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bluebird bio: Starbeam Treatment Protocol Overview

The following excerpt is from the company's SEC filing.

Subject and Cellular Product Characteristics N=18 consented (formally screened) and N=17 infused subjects Baseline Characteristics Parameter Median (range) Age at consent (years) 6.0 (4-13) Time from CALD diagnosis to treatment (months) 6.8 (2.5-65.8) Time from enrollment to treatment (months) 2.2 (1.9-2.9) Baseline Loes score 2.0 (1.0-7.5) Baseline NFS score 0 (0-0) MFDs present at baseline None 17 subjects treated between October 2013 and July 2015 Median follow-up 16 months (6-24m) as of March 31, 2016 3 completed 24 months 10 with 12 to <24 months follow-up 4 with ≥6 to <12 months follow-up Enrolled n=18 Stem Cell Harvest (apheresis) n=17 Treated n=17 Ineligible n=1 (Loes Score >9) Data as of March 31, 2016

Successful Autologous HSCT Procedure Study procedures generally well tolerated No graft failure Integration site analysis: polyclonal reconstitution in all subjects, no molecular evidence of clonal dominance No replication competent lentivirus detected to date Parameter Median (range) Drug Product Cell Dose (x106/kg) 10.5 (6.0-19.4) Neutrophil engraftment1 (with and without GCSF) Day +13 (11-39) Platelet engraftment2 Day +29 (16-55) Adverse events consistent with myeloablative conditioning Two AEs considered possibly related to drug product: BK-mediated viral cystitis (SAE, grade 3) and tachycardia (grade 1) All AEs and SAEs resolved with standard measures Data as of March 31, 2016 Grade ≥3 non-lab AEs in >1 subject D-10 to D+43 (N=17) ≥D+44 (N=17) Febrile neutropenia 13 (76%) 0 Stomatitis 5 (29%) 0 Nausea 3 (18%) 0 Vomiting 2 (12%) 0 Decreased appetite 6 (35%) 0 Epistaxis 2 (12%) 0 Study procedures generally well tolerated No graft failure or GvHD No molecular evidence of clonal dominance or insertional oncogenesis No replication competent lentivirus detected to date 1. 3 consecutive ANC values of ≥ 0.5 x 109/L obtained on different days. 2. 3 consecutive platelet values ≥ 20 x 109 /L obtained on different days (unsupported)

All Patients Remain Free from Major Functional Disabilities, 16/17 Have Stable NFS1 NFS Progression in Untreated Patients Historical Controls (n=13), Study ALD-1012 1. Stable NFS: change of <3 points and an absolute NFS ≤ 4. 2. GdE+ untreated subjects regardless of stage of disease with more than 1 NFS score reported (N=13), from time of first GdE+ MRI. Most recent score within 1 year prior to HCT reported as screening score. bluebird bio study ALD-101, manuscript in preparation. Data as of March 31, 2016 months since treatment Neurologic Function Scores Starbeam Study

All Patients Remain Free from Major Functional Disabilities, 16/17 Have Stable NFS1 1. Stable NFS: change of <3 points and an absolute NFS ≤ 4. 2. GdE+ HCT treated (N=27) subjects with Loes score ≤9 & NFS ≤1, from time of HCT, all donor sources. Most recent score within 1 year prior to HCT reported as screening score. bluebird bio study ALD-101, manuscript in preparation. Data as of March 31, 2016 months since treatment Neurologic Function Scores Starbeam Study NFS Progression in BMT-Treated Patients Historical Controls eligible for ALD-102 (n=27), Study ALD-1012

Loes Scores Have Stabilized1 in 14/17 Patients Data as of March 31, 2016 1. Stable Loes score: change of ≤ 5 points or an absolute Loes Score ≤ 9. 2. GdE+ HCT-treated (N=27) subjects with Loes score ≤9 & NFS ≤1, from time of HCT, all donor sources. bluebird bio study ALD-101, manuscript in preparation. months since treatment Loes Scores in BMT-Treated Patients Historical Controls eligible for ALD-102 (n=27), Study ALD-1012 Loes Scores in Starbeam Study

