BLUE - Genetix Pharmaceuticals Inc
Form 424B4 Period: JUN.19.13 Date Filed: JUN.19.13
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  Form 424B4 (Entire Filing)

Filing Body

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

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Page 251: 5,941,176 shares

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