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General and administrative

^[1]

$ 328

$ 1,358

Operating expenses:

Research and development

^[2]

33,822

19,519

14,929

8,836

Total operating expenses

48,751

28,355

Loss from operations

(48,423)

(26,997)

Other income (expense):

Loss from equity method investment

(1,025)

(1,091)

Other income (expense), net

1,125

(126)

Total other income (expense), net

100

(1,217)

Net loss before income taxes

(48,323)

(28,214)

Provision for income taxes

(85)

(86)

Net loss

(48,408)

(28,300)

Foreign currency translation adjustment

Comprehensive loss

(48,400)

(28,288)

Reconciliation of net loss to net loss attributable to common shareholders:

Net loss

$ (48,408)

$ (28,300)

Net loss per share attributable to common shareholders—basic and diluted

$ (0.93)

$ (0.62)

Weighted-average common shares outstanding used in net loss per share attributable to common shareholders—basic and diluted

52,093,208

45,877,428

[1]	Including the following revenue from a related party, see Notes 6 & 11:
[2]	Including the following research and development expense with a related party, see Notes 6 & 11:

Condensed Consolidated Statements of Operations and Comprehensive Loss (Parenthetical) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Income Statement [Abstract]
Revenue from related party	$ 328	$ 1,082
Research and development expense with a related party	$ 7,587	$ 1,106

Consolidated Statements of Shareholders' Equity - USD ($) $ in Thousands	Total	Common Shares [Member]	Treasury Shares [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Accumulated Other Comprehensive Income (Loss) [Member]
Beginning balance at Dec. 31, 2017	$ 187,832	$ 1,240		$ 312,018	$ (125,440)	$ 14
Beginning balance (in shares) at Dec. 31, 2017		40,592,248	444,873
Cumulative effect of ASC 606 adoption \| ASU 2014-09 [Member]	(1,148)				(1,148)
Issuance of common stock, net of issuance costs	122,597	$ 174		122,423
Issuance of common stock, net of issuance costs (in shares)		5,750,000
Vesting of restricted shares, value	13			13
Vesting of restricted shares (in shares)		10,042
Exercise of vested options, value	2,656	$ 9		2,647
Exercise of vested options (in shares)		328,525	(6,253)
Stock-based compensation expense	6,673			6,673
Other comprehensive loss	12					12
Net loss	(28,300)				(28,300)
Ending balance at Mar. 31, 2018	290,335	$ 1,423		443,774	(154,888)	26
Ending balance (in shares) at Mar. 31, 2018		46,680,815	438,620
Beginning balance at Dec. 31, 2018	392,195	$ 1,584	$ (57)	682,245	(291,569)	(8)
Beginning balance (in shares) at Dec. 31, 2018		51,852,862	307,936
Issuance of common stock, net of issuance costs	23,472			23,472
Issuance of common stock, net of issuance costs (in shares)		631,580
Vesting of restricted shares, value	15			15
Vesting of restricted shares (in shares)		9,288
Exercise of vested options, value	$ 1,832	$ 5		1,827
Exercise of vested options (in shares)	148,743	141,915
Stock-based compensation expense	$ 10,696			10,696
Other comprehensive loss	8					$ 8
Net loss	(48,408)				(48,408)
Ending balance at Mar. 31, 2019	$ 379,810	$ 1,589	$ (57)	$ 718,255	$ (339,977)
Ending balance (in shares) at Mar. 31, 2019		52,635,645	307,936

Consolidated Statements of Shareholders' Equity (Parenthetical) $ in Millions	3 Months Ended
	Mar. 31, 2019 USD ($)	Mar. 31, 2018 USD ($)	Mar. 31, 2019 SFr / shares	Mar. 31, 2018 SFr / shares
Common shares, par value			SFr 0.03
Common Shares [Member]
Issuance costs \| $	$ 1.2	$ 8.2
Common shares, par value			SFr 0.03	SFr 0.03

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Operating activities:
Net loss	$ (48,408)	$ (28,300)
Reconciliation of net loss to net cash used in operating activities:
Depreciation and amortization	812	826
Equity-based compensation	9,671	5,582
Loss from equity method investment	1,025	1,091
Changes in:
Accounts receivable	(243)	1,366
Prepaid expenses and other assets	(3,051)	(2,426)
Accounts payable and accrued expenses	(3,324)	(439)
Deferred revenue	(25)	(218)
Deferred rent		(195)
Other liabilities, net	(194)	25
Net cash used in operating activities	(43,737)	(22,688)
Investing activities:
Purchase of property and equipment	(1,097)	(555)
Net cash used in investing activities	(1,097)	(555)
Financing activities:
Proceeds from issuance of common shares, net of issuance costs	23,894	122,597
Proceeds from exercise of options	1,832	2,654
Net cash provided by financing activities	25,726	125,251
Effect of exchange rate changes on cash	8	12
(Decrease) increase in cash	(19,100)	102,020
Cash, cash equivalents and restricted cash, beginning of period	459,812	242,912
Cash, cash equivalents and restricted cash, end of period	440,712	344,932
Supplemental disclosure of non-cash investing and financing activities
Property and equipment purchases in accounts payable and accrued expenses	579	$ 307
Equity issuance costs in accounts payable and accrued expenses	$ 181

Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018	Mar. 31, 2018	Dec. 31, 2017
Reconciliation to amounts within the condensed consolidated balance sheets
Cash and cash equivalents	$ 437,549	$ 456,649	$ 341,767
Restricted cash	3,163	3,163	3,165
Cash, cash equivalents and restricted cash at end of period	$ 440,712	$ 459,812	$ 344,932	$ 242,912

Basis of Presentation and Significant Accounting Policies

3 Months Ended

Mar. 31, 2019

Accounting Policies [Abstract]

Basis of Presentation and Significant Accounting Policies

1. Basis of Presentation and Significant Accounting Policies

Basis of Presentation

The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America (“GAAP”).

The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. These interim financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the financial position and results of operations for the interim periods ended March 31, 2019 and 2018.

The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2018, which are contained in the 2018 Annual Report on Form 10-K.

Use of Estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses and equity-based compensation expense. The Company bases its estimates on historical experience and various other assumptions, including in certain circumstances future projections that management believes to be reasonable under the circumstances. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known.

