What changed in this filing

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Item 9A. Controls and Procedures

2021-02-11 compared with 2020-02-13 · 47 added, 32 removed, 242 unchanged (25% of the section changed)

17 unchanged sentences

Based on our assessment, our management concluded that, as of December 31, 2020, our internal control over financial reporting is effective based on those criteria.

The effectiveness of our internal control over financial reporting as of December 31, 2019 has been audited by PricewaterhouseCoopers LLP, an independent registered public accounting firm, as stated in their report.

This report appears on pages 79 and 80.

The effectiveness of our internal control over financial reporting as of December 31, 2020 has been audited by PricewaterhouseCoopers LLP, an independent registered public accounting firm, as stated in their report, which is included herein.

Changes in Internal Control

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Incorporated by reference from the information in our Proxy Statement for our 2021 Annual Meeting of Stockholders, which we will file with the SEC within 120 days of the end of the fiscal year to which this annual report on Form 10-K relates.

PRINCIPAL ACCOUNTANT FEES AND SERVICES

EXHIBITS AND FINANCIAL STATEMENT SCHEDULES

2 unchanged sentences

Report of Independent Registered Public Accounting Firm

Consolidated Balance Sheets a s of December 31, 201 9 and 201 8

Consolidated Balance Sheets as of December 31, 2020 and 2019

Consolidated Statements of Operations and Comprehensive Loss for the Years Ended December 31, 2020, 2019 and 2018

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001-36407) and incorporated herein by reference)

3.2# Amended and Restated Bylaws of the Registrant, as amended

3.2 Second Amended and Restated Bylaws of the Registrant, as amended (filed as Exhibit 3.1 to the Registrant’s Quarterly Report on Form 10-Q filed on November 5, 2020 (File No.

001-36407) for the quarterly period ended September 30, 2020 and incorporated herein by reference)

4.1 Specimen certificate evidencing shares of common stock (filed as Exhibit 4.1 to the Registrant’s Registration Statement on Form S-1 (File No.

333-113162) and incorporated herein by reference)

4.2# Description of Capital Stock

4.2 Description of Capital Stock (filed as Exhibit 4.2 to the Registrant’s Annual Report on Form 10-K filed on February 13, 2020 (File No.

001-36407) for the year ended December 31, 2019 and incorporated herein by reference)

10.1** Amended and Restated 2004 Stock Incentive Plan (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on August 8, 2014 (File No.

8 unchanged sentences

001-36407) for the quarterly period ended September 30, 2016 and incorporated herein by reference)

10.6** Amended and Restated 2004 Employee Stock Purchase Plan (filed as Exhibit 10.

1 to the Registrant’s Quarterly Report on Form 10-Q filed on May 2 , 201 9 (File No.

10.6** Amended and Restated 2004 Employee Stock Purchase Plan (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on May 2, 2019 (File No.

001-36407) for the quarterly period ended March 31, 2019 and incorporated herein by reference)

10.7** Amendment to Amended and Restated 2004 Employee Stock Purchase Plan, as amended (filed as Exhibit 10.6 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2020 (File No.

001-36407) for the quarterly period ended June 30, 2020 and incorporated herein by reference)

10.8** 2018 Stock Incentive Plan, as amended (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

001-36407) for the quarterly period ended June 30, 2019 and incorporated herein by reference)

10.9** Second Amendment to 2018 Stock Incentive Plan, as amended (filed as Exhibit 10.5 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2020 (File No.

001-36407) for the quarterly period ended June 30, 2020 and incorporated herein by reference)

10.10** Forms of Incentive Stock Option Agreement, Nonstatutory Stock Option Agreements, Restricted Stock Agreement and Restricted Stock Unit Award Agreement under 2018 Stock Incentive Plan, as amended (filed as Exhibit 10.2 to the Registrant’s Quarterly Report on Form 10-Q filed on August 2, 2018 (File No.

2 unchanged sentences

001-36407) for the quarterly period ended June 30, 2019 and incorporated herein by reference)

10.10**# Amended and Restated Annual Incentive Program

10.12**# Amended and Restated Annual Incentive Program, as amended

10.13** Letter Agreement between the Registrant and John M.

3 unchanged sentences

10.14** Letter Agreement between the Registrant and Barry E.

Greene dated September 29, 2003 (filed as Exhibit 10.10 to the Registrant’s Registration Statement on Form S-1 (File No.

333-113162) and incorporated herein by reference)

Greene dated August 26, 2020 (filed as Exhibit 10.4 to the Registrant’s Quarterly Report on Form 10-Q filed on November 5, 2020 (File No.

