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Bluebird Bio Releases Data on Current Gene Therapy Trials

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Canadian_Family:
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.

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

Parin:
Thanks Andy.

Is it 204, 205 and 206 are beta Zero?

Canadian_Family:
Thanks for the update Andy. Helpful.

Canadian_Family:
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.

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