Significant Accounting Policies

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2019 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2018 Annual Report on Form 10-K, except with respect to the Company’s lease accounting policy noted within the “Recently adopted accounting standards” section below.

Recently Adopted Accounting Standards

The Company adopted ASC 842, Leases (“ASC 842”), using the required modified retrospective approach, effective January 1, 2019. The Company chose to apply the transition provisions as of the period of adoption. The Company elected the package of practical expedients permitted under the transition guidance within the new standard, which, among other things, allowed the Company to carry forward the historical lease classification. In addition, the Company elected the practical expedient not to apply the recognition requirements in the lease standard to short-term leases (a lease that at commencement date has a lease term of 12 months or less and does not contain a purchase option that it is reasonably certain to exercise) and the practical expedient that permits lessees to make an accounting policy election (by class of underlying asset) to account for each separate lease component of a contract and its associated non-lease components as a single lease component. The adoption of the new standard resulted in the recording net lease assets and lease liabilities of $26.1 million and $37.6 million, respectively, as of January 1, 2019. The difference between the additional lease assets and lease liabilities, is primarily due to the change in classification of lease incentives from liabilities to a reduction in our net lease assets. The standard had no impact on our net loss or cash flows.

Impact of Adopting ASC 842 on the Financial Statements

	January 1, 2019 Prior to ASC 842 Adoption		ASC 842 Adjustment			January 1, 2019 As Adjusted
Consolidated Balance Sheet Data (in thousands):
Prepaid expenses and other current assets⁽¹⁾	$	9,658	$	(553	)	$	9,105
Operating lease assets⁽²⁾	$	—	$	26,087		$	26,087
Deferred rent⁽³⁾⁽⁴⁾	$	1,026	$	(1,026	)	$	—
Deferred rent non-current⁽³⁾	$	11,052	$	(11,052	)	$	—
Operating lease liabilities⁽⁵⁾	$	—	$	4,930		$	4,930
Non-current operating lease liabilities⁽⁵⁾	$	—	$	32,682		$	32,682

(1) Represents reclassification of prepaid rent to operating lease assets.
(2) Represents capitalization of operating lease assets and reclassification of equipment licenses from prepaid expenses to operating lease assets, offset by reclassification of deferred rent to operating lease assets.
(3) Represents reclassification of deferred rent and tenant incentives to operating lease assets.
(4) As of December 31, 2018, the deferred rent balance was $1,202, which included $176 of sublease income received prior to year-end but not due until January 1, 2019.
(5) Represents recognition of operating lease liabilities.

Property and Equipment, net

3 Months Ended

Mar. 31, 2019

Property Plant And Equipment [Abstract]

Property and Equipment, net

2. Property and Equipment, net

Property and equipment, net, consists of the following (in thousands):

	As of
	March 31,			December 31,
	2019			2018
Computer equipment	$	592		$	443
Furniture, fixtures and other		2,453			2,453
Laboratory equipment		9,571			8,964
Leasehold improvements		13,776			13,776
Construction work in process		825			239
Total property and equipment, gross		27,217			25,875
Accumulated depreciation		(8,346	)		(7,375	)
Total property and equipment, net	$	18,871		$	18,500

Depreciation expense for the three months ended March 31, 2019 and 2018 was $1.0 million and $0.8 million, respectively.

Accrued Expenses

3 Months Ended

Mar. 31, 2019

Payables And Accruals [Abstract]

Accrued Expenses

3. Accrued Expenses

Accrued expenses consist of the following (in thousands):

	As of
	March 31,		December 31,
	2019		2018
Payroll and employee-related costs	$	3,834	$	7,321
Research costs		8,023		7,973
Licensing fees		105		625
Professional fees		2,446		1,848
Intellectual property costs		1,101		2,193
Accrued property and equipment		103		294
Other		68		598
Total	$	15,680	$	20,852

Commitments and Contingencies

3 Months Ended

Mar. 31, 2019

Commitments And Contingencies Disclosure [Abstract]

Commitments and Contingencies

4. Commitments and Contingencies

Litigation

In the ordinary course of business, we are from time to time involved in lawsuits, claims, investigations, proceedings, and threats of litigation relating to intellectual property, commercial arrangements, employment and other matters. While the outcome of these proceedings and claims cannot be predicted with certainty, we are not party to any legal or arbitration proceedings that may have significant effects on our financial position. We are not a party to any material proceedings in which any director, member of executive management or affiliate of ours is either a party adverse to us or our subsidiaries or has a material interest adverse to us or our subsidiaries.

Letters of Credit

As of March 31, 2019, the Company had restricted cash of $3.2 million representing letters of credit securing the Company’s obligations under certain leased facilities in Cambridge, Massachusetts, as well as certain credit card arrangements. The letters of credit are secured by cash held in a restricted depository account. The cash deposit is recorded in restricted cash in the accompanying consolidated balance sheet as of March 31, 2019.

Research Agreements

The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the gene-editing technology. The Company is also a party to a number of research license agreements which require significant upfront payments, future royalty payments and potential milestone payments from time to time, as well as intellectual property agreements, which require maintenance and milestone payments from time to time. In association with these agreements, the Company has committed to making payments of $2.1 million and $1.9 million in 2019 and 2020, respectively. For the three months ended March 31, 2019, the Company paid $1.7 million related to these research agreements.

The Company is also a party to a number of manufacturing agreements that require upfront payments for the future performance of services. In connection with these agreements, the Company paid $4.0 million in upfront payments, which were recorded as prepaid expenses as of March 31, 2019. The Company will amortize the prepaid balance as services are performed.

Leases

3 Months Ended

Mar. 31, 2019

Leases [Abstract]

Leases

5. Leases

In June 2015, the Company entered into a lease agreement for the lease of approximately 19,817 square feet of research facility space with a commencement date of November 15, 2015 (the “2015 Lease”). The lease expires in February 2022. The 2015 Lease contains escalating rent clauses which require higher rent payments in future years. With the adoption of ASC 842, the Company has recorded a right-of-use asset and corresponding lease liability.

