■ | 11,143,761 shares of our common stock issuable upon the exercise of stock options outstanding as of December 31, 2021, having a weighted average exercise price of $14.51 per share; |
■ | 1,218,038 shares of our common stock that have been reserved for issuance upon exercise of outstanding warrants as of December 31, 2021, having a weighted average exercise price of $32.04 per share; and |
■ | 23,500 shares of common stock that have been reserved for issuance upon exercise of Restricted Stock Units as of December 31, 2021. |
■ | federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the U.S. Food and Drug Administration (“FDA”); |
■ | the timing of and our ability to submit regulatory filings with the FDA and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates; |
■ | our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting; |
■ | whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval or further development of any of our product candidates; |
■ | our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product candidates, within currently anticipated timeframes, or at all; |
■ | our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates; |
■ | our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates; |
■ | our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances, or joint ventures; |
■ | our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire; |
■ | unanticipated delays due to manufacturing difficulties, including the development of our direct manufacturing capabilities for our AAV programs, and any supply constraints or changes in the regulatory environment; our ability to successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws; |
■ | uncertainties associated with obtaining and enforcing patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims; |
■ | anticipated trends and challenges in our business and the markets in which we operate; |
■ | natural and manmade disasters, including pandemics such as COVID-19, and other force majeures, which could impact our operations, and those of our partners and other participants in the health care industry, and which could adversely impact our clinical studies, preclinical research activities, and drug supply; |
■ | our estimates regarding our capital requirements; and |
■ | our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities; and |
■ | our expected use of proceeds from any offering under this prospectus supplement. |
Assumed public offering price per share | | | | | $17.85 | |
Net tangible book value per share of as December 31, 2021 | | | $6.57 | | | |
Increase in net tangible book value per share attributable to this offering | | | $1.59 | | | |
As adjusted net tangible book value per share as of December 31, 2021, after giving effect to this offering | | | | | $8.15 | |
Dilution per share to investors purchasing our common stock in this offering | | | | | $9.70 |
■ | 11,143,761 shares of our common stock issuable upon the exercise of stock options outstanding as of December 31, 2021, having a weighted average exercise price of $14.51 per share; |
■ | 1,218,038 shares of our common stock that have been reserved for issuance upon exercise of outstanding warrants as of December 31, 2021, having a weighted average exercise price of $32.04 per share; and |
■ | 23,500 shares of common stock that have been reserved for issuance upon exercise of Restricted Stock Units as of December 31, 2021. |
■ | our Annual Report on Form 10-K for the year ended December 31, 2021 filed with the SEC on February 28, 2022, including the information that will be incorporated by reference therein upon the filing of our Definitive Proxy Statement on Schedule 14A to be filed with the SEC; |
■ | the description of the Company’s common stock contained in its registration statement on Form 8-A, which was filed with the SEC on February 2, 2015, and amended on January 11, 2018, including any amendment or report filed for the purpose of updating such description. |
• | federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the U.S. Food and Drug Administration (“FDA”); |
• | the timing of and our ability to submit regulatory filings with the FDA and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates; |
• | our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting; |
• | whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval or further development of any of our product candidates; |
• | our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product candidates, within currently anticipated timeframes, or at all; |
• | our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates; |
• | our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates; |
• | our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances, or joint ventures; |
• | our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire; |
• | unanticipated delays due to manufacturing difficulties, including the development of our direct manufacturing capabilities for our adeno-associated virus programs, and any supply constraints or changes in the regulatory environment; our ability to successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws; |
• | uncertainties associated with obtaining and enforcing patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims; |
• | anticipated trends and challenges in our business and the markets in which we operate; |
• | natural and manmade disasters, including pandemics such as COVID-19, and other force majeures, which could impact our operations, and those of our partners and other participants in the health care industry, and which could adversely impact our clinical studies, preclinical research activities, and drug supply; |
• | our estimates regarding our capital requirements; and |
• | our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities. |
• | do not limit the amount of debt securities that we may issue; |
• | allow us to issue debt securities in one or more series; |
• | do not require us to issue all of the debt securities of a series at the same time; and |
• | allow us to reopen a series to issue additional debt securities without the consent of the holders of the debt securities of such series. |
• | the title of the debt securities and whether they are senior or subordinated; |
• | any limit upon the aggregate principal amount of the debt securities of that series; |
