Product and Service [Axis]
Product [Member]
License [Member]
Equity Components [Axis]
Common Stock [Member]
Additional Paid-in Capital [Member]
Retained Earnings [Member]
AOCI Attributable to Parent [Member]
Plan Name [Axis]
Pre Funded Warrants 2024 [Member]
Loan Agreement Warrants 2024 [Member]
Collaborative Arrangement and Arrangement Other than Collaborative [Axis]
Loan Agreement [Member]
Pre Funded Warrants 2023 [Member]
Antidilutive Securities [Axis]
Share-Based Payment Arrangement, Option [Member]
Restricted Stock [Member]
Warrant [Member]
Concentration Risk Benchmark [Axis]
Revenue Benchmark [Member]
Concentration Risk Type [Axis]
Customer Concentration Risk [Member]
Customer [Axis]
Customer One [Member]
Accounts Receivable [Member]
Customer Two [Member]
Financial Instrument [Axis]
US Treasury Securities [Member]
US Government Corporations and Agencies Securities [Member]
Certificates of Deposit [Member]
Long-Lived Tangible Asset [Axis]
Laboratory Equipment [Member]
Furniture, Software and Office Equipment [Member]
Statistical Measurement [Axis]
Minimum [Member]
Maximum [Member]
Leasehold Improvements [Member]
Construction in Progress [Member]
Equipment [Member]
Loan and Security Agreement [Member]
Measurement Frequency [Axis]
Fair Value, Recurring [Member]
Asset Class [Axis]
Money Market Funds [Member]
Fair Value Hierarchy and NAV [Axis]
Fair Value, Inputs, Level 1 [Member]
Fair Value, Inputs, Level 2 [Member]
Fair Value, Inputs, Level 3 [Member]
Money Market Deposit Account [Member]
Warrant Liabilities [Member]
Class of Warrant or Right [Axis]
2021 Public Offering Warrants [Member]
2022 Private Placement Offering [Member]
2024 Loan Agreement [Member]
2024 Loan Agreement One [Member]
Measurement Input Type [Axis]
Measurement Input, Share Price [Member]
Measurement Input, Expected Term [Member]
Measurement Input, Risk Free Interest Rate [Member]
Measurement Input, Price Volatility [Member]
Measurement Input, Expected Dividend Rate [Member]
Lease Agreement [Member]
Sub Lease Agreement [Member]
Vesting [Axis]
Tranche 1 [Member]
Tranche 2 [Member]
Discretionary Tranche [Member]
Debt Instrument [Axis]
Senior Secured Term Loan [Member]
Award Type [Axis]
July 2025 Avenue Warrants [Member]
Legal Entity [Axis]
Avenue [Member]
Avenue 2 [Member]
2024 Pre-Funded Warrants [Member]
Open Market Sale Agreements [Member]
Jefferies LLC [Member]
Sale of Stock [Axis]
Private Placement Offerings [Member]
Pre Funded Warrant [Member]
Common Stock and Accompanying Warrant [Member]
Pre-Funded Warrant and Accompanying Warrant [Member]
Location, Statement of Income, Balance [Axis]
General and Administrative Expense [Member]
Private Placement [Member]
2023 Pre-Funded Warrants [Member]
2023 Equity Incentive Plan [Member]
Inducement Plan [Member]
2015 Equity Incentive Plan [Member]
Research and Development Expense [Member]
Selling, General and Administrative Expenses [Member]
Exercise Price Range [Axis]
Range One [Member]
Range Two [Member]
Range Three [Member]
Range Four [Member]
Research and Development Arrangement, Contract to Perform for Others, Type [Axis]
License Agreement [Member]
License Agreement Relating to AAV Capsids [Member]
Event Based Milestone Payments [Member]
Sales Based Milestone Payments [Member]
Sublicense And Inventory Purchase Agreements [Member]
Sublicense Agreement Relating to Rett Syndrome [Member]
License Agreement Terms [Member]
Retirement Plan Type [Axis]
Under Age 50 [Member]
Tax Period [Axis]
Tax Year 2032 [Member]
Income Tax Jurisdiction [Axis]
State and Local Jurisdiction [Member]
Definitive Asset Purchase Agreement [Member]
Title and Position [Axis]
Director [Member]
Subsequent Event Type [Axis]
Subsequent Event [Member]
Three Equal Annual Installments [Member]
Cover [Abstract]
Document Type
Amendment Flag
Amendment Description
Document Registration Statement
Document Annual Report
Document Quarterly Report
Document Transition Report
Document Shell Company Report
Document Shell Company Event Date
Document Period Start Date
Document Period End Date
Document Fiscal Period Focus
Document Fiscal Year Focus
Current Fiscal Year End Date
Entity File Number
Entity Registrant Name
Entity Central Index Key
Entity Primary SIC Number
Entity Tax Identification Number
Entity Incorporation, State or Country Code
Entity Address, Address Line One
Entity Address, Address Line Two
Entity Address, Address Line Three
Entity Address, City or Town
Entity Address, State or Province
Entity Address, Country
Entity Address, Postal Zip Code
Country Region
City Area Code
Local Phone Number
Extension
Written Communications
Soliciting Material
Pre-commencement Tender Offer
Pre-commencement Issuer Tender Offer
Title of 12(b) Security
No Trading Symbol Flag
Trading Symbol