001-36407) for the quarterly period ended September 30, 2020 and incorporated herein by reference)

10.15** Letter Agreement between the Registrant and Yvonne L.

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001-36407) for the quarterly period ended September 30, 2017 and incorporated herein by reference)

10.20**# Change in Control Agreement dated November 2, 2020 by and between the Registrant and John M.

10.21 Lease, dated as of September 26, 2003 by and between the Registrant and Three Hundred Third Street LLC (filed as Exhibit 10.15 to the Registrant’s Registration Statement on Form S-1 (File No.

32 unchanged sentences

001-36407) for the quarterly period ended June 30, 2015 and incorporated herein by reference)

10.33 First Amendment to Lease entered into between the Registrant and RREEF America REIT II CORP.

PPP dated September 30, 2020.

(filed as Exhibit 10.3 to the Registrant’s Quarterly Report on Form 10-Q filed on November 5, 2020 (File No.

001-36407) for the quarterly period ended September 30, 2020 and incorporated herein by reference)

10.34 Lease entered into as of April 3, 2015 by and between BMR-675 West Kendall Street LLC and the Registrant (filed as Exhibit 10.7 to the Registrant’s Quarterly Report on Form 10-Q filed on August 7, 2015 (File No.

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000-50743) for the quarterly period ended March 31, 2011 and incorporated herein by reference)

10.38† License and Collaboration Agreement entered into as of May 27, 2008 by and among Takeda Pharmaceutical Company Limited and the Registrant (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on August 8, 2008 (File No.

000-50743) for the quarterly period ended June 30, 2008 and incorporated herein by reference)

10.39† Amended and Restated License and Collaboration Agreement, entered into as of January 1, 2009, by and among the Registrant, Ionis Pharmaceuticals, Inc.

(formerly Isis Pharmaceuticals, Inc.) and Regulus Therapeutics Inc.

(filed as Exhibit 10.3 to the Registrant’s Quarterly Report on Form 10-Q filed on May 8, 2009 (File No.

000-50743) for the quarterly period ended March 31, 2009 and incorporated herein by reference)

10.42† Sublicense Agreement dated effective January 8, 2007 among the Registrant and INEX Pharmaceuticals Corporation (now Arbutus Biopharma Corporation, as successor in interest) (filed as Exhibit 10.38 to the Registrant’s Annual Report on Form 10-K filed on February 18, 2011 (File No.

27 unchanged sentences

000-50743) for the quarterly period ended March 31, 2013 and incorporated herein by reference)

10.50# Amendment to License and Collaboration Agreement, dated as of November 22, 2019 between the Registrant and The Medicines Company

10.51 Stock Purchase Agreement dated as of January 11, 2014 by and between the Registrant and Sanofi Genzyme (formerly Genzyme Corporation) (filed as Exhibit 10.2 to the Registrant’s Quarterly Report on Form 10-Q filed on May 9, 2014 (File No.

001-36407) for the quarterly period ended March 31, 2014 and incorporated herein by reference)

10.52† Amended and Restated Investor Agreement dated as of April 8, 2019 by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.5 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

001-36407) for the quarterly period ended June 30, 2019 and incorporated herein by reference)

10.53† Master Collaboration Agreement dated as of January 11, 2014 by and between the Registrant and Sanofi Genzyme (formerly Genzyme Corporation), including the Regional, Global and Co-Co License Terms Appended thereto (filed as Exhibit 10.4 to the Registrant’s Quarterly Report on Form 10-Q filed on May 9, 2014 (File No.

10.52 Amendment to License and Collaboration Agreement, dated as of November 22, 2019 between the Registrant and The Medicines Company (filed as Exhibit 10.50 to the Registrant’s Annual Report on Form 10-K filed on February 13, 2020 (File No.

001-36407) for the year ended December 31, 2019 and incorporated herein by reference)

10.53† Master Collaboration Agreement dated as of January 11, 2014 by and between the Registrant and Sanofi Genzyme (formerly Genzyme Corporation) (filed as Exhibit 10.4 to the Registrant’s Quarterly Report on Form 10-Q filed on May 9, 2014 (File No.

001-36407) for the quarterly period ended March 31, 2014 and incorporated herein by reference)

10.54† Amendment No.

1 effective as of July 1, 2015 to Master Collaboration Agreement dated as of January 11, 2014, including certain Regional, Global and Co-Co License Terms attached thereto, by and between the Registrant and Sanofi Genzyme (formerly Genzyme Corporation) (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on November 9, 2015 (File No.

1 effective as of July 1, 2015 to Master Collaboration Agreement dated as of January 11, 2014 by and between the Registrant and Sanofi Genzyme (formerly Genzyme Corporation) (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on November 9, 2015 (File No.

