What happened
Shares of biotech bluebird bio (BLUE +0.00%) are up 7.3% at 12:24 p.m. EDT, having been up as much as 16.3% today, after the company disclosed data in its abstracts for the American Society of Hematology (ASH) meeting in December. Being focused on blood diseases, basically the biotech's entire pipeline is being covered at ASH, from gene therapy for sickle-cell disease and beta-thalassemia to its CAR-T bb2121 program.
So what
Arguably the most important data comes from Bluebird's LentiGlobin gene therapy for sickle-cell disease. Bluebird changed the manufacturing protocol for the therapy to increase the number of copies of the gene inserted into the cell, and improve the ability of the treated cells to be incorporated into the patient, which seems to be having a positive effect.
Two patients have been treated under the new protocol, both of whom had higher copy numbers than were typically produced by the old protocol. Three months after infusion, one of the patients had 1.5 g/dL of the protein expressed by the gene, which is the highest level seen to date in the study.
Image source: Getty Images.
Additionally, Bluebird's CAR-T bb2121, which is further behind but coming up strong, continues to post promising data. The phase 1 trial has now enrolled 21 patients with relapsed and/or refractory multiple myeloma. Of the 18 that could be evaluated for efficacy, the overall response rate (ORR) was 89%. And excluding the three patients treated at the lowest dose, the ORR goes up to 100%. At this point, it's clear bb2121 is working, and Bluebird and partner Celgene (CELG +0.00%) are just trying to figure out the proper dose to take into a pivotal trial.
Now what
Today's disclosures are good news, mostly because they show Bluebird is back on track with its gene therapy and there aren't any hiccups with the CAR-T program. We'll get updated clinical data at ASH. Hopefully it will show that the higher copy number and protein levels are helping reduce or eliminate patients' symptoms of sickle-cell disease, which is the ultimate goal of the treatment.
