Nature of the Business and Basis of Presentation - Additional Information (Detail) - USD ($) $ in Thousands |
3 Months Ended | ||
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Mar. 31, 2022 |
Mar. 31, 2021 |
Dec. 31, 2021 |
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Organization Consolidation And Presentation Of Financial Statements [Abstract] | |||
Company's mission description | The Company’s mission is to cure Duchenne muscular dystrophy (“Duchenne”), a genetic muscle-wasting disease predominantly affecting boys. | ||
Medicine description | In March 2022, the Company reported two-year interim safety and efficacy data from the first three patients treated with SGT-001 in the 2E14 vg/kg dose cohort of IGNITE DMD, which results suggested durable benefit compared with natural history trajectories 24 months post-administration of SGT-001 across functional, pulmonary and patient reported outcome measures. In addition, no new drug-related safety findings have been identified in patients treated with SGT-001 in IGNITE DMD in post-dosing periods of 90 days to approximately four years. In April 2022, the Company announced that it had concluded enrollment in IGNITE DMD. | ||
Net loss | $ 25,328 | $ 16,900 | |
Accumulated deficit | 502,085 | $ 476,757 | |
Net cash used in operating activities | 27,190 | $ 21,366 | |
Cash, cash equivalents and available-for-sale securities | $ 180,050 |
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- Definition Medicine description. No definition available.
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- Definition Mission description. No definition available.
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- Definition Cash includes currency on hand as well as demand deposits with banks or financial institutions. It also includes other kinds of accounts that have the general characteristics of demand deposits in that the customer may deposit additional funds at any time and effectively may withdraw funds at any time without prior notice or penalty. Cash equivalents, excluding items classified as marketable securities, include short-term, highly liquid Investments that are both readily convertible to known amounts of cash, and so near their maturity that they present minimal risk of changes in value because of changes in interest rates. Generally, only investments with original maturities of three months or less qualify under that definition. Original maturity means original maturity to the entity holding the investment. For example, both a three-month US Treasury bill and a three-year Treasury note purchased three months from maturity qualify as cash equivalents. However, a Treasury note purchased three years ago does not become a cash equivalent when its remaining maturity is three months. Short-term investments, exclusive of cash equivalents, generally consist of marketable securities intended to be sold within one year (or the normal operating cycle if longer) and may include trading securities, available-for-sale securities, or held-to-maturity securities (if maturing within one year), as applicable. Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
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- Definition Amount of cash inflow (outflow) from operating activities, including discontinued operations. Operating activity cash flows include transactions, adjustments, and changes in value not defined as investing or financing activities. Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
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- References No definition available.
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- Definition The consolidated profit or loss for the period, net of income taxes, including the portion attributable to the noncontrolling interest. Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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- Definition The cumulative amount of the reporting entity's undistributed earnings or deficit. Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
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