Bluebird Presents At ASH 12/15

The summary of Bluebird's presentation at ASH is now available. We have seen most of this information previously, but there is one very important addition to this info. A new study of beta zero patients will be designed and conducted. As I have suggested previously, there are alterations that can be done to preparation and dosing that may alter the outcome in beta zero patients. I hope to attend the Bluebird webinar on Thursday to hopefully get more information.

Summary of ASH Data from bluebird bio
The results shared at ASH continue to support the potential of LentiGlobin BB305 gene therapy in patients with beta-thalassemia major and severe sickle cell disease. No severe drug-product related adverse events have been observed.
 
Beta-thalassemia
·         People living with beta-thalassemia major can have different genotypes. Differences in outcomes among patients treated with LentiGlobin BB305 gene therapy have been noted among the genotypes.
·         In the Northstar (HGB-204) study, transfusion independence has been achieved in all five of the beta-thalassemia patients with non-beta-0/beta-0 genotype treated at least six months ago. Four other patients with the beta-0/beta-0 genotype have had reduced transfusion needs (33 to 100% decrease in volume), but are not transfusion-free.
·         In the HGB-205 study, two patients with the beta-E/beta-0 genotype have remained transfusion-free for 18 and 21 months since treatment, respectively.
·         Several other patients have been treated in both studies, but it’s too early to know their outcomes.
·        bluebird bio plans to open a new study for patients with the beta-0/beta-0 genotype and plans to move forward with the HGB-207 and HGB-208 studies only in patients with the non-beta-0/beta-0 genotype.
 
Sickle cell disease
·         In the HGB-205 study that also included patients with severe sickle cell disease, the first patient treated with LentiGlobin BB305 is producing more anti-sickling hemoglobin (49% of total hemoglobin), has been transfusion-free for nine months, and has not had a post-treatment hospitalization for a sickle cell disease complication.
·         In the HGB-206 study, three patients have been treated and additional patients are undergoing screening. Early data on two of these patients shows a gradual increase in anti-sickling hemoglobin.
 
Multiple Myeloma
·         Three abstracts describe pre-clinical data supporting development of the chimeric antigen receptor (CAR) T cell therapy bb2121 in multiple myeloma, including insights into the manufacturing platform and engineering of more potent CARs.

Encouraging results.

Is it that Northstar HGB204 study, Bluebird treated 4 patients with beta 0/beta0 genotype and found reduced tansfusion needs, which encourages them to go with HGB207 studies.

Thanks for the update.

I'm wondering more about the success with the sickle cell patient, because the dosing and conditioning are different. There may be something that can be applied to beta zero patients.

Point well taken.

Things seems to be moving in the right direction for both thal and sickle.

Thank you for sharing Andy.  This is all positive news of progress.  

Andy, Thanks for posting the summary. Lots of hope are tied to Bluebird's success......Hoping for continued good news from them.