Bluebird Bio Releases Data on Current Gene Therapy Trials

Thank you

BlueBird announced its financial results yesterday. Important announcement for us was below:

“In the second half of 2016, we look forward to continued progress on our clinical programs, including initiation of the LentiGlobin HGB-207 Phase 3 study in non-β0/β0 transfusion-dependent thalassemia (TDT), the integration of manufacturing process improvements into our LentiGlobin clinical trials, and presenting updated LentiGlobin clinical data at ASH.”

I only question if they have any success with B0/B0 transfusion dependent thalassemia. I did not see any reference to that in the conference call.

The beta 0 trials have been split off into their own trials now, with their unique protocol. I don't know if we'll see any updates soon on it. Maybe at ASH in December. My understanding is that the doctors running trials are confident in the eventual success of gene therapy in all forms of thalassemia. My own observation on this process since it began years ago is that there is constant refining and improvement of the vector and the understanding of both dosing and preparation regimen. SK has a new vector that is unique and will give them control over the new genes so they can't get out of control, removing the main concern about gene therapy having any potential dangers. In the long term, it looks quite good.

Thanks Andy.

Appreciate the clarification.

Thanks Andy and Canadian Family for the updates...

bluebird bio Inc Opens Phase 3 Study of LentiGlobin™ Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia

http://www.smarteranalyst.com/2016/09/08/company-update-nasdaqblue-bluebird-bio-inc-opens-phase-3-study-lentiglobin-drug-product-patients-transfusion-dependent-beta-thalassemia/

Thank you Canadian_Family for sharing...Its good news for all patients and parents.

In this study, the process by which the patient’s cells are transduced with LentiGlobin will be modified by the addition of two additives during the transduction step of the manufacturing process, intended to increase vector copy number and the percentage of cells successfully transduced.

I know some get discouraged quickly over early results in some patients, but this process continues to be refined and improved. My understanding is that the doctors running the trials are very optimistic about the eventual outcome, including that for beta zero patients. Patience is needed.

Andy,

Can you please explain this more..i didnt get that. "addition of two additives during the transduction"

The hoped for result of these new additives to the process is "intended to increase vector copy number and the percentage of cells successfully transduced" What this means is that more beta globin should be produced with these changes, which would lead to a greater increase in Hb. Even though this particular study is for non beta zero patients, these changes should mean that the process will also mean a higher increase in Hb in beta zero patients.

Andy
If this medicine comes in 4 or 5 years and takes care of transfusion, will there be need for chelation for Thal intermedia
Can we do anything to help expedite?

Thanks Andy, i hope  beta zero patients could use this therapy soon.

BabyRiya, if a patient undergoes gene therapy, they will need chelation if they were already carrying an iron load. They will not be building a new iron load, though.

Thx Andy

Hi Andy,

I just browsed through ASH conference preliminary program in 2016. I did not see any participation from Bluebird. Do you know if they dropped out from ASH.

http://www.hematology.org/Annual-Meeting/Program/

I see Bluebird is presenting in Gene Therapy Day seminar on October 13, 2016.

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-calendar

Keeping fingers crossed.....??