Bluebird Bio Releases Data on Current Gene Therapy Trials

October 13, 2016 highlights. Read for full details....

http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2211455

Important update:

LentiGlobin Manufacturing Data: A Head-to-Head In Vitro Comparison of Process 1 and Process 2
bluebird bio has recently modified the process by which the patient’s cells are transduced in LentiGlobin clinical studies with the addition of enhancers during the manufacturing process. The goal of manufacturing Process 2 is to increase the percentage of cells successfully transduced, thereby increasing vector copy number (VCN) in the drug product that is given to the patient.

Using retained samples of CD34+ stem cells collected from patients in the HGB-204 (Northstar) and HGB-206 studies, the company was able to demonstrate in a head-to-head in vitro comparison that manufacturing Process 2 substantially increased the percentage of cells transduced and VCN, as compared to manufacturing Process 1.

This in vitro data from Process 2 showed an average increase of approximately three-fold in vector-positive cells and VCN across all patient samples tested. Process 2 has been successfully scaled up for clinical manufacturing, and all LentiGlobin clinical trials moving forward will use manufacturing Process 2, including the Phase 3 HGB-207 (Northstar-2) trial and the Phase 1 HGB-206 clinical trial.

bluebird bio also highlighted progress it has made in moving from adherent manufacturing of lentiviral vectors to potentially more efficient suspension manufacturing, consistently achieving the targeted potency, purity and VCNs at increased scale.

That's some good news, hopefully bluebird gets there quickly.

regards,
R

Wow, things are moving with some pace

I emailed the new rep. Hopefully, I will get a reply.

Canadian_Family,

Thank you kindly for sharing this is great news for all thal patients - especially beta zeros!

My personal take on the news is that there is not much to report in December during ASH conference. The enhancers will take time and new data will not be available until 2017 and beyond. The catalyst that Blue will show positive data from process 1 is wasted. Looking forward to 2017 and beyond.

Bluebird will be presenting on 204, 205 and 206 at ASH

Thanks Andy.

Is it 204, 205 and 206 are beta Zero?

Thanks for the update Andy. Helpful.

Update Nov 3, 2016

In Northstar, the largest study of gene therapy in patients with TDT to date, all patients have demonstrated robust expression of therapeutic hemoglobin, or HbAT87Q, with a safety profile that is consistent with autologous transplantation. We are particularly pleased to see that, across the Northstar and HGB-205 studies, all patients with non-β0/β0 genotypes and at least 12 months of follow-up remained free of transfusions for up to 31 months, adding to the growing body of evidence supporting the potential for LentiGlobin to become a transformative therapy for these patients. Additionally, we have seen clinically meaningful reductions in transfusions for patients with β0/β0 genotypes. We believe that the implementation of manufacturing process 2 going forward has the potential to further reduce transfusion requirements for patients with β0/β0 genotypes, and we will evaluate that hypothesis in our planned Phase 3 HGB-212 trial.

Details http://www.streetinsider.com/

Abstract Results, as of June 27, 2016 Data Cut-off:

Eighteen patients have received LentiGlobin drug product: eight with β0/β0 genotypes and 10 with non-β0/β0 genotypes
The median vector copy number (VCN) in drug product (DP) was 0.7 (range: 0.3 – 1.5 copies/diploid genome) and the median cell dose was 8.1 x 106 CD34+ cells/kg (range: 5.2 – 18.1 x 106 cells/kg)
The median in vivo VCN in patients with at least six months of follow-up was 0.4 copies/diploid genome (range: 0.2-1.0; n=13)
Patients of all genotypes with at least six months of follow-up (n=14) achieved a median HbAT87Q level of 4.7 g/dl and robust HbAT87Q production was sustained in the 10 patients with ≥12 months of follow-up
All patients with non-β0/β0 genotypes and at least 12 months follow-up (n=5) have discontinued transfusions and remain free of transfusions (median 19.4 months without transfusion; range: 15.3 to 24.0 months)
All patients with β0/β0 genotypes have considerably reduced transfusion requirements (median 60% reduction; from median 171.9 ml/kg/year at baseline [range: 168.1- 223.2 ml/kg/year] to 67.8 ml/kg/year post-treatment [range: 14.8 to 123.7 ml/kg/year])
The safety profile remains consistent with autologous stem cell transplantation with no ≥ Grade 3 drug product-related adverse events (AEs) reported.

Bluebird's link on this announcement. http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2219192

bluebird bio Announces First Patient Treated with LentiGlobinTM Drug Product in Northstar-2 (HGB-207) Phase 3 Trial of Patients with Transfusion-Dependent β-Thalassemia

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 14, 2016-- bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced treatment of the first patient in Northstar-2, the Phase 3 study of its LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. This study will use LentiGlobin drug product manufactured with the addition of transduction enhancers intended to increase the drug product vector copy number and percent of cells transduced. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study.

“The opening of our first Phase 3 trial in TDT is an exciting milestone for bluebird,” said David Davidson, chief medical officer. “At ASH we presented interim LentiGlobin data from the Northstar study showing substantial and durable treatment effects, with all patients with non-β0/β0 genotypes and at least twelve months of follow-up achieving freedom from transfusions. We are hopeful that our new manufacturing process incorporating transduction enhancers will build upon these results and may provide even better outcomes for patients. It is an encouraging start for Northstar-2 to have achieved a robust drug product VCN of 2.9 c/dg and 77% LVV+ for our first treated patient. We are tremendously grateful to the patients, families, study investigators, and site staff who have participated in our LentiGlobin program and enabled progress toward our goal of improving the lives of patients with TDT.”

“The current results of the HGB-204 and HGB-205 LentiGlobin studies show a reduction, and in some cases, elimination of RBC transfusions in patients with TDT,” said Mark C. Walters, M.D., UCSF Benioff Children’s Hospital Oakland, a principal investigator on the study. “The current study, Northstar-2, will test a new manufacturing method to increase the VCN in the drug product. If it is successful, this new enhanced transduction method could expand the number of patients who no longer need transfusions after the gene therapy treatment.”

bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β0/β0 genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction.


http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2229663

First SCD patient treated under the amended study protocol in HGB-206.  For details pl. follow the link.

http://finance.yahoo.com/news/bluebird-bio-announces-first-patient-130000772.html

Thanks for sharing..its good news.

canadian Family. pl inform when will Thal cure. if not fully cured the transfusion burden may b reduced. v r tired now. believe me