Security Exchange Name
Title of 12(g) Security
Security Reporting Obligation
Annual Information Form
Audited Annual Financial Statements
Entity Well-known Seasoned Issuer
Entity Voluntary Filers
Entity Current Reporting Status
Entity Interactive Data Current
Entity Filer Category
Entity Small Business
Entity Emerging Growth Company
Elected Not To Use the Extended Transition Period
Document Accounting Standard
Other Reporting Standard Item Number
Entity Shell Company
Entity Public Float
Entity Bankruptcy Proceedings, Reporting Current
Entity Common Stock, Shares Outstanding
Documents Incorporated by Reference [Text Block]
ICFR Auditor Attestation Flag
Document Financial Statement Error Correction [Flag]
Auditor Firm ID
Auditor Opinion [Text Block]
Auditor Name
Auditor Location
Statement of Financial Position [Abstract]
ASSETS
Current assets:
Cash and cash equivalents
Short-term investments
Restricted cash
Accounts receivable, net
Inventory
Other receivables
Prepaid expenses and other current assets
Total current assets
Property and equipment, net
Operating lease right-of-use assets
Other assets
Total assets
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable
Accrued expenses
Current portion of long-term debt
Current portion of operating lease liability
Accrued taxes
Other current liabilities
Total current liabilities
Long-term operating lease liabilities
Long-term debt
Warrant liabilities
Total liabilities
Commitments and contingencies
Stockholders’ equity:
Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2025 and 2024, respectively
Common stock - $0.01 par value; authorized 200,000,000 shares; 55,043,413 and 45,644,091 shares issued and outstanding as of December 31, 2025 and 2024, respectively
Additional paid-in capital
Accumulated deficit
Accumulated other comprehensive loss
Total stockholders’ equity
Total liabilities and stockholders’ equity
Preferred stock, par value
Preferred stock, shares authorized
Preferred stock, shares issued
Preferred stock, shares outstanding
Common stock, par value
Common stock, shares authorized
Common stock, shares issued
Common stock, shares outstanding
Statement [Table]
Statement [Line Items]
Revenues:
Total revenues
Costs and expenses:
Cost of sales
Royalties
Research and development
Selling, general and administrative
Total costs and expenses
Loss from operations
Interest income
Interest expense
Change in fair value of warrant and derivative liabilities
Gain from sale of priority review voucher, net
Other income, net
Income (loss) before income taxes
Income tax (benefit) expense
Net income (loss)
Basic income (loss) per common share
Dilutive income (loss) per common share
Weighted average number of common shares outstanding:
Basic
Dilutive
Other comprehensive income (loss):
Change in unrealized gains related to available-for-sale debt securities
Comprehensive income (loss)
Balance
Balance, shares
Stock-based compensation expense
Issuance of common stock in connection with restricted share awards, net of cancellations and shares settled for tax withholding settlement
Issuance of common stock in connection with restricted share awards, net of cancellations and shares settled for tax withholding settlement, shares
Issuance of common stock, net of offering costs under open market sale agreement (ATM)
Issuance of common stock, net of offering costs under open market sale agreement (ATM), shares
Issuance of common stock in connection with public offering, net of offering costs
Issuance of common stock in connection with public offering, net of offering costs, shares
Issuance of common stock upon exercise of pre-funded warrants, net of shares settled
Issuance of common stock upon exercise of pre-funded warrants, net of shares settled, shares
Reclassification of warrant liability
Net income (loss)
Other comprehensive income
Issuance of common stock upon exercise of warrants
Issuance of common stock upon exercise of warrants, shares
Balance
Balance, shares
Statement of Cash Flows [Abstract]
Cash flows from operating activities:
Adjustments to reconcile net income (loss) to cash used in operating activities:
Depreciation and amortization
Stock-based compensation expense
Change in fair value of warrant and derivative liabilities
Accretion and interest on short-term investments
Amortization of right-of-use lease assets
Non-cash interest
Gain on disposal of property and equipment
Gain from sale of priority review voucher
Change in operating assets and liabilities:
Accounts receivable
Inventory
Other receivables
Prepaid expenses and other current assets
Other assets
Accounts payable and accrued expenses
Accrued taxes
Lease liabilities
Change in payable to licensor
Other current liabilities