In May 2016, the Company entered into a sublease agreement for its primary office and research facility in Cambridge, Massachusetts, with a commencement date of December 23, 2016 (the “2016 Sublease”). The sublease expires in December 2026 with an option to extend the term of sublease for an additional five-year period if, at the time of expiration of the initial term, the sublessor does not intend to utilize the space for itself or its affiliates. The 2016 Sublease contains escalating rent clauses which require higher rent payments in future years. With the adoption of ASC 842, the Company has recorded a right-of-use asset and corresponding lease liability. The right-of-use asset and corresponding lease liability does not include the additional five-year period under the option.

In addition, the Company rents certain offices space in Zug, Switzerland, on a short-term basis for which a right-of-use asset and liability are not recorded, in accordance with the practical expedient elected.

The Company has embedded leases in certain research and license agreements for which the Company has recorded a right of use asset and liability. These arrangements are not significant in comparison to the Company’s total operating lease assets and liabilities. In addition, the Company has identified certain short-term leases embedded within its manufacturing contracts which are not recorded on the Company’s balance sheet in accordance with the practical expedient elected.

The Company identified and assessed the following estimates in recognizing the right-of-use asset and corresponding liability:

•

Expected lease term: The expected lease term for those leases commencing prior to January 1, 2019 did not change with the adoption of ASC 842. The expected lease term for leases commencing after the adoption of ASC 842 includes noncancelable lease periods and, when applicable, periods covered by an option to extend the lease if the Company is reasonably certain to exercise that option, as well as periods covered by an option to terminate the lease if the Company is reasonably certain not to exercise that option.

•

Incremental borrowing rate: As the discount rates in the Company’s lease are not implicit, the Company estimated the incremental borrowing rate based on the rate of interest the Company would have to pay to borrow a similar amount on a collateralized basis over a similar term.

The following table summarizes the lease assets and liabilities as of March 31, 2019 (in thousands):

	As of March 31, 2019
Assets
Operating lease assets	$	25,098
Total lease assets		25,098
Liabilities
Current
Operating lease liabilities		4,899
Non-current
Operating lease liabilities, net of current portion		31,728
Total lease liabilities	$	36,627

The following table summarizes operating lease costs included in research and development and general and administrative expense, as well as sublease income for the three months ended March 31, 2019 (in thousands):

	Three months ended March 31, 2019
Operating lease costs	$	1,822
Short-term lease costs		1,138
Variable lease costs		730
Sublease income		(525	)
Net lease cost	$	3,165

The following table summarizes the maturity of undiscounted payments due under lease liabilities and the present value of those liabilities as of March 31, 2019 (in thousands):

	Total
2019	$	6,367
2020		6,866
2021		7,072
2022		5,874
2023		5,855
Thereafter		18,639
Total	$	50,673
Present value adjustment		(14,046	)
Present value of lease liabilities	$	36,627

The following table summarizes the lease term and discount rate as of March 31, 2019:

	As of March 31, 2019
Weighted-average remaining lease term (years)
Operating leases		7.2
Weighted-average discount rate
Operating leases		9.7	%

The following table summarizes the cash paid for amounts included in the measurement of lease liabilities for the three months ended March 31, 2019 (in thousands):

	Three months ended March 31
	2019
Cash paid for amounts included in the measurement of lease liabilities	$	1,818
Operating cash flows from operating leases	$	1,818

Significant Contracts

3 Months Ended

Mar. 31, 2019

Organization Consolidation And Presentation Of Financial Statements [Abstract]

Significant Contracts

6. Significant Contracts

Collaboration Agreement with and Joint Development Agreement with Vertex Pharmaceuticals, Inc.

Summary of Agreement

On October 26, 2015, the Company entered into a strategic collaboration, option and license agreement (“Collaboration Agreement”) with Vertex Pharmaceuticals, Inc. (“Vertex”). The Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. On December 12, 2017, the Company and Vertex entered into Amendment No. 1 to the Collaboration Agreement (the “Amendment”) and the Joint Development Agreement (the “JDA”). The Amendment, among other things, modified certain definitions and provisions of the Collaboration Agreement to make them consistent with the JDA and clarified how many options are exercised (or deemed exercised) in connection with certain targets specified under the Collaboration Agreement. The Amendment also amended other provisions of the Collaboration Agreement, including the expiration terms.

In connection with the Collaboration Agreement, Vertex made a nonrefundable upfront payment of $75.0 million. Under the Collaboration Agreement, Vertex will fund the discovery activities conducted pursuant to the agreement while retaining options to co-exclusive and exclusive licenses. In December 2017, upon execution of the JDA and Amendment, Vertex exercised its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets. As such, for potential hemoglobinopathy treatments, including treatments for sickle cell disease, the Company and Vertex will share equally all research and development costs and worldwide revenues. For other targets that Vertex elects to license, Vertex will lead development and global commercialization activities. For each of up to four remaining targets that Vertex elects to license, the Company has the potential to receive up to $420.0 million in development, regulatory and commercial milestones and royalties on net product sales.

In connection with the JDA, the Company received a $7.0 million up-front payment from Vertex and is eligible for a one-time low seven-digit milestone payment upon the dosing of the second patient in a clinical trial with the initial product candidate. The net profits and net losses, as applicable, incurred under the JDA will be shared equally between the Company and Vertex.

Accounting for the Collaboration Agreement, Amendment and JDA

The arrangements include components of a customer-vendor relationship and collaborative arrangements as defined under ASC 808, Collaborative Arrangements. The Company applies the guidance of ASC 606, Revenue from Contracts with Customers (“ASC 606”) by analogy to the vendor-customer performance obligations of the Collaboration Agreement and the performance obligations of the JDA subject to ASC 606 as outlined below. The Company applies the guidance of ASC 808 to those elements in which there is a collaboration relationship in which both parties share equally in the risks and rewards of the research and development as outlined below.

Accounting Analysis Under ASC 606

As the overall arrangement was modified in December 2017, the Company elected a practical expedient within ASC 606 that allowed entities to reflect the aggregate effect of all contract modifications when identifying the satisfied and unsatisfied performance obligations for contracts that were modified prior to the adoption of ASC 606. As of the December 2017 contract modification date, the Company concluded the arrangement contained the following performance obligations: (i) the non-exclusive research license; (ii) four material rights representing the option for up to four exclusive licenses to develop and commercialize the collaboration targets; (iii) a combined performance obligation representing the co-exclusive research license, and a development and commercialization license to develop and commercialize hemoglobinopathies and beta-globin targets; and (iv) the performance of research and development (“R&D Services”).

