Page 1: Filed Pursuant to Rule 424(b)(4)
Page 2: Table of contents
Page 3: Prospectus summary
Page 4: Our gene therapy platform and process
Page 5: Our product candidate pipeline
Page 6: Our Lenti-D product candidate
Page 7: Our LentiGlobin product candidate
Page 8: Our strategic alliance with Celgene
Page 9: Corporate information
Page 10: N/A
Page 11: The offering
Page 12: N/A
Page 13: Summary consolidated financial data
Page 14: N/A
Page 15: Risk factors
Page 16: We have never generated any revenue from product sales and may never be profitable.
Page 17: Even if this offering is successful, we will need to raise additional funding, which may not be avai
Page 18: Risks related to the discovery and development of our product candidates
Page 19: We may find it difficult to enroll patients in our clinical studies, which could delay or prevent cl
Page 20: We may encounter substantial delays in our clinical studies or we may fail to demonstrate safety and
Page 21: N/A
Page 22: We have not tested any of our current viral vectors or product candidates derived from these viral v
Page 23: In previous clinical studies involving viral vectors for gene therapy, some subjects experienced ser
Page 24: ex vivo
Page 25: Risks related to our reliance on third parties
Page 26: We and our contract manufacturers are subject to significant regulation with respect to manufacturin
Page 27: We expect to rely on third parties to conduct, supervise and monitor our clinical studies, and if th
Page 28: Our reliance on third parties requires us to share our trade secrets, which increases the possibilit
Page 29: Risks related to commercialization of our product candidates
Page 30: If we are unable to establish sales and marketing capabilities or enter into agreements with third p
Page 31: The commercial success of any current or future product candidate will depend upon the degree of mar
Page 32: If we obtain approval to commercialize our product candidates outside of the United States, a variet
Page 33: The insurance coverage and reimbursement status of newly-approved products is uncertain. Failure to
Page 34: Due to the novel nature of our technology and the potential for our product candidates to offer ther
Page 35: Our future success depends on our ability to retain key employees, consultants and advisors and to a
Page 36: Our employees, principal investigators, consultants and commercial partners may engage in misconduct
Page 37: If we fail to comply with environmental, health and safety laws and regulations, we could become sub
Page 38: We may not be successful in our efforts to identify or discover additional product candidates.
Page 39: Unfavorable global economic conditions could adversely affect our business, financial condition or r
Page 40: N/A
Page 41: Third-party claims of intellectual property infringement may prevent or delay our development and co
Page 42: We may not be successful in obtaining or maintaining necessary rights to gene therapy product compon
Page 43: If we fail to comply with our obligations in the agreements under which we license intellectual prop
Page 44: We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, wh
Page 45: We may be subject to claims that our employees, consultants or independent contractors have wrongful
Page 46: Issued patents covering our product candidates could be found invalid or unenforceable if challenged
Page 47: We may not be able to protect our intellectual property rights throughout the world.
Page 48: N/A
Page 49: Our principal stockholders and management own a significant percentage of our stock and will be able
Page 50: If you purchase our common stock in this offering, you will incur immediate and substantial dilution
Page 51: Future sales and issuances of our common stock or rights to purchase common stock, including pursuan
Page 52: Our ability to use our net operating loss carryforwards and certain other tax attributes may be limi
Page 53: Provisions in our collaboration agreement with Celgene Corporation may prevent or delay a change in
Page 54: N/A
Page 55: Cautionary note regarding forward-looking statements
Page 56: N/A
Page 57: Use of proceeds
Page 58: Dividend policy
Page 59: Capitalization
Page 60: N/A
Page 61: Dilution
Page 62: N/A
Page 63: Selected consolidated financial data
Page 64: N/A
Page 65: Management s discussion and analysis of financial condition and results of operations
Page 66: Financial operations overview
Page 67: Revenue Recognition
Page 68: N/A
Page 69: General and administrative expenses
Page 70: Critical accounting policies and significant judgments and estimates
Page 71: Revenue Recognition-Multiple-Element Arrangements
Page 72: N/A
Page 73: Revenue Recognition-Milestone Method
Page 74: Stock-based compensation
Page 75: Fair value of stock options
Page 76: April 21, 2011 valuation
Page 77: April 15, 2012 valuation
Page 78: July 23, 2012 valuation
Page 79: N/A
