Bluebird bio has seen its share price soar over the past year on the promise that its in-development gene therapy could be a functional cure for a rare blood disease. But new clinical data reveal that not all patients respond the same way, possibly dimming the potential of the company's lead treatment.
Shares of bluebird bio took a hit midday Monday after the closely watched biotech noted that one of the patients in a pioneering gene therapy study for beta-thalassemia required two blood transfusions after demonstrating symptoms of anemia following 7 years of being transfusion-free.
Immuno-oncology innovator Kite Pharma is joining gene therapy luminary bluebird bio to craft new treatments for HPV-related cancers, combining two high-profile technologies in hopes of developing targeted therapies.
Bluebird bio took another step forward today in its carefully planned march toward a possible accelerated approval for its lead program, noting that their first sickle cell disease patient has been responding remarkably well to their therapy while adding evidence of improved durability in responses for beta-thalassemia.
Two years after Celgene dangled a selection of $225 million milestone prizes in front of bluebird bio in exchange for collaborating on a range of CAR-T cancer drug programs, the Big Biotech has narrowed its sights to a single target and chipped in a $25 million payment to move their lead candidate into the clinic soon.
Bluebird bio, already making headlines with its promising gene therapy, is expanding its efforts in immuno-oncology, pairing up with Five Prime Therapeutics to develop cell therapies for cancer.
Bluebird bio, at work on a gene therapy for a rare blood disorder, unveiled some early but promising data on the one-time treatment's secondary indication of curing sickle cell disease, sending the biotech's value further skyward.
Bluebird bio, developing a potential cure for a rare blood disorder, is angling for an accelerated approval as it works through clinical trials, setting out a regulatory framework that could get the gene therapy on the market sooner than expected.
Bluebird bio nabbed the FDA's breakthrough drug title for LentiGlobin BB305 as a new treatment for beta-thalassemia major, putting one of the industry's top experimental gene therapies back squarely in a promising spotlight.
Bluebird bio's experimental therapy for the genetic blood disorder beta-thalassemia major is working just as the biotech planned, using a single dose to wean two more patients off of the chronic transfusions required to treat the disease.