The selling price of each performance obligation was determined based on the Company’s estimated standalone selling price (the “ESSP”). The Company developed the ESSP for all the performance obligations included in the Collaboration Agreement and JDA with the objective of determining the price at which it would sell such an item if it were to be sold regularly on a standalone basis. The ESSP for material rights was determined based on the incremental discount given to Vertex based on the ESSP of the four remaining exclusive licenses and the exercise price paid at the time of exercise.

As the Company has a right to consideration from Vertex in an amount that corresponds directly with the value of the Company’s performance completed to date for the R&D Services, the Company recognizes revenue related to the R&D Services as invoiced, in line with the practical expedient in ASC 606-10-55-18.

The transaction price was comprised of: (i) original upfront payment of $75.0 million, (ii) an upfront payment of $7.0 million under the JDA and (iii) $19.3 million of variable consideration associated with the R&D Services. The R&D Services revenue is recognized as invoiced and specifically allocated to the R&D Services performance obligation. The remaining transaction price of $82.0 million was allocated among the performance obligations using the relative selling price method as follows: (i) a non-exclusive research license: $0.5 million; (ii) a material right to discounts for exclusive licenses for up to four Collaboration Targets: $22.2 million, $18.7 million, $8.4 million and $8.4 million for a total of $57.7 million; and (iii) co-exclusive development and commercialization licenses for hemoglobinopathy and beta-globin targets identified in the JDA and co-exclusive research license for the follow-on products: $23.8 million.

The Company recognized $0.1 million and $0.2 million of revenue related to the collaboration with Vertex for the three months ended March 31, 2019 and 2018, respectively. As of March 31, 2019, there was $57.8 million of non-current deferred revenue related to the collaboration with Vertex, which is unchanged from December 31, 2018. The transaction price allocated to the remaining performance obligations is $57.9 million. The remaining performance obligations will be recognized as follows: four material rights to obtain an exclusive commercialization and development license at a point in time, upon exercise; and the non-exclusive research license ratably over/within the remaining two-and-a-half-year research term. As of March 31, 2019, the remaining amount to be recognized for the non-exclusive research license is not significant. R&D Services are recognized as invoiced under the practical expedient and, as such, are not disclosed within the remaining performance obligation balance.

Milestones under the Collaboration Agreement

The Company evaluated the milestones that may be received in connection with the Collaboration Agreement and JDA. The first potential milestone the Company will be entitled to receive is the milestone in the JDA to receive a one-time low seven-digit milestone payment in any clinical trial in the initial shared product and is currently fully constrained. The remaining milestones are predominately related to the development and commercialization of a product resulting from the arrangement and are payable with respect to each selected exclusive license which have yet to be exercised and are not currently included in the determination of the transaction price. Each milestone is payable only once per collaboration target, regardless of the number of products directed to such collaboration target that achieve the relevant milestone event. There are nine remaining clinical development and regulatory approval milestones which may trigger proceeds of up to $90.0 million and $235.0 million, respectively, for each selected exclusive license, and two commercial milestones which may trigger proceeds of up to $75.0 million for each selected exclusive license (which, when combined with the $10.0 million due upon exercise of the exclusive option and the $10.0 million development milestone associated with an Investigational New Drug- enabling application, total $420.0 million for each selected Exclusive License), as follows:

Developmental Milestone Events

Initiation of the first Clinical Trial of a Product

Establishment of Proof of Concept for a Product

Initiation of the first Phase 3 Clinical Trial of a Product

Acceptance of Approval Application by the U.S. Food and Drug Administration for a Product

Acceptance of Approval Application by the European Medicines Agency for a Product

Acceptance of Approval Application by a Regulatory Authority in Japan for a Product

Marketing Approval in the U.S. for a Product

Marketing Approval in the EU for a Product

Marketing Approval in Japan for a Product

Commercial Milestone Events

Annual Net Sales for Products with respect to a Collaboration Target exceed $500 million; and

Annual Net Sales for Products with respect to a Collaboration Target exceed $1.0 billion

There is uncertainty that the events to obtain the developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. Upon exercise of the exclusive license options, developmental milestones will be constrained until the Company is sure that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.

Accounting Analysis under ASC 808

The Company identified the following collaborative elements which are accounted for under ASC 808: (i) development and commercialization services for shared products; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing associated with research and development is included in research and development expense. Expenses related to services performed by the Company are classified as research and development expense. Payments received from Vertex for partial reimbursement of expenses are recorded as a reduction of research and development expense.

The Company recognized $7.1 million and $4.4 million of research and development expense related to the collaboration with Vertex for the three months ended March 31, 2019 and 2018, respectively. Research and development expense for the three months ended March 31, 2019 and 2018 was net of $4.5 million and $3.1 million of reimbursements from Vertex, respectively.

Joint Venture with Bayer Healthcare LLC

On December 19, 2015, the Company entered into an agreement with Bayer Healthcare LLC (“Bayer”), to establish a joint venture (“Bayer Joint Venture”) to focus on the research the development of new therapeutics to cure blood disorders, blindness and congenital heart disease. On February 12, 2016, the Company and Bayer completed the formation of the joint venture entity, Casebia Therapeutics LLP (“Casebia”). Bayer and the Company each received a 50% equity interest in the entity in exchange for their respective contributions to the entity. The Company contributed $0.1 million in cash and licensed its proprietary CRISPR/Cas9 gene editing technology and intellectual property for selected disease indications. Bayer contributed its protein engineering expertise and relevant disease know-how. Under the agreement, Casebia paid the Company $35.0 million in exchange for a worldwide, exclusive license to commercialize the Company’s gene-editing technology specifically for the indications covered by the license. There are no milestone, royalties or other payments due to the Company under this aspect of the agreement. The Company also entered into a separate services agreement with Casebia, under which the Company agreed to provide compensated research and development services.

During 2016, the Company recorded an equity method investment of $36.5 million equal to the fair value of the Company’s interest in Casebia and subsequently recorded unrealized equity method losses for the same amount. The Company has no further contractual obligations to provide cash financing to Casebia and accordingly, no additional losses have been recorded beyond the initial equity amount. Casebia’s net losses were $14.2 million and $12.5 million for the three months ended March 31, 2019 and 2018, respectively. Unrecognized equity method losses in excess of the Company’s equity investment in Casebia were $51.9 million and $45.3 million as of March 31, 2019 and December 31, 2018, respectively.

