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CRISPR Therapeutics's Leading Thalassemia And Sickle Cell Disease Investigational Therapy

Apr. 06, 2021 10:35 AM ETCRISPR Therapeutics AG (CRSP)BEAM, BLUE, EDIT, NTLA, SRPT11 Comments
Bashar Issa profile picture
Bashar Issa


  • CRSP's leading investigational therapy CTX001's mode of action creates a situation where the company is killing two birds with one stone, treating both beta-thalassemia and sickle cell disease patients.
  • Although the beta-thalassemia market size is small, the combined patient pool of Sickle Cell Disease and beta-thalassemia is enough for a commercially viable launch once the therapy is approved.
  • The latest update on CTX001 was in December last year. Given the trial's three-month endpoint, investors should expect an update for the newly enrolled patients soon.

Thalassemia test
Photo by jarun011/iStock via Getty Images

Investment Thesis

CRISPR Therapeutics' (NASDAQ:CRSP) leading investigational drug showed promising clinical trials result. All beta-thalassemia patients treated by CTX001 are now transfusion-independent, which is remarkable, given that the average monthly transfusion before treatment was 3

This article was written by

Bashar Issa profile picture
Bashar is a contributing writer at Seeking Alpha, focusing on Long/Short investment ideas, with a geographic focus in North America. Before that, Bashar worked at an Investment Fund in the United Kingdom. He has a Master's degree in Finance from the Queen Mary University of London and a Bachelor's degree in Economics from Middlesex University.

Analyst’s Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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