• | the date or dates on which the principal of the debt securities of the series is payable; |
• | the price at which the debt securities will be issued, expressed as a percentage of the principal and, if other than the principal amount thereof, the portion of the principal amount thereof payable upon declaration of acceleration of the maturity thereof or, if applicable, the portion of the principal amount of such debt securities that is convertible into another security of ours or the method by which any such portion shall be determined; |
• | the rate or rates at which the debt securities of the series shall bear interest or the manner of calculation of such rate or rates, if any; |
• | the date or dates from which interest will accrue, the interest payment dates on which such interest will be payable or the manner of determination of such interest payment dates, the place(s) of payment, and the record date for the determination of holders to whom interest is payable on any such interest payment dates or the manner of determination of such record dates; |
• | the right, if any, to extend the interest payment periods and the duration of such extension; |
• | the period or periods within which, the price or prices at which and the terms and conditions |
• | upon which debt securities of the series may be redeemed, converted or exchanged, in whole or in part; |
• | our obligation, if any, to redeem or purchase debt securities of the series pursuant to any sinking fund, mandatory redemption, or analogous provisions (including payments made in cash in satisfaction of future sinking fund obligations) or at the option of a holder thereof and the period or periods within which, the price or prices at which, and the terms and conditions upon which, debt securities of the series shall be redeemed or purchased, in whole or in part, pursuant to such obligation; |
• | the form of the debt securities of the series including the form of the Certificate of Authentication for such series; |
• | if other than minimum denominations of one thousand U.S. dollars ($1,000) or any integral multiple of $1,000 thereof, the denominations in which the debt securities of the series shall be issuable; |
• | whether the debt securities of the series shall be issued in whole or in part in the form of a global debt security or global debt securities; the terms and conditions, if any, upon which such global debt security or global debt securities may be exchanged in whole or in part for other individual debt securities; and the depositary for such global debt security or global debt securities; |
• | whether the debt securities will be convertible into or exchangeable for common stock or other securities of ours or any other Person and, if so, the terms and conditions upon which such debt securities will be so convertible or exchangeable, including the conversion or exchange price, as applicable, or how it will be calculated and may be adjusted, any mandatory or optional (at our option or the holders’ option) conversion or exchange features, and the applicable conversion or exchange period; |
• | any additional or alternative events of default to those set forth in the indenture; |
• | any additional or alternative covenants to those set forth in the indenture; |
• | the currency or currencies including composite currencies, in which payment of the principal of (and premium, if any) and interest, if any, on such debt securities shall be payable (if other than the currency of the United States of America), which unless otherwise specified shall be the currency of the United States of America as at the time of payment is legal tender for payment of public or private debts; |
• | if the principal of (and premium, if any), or interest, if any, on such debt securities is to be payable, at our election or at the election of any holder thereof, in a coin or currency other than that in which such debt securities are stated to be payable, then the period or periods within which, and the terms and conditions upon which, such election may be made; |
• | whether interest will be payable in cash or additional debt securities at our or the holders’ option and the terms and conditions upon which the election may be made; |
• | the terms and conditions, if any, upon which we will pay amounts in addition to the stated interest, premium, if any and principal amounts of the debt securities of the series to any holder that is not a “United States person” for federal tax purposes; |
• | additional or alternative provisions, if any, related to defeasance and discharge of the offered debt securities than those set forth in the indenture; |
• | the applicability of any guarantees; |
• | any restrictions on transfer, sale or assignment of the debt securities of the series; and |
• | any other terms of the debt securities (which may supplement, modify or delete any provision of the indenture insofar as it applies to such series). |
• | either we are the continuing entity, or the successor entity, if other than us, assumes the obligations (a) to pay the principal of, and any premium, and interest on, all of the debt securities and (b) to duly perform and observe all of the covenants and conditions contained in the applicable indenture; and in the event the debt securities are convertible into or exchangeable for common stock or other securities of ours, such successor entity will, by such supplemental indenture, make provision so that the holders of debt securities of that series shall thereafter be entitled to receive upon conversion or exchange of such debt securities the number of securities or property to which a holder of the number of common stock or other securities of ours deliverable upon conversion or exchange of those debt securities would have been entitled had such conversion or exchange occurred immediately prior to such consolidation, merger, sale, conveyance, transfer or other disposition; and |
• | an officers’ certificate and legal opinion covering such conditions are delivered to each applicable trustee. |
• | default in the payment of any installment of interest on any debt security of such series continuing for 90 days unless such date has been extended or deferred; |
• | default in the payment of principal of, or any premium on, any debt security of such series when due and payable unless such date has been extended or deferred; |