Net cash used in operating activities
Cash flows from investing activities:
Proceeds from sale of priority review voucher, net of transaction costs of $2.6 million
Capital expenditures
Proceeds from disposal of property and equipment
Purchases of short-term investments
Proceeds from maturities of short-term investments
Net cash provided by (used in) investing activities
Cash flows from financing activities:
Proceeds from ATM sales of common stock, net of issuance costs
Payments related to net settlement of restricted share awards
Proceeds from underwritten sales of common stock, net of issuance costs
Proceeds from exercise of warrants
Proceeds from issuance of long-term debt
Payment of debt issuance costs
Net cash provided by financing activities
Net increase in cash, cash equivalents and restricted cash
Cash, cash equivalents and restricted cash at beginning of period
Cash, cash equivalents and restricted cash at end of period
Supplemental cash flow information:
Cash and cash equivalents
Restricted cash
Total cash, cash equivalents and restricted cash
Supplemental non-cash flow information:
Right-of-use asset obtained in exchange for new operating lease liabilities
Derivative and warrant additions associated with loan and security agreement
Reclassification of derivative and warrant liability to equity
Changes in accrued property and equipment
Cash paid for interest
Cash paid for taxes
Transaction costs
Pay vs Performance Disclosure [Table]
Executive Category [Axis]
Individual [Axis]
Adjustment to Compensation [Axis]
Measure [Axis]
Pay vs Performance Disclosure, Table
Company Selected Measure Name
Named Executive Officers, Footnote
Peer Group Issuers, Footnote
Changed Peer Group, Footnote
PEO Total Compensation Amount
PEO Actually Paid Compensation Amount
Adjustment To PEO Compensation, Footnote
Non-PEO NEO Average Total Compensation Amount
Non-PEO NEO Average Compensation Actually Paid Amount
Adjustment to Non-PEO NEO Compensation Footnote
Equity Valuation Assumption Difference, Footnote
Compensation Actually Paid vs. Total Shareholder Return
Compensation Actually Paid vs. Net Income
Compensation Actually Paid vs. Company Selected Measure
Total Shareholder Return Vs Peer Group
Compensation Actually Paid vs. Other Measure
Tabular List, Table
Total Shareholder Return Amount
Peer Group Total Shareholder Return Amount
Net Income (Loss)
Company Selected Measure Amount
Other Performance Measure, Amount
Adjustment to Compensation, Amount
PEO Name
Name
Non-GAAP Measure Description
Additional 402(v) Disclosure
Pension Benefits Adjustments, Footnote
Equity Awards Adjustments, Footnote
Erroneously Awarded Compensation Recovery [Table]
Restatement Determination Date [Axis]
Restatement Determination Date
Aggregate Erroneous Compensation Amount
Erroneous Compensation Analysis
Stock Price or TSR Estimation Method
Outstanding Aggregate Erroneous Compensation Amount
Aggregate Erroneous Compensation Not Yet Determined
Name
Forgone Recovery due to Expense of Enforcement, Amount
Forgone Recovery due to Violation of Home Country Law, Amount
Forgone Recovery due to Disqualification of Tax Benefits, Amount
Forgone Recovery, Explanation of Impracticability
Name
Compensation Amount
Restatement does not require Recovery
Awards Close in Time to MNPI Disclosures [Table]
Award Timing MNPI Disclosure
Award Timing Method
Award Timing Predetermined
Award Timing MNPI Considered
Award Timing, How MNPI Considered
MNPI Disclosure Timed for Compensation Value
Awards Close in Time to MNPI Disclosures, Table
Name
Underlying Securities
Exercise Price
Fair Value as of Grant Date
Underlying Security Market Price Change
Insider Trading Arrangements [Line Items]
Material Terms of Trading Arrangement
Name
Title
Rule 10b5-1 Arrangement Adopted
Non-Rule 10b5-1 Arrangement Adopted
Adoption Date
Rule 10b5-1 Arrangement Terminated
Non-Rule 10b5-1 Arrangement Terminated
Termination Date
Expiration Date
Arrangement Duration
Aggregate Available
Rule 10b5-1 Arrangement Modified
Non-Rule 10b5-1 Arrangement Modified
Insider Trading Policies and Procedures [Line Items]
Insider Trading Policies and Procedures Adopted
Insider Trading Policies and Procedures Not Adopted
Cybersecurity Risk Management, Strategy, and Governance [Abstract]
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]
Cybersecurity Risk Management Processes Integrated [Flag]
Cybersecurity Risk Management Processes Integrated [Text Block]
Cybersecurity Risk Management Third Party Engaged [Flag]
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag]
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag]
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Text Block]
Cybersecurity Risk Board of Directors Oversight [Text Block]