Neuroimaging Outcomes Demonstrate Halting of Disease Progression after Lenti-D Treatment pre treatment Data as of March 31, 2016 1 year after Lenti-D 2 years after Lenti-D Representative Untreated patient Subject 2001: First Patient Treated in Starbeam Study Loes score = 2 Loes score = 3 Loes score = 2 T1 Post Flair

Resolution of Gadolinium Enhancement 2001 2002 2003 2004 2005 2006 2007 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 Data as of March 31, 2016. GdE status (+/-) assigned by central MRI reader blinded to subject and time point GdE- GdE+ Subject ID Screening Month 1 Month 6 Month 12 Month 18 Month 24 #2005 Screening 12 months #2004 #2014 #2013 #2009

Persistence of Vector-Marked Cells and ALDP Expression ALD Protein Expression in CD14+ cells Vector Copy Number in Peripheral Blood Data as of March 31, 2016 Median VCN in drug product: 1.0 (0.5-2.5) All samples have detectable VCN and ALDP expression at latest follow-up N=11 N=13 N=13 N=14 N=13 N=6 N=5 Vector copies per diploid genome Months since treatment Months since treatment % CD14 cells positive for ALDP Monocytes (CD14+) differentiate into microglia Median, IQR, min, max

Summary Data as of March 31, 2016 Initial Starbeam results suggest early treatment with Lenti-D gene therapy may halt neuro-inflammation and demyelination in most CALD patients, with promising safety All subjects remain free of Major Functional Disabilities (MFD) to date Stabilization of NFS achieved in 94% (16/17) and Loes score achieved in 82% (14/17) Resolution of gadolinium enhancement by month 6 in 94% (16/17) Re-appearance of diffuse gadolinium enhancement in 5 subjects, resolved in those (n=2) who have later follow-up No deaths, graft failure, or GvHD reported to date AE profile consistent with myeloablative conditioning with busulfan and cyclophosphamide Lenti-D gene therapy may offer an alternative to allogeneic bone marrow transplant, particularly for patients with no matched sibling donor Additional follow-up is needed to fully assess efficacy, durability of effect and long-term safety

Newborn Screening for ALD Early detection is critical for boys with CALD Rapidly degenerative disease; treatment is most successful in boys who are diagnosed early Newborn screening allows boys born with ALD to be monitored for development of symptoms and treated early The federal government now recommends that states add ALD to their newborn screening panels, and it is up to the states to implement screening. Advocates continue to work on a state-by-state basis to get broader adoption. New York has already begun screening Total number of newborns screened in 2014 and 2015 was 469,515 Connecticut recently started screening California, Illinois, New Jersey have agreed to add ALD to their state’s newborn screening panels Note: not all boys identified with ALD will convert to cerebral ALD. Literature suggests that 30 to 40% of ALD cases will convert to CALD.

Anticipated Significant 2016 Milestones FIRST HALF 2016 SECOND HALF 2016 LentiGlobin Thal and SCD Data Update at ASH Initiated CRB-401 Study of bb2121 (Q1) CALD Data Update at AAN (April) Initiate HGB-207 Study of LentiGlobin Cash Runway Through 2018

Transforming the Lives of Patients with Severe Genetic and Rare Diseases Making Hope a Reality

The above information was disclosed in a filing to the SEC. To see the filing, click here.

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Other recent filings from the company include the following:

Bluebird Bio Reports Interim Clinical Data From Starbeam Study Of Lenti-D™ At Aan 2016 Annual Meeting - April 20, 2016
bluebird bio, Inc.'s Chief Medical Officer just cashed-in 1,000 options - April 19, 2016
Bluebird Bio To Present Immuno-Oncology And Gene Therapy Data At The Asgct 19 Annual Meeting CAMBRIDGE, Mass - April 18, 2016