The remaining performance obligations include research and development services, which are recorded as revenue under ASC 606 and cost sharing activities with Casebia related to shared research and technology licenses are accounted for as a cost/profit sharing arrangement under ASC 808, with the related impact of the cost sharing included as research and development expense. During the three months ended March 31, 2019 and 2018, the Company recognized $0.2 million and $1.1 million of revenue, respectively, related to the collaboration with Casebia. During the three months ended March 31, 2019 and 2018, the Company recognized $0.5 million and $1.1 million, respectively, of research and development expense, related to the collaboration with Casebia. During the three months ended March 31, 2019 and 2018, the Company recognized a loss from equity method investment of $1.0 million and $1.1 million, respectively, related to stock-based compensation expense for Casebia employees.

Share Capital

3 Months Ended

Mar. 31, 2019

Equity [Abstract]

Share Capital

7. Share Capital

The Company had 53,325,963 authorized common shares as of March 31, 2019, with a par value of CHF 0.03 per share. Included in the authorized common shares as of March 31, 2019 are 13,962 shares of unvested restricted stock awards, 307,936 treasury shares which are legally outstanding but not considered outstanding for accounting purposes and 368,420 shares registered and reserved for future issuance. The Company had conditional capital reserved for future issuance of 15,579,296 common shares for employee benefit plans and 4,919,700 common shares for debt instruments as of March 31, 2019. Under Swiss law, authorized share capital consisted of 17,756,799 common shares as of March 31, 2019.

At-the-Market Offering

In August 2018, the Company entered into an At-The-Market (“ATM”) sales agreement with Jefferies LLC (“Jefferies”), under which Jefferies may offer and sell, from time to time, common shares having aggregate gross proceeds of up to $125.0 million. In the first quarter of 2019, we began to issue and sell securities under this sales agreement. During the three months ended March 31, 2019, the Company sold 631,580 shares of common stock for net cash proceeds of $23.9 million, after deducting commission fees of $0.7 million. The Company sold an additional 493,017 shares of common stock under this agreement subsequent to March 31, 2019 through April 29, 2019, resulting in net cash proceeds of approximately $18.7 million.

Stock-based Compensation

3 Months Ended

Mar. 31, 2019

Disclosure Of Compensation Related Costs Sharebased Payments [Abstract]

Stock-based Compensation

8. Stock-based Compensation

During the three months ended March 31, 2019 and 2018, the Company recognized the following stock-based compensation expense (in thousands):

	Three Months Ended March 31,
	2019		2018
Research and development	$	4,962	$	2,916
General and administrative		4,709		2,666
Loss from equity method investment		1,025		1,091
Total	$	10,696	$	6,673

Stock option activity

The following table summarizes stock option activity for the three months ended March 31, 2019 (intrinsic value in thousands):

	Stock Options			Weighted-Average Exercise Price		Weighted-Average Remaining Contractual Term (years)		Aggregate Intrinsic Value
Outstanding at December 31, 2018		6,689,311		$	25.42		8.3	$	68,572
Granted		1,545,573		$	35.25
Exercised		(148,743	)	$	13.35
Cancelled or forfeited		(53,559	)	$	39.57
Outstanding at March 31, 2019		8,032,582		$	27.44		8.5	$	102,371
Exercisable at March 31, 2019		2,798,109		$	17.17		7.7	$	58,010
Vested and expected to vest at March 31, 2019		8,032,582		$	27.44		8.5	$	102,371

The Company estimated the fair value of each stock option award using the Black-Scholes option-pricing model based on the following assumptions:

	Three Months Ended March 31,
	2019			2018
Assumptions
Weighted-average expected volatility		69.3	%		70.5	%
Expected term (in years)		6.0			6.0
Risk-free interest rate		2.5	%		2.7	%
Expected dividend yield		0.0	%		0.0	%

As of March 31, 2019, total unrecognized compensation expense related to stock options was $107.6 million which the Company expects to recognize over a remaining weighted-average period of 3.1 years.

Restricted stock activity

The following table summarizes restricted stock activity for the three months ended March 31, 2019:

	Restricted Stock			Weighted- Average Grant Date Fair Value
Unvested balance as of December 31, 2018		327,342		$	36.72
Granted		40,000			32.06
Vested		(10,046	)		4.88
Cancelled or forfeited		(12,000	)		38.33
Unvested balance as of March 31, 2019		345,296		$	37.05

As of March 31, 2019, total unrecognized compensation expense related to unvested restricted common shares was $9.4 million which the Company expects to recognize over a remaining weighted-average vesting period of 1.6 years.

Net Loss Per Share Attributable to Common Shareholders

3 Months Ended

Mar. 31, 2019

Earnings Per Share [Abstract]

Net Loss Per Share Attributable to Common Shareholders

9. Net Loss Per Share Attributable to Common Shareholders

Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net income per share is calculated by dividing the net income attributable to common shareholders by the weighted-average number of common equivalent shares outstanding for the period, including any dilutive effect from outstanding stock options and warrants using the treasury stock method.

The following common share equivalents, presented on an as-converted basis, were excluded from the calculation of net loss per share for the periods presented due to their anti-dilutive effect (in common stock equivalent shares):

	As of
	March 31, 2019		March 31, 2018
Outstanding options		8,032,582		6,638,120
Unvested restricted common shares		345,296		147,472
Total		8,377,878		6,785,592

Income Taxes

3 Months Ended

Mar. 31, 2019

Income Tax Disclosure [Abstract]

Income Taxes

10. Income Taxes

During the three months ended March 31, 2019 and 2018, the Company recorded an income tax provision of $0.1 million and $0.1 million, respectively, representing an effective tax rate of -0.2% and -0.3%, respectively. The income tax provision is primarily attributable to the year-to-date pre-tax income earned by the Company’s U.S. subsidiary. The difference in the statutory tax rate and effective tax rate is primarily a result of the jurisdictional mix of earnings and losses that are not benefited. The Company maintains a valuation allowance against certain deferred tax assets that are not more-likely-than-not realizable. As a result, the Company has not recognized a tax benefit related to losses generated in Switzerland in the current periods.