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block]
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]
Cybersecurity Risk Role of Management [Text Block]
Cybersecurity Risk Management Positions or Committees Responsible [Flag]
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]
Cybersecurity Risk Management Expertise of Management Responsible [Text Block]
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block]
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag]
Material Cybersecurity Incident [Abstract]
Material Cybersecurity Incident Nature [Text Block]
Material Cybersecurity Incident Scope [Text Block]
Material Cybersecurity Incident Timing [Text Block]
Material Cybersecurity Incident Material Impact or Reasonably Likely Material Impact [Text Block]
Material Cybersecurity Incident Information Not Available or Undetermined [Text Block]
Organization, Consolidation and Presentation of Financial Statements [Abstract]
NATURE OF OPERATIONS
Accounting Policies [Abstract]
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES
Revenue from Contract with Customer [Abstract]
REVENUE
Investments, Debt and Equity Securities [Abstract]
SHORT-TERM INVESTMENTS
Inventory Disclosure [Abstract]
INVENTORY
Property, Plant and Equipment [Abstract]
PROPERTY AND EQUIPMENT
Fair Value Disclosures [Abstract]
FAIR VALUE MEASUREMENTS
Payables and Accruals [Abstract]
ACCRUED EXPENSES
Leases
LEASES
Debt Disclosure [Abstract]
DEBT
Equity [Abstract]
EQUITY
Share-Based Payment Arrangement [Abstract]
STOCK-BASED COMPENSATION
LICENSE/SUPPLIER AGREEMENTS
Retirement Benefits [Abstract]
401(k) PLAN
Income Tax Disclosure [Abstract]
INCOME TAXES
Commitments and Contingencies Disclosure [Abstract]
COMMITMENTS AND CONTINGENCIES
Segment Reporting [Abstract]
SEGMENT INFORMATION
Sale Of Nonfinancial Assets
SALE OF NONFINANCIAL ASSETS
Subsequent Events [Abstract]
SUBSEQUENT EVENTS
Principles of Consolidation
Use of Estimates
Cash and Cash Equivalents
Restricted Cash
Short-term Investments
Accounts Receivable
Other Receivables
Concentration of Credit Risk and Off-Balance Sheet Risk
Inventory and Costs of Sales
Property and Equipment
Leases
Impairment of Long-Lived Assets
Credit Losses
Segments
Revenue Recognition
Royalties
Research and Development Expenses
Selling, General and Administrative Expenses
Income Taxes
Net Income (Loss) Per Share
Stock-Based Compensation
Derivative Liability
Warrants
Recently Adopted Accounting Pronouncements
Recently Issued Accounting Pronouncements
SCHEDULE OF ACCOUNTS RECEIVABLE
SCHEDULE OF NUMERATORS AND DENOMINATORS OF BASIC AND DILUTED NET INCOME (LOSS) PER SHARE AMOUNTS
SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE
SCHEDULE OF REVENUE
SCHEDULE OF CONCENTRATION OF CREDIT RISK
SCHEDULE OF AVAILABLE FOR SALE SHORT-TERM INVESTMENTS
SCHEDULE OF INVENTORY
SCHEDULE OF PROPERTY AND EQUIPMENT
SCHEDULE OF FAIR VALUE, ASSETS AND LIABILITIES MEASURED ON RECURRING AND NON-RECURRING BASIS
SCHEDULE OF OUTSTANDING WARRANT LIABILITIES
SCHEDULE OF ACTIVITY OF WARRANT LIABILITIES
SCHEDULE OF ESTIMATE FAIR VALUE OF WARRANTS
SCHEDULE OF ACTIVITY OF DERIVATIVE LIABILITIES
SCHEDULE OF ACCRUED EXPENSES
SUMMARY OF OPERATING LEASE LIABILITIES
SCHEDULE OF COMPONENTS OF LEASE COST
SCHEDULE OF MATURITIES OF OPERATING LEASE LIABILITIES
SCHEDULE OF DEBT AND NET OF DEBT ISSUANCE COSTS
SCHEDULE OF FUTURE PAYMENT OBLIGATIONS
Schedule of Share-Based Compensation Arrangements by Share-Based Payment Award [Table]
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
SCHEDULE OF STOCK BASED COMPENSATION
SCHEDULE OF STOCK OPTION ACTIVITY
SCHEDULE OF OPTIONS OUTSTANDING AND EXERCISABLE
SCHEDULE OF RESTRICTED STOCK AWARD ACTIVITY
SCHEDULE OF PROVISION FOR INCOME TAXES
SCHEDULE OF INCOME TAX RATE AND TAX PROVISION
SCHEDULE OF DEFERRED TAX ASSETS AND LIABILITIES
SCHEDULE OF SIGNIFICANT EXPENSE CATEGORIES
Product sales
License revenues
Total accounts receivable, net
SummaryOfSignificantAccountingPoliciesTable [Table]
Other Receivables
Deferred revenue
Unfounded exercise price per share
Warrants to purchase shares
Exercise price
Exercise of warrants
Exercise of warrants
Fair value of warrants
Share price
Net income (loss) used for basic net income (loss) per share
Fair value adjustments for warrant liabilities
Numerator for dilutive net income (loss) per share - net income (loss) available for common shareholders’ after the effect of dilutive securities
Weighted average number of common shares outstanding - basic
Shares of common stock issuable upon exercise of stock options
Shares of common stock underlying restricted stock
Shares of common stock issuable upon exercise of warrants
Shares of common stock issuable upon exercise of conversion feature of loan agreement
Dilutive potential common shares