• | default in the performance or breach of any covenant or warranty in the debt securities or in the indenture by us continuing for 90 days after written notice described below; |
• | bankruptcy, insolvency or reorganization, or court appointment of a receiver, liquidator or trustee of us; and |
• | any other event of default provided with respect to a particular series of debt securities. |
• | we have deposited with the applicable trustee all required payments of the principal, any premium, interest and, to the extent permitted by law, interest on overdue installment of interest, plus applicable fees, expenses, disbursements and advances of the applicable trustee; and |
• | all events of default, other than the non-payment of accelerated principal, or a specified portion thereof, and any premium, have been cured or waived. |
• | is in conflict with any law or the applicable indenture; |
• | may involve the trustee in personal liability; or |
• | may be unduly prejudicial to the holders of debt securities of the series not joining the proceeding. |
• | to cure any ambiguity, defect, or inconsistency in the applicable indenture or in the Securities of any series; |
• | to comply with the covenant described above under “—Merger, Consolidation or Sale of Assets”; |
• | to provide for uncertificated debt securities in addition to or in place of certificated debt securities; |
• | to add events of default for the benefit of the holders of all or any series of debt securities; |
• | to add the covenants, restrictions, conditions or provisions relating to us for the benefit of the holders of all or any series of debt securities (and if such covenants, restrictions, conditions or provisions are to be for the benefit of less than all series of debt securities, stating that such covenants, restrictions, conditions or provisions are expressly being included solely for the benefit of such series), to make the occurrence, |
• | or the occurrence and the continuance, of a default in any such additional covenants, restrictions, conditions or provisions an event of default, or to surrender any right or power in the applicable indenture conferred upon us; |
• | to add to, delete from, or revise the conditions, limitations, and restrictions on the authorized amount, terms, or purposes of issue, authentication, and delivery of debt securities, as set forth in the applicable indenture; |
• | to make any change that does not adversely affect the rights of any holder of notes under the applicable indenture in any material respect; |
• | to provide for the issuance of and establish the form and terms and conditions of the debt securities of any series as provided in the applicable indenture, to establish the form of any certifications required to be furnished pursuant to the terms of the applicable indenture or any series of debt securities under the applicable indenture, or to add to the rights of the holders of any series of debt securities; |
• | to evidence and provide for the acceptance of appointment under the applicable indenture by a successor trustee or to appoint a separate trustee with respect to any series; |
• | to comply with any requirements of the SEC or any successor in connection with the qualification of the indenture under the Trust Indenture Act of 1939, as amended, or the Trust Indenture Act; or |
• | to conform the applicable indenture to this “—Description of Debt Securities” or any other similarly titled section in any prospectus supplement or other offering document relating to a series of debt securities. |
• | either (i) all securities of such series have already been delivered to the applicable trustee for cancellation; or (ii) all securities of such series have not already been delivered to the applicable trustee for cancellation but (a) have become due and payable, (b) will become due and payable within one year, or (c) if redeemable at our option, are to be redeemed within one year, and we have irrevocably deposited with the applicable trustee, in trust, funds in such currency or currencies, or governmental obligations in an amount sufficient to pay the entire indebtedness on such debt securities in respect of principal and any premium, and interest to the date of such deposit if such debt securities have become due and payable or, if they have not, to the stated maturity or redemption date; |
• | we have paid or caused to be paid all other sums payable. |
• | the offering price and aggregate number of warrants offered; |
• | the currency for which the warrants may be purchased; |
• | if applicable, the designation and terms of the securities with which the warrants are issued and the number of warrants issued with each such security or each principal amount of such security; |
• | if applicable, the date on and after which the warrants and the related securities will be separately transferable; |
• | in the case of warrants to purchase debt securities, the principal amount of debt securities purchasable upon exercise of one warrant and the price at, and currency in which, this principal amount of debt securities may be purchased upon such exercise; |
• | in the case of warrants to purchase common stock or preferred stock, the number of shares of common stock or preferred stock, as the case may be, purchasable upon the exercise of one warrant and the price at which these shares may be purchased upon such exercise; |
• | the effect of any merger, consolidation, sale or other disposition of our business on the warrant agreement and the warrants; |
• | the terms of any rights to redeem or call the warrants; |
• | any provisions for changes to or adjustments in the exercise price or number of securities issuable upon exercise of the warrants; |
• | the periods during which, and places at which, the warrants are exercisable; |
• | the manner of exercise; |
• | the dates on which the right to exercise the warrants will commence and expire; |
• | the manner in which the warrant agreement and warrants may be modified; |
• | federal income tax consequences of holding or exercising the warrants; |
• | the terms of the securities issuable upon exercise of the warrants; and |
• | any other specific terms, preferences, rights or limitations of or restrictions on the warrants. |
