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Income Taxes (Tables)
12 Months Ended
Dec. 31, 2019
Income Tax Disclosure [Abstract]  
Components of the Company's Deferred Income Tax Assets (Liabilities)
The significant components of the Company's deferred income tax assets (liabilities) were as follows (in thousands):
December 31,
20192018
Deferred income tax assets:
Federal U.S. net operating loss carryforward$116,436  $87,284  
State net operating loss carryforward31,110  22,809  
Research and development credit, net35,580  29,750  
Orphan drug credit, net22,881  22,580  
Operating lease liabilities8,413  —  
Deferred revenue367  3,736  
Other9,123  9,972  
Gross deferred income tax assets223,910  176,131  
Valuation allowance(214,893) (172,457) 
Net deferred income tax assets9,017  3,674  
Deferred income tax liabilities:
Depreciation(438) (1,911) 
Operating lease ROU assets(5,547) —  
Prepaid expenditures(3,032) (1,763) 
Gross deferred income tax liabilities(9,017) (3,674) 
Net deferred income tax asset/(liability)$—  $—  
Reconciliation of Reported Estimated Income Tax Benefit
The reconciliation of the reported estimated income tax benefit to the amount that would result by applying the U.S. federal statutory tax rate to the net income is as follows (in thousands):
Year Ended December 31,
201920182017
United States federal tax at statutory rate$(31,880) $(36,005) $(6,869) 
State taxes (net of federal benefit)(9,524) (11,133) (735) 
Deferred income tax adjustments2,004  (4,435) 607  
Deferred state blended rate adjustments—  —  (485) 
Deferred federal rate change reduction in corporate rate—  —  39,447  
Research credit, net(5,830) (8,466) (8,455) 
Orphan drug credit, net(301) (872) (1,853) 
Other permanent items1,206  148  276  
Equity-based compensation1,889  758  2,067  
Change in valuation allowance42,436  60,005  (24,000) 
Income tax expense/(benefit)$—  $—  $—  
Reconciliation of the Beginning and Ending Amount of Gross Unrecognized Tax Benefits
A reconciliation of the beginning and ending amount of gross unrecognized tax benefits is as follows (in thousands):
Year Ended December 31,
201920182017
Beginning balance$4,318  $3,395  $2,465  
Increases for current year tax positions637  642  569  
Increases/(decreases) for prior year tax positions(5) 281  361  
Ending balance$4,950  $4,318  $3,395