001-36407) for the quarterly period ended September 30, 2015 and incorporated herein by reference)

1 unchanged sentence

2 entered into as of January 6, 2018 to the Master Collaboration Agreement dated as of January 11, 2014, as amended by Amendment No.

1, including certain Regional, Global and Co-Co License Terms attached thereto, by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on May 4, 2018 (File No.

1, by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on May 4, 2018 (File No.

001-36407) for the quarterly period ended March 31, 2018 and incorporated herein by reference)

2 unchanged sentences

1 and Amendment No.

2, including certain Regional, Global and Co-Co License Terms attached thereto, by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.3 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

2 by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.3 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

001-36407) for the quarterly period ended June 30, 2019 and incorporated herein by reference)

1 unchanged sentence

001-36407) for the quarterly period ended March 31, 2018 and incorporated herein by reference)

10.58† Amended and Restated ALN-AT3 Global License Terms entered into as of April 8, 2019 by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.

4 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

10.58† Amended and Restated ALN-AT3 Global License Terms entered into as of April 8, 2019 by and between the Registrant and Genzyme Corporation (filed as Exhibit 10.4 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2019 (File No.

001-36407) for the quarterly period ended June 30, 2019 and incorporated herein by reference)

12 unchanged sentences

001-36407) for the quarterly period ended March 31, 2018 and incorporated herein by reference)

10.63† Purchase and Sale Agreement dated April 10, 2020 between BX Bodyguard Royalties L.P.

and the Registrant (filed as Exhibit 10.2 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2020 (File No.

001-36407) for the quarterly period ended June 30, 2020 and incorporated herein by reference)

10.64* Credit Agreement dated April 10, 2020 by and among the Registrant, as Borrower, the Guarantors from time to time party thereto, the Lenders from time to time party thereto, and Wilmington Trust, National Association, as Administrative Agent (filed as Exhibit 10.3 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2020 (File No.

001-36407) for the quarterly period ended June 30, 2020 and incorporated herein by reference)

10.65* First Amendment to Credit Agreement by and among the Registrant, as Borrower, the Guarantors from time to time party thereto, the Lenders from time to time party thereto, and Wilmington Trust, National Association, as Administrative Agent dated August 15, 2020 (filed as Exhibit 10.2 to the Registrant’s Quarterly Report on Form 10-Q filed on November 5, 2020 (File No.

001-36407) for the quarterly period ended September 30, 2020 and incorporated herein by reference)

10.66 Stock Purchase Agreement by and among the Registrant, as Borrower, and the investors listed in Exhibit A thereto, dated April 10, 2020 (filed as Exhibit 4.2 to the Registrant’s Registration Statement on Form S-3 filed on June 5, 2020 (File No.

333-238989) and incorporated herein by reference).

10.67*† Co-Development Agreement between the Registrant and BXLS V Bodyguard – PCP L.P.

and BXLS Family Investment Partnership V – ESC L.P.

dated August 15, 2020 (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on November 5, 2020 (File No.

001-36407) for the quarterly period ended September 30, 2020 and incorporated herein by reference)

10.68† Patent Cross-License Agreement dated April 3, 2020 between Dicerna Pharmaceuticals, Inc.

and the Registrant (filed as Exhibit 10.1 to the Registrant’s Quarterly Report on Form 10-Q filed on August 6, 2020 (File No.

001-36407) for the quarterly period ended June 30, 2020 and incorporated herein by reference)

21.1# Subsidiaries of the Registrant

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** Management contracts or compensatory plans or arrangements required to be filed as an exhibit hereto pursuant to Item 15(a) of Form 10-K.

† Portions of this exhibit (indicated by asterisks) have been omitted in accordance with the rules of the Securities and Exchange Commission.

† Portions of this exhibit (indicated by asterisks) have been omitted in accordance with the rules of the Securities and Exchange Commission because such information (i) is not material and (ii) would likely cause competitive harm to the Registrant if publicly disclosed.

# Filed herewith.

PRINCIPAL ACCOUNTANT FEES AND SERVICES

Incorporated by reference from the information in our Proxy Statement for our 2020 Annual Meeting of Stockholders, which we will file with the SEC within 120 days of the end of the fiscal year to which this annual report on Form 10-K relates.

FORM 10-K SUMMARY

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Bonney Director

/s/ Olivier Brandicourt, M.D.

Olivier Brandicourt, M.D.

/s/ Marsha H.

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Reitan Director

Schimmel, Ph.D.

Schulman Director

1 unchanged sentence

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 9A. Controls and Procedures