Denominator for dilutive net income (loss) per share - adjusted weighted average shares used in computing net income (loss) per share - dilutive
Earnings per share:
Antidilutive Security, Excluded EPS Calculation [Table]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total
Disaggregation of Revenue [Table]
Disaggregation of Revenue [Line Items]
Concentration risk, percentage
Revenue reductions of government rebates
Marketable Securities [Table]
Marketable Securities [Line Items]
Amortized cost
Gross Unrealized Gain
Gross Unrealized Loss
Fair Value
Available-for-sale securities realized gain loss
Raw materials
Work-in-progress
Finished goods
Total inventory
Inventory write-down
Property, Plant and Equipment [Table]
Property, Plant and Equipment [Line Items]
Useful lives (years)
Subtotal
Property, Plant, and Equipment, Useful Life, Term, Description [Extensible Enumeration]
Less: accumulated depreciation
Total property and equipment, net
Depreciation expense
Gain (Loss) on Disposition of Property Plant Equipment, Excluding Oil and Gas Property and Timber Property
Fair Value, Recurring and Nonrecurring [Table]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents fair value
Short-term investments fair value
Total assets measured at fair value
Fair value, liabilities
Total liabilities measured at fair value
Class of Warrant or Right [Table]
Class of Warrant or Right [Line Items]
Outstanding warrants liabilities
Warrants, exercise price
Beginning warrant liabilities
Issuance of warrants
Reclassification of warrants to equity as part of warrant exercise
(Gain) loss recognized in earnings from change in fair value
Ending warrant liabilities
Fair Value Measurement Inputs and Valuation Techniques [Table]
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Warrants measurement input
Warrants measurement input
Beginning derivative liabilities
Fair value of derivatives issued in connection with Loan Agreement
Loss recognized in earnings from change in fair value
Derivative, Loss, Statement of Income or Comprehensive Income [Extensible Enumeration]
Reclassification of derivative liability in connection with the Loan Agreement
Ending derivative liabilities
Collaborative Arrangement and Arrangement Other than Collaborative [Table]
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Estimated fair value of loan agreement
Warrants to purchase
Class of warrant
Conversion price
Accrued employee compensation
Accrued contracted services and other
Accrued rebates
Total accrued expenses
Summary Of Operating Lease Liabilities
Current operating lease liability
Non-current operating lease liability
Total operating lease liability
Schedule Of Components Of Lease Cost
Operating lease cost
Variable lease cost
Short-term lease cost
Total operating lease costs
2026
2027
2028
2029
2030
Total undiscounted operating lease payments
Less: imputed interest
Present value of operating lease liabilities
Lease cost
Annual lease payments
Total lease payments
Operating lease assets and liabilities
Operating lease expense
Leasehold improvement allowance
Weighted average remaining term
Weighted average discount rate
Sublease income
Loan Agreement Principal
Accreted final payment fee
Unamortized debt issuance costs and discounts
Total long-term debt
Less: current maturities
Long-term debt, net of current maturities
2026
2027
Total principal
Schedule of Long-Term Debt Instruments [Table]
Debt Instrument [Line Items]
Aggregate principal amount
Aggregate principal amount
Effective interest rate
Interest rate
Stated interest rate
Purchase of warrants
Exercise price
Percentage fee, description
Unrestricted cash
Outstanding principal amount of loan
Exercise price of warrants percent
Fair value of conversion right
Conversion Price
Derivative liabilities
Aggregate transaction amount
Participation right description
Warrants purchase amount
Preferred stock shares authorized
Preferred stock par value
Preferred stock shares outstanding
Shares issued
Warrants purchase, shares
Warrants exercise price
Proceeds from public offering
Stock issuance costs
Additional paid-in capital
Warrant liabilities
Warrants maturity date
Warrants exercised
Purchase price of common stock
Shares issuance costs
Offering price
Net proceeds
Exercise price
Additional paid in capital stock reduction
Proceeds from warrant liability
Warrant outstanding
Warrant exercised
Exercise price per share
Total stock-based compensation expense
Number of options, outstanding balance
Weighted average exercise price, outstanding balance
Weighted average remaining contractual term (years), outstanding
Aggregate intrinsic value, outstanding balance
Number of options, granted
Weighted average exercise price, granted
