Ahead of 2-day ad­comm, FDA sounds pos­i­tive on one blue­bird gene ther­a­py but ques­tions re­main for an­oth­er

In what may be make-or-break meet­ings for blue­bird bio, the FDA’s cell and gene ther­a­py ad­vi­so­ry com­mit­tee will meet Thurs­day and Fri­day to dis­cuss the com­pa­ny’s two po­ten­tial new gene ther­a­pies, with the agency sig­nal­ing a like­ly green light for one to treat a rare blood dis­or­der and ques­tions for the oth­er.

The agency sig­naled in brief­ing doc­u­ments re­leased Tues­day that it’s ready to ap­prove the com­pa­ny’s like­ly ex­pen­sive beti-cel gene ther­a­py to treat β-tha­lassemia for those who re­quire reg­u­lar blood trans­fu­sions.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER