T

he field of gene therapy is making significant strides towards the day when a one-time treatment ― inserting a healthy gene into patients ― could cure a host of inherited, often fatal diseases. But it’s been a rocky road. And new clinical data presented this morning by Bluebird Bio make clear that success is far from guaranteed. Bluebird is testing a gene therapy for two inherited blood disorders ― and the data managed both to impress and to leave important questions unanswered.

On the impressive side: A patient with the inherited blood disease beta-thalassemia, which causes severe anemia, was able to stop regular blood transfusions one month after treatment and achieved a normal level of hemoglobin in six months, according to interim data from an ongoing phase 3 clinical trial. The positive outcome for this patient suggests, but does not yet prove, that manufacturing changes made by Bluebird last year to improve the efficacy of its investigational therapy, Lentiglobin, are having the desired effect.

Unlock this article by subscribing to STAT Plus today. Try it FREE for 30 days and cancel anytime!

SUBSCRIBE TODAY

What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • Impressive results? Compared to what? Bellicum Pharma data today at EHA on Sickle Cell and Thalassemia report 100% of patients transfusion free months and in some cases years after treatment. Bluebird Thala and Sickle cell programs are obsolete and nothing but fraudulent promotion by a company that misrepresents trial data and sells stock for a living.

Sign up for our Morning Rounds newsletter

Your daily dose of what’s new in health and medicine.

Privacy Policy