Number of options, cancelled/forfeited
Weighted average exercise price, cancelled/forfeited
Number of options, exercised
Weighted average exercise price, exercised
Number of options, outstanding balance
Weighted average exercise price, outstanding balance
Aggregate intrinsic value, outstanding balance
Number of options, exercisable
Weighted average exercise price, exercisable
Weighted average remaining contractual term (years), exercisable
Aggregate intrinsic value, exercisable
Number of options, unvested
Weighted average exercise price, unvested
Weighted average remaining contractual term (years), unvested
Aggregate intrinsic value, unvested
Range of exercise price, lower limit
Range of exercise price, upper limit
Number of options outstanding
Weighted-average remaining life in years
Weighted-average exercise price
Number of options exercisable
Weighted-average remaining life in years, exercisable
Weighted-average exercise price, exercisable
Restricted common stock awards, balance
Weighted-average grant date fair value, balance
Restricted common stock awards, granted
Weighted-average grant date fair value, granted
Restricted common stock awards, cancelled/forfeited
Weighted-average grant date fair value, cancelled/forfeited
Restricted common stock awards, vested
Weighted-average grant date fair value, vested
Restricted common stock awards, balance
Weighted-average grant date fair value, balance
Increase in shares authorized
Shares available for grant
Granted
Compensation cost related to non-vested options not recognized
Weighted average period total compensation costs related to non-vested options
Compensation cost related to restricted common stock not recognized
Weighted average compensation costs related to restricted common stock
Fair value of restricted stock awards that vested
Remaining milestone payments due
Accrued royalities
Royalty payment
Royalty payment
Fixed consideration
Variable consideration
Revenue recognized
Contract assets
Accounts receivable
Milestone payments
Defined Benefit Plan [Table]
Defined Benefit Plan Disclosure [Line Items]
Maximum annual contribution per employee under 401(k) plan
Number of investment options
Company contributions under 401 (k) Plan
Current:
U.S. federal
State and local
Total current income tax expense
Deferred:
U.S. federal
State and local
Total deferred income tax expense
Total income tax expense
Income taxes at U.S. statutory rate
U.S. federal statutory tax rate, percentage
State tax, net of federal benefit
State and Local Income Taxes, Net of Federal Income Tax Effect, percentage
Research and development credit
Research and development tax credits, percentage
Deferred true ups
Change in fair value of warrant liabilities
Expired tax losses and credits
Permanent differences
R&D credit expired (under statute or 382 study)
Research and development tax credits expired, percentage
Valuation allowance
Change in valuation allowance, percentage
Change in FV of warrant liabilities
Change in fair value, percentage
Other
Other adjustments, percentage
Federal NOL’s expired (under statute or 382 limitation)
Federal nol Expired, percentage
Share-based awards
Share based Awards, percentage
Other
Other, percentage
Effective income tax rate, percentage
Deferred tax assets:
Net operating loss carryforwards
General business credit carryforwards
State credits
Property and equipment
Stock based compensation
Intangible assets
Accrual to cash conversion
Accruals
Capitalized research and development
Operating lease liabilities
Other
Deferred tax assets before valuation allowance
Valuation allowance
Total deferred tax assets
Deferred tax liabilities:
Property and equipment
Right-of-use asset
Total deferred tax liabilities
Net deferred tax asset (liability)
Operating Loss Carryforwards [Table]
Operating Loss Carryforwards [Line Items]
Federal operating loss carryforwards
Operating loss carry forwards
State net operating loss carryforwards
Operating loss taxable percentage
Income tax description
Deferred tax assets
Valuation allowance
Segment Reporting, Revenue from External Customer, Product and Service [Table]
Revenue from External Customer [Line Items]
Research and development costs:
Salaries & related costs
Non-cash stock-based compensation
Other selling, general and administrative costs
Total research and development costs
Selling, general and administrative costs:
Commercial costs
Total selling, general and administrative costs
Other segment items, net
Gross proceeds
Sale of priority
Subsequent Event [Table]
Subsequent Event [Line Items]
One Annual Installment [Member]
Restricted stock awards, issued
Estimated total stock compensation
Rule 10b5-1 Arrangement Modified
Non-Rule 10b5-1 Arrangement Modified
Warrant liabilities.
