD at EMA 2021

 Vertex and CRISPR Therapeutics Present New Data of CTX001 from 22 Patients with TDT and SCD at EMA 2021

Vertex and CRISPR Therapeutics Present New Data of CTX001 from 22 Patients with TDT and SCD at EMA 2021

Shots:

  • The ongoing two P-I/ II studies, CLIMB-Thal-111 & CLIMB-SCD-121 evaluating CTX001 in 45 patients with a follow-up of ~2yrs. after infusion aged 18-35yrs to treat transfusion-dependent beta-thalassemia & sickle cell disease
  • The results from 22 patients with a follow-up of >3 mos., i.e 15 patients with TDT are transfusion-free after CTX001 infusion @ follow-up of 4 to 26 mos., improvement in total hemoglobin & fetal hemoglobin
  • Remaining 7 patients with SCD showed free of VOC’s after CTX001 infusion @ follow-up of 5 to 22 mos., improvements in total hemoglobin & fetal hemoglobin levels. Additioanlly, 5 patients with TDT & 2 patients with SCD with follow-up of > 1yr. showed a stable & durable response

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Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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