Related Party Transactions

3 Months Ended

Mar. 31, 2019

Related Party Transactions

11. Related Party Transactions

In the fourth quarter of 2018, upon becoming owners of record of more than 10% of the voting interest of the Company, Vertex became a related party under ASC 850, Related party disclosures. Refer to Note 6, “Collaboration Agreement with and Joint Development Agreement with Vertex Pharmaceuticals, Inc.” and “Joint Venture with Bayer Healthcare LLC” for discussion of transactions with Casebia and Vertex, related parties.

Basis of Presentation and Significant Accounting Policies (Policies)

3 Months Ended

Mar. 31, 2019

Accounting Policies [Abstract]

Basis of Presentation

Use of Estimates

Recently Adopted Accounting Standards

Impact of Adopting ASC 842 on the Financial Statements

	January 1, 2019 Prior to ASC 842 Adoption		ASC 842 Adjustment			January 1, 2019 As Adjusted
Consolidated Balance Sheet Data (in thousands):
Prepaid expenses and other current assets⁽¹⁾	$	9,658	$	(553	)	$	9,105
Operating lease assets⁽²⁾	$	—	$	26,087		$	26,087
Deferred rent⁽³⁾⁽⁴⁾	$	1,026	$	(1,026	)	$	—
Deferred rent non-current⁽³⁾	$	11,052	$	(11,052	)	$	—
Operating lease liabilities⁽⁵⁾	$	—	$	4,930		$	4,930
Non-current operating lease liabilities⁽⁵⁾	$	—	$	32,682		$	32,682

(1) Represents reclassification of prepaid rent to operating lease assets.
(2) Represents capitalization of operating lease assets and reclassification of equipment licenses from prepaid expenses to operating lease assets, offset by reclassification of deferred rent to operating lease assets.
(3) Represents reclassification of deferred rent and tenant incentives to operating lease assets.
(4) As of December 31, 2018, the deferred rent balance was $1,202, which included $176 of sublease income received prior to year-end but not due until January 1, 2019.
(5) Represents recognition of operating lease liabilities.

Basis of Presentation and Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2019

ASC 842 [Member]

Impact of Adoption ASC 842 on the Financial Statements

Impact of Adopting ASC 842 on the Financial Statements

	January 1, 2019 Prior to ASC 842 Adoption		ASC 842 Adjustment			January 1, 2019 As Adjusted
Consolidated Balance Sheet Data (in thousands):
Prepaid expenses and other current assets⁽¹⁾	$	9,658	$	(553	)	$	9,105
Operating lease assets⁽²⁾	$	—	$	26,087		$	26,087
Deferred rent⁽³⁾⁽⁴⁾	$	1,026	$	(1,026	)	$	—
Deferred rent non-current⁽³⁾	$	11,052	$	(11,052	)	$	—
Operating lease liabilities⁽⁵⁾	$	—	$	4,930		$	4,930
Non-current operating lease liabilities⁽⁵⁾	$	—	$	32,682		$	32,682

(1) Represents reclassification of prepaid rent to operating lease assets.
(2) Represents capitalization of operating lease assets and reclassification of equipment licenses from prepaid expenses to operating lease assets, offset by reclassification of deferred rent to operating lease assets.
(3) Represents reclassification of deferred rent and tenant incentives to operating lease assets.
(4) As of December 31, 2018, the deferred rent balance was $1,202, which included $176 of sublease income received prior to year-end but not due until January 1, 2019.
(5) Represents recognition of operating lease liabilities.

Property and Equipment, net (Tables)

3 Months Ended

Mar. 31, 2019

Property Plant And Equipment [Abstract]

Summary of Property and Equipment, Net

Property and equipment, net, consists of the following (in thousands):

	As of
	March 31,			December 31,
	2019			2018
Computer equipment	$	592		$	443
Furniture, fixtures and other		2,453			2,453
Laboratory equipment		9,571			8,964
Leasehold improvements		13,776			13,776
Construction work in process		825			239
Total property and equipment, gross		27,217			25,875
Accumulated depreciation		(8,346	)		(7,375	)
Total property and equipment, net	$	18,871		$	18,500

Accrued Expenses (Tables)

3 Months Ended

Mar. 31, 2019

Payables And Accruals [Abstract]

Schedule of Accrued Expenses

Accrued expenses consist of the following (in thousands):

	As of
	March 31,		December 31,
	2019		2018
Payroll and employee-related costs	$	3,834	$	7,321
Research costs		8,023		7,973
Licensing fees		105		625
Professional fees		2,446		1,848
Intellectual property costs		1,101		2,193
Accrued property and equipment		103		294
Other		68		598
Total	$	15,680	$	20,852

Leases (Tables)

3 Months Ended

Mar. 31, 2019

Leases [Abstract]

Summary of Lease Assets and Liabilities

The following table summarizes the lease assets and liabilities as of March 31, 2019 (in thousands):

	As of March 31, 2019
Assets
Operating lease assets	$	25,098
Total lease assets		25,098
Liabilities
Current
Operating lease liabilities		4,899
Non-current
Operating lease liabilities, net of current portion		31,728
Total lease liabilities	$	36,627

Summary of Operating Lease Costs Included in Research and Development and General and Administrative Expense, as well as Sublease Income

	Three months ended March 31, 2019
Operating lease costs	$	1,822
Short-term lease costs		1,138
Variable lease costs		730
Sublease income		(525	)
Net lease cost	$	3,165

Summary of Maturity of Undiscounted Payments Due under Lease Liabilities and Present Value of Those Liabilities

The following table summarizes the maturity of undiscounted payments due under lease liabilities and the present value of those liabilities as of March 31, 2019 (in thousands):

	Total
2019	$	6,367
2020		6,866
2021		7,072
2022		5,874
2023		5,855
Thereafter		18,639
Total	$	50,673
Present value adjustment		(14,046	)
Present value of lease liabilities	$	36,627

Summary of Lease Term and Discount Rate

The following table summarizes the lease term and discount rate as of March 31, 2019:

	As of March 31, 2019
Weighted-average remaining lease term (years)
Operating leases		7.2
Weighted-average discount rate
Operating leases		9.7	%

Summary of Cash Paid for Amounts Included in Measurement of Lease Liabilities

The following table summarizes the cash paid for amounts included in the measurement of lease liabilities for the three months ended March 31, 2019 (in thousands):