Royalty Policy [Text Block]
Lease Agreement [Member]
Schedule of Operating Lease Liabilities [Table Text Block]
Fair value adjustments for warrant and derivative liabilities.
Loan Agreement [Member]
Sub Lease Agreement [Member]
Derivative Liabilty [Policy Text Block]
Warrants [Policy Text Block]
Pre Funded Warrants 2024 [Member]
Number of warrants or rights exercised.
Loan Agreement Warrants 2024 [Member]
Gain from sale of priority review voucher net.
Accreted final payment fee.
Pre Funded Warrants 2023 [Member]
Recently Adopted Accounting Pronouncements [Policy Text Block]
Schedule Of Revenue [Table Text Block]
Loan and Security Agreement [Member]
Tranche 1 [Member]
Tranche 2 [Member]
Discretionary Tranche [Member]
Senior Secured Term Loan [Member]
July 2025 Avenue Warrants [Member]
Exercise price of warrants percent.
Fair value of conversion right.
Participation right description.
Avenue [Member]
Avenue 2 [Member]
Issuance of common stock in connection with public offering.
Stock issued during period value of common stock upon exercise of prefunded warrants net of shares settled.
Stock issued during period shares of common stock upon exercise of prefunded warrants net of shares settled.
Issuance of common stock shares in connection with public offering.
Fair value warrant liabilities.
2021 Public Offering Warrants [Member]
2024 Pre-Funded Warrants [Member]
Gross proceeds from issuance of common stock.
Open Market Sale Agreements [Member]
Jefferies LLC [Member]
Private Placement Offerings [Member]
Pre Funded Warrant [Member]
Common Stock And Accompanying Warrant [Member]
Pre Funded Warrant And Accompanying Warrant [Member]
2023 Pre-Funded Warrants [Member]
2023 Equity Incentive Plan [Member]
Inducement Plan [Member]
Customer One [Member]
Customer Two [Member]
Gain from sale of priority review voucher gross.
Share based compensation arrangement by share based payment award options unvested weighted average remaining contractual term1.
Sharebased compensation arrangement by sharebased payment award options unvested intrinsic value1 .
2015 Equity Incentive Plan [Member]
Range One [Member]
Range Two [Member]
Range Three [Member]
Range Four [Member]
Laboratory Equipment [Member]
Furniture, Software and Office Equipment [Member]
Proceeds from repayment settlement of restricted share awards.
Derivative and warrant additions associated with loan and security agreement.
Reclassification of derivative liability to equity.
Sharebased compensation arrangement by sharebased payment award other than options vested in period fair value.
Remaining milestone payments due.
License Agreement [Member]
Money Market Deposit Account [Member]
License Agreement Relating to AAV Capsids [Member]
Event-Based Milestone Payments [Member]
Sales-Based Milestone Payments [Member]
Changes in accrued property and equipment.
Sublicense And Inventory Purchase Agreements [Member]
Warrant Liabilities [Member]
Sublicense Agreement Relating to Rett Syndrome [Member]
Under Age 50 [Member]
Number of investment options.
Schedule Of Outstanding Warrant Liabilities [Table Text Block]
2022 Private Placement Offering [Member]
2024 Loan Agreement [Member]
2024 Loan Agreement One [Member]
Schedule Of Activity Warranty Liability [Table Text Block]
Gain (loss) recognized in earnings from change in fair value.
Gain recognized in earnings from change in fair value.
Income tax reconciliation tax credits research expired.
Effective income tax rate reconciliation tax credits research expired.
Amount of the difference between reported income tax expense (benefit) and expected income tax expense (benefit) computed by applying the domestic federal statutory income tax rates to pretax income (loss) from continuing operations attributable to nondeductible research and development credit.
Income tax reconciliation change in deferred tax assets true ups.
Income tax reconciliation income tax expense benefit changes of warrant liabilities.
Income tax reconciliation income tax expense benefit expired tax lossed and credit tax rate.
Income tax reconciliation income tax expense benefit permanent differences.
Deferred tax assets property plant and equipments and goodwill.
Deferred tax assets tax deferred expense reserves and accrual accrued liabilities.
Deferred tax assets operating lease liabilities.
Fair value of derivatives issued.
Deferred tax assets right of use assets.
Reclassification of derivative liablity in connection with loan agreement.
Tax Year 2032 [Member]
Precommercial preparation costs.
Other segments.
Sale of Non financial Assets [TextBlock]
Definitive Asset Purchase Agreement [Member]
Three Equal Annual Installments [Member]
One Annual Installment [Member]
Deferred tax assets others.
Royalty expenses.