Page 80: December 31, 2012 valuation
Page 81: March 31, 2013 valuation
Page 82: N/A
Page 83: Initial public offering price
Page 84: Convertible preferred stock warrants
Page 85: Emerging growth company status
Page 86: Results of operations
Page 87: Comparison of the years ended December 31, 2011 and 2012
Page 88: Liquidity and capital resources
Page 89: Investing activities
Page 90: Operating capital requirements
Page 91: Contractual obligations and commitments
Page 92: N/A
Page 93: Off-balance sheet arrangements
Page 94: Business
Page 95: N/A
Page 96: The potential of gene therapy to address severe genetic and orphan diseases
Page 97: Our gene therapy platform and process
Page 98: Our proprietary lentiviral vectors
Page 99: Our focus on Hematopoietic Stem Cells (HSCs)
Page 100: Our therapeutic approach
Page 101: N/A
Page 102: Our strategy
Page 103: N/A
Page 104: Our product candidate pipeline
Page 105: Adrenoleukodystrophy
Page 106: Our Lenti-D product candidate
Page 107: Clinical development of our Lenti-D product candidate
Page 108: Three primary clinical measurements of CCALD disease progression
Page 109: Summary of findings
Page 110: N/A
Page 111: Previous clinical experience with lentiviral gene therapy for CCALD (the TG04.06.01 Study)
Page 112: N/A
Page 113: Phase II/III clinical study (the ALD-102 Study)
Page 114: Preclinical evaluation of our Lenti-D product candidate
Page 115: Additional potential clinical indications for Lenti-D
Page 116: Limitations of current treatment options
Page 117: Limitations of current treatment options
Page 118: Clinical development of our LentiGlobin product candidate
Page 119: Phase I/II clinical study for -thalassemia major and sickle cell disease (the HGB-205 Study)
Page 120: Phase I/II clinical study for -thalassemia major (the HGB-204 Study)
Page 121: Preclinical evaluation of our LentiGlobin product candidate
Page 122: Previous preclinical experience with lentiviral gene therapy for sickle cell disease
Page 123: Our target cell transduction process creating the gene-modified cells (our drug product)
Page 124: Future applications and opportunities
Page 125: Strategic collaborations
Page 126: N/A
Page 127: Baylor College of Medicine
Page 128: Intellectual property
Page 129: Childhood Cerebral Adrenoleukodystrophy (CCALD)
Page 130: -thalassemia/SCD
Page 131: N/A
Page 132: License agreements
Page 133: Stanford University
Page 134: Massachusetts Institute of Technology
Page 135: Research Development Foundation
Page 136: Competition
Page 137: N/A
Page 138: Government regulation
Page 139: U.S. biological products development process
Page 140: N/A
Page 141: N/A
Page 142: U.S. review and approval processes
Page 143: N/A
Page 144: Orphan drug designation
Page 145: Post-approval requirements
Page 146: U.S. patent term restoration and marketing exclusivity
Page 147: Additional regulation
Page 148: N/A
Page 149: Facilities
Page 150: Employees
Page 151: Management
Page 152: Jeffrey T. Walsh
Page 153: Linda C. Bain, CPA
Page 154: Steven Gillis, Ph.D.
Page 155: John M. Maraganore, Ph.D.
Page 156: David P. Schenkein, M.D.
Page 157: Board composition
Page 158: Compensation committee
Page 159: Nominating and corporate governance committee
Page 160: Compensation committee interlocks and insider participation
Page 161: Executive and director compensation
Page 162: Involuntary termination of employment
Page 163: Definitions
Page 164: Equity compensation
Page 165: Director compensation
Page 166: Compensation risk assessment
Page 167: N/A
Page 168: N/A
Page 169: 2013 Employee Stock Purchase Plan
Page 170: N/A
Page 171: 2002 Employee, Director and Consultant Plan
Page 172: 401(k) plan
Page 173: Certain relationships and related party transactions
Page 174: Series C financing
Page 175: Director and executive officer compensation
Page 176: Principal stockholders
Page 177: N/A
Page 178: N/A
Page 179: N/A
Page 180: Description of capital stock
Page 181: Warrants
Page 182: Demand registration rights
Page 183: No written consent of stockholders
Page 184: Blank check preferred stock
Page 185: Exclusive jurisdiction of certain actions
Page 186: Shares eligible for future sale
Page 187: Rule 144
Page 188: Rule 701
Page 189: Material U.S. federal income tax considerations for non-U.S. holders
Page 190: Non-U.S. holder defined
Page 191: Gain on disposition of common stock
Page 192: FATCA withholding and information reporting
Page 193: Underwriting
Page 194: N/A
Page 195: N/A
Page 196: N/A
Page 197: Notice to prospective investors in Switzerland
Page 198: Notice to prospective investors in the Dubai International Financial Centre
Page 199: Legal matters
Page 200: Glossary
Page 201: N/A
Page 202: bluebird bio, Inc.
Page 203: Report of independent registered public accounting firm
Page 204: Report of independent registered public accounting firm
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Page 251: 5,941,176 shares