	Three months ended March 31
	2019
Cash paid for amounts included in the measurement of lease liabilities	$	1,818
Operating cash flows from operating leases	$	1,818

Stock-based Compensation (Tables)

3 Months Ended

Mar. 31, 2019

Disclosure Of Compensation Related Costs Sharebased Payments [Abstract]

Schedule of Stock-Based Compensation Expense

During the three months ended March 31, 2019 and 2018, the Company recognized the following stock-based compensation expense (in thousands):

	Three Months Ended March 31,
	2019		2018
Research and development	$	4,962	$	2,916
General and administrative		4,709		2,666
Loss from equity method investment		1,025		1,091
Total	$	10,696	$	6,673

Summary of Stock Option Activity

The following table summarizes stock option activity for the three months ended March 31, 2019 (intrinsic value in thousands):

	Stock Options			Weighted-Average Exercise Price		Weighted-Average Remaining Contractual Term (years)		Aggregate Intrinsic Value
Outstanding at December 31, 2018		6,689,311		$	25.42		8.3	$	68,572
Granted		1,545,573		$	35.25
Exercised		(148,743	)	$	13.35
Cancelled or forfeited		(53,559	)	$	39.57
Outstanding at March 31, 2019		8,032,582		$	27.44		8.5	$	102,371
Exercisable at March 31, 2019		2,798,109		$	17.17		7.7	$	58,010
Vested and expected to vest at March 31, 2019		8,032,582		$	27.44		8.5	$	102,371

Assumptions to Estimate Fair Value of Stock Option Award Using the Black-Scholes Option-Pricing Model

The Company estimated the fair value of each stock option award using the Black-Scholes option-pricing model based on the following assumptions:

	Three Months Ended March 31,
	2019			2018
Assumptions
Weighted-average expected volatility		69.3	%		70.5	%
Expected term (in years)		6.0			6.0
Risk-free interest rate		2.5	%		2.7	%
Expected dividend yield		0.0	%		0.0	%

Summary of Restricted Stock Activity

The following table summarizes restricted stock activity for the three months ended March 31, 2019:

	Restricted Stock			Weighted- Average Grant Date Fair Value
Unvested balance as of December 31, 2018		327,342		$	36.72
Granted		40,000			32.06
Vested		(10,046	)		4.88
Cancelled or forfeited		(12,000	)		38.33
Unvested balance as of March 31, 2019		345,296		$	37.05

Net Loss Per Share Attributable to Common Shareholders (Tables)

3 Months Ended

Mar. 31, 2019

Earnings Per Share [Abstract]

Schedule of Antidilutive Securities Excluded from Calculation of Net Loss per Share

	As of
	March 31, 2019		March 31, 2018
Outstanding options		8,032,582		6,638,120
Unvested restricted common shares		345,296		147,472
Total		8,377,878		6,785,592

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands	Mar. 31, 2019	Jan. 01, 2019
Summary Of Significant Accounting Policies [Line Items]
Net lease assets	$ 25,098
Net lease liabilities	$ 36,627
ASC 842 [Member]
Summary Of Significant Accounting Policies [Line Items]
Net lease assets		$ 26,087
Net lease liabilities		$ 37,600

Basis of Presentation and Significant Accounting Policies - Impact of Adopting ASC 842 on Financial Statements (Detail) - USD ($) $ in Thousands	Mar. 31, 2019	Jan. 01, 2019	Dec. 31, 2018
New Accounting Pronouncements Or Change In Accounting Principle [Line Items]
Prepaid expenses and other current assets	$ 11,916		$ 9,658
Operating lease assets	25,098
Deferred rent			1,202
Deferred rent non-current			$ 11,052
Operating lease liabilities	4,899
Non-current operating lease liabilities	$ 31,728
ASC 842 [Member]
New Accounting Pronouncements Or Change In Accounting Principle [Line Items]
Prepaid expenses and other current assets		$ 9,105
Operating lease assets		26,087
Operating lease liabilities		4,930
Non-current operating lease liabilities		32,682
Prior [Member] \| ASC 842 [Member]
New Accounting Pronouncements Or Change In Accounting Principle [Line Items]
Prepaid expenses and other current assets		9,658
Deferred rent		1,026
Deferred rent non-current		11,052
Adjustment [Member] \| ASC 842 [Member]
New Accounting Pronouncements Or Change In Accounting Principle [Line Items]
Prepaid expenses and other current assets		(553)
Operating lease assets		26,087
Deferred rent		(1,026)
Deferred rent non-current		(11,052)
Operating lease liabilities		4,930
Non-current operating lease liabilities		$ 32,682

Basis of Presentation and Significant Accounting Policies - Impact of Adopting ASC 842 on Financial Statements (Parenthetical) (Detail) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2019	Dec. 31, 2018
Accounting Policies [Abstract]
Deferred rent		$ 1,202
Sublease income	$ 525	$ 176

Property and Equipment, net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 27,217	$ 25,875
Accumulated depreciation	(8,346)	(7,375)
Total property and equipment, net	18,871	18,500
Computer Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	592	443
Furniture, Fixtures and Other [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	2,453	2,453
Laboratory Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	9,571	8,964
Leasehold Improvements [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	13,776	13,776
Construction Work in Process [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 825	$ 239

Property and Equipment, net - Additional Information (Detail) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2019	Mar. 31, 2018
Property Plant And Equipment [Abstract]
Depreciation expense	$ 1.0	$ 0.8

Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands	Mar. 31, 2019	Dec. 31, 2018
Accrued Liabilities Current [Abstract]
Payroll and employee-related costs	$ 3,834	$ 7,321
Research costs	8,023	7,973
Licensing fees	105	625
Professional fees	2,446	1,848
Intellectual property costs	1,101	2,193
Accrued property and equipment	103	294
Other	68	598
Total	$ 15,680	$ 20,852

Commitments and Contingencies - Additional Information (Detail) - USD ($)
$ in Thousands

3 Months Ended

12 Months Ended

Mar. 31, 2019

Mar. 31, 2018

Dec. 31, 2020

Dec. 31, 2019

Research Agreements [Member]

^[1]

$ 33,822

$ 19,519

Research Agreements [Member] | Scenario Forecast [Member]

