Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Blue­bird bio an­nounced it com­plet­ed the rolling sub­mis­sion of its BLA to the FDA for betibeglo­gene au­totem­cel gene ther­a­py.

The ther­a­py, de­signed for pa­tients with be­ta-tha­lassemia who re­quire reg­u­lar red blood cell trans­fu­sions, was pre­vi­ous­ly grant­ed break­through ther­a­py des­ig­na­tion for treat­ing trans­fu­sion-de­pen­dent be­ta-tha­lassemia (TDT). If ap­proved, beti-cel will be the first hematopoi­et­ic stem cell ex-vi­vo gene ther­a­py for pa­tients in the US.

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