Schedule of Effective Income Tax Rate Reconciliations [Table Text Block]
Change in fair value, amount.
Operating loss carryforwards taxable percentage.
Operating lease right of use asset and liabilities.
Location, Statement of Income, Balance [Axis]
General and Administrative Expense [Member]
Research and Development Expense [Member]
Selling, General and Administrative Expenses [Member]
Useful Life, Lease Term [Member]
Assets, Current
Assets
Liabilities, Current
Liabilities
Equity, Attributable to Parent
Liabilities and Equity
Costs and Expenses
Operating Income (Loss)
Interest Expense, Operating and Nonoperating
Income (Loss) from Continuing Operations before Income Taxes, Noncontrolling Interest
Comprehensive Income (Loss), Net of Tax, Attributable to Parent
Shares, Outstanding
Accretion (Amortization) of Discounts and Premiums, Investments
Gain (Loss) on Disposition of Property Plant Equipment
GainFromSaleOfPriorityReviewVoucherGross
Increase (Decrease) in Accounts Receivable
Increase (Decrease) in Inventories
Increase (Decrease) in Other Receivables
Increase (Decrease) in Prepaid Expense and Other Assets
Increase (Decrease) in Other Noncurrent Assets
Increase (Decrease) in Accrued Taxes Payable
Increase (Decrease) in Other Current Liabilities
Cash Provided by (Used in) Operating Activity, Including Discontinued Operation
Payments to Acquire Property, Plant, and Equipment
Payments to Acquire Short-Term Investments
Cash Provided by (Used in) Investing Activity, Including Discontinued Operation
Payments of Debt Issuance Costs
Cash Provided by (Used in) Financing Activity, Including Discontinued Operation
Cash, Cash Equivalent, Restricted Cash, and Restricted Cash Equivalent, Period Increase (Decrease), Including Exchange Rate Effect and Discontinued Operation
Cash, Cash Equivalent, Restricted Cash, and Restricted Cash Equivalent, Including Discontinued Operation
Restricted Cash and Cash Equivalent
Cash, Cash Equivalent, Restricted Cash, and Restricted Cash Equivalent, Continuing Operation
Forgone Recovery, Individual Name
Outstanding Recovery, Individual Name
Awards Close in Time to MNPI Disclosures, Individual Name
Trading Arrangement, Individual Name
Lessee, Leases [Policy Text Block]
Royalty Policy [Text Block]
Other Receivables [Default Label]
Debt Securities, Available-for-Sale, Unrealized Loss
Accumulated Depreciation, Depletion and Amortization, Property, Plant, and Equipment
WarrantLiabilities
GainLossRecognizedInEarningsFromChangeInFairValue
GainRecognizedInEarningsFromChangeInFairValue
Warrants and Rights Outstanding, Term
Derivative Liability, Noncurrent
ReclassificationOfDerivativeLiablityInConnectionWithLoanAgreement
Lease, Cost
Lessee, Operating Lease, Liability, to be Paid
Debt Instrument, Unamortized Discount (Premium) and Debt Issuance Costs, Net
Long-Term Debt
Long-Term Debt, Maturity, Year One
Long-Term Debt, Maturity, Year Two
Debt Instrument, Periodic Payment, Principal
Sale of Stock, Price Per Share
Additional Paid in Capital, Common Stock
FairValueWarrantLiabilities
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Intrinsic Value
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Forfeitures and Expirations in Period
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Forfeited in Period
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Vested in Period
Current Income Tax Expense (Benefit)
Deferred Federal Income Tax Expense (Benefit)
Deferred State and Local Income Tax Expense (Benefit)
Deferred Income Tax Expense (Benefit)
IncomeTaxReconciliationNondeductibleExpenseResearchAndDevelopmentCredit
Effective Income Tax Rate Reconciliation, Tax Credit, Research, Percent
IncomeTaxReconciliationTaxCreditsResearchExpired
Effective Income Tax Rate Reconciliation, Nondeductible Expense, Share-Based Payment Arrangement, Percent
Effective Income Tax Rate Reconciliation, Tax Exempt Income, Amount
Effective Income Tax Rate Reconciliation, Tax Exempt Income, Percent
Effective Income Tax Rate Reconciliation, Other Adjustments, Amount
Deferred Tax Assets, Other
Deferred Tax Assets, Gross
Deferred Tax Assets, Valuation Allowance
Deferred Tax Assets, Net of Valuation Allowance
Deferred Tax Liabilities, Property, Plant and Equipment
Deferred Tax Liabilities, Regulatory Assets and Liabilities
Deferred Tax Liabilities, Net
Deferred Tax Assets, Net
Valuation Allowance, Deferred Tax Asset, Increase (Decrease), Amount