1,700

Manufacturing Agreements [Member] | Prepaid Expenses [Member]

$ 1,900

$ 2,100

Upfront payment recorded as prepaid expense

4,000

Letter of Credit [Member]

Letters of credit secured by cash held in restricted depository account

$ 3,200

[1]	Including the following research and development expense with a related party, see Notes 6 & 11:

Leases - Additional Information (Detail) - ft²	1 Months Ended		3 Months Ended
Leases - Additional Information (Detail) - ft²	May 31, 2016	Jun. 30, 2015	Mar. 31, 2019
2015 Lease [Member]
Lessee Lease Description [Line Items]
Lease commencement date		Nov. 15, 2015
Lease expiration date		Feb. 28, 2022
2016 Sublease [Member]
Lessee Lease Description [Line Items]
Lease commencement date	Dec. 23, 2016
Lease expiration date	Dec. 31, 2026
Lessee, operating sublease, option to extend			an option to extend the term of sublease for an additional five-year period if, at the time of expiration of the initial term
Lessee, operating sublease, existence of option to extend	true
Extended term of lease expiration	5 years
Operating Lease, Research Facility Space [Member]
Lessee Lease Description [Line Items]
Leased space		19,817

Leases - Summary of Lease Assets and Liabilities (Detail) $ in Thousands	Mar. 31, 2019 USD ($)
Assets
Operating lease assets	$ 25,098
Total lease assets	25,098
Liabilities
Operating lease liabilities, current	4,899
Operating lease liabilities, net of current portion	31,728
Total lease liabilities	$ 36,627

Leases - Summary of Operating Lease Costs Included in Research and Development and General and Administrative Expense, as well as Sublease Income (Detail) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
	Mar. 31, 2019	Dec. 31, 2018
Leases [Abstract]
Operating lease costs	$ 1,822
Short-term lease costs	1,138
Variable lease costs	730
Sublease income	(525)	$ (176)
Net lease cost	$ 3,165

Leases - Summary of Maturity of Undiscounted Payments Due under Lease Liabilities and Present Value of Those Liabilities (Detail) $ in Thousands	Mar. 31, 2019 USD ($)
Leases [Abstract]
2019	$ 6,367
2020	6,866
2021	7,072
2022	5,874
2023	5,855
Thereafter	18,639
Total	50,673
Present value adjustment	(14,046)
Present value of lease liabilities	$ 36,627

Leases - Summary of Lease Term and Discount Rate (Detail)	Mar. 31, 2019
Leases [Abstract]
Operating leases, weighted-average remaining lease term (years)	7 years 2 months 12 days
Operating leases, weighted-average discount rate	9.70%

Leases - Summary of Cash Paid for Amounts Included in Measurement of Lease Liabilities (Detail) $ in Thousands	3 Months Ended
	Mar. 31, 2019 USD ($)
Leases [Abstract]
Cash paid for amounts included in the measurement of lease liabilities	$ 1,818
Operating cash flows from operating leases	$ 1,818

Significant Contracts - Additional Information (Detail)

3 Months Ended

12 Months Ended

Feb. 12, 2016

USD ($)

Oct. 26, 2015

USD ($)

Milestone

Mar. 31, 2019

USD ($)

Mar. 31, 2018

USD ($)

Dec. 31, 2018

USD ($)

Dec. 31, 2016

USD ($)

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Non-current deferred revenue

^[1]

$ 328,000

$ 1,358,000

57,755,000

$ 57,780,000

Date of joint venture agreement

^[2]

$ 33,822,000

19,519,000

Dec. 19, 2015

Equity method investment

$ 36,500,000

Unrealized equity method losses

$ 1,025,000

1,091,000

$ 36,500,000

Casebia Therapeutics LLP [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

200,000

1,100,000

Date of formation of joint venture entity

$ 500,000

1,100,000

Feb. 12, 2016

Equity method investment, ownership percentage

50.00%

Cash contribution

$ 100,000

Other payments due to company from joint venture entity

Unrealized equity method losses

$ 1,000,000

1,100,000

Net loss of joint venture

14,200,000

12,500,000

Unrecognized equity method losses in excess of Company's interest

51,900,000

$ 45,300,000

License and Service [Member] | Casebia Therapeutics LLP [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Royalty [Member] | Casebia Therapeutics LLP [Member]

35,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member]

$ 0

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Nonrefundable upfront payment received

$ 75,000,000

420,000,000

Non-current deferred revenue

82,000,000

57,900,000

57,800,000

Reimbursements from research and license agreements

7,100,000

4,400,000

4,500,000

3,100,000

Vertex Pharmaceuticals Inc [Member] | License and Service [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Research and Development [Member]

100,000

$ 200,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Variable consideration received

19,300,000

Vertex Pharmaceuticals Inc [Member] | Joint Development Agreement [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Nonrefundable upfront payment received

7,000,000

Up-front payment received

$ 7,000,000

Agreement description

Vertex Pharmaceuticals Inc [Member] | Non-Exclusive Research License [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Material Right to Discounts for Exclusive Licenses [Member]

500,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Material Right to Discounts for Exclusive Licenses [Member] | Unit One [Member]

57,700,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Material Right to Discounts for Exclusive Licenses [Member] | Unit Two [Member]

22,200,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Material Right to Discounts for Exclusive Licenses [Member] | Unit Three [Member]

18,700,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Material Right to Discounts for Exclusive Licenses [Member] | Unit Four [Member]

8,400,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Co-exclusive Development and Commercialization Licenses and Research License [Member]

8,400,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Clinical Development And Regulatory Milestone [Member]

$ 23,800,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Number of developmental milestone events | Milestone

Vertex Pharmaceuticals Inc [Member] | Clinical Development Milestones [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Regulatory Approval Milestone [Member]

$ 90,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Commercial Milestones [Member]

235,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Number of developmental milestone events | Milestone

$ 75,000,000

Vertex Pharmaceuticals Inc [Member] | Exercise of Exclusive Option [Member]

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Collaborative Arrangement Copromotion [Member]

$ 10,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Commercial Milestone Event One [Member] | Minimum [Member]

10,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Vertex Pharmaceuticals Inc [Member] | Commercial Milestone Event Two [Member] | Minimum [Member]

500,000,000

Research and Development Arrangement, Contract to Perform for Others [Line Items]