• | the designation and terms of the units and of the securities comprising the units, including whether and under what circumstances those securities may be held or transferred separately; |
• | any provisions of the governing unit agreement; |
• | the price or prices at which the units will be issued; |
• | the applicable United States federal income tax considerations relating to the units; |
• | any provisions for the issuance, payment, settlement, transfer or exchange of the units or of the securities comprising the units; and |
• | any other terms of the units and of the securities comprising the units. |
• | to cure any ambiguity, including modifying any provisions of the governing unit agreement that differ from those described below; |
• | to correct or supplement any defective or inconsistent provision; or |
• | to make any other change that we believe is necessary or desirable and will not adversely affect the interests of the affected holders in any material respect. |
• | impair any right of the holder to exercise or enforce any right under a security included in the unit if the terms of that security require the consent of the holder to any changes that would impair the exercise or enforcement of that right; or |
• | reduce the percentage of outstanding units or any series or class the consent of whose holders is required to amend that series or class, or the applicable unit agreement with respect to that series or class, as described below. |
• | If the change affects only the units of a particular series issued under that agreement, the change must be approved by the holders of a majority of the outstanding units of that series; or |
• | If the change affects the units of more than one series issued under that agreement, it must be approved by the holders of a majority of all outstanding units of all series affected by the change, with the units of all the affected series voting together as one class for this purpose. |
• | The units will be issued in the denominations stated in the applicable prospectus supplement. Holders may exchange their units for units of smaller denominations or may request that their units be combined into fewer units of larger denominations, as long as the total amount of units held by a holder is not changed. |
• | Holders may exchange or transfer their units at the office of the unit agent. Holders may also replace lost, stolen, destroyed or mutilated units at that office. We may appoint another entity to perform these functions or perform them ourselves. |
• | Holders will not be required to pay a service charge to transfer or exchange their units, but they may be required to pay for any tax or other governmental charge associated with the transfer or exchange. The transfer or exchange, and any replacement, will be made only if our transfer agent is satisfied with the holder’s proof of legal ownership. The transfer agent may also require an indemnity before replacing any units. |
• | If we have the right to redeem, accelerate or settle any units before their maturity, and we exercise our right as to less than all those units or other securities, we may block the exchange or transfer of those units during the period beginning 15 days before the day we mail the notice of exercise and ending on the day of that mailing, in order to freeze the list of holders to prepare the mailing. We may also refuse to register transfers of or exchange any unit selected for early settlement, except that we will continue to permit transfers and exchanges of the unsettled portion of any unit being partially settled. We may also block the transfer or exchange of any unit in this manner if the unit includes securities that are or may be selected for early settlement. |
• | before the stockholder became interested, our Board of Directors approved either the business combination or the transaction which resulted in the stockholder becoming an interested stockholder; |
• | upon consummation of the transaction which resulted in the stockholder becoming an interested stockholder, the interested stockholder owned at least 85% of the voting stock of the corporation outstanding at the time the transaction commenced, excluding for purposes of determining the voting stock outstanding, shares owned by persons who are directors and also officers, and employee stock plans, in some instances, but not the outstanding voting stock owned by the interested stockholder; or |
• | at or after the time the stockholder became interested, the business combination was approved by our Board of Directors and authorized at an annual or special meeting of the stockholders by the affirmative vote of at least two-thirds of the outstanding voting stock which is not owned by the interested stockholder. |
• | any merger or consolidation involving the corporation and the interested stockholder; |
• | any sale, transfer, lease, pledge or other disposition involving the interested stockholder of 10% or more of the assets of the corporation; |
• | subject to exceptions, any transaction that results in the issuance or transfer by the corporation of any stock of the corporation to the interested stockholder; |
• | subject to exceptions, any transaction involving the corporation that has the effect of increasing the proportionate share of the stock of any class or series of the corporation beneficially owned by the interested stockholder; and |
• | the receipt by the interested stockholder of the benefit of any loans, advances, guarantees, pledges or other financial benefits provided by or through the corporation. |
• | on or through the facilities of The Nasdaq Global Select Market or any other securities exchange or quotation or trading service on which such securities may be listed, quoted or traded at the time of sale; and/or |
• | to or through a market maker otherwise than on The Nasdaq Global Select Market or such other securities exchanges or quotation or trading services. |
• | the name of the agent or any underwriters; |
• | the public offering or purchase price; |
• | any discounts and commissions to be allowed or paid to the agent or underwriters; |
• | all other items constituting underwriting compensation; |
• | any discounts and commissions to be allowed or paid to dealers; and |
• | any exchanges on which the securities will be listed. |
• | the purchase by an institution of the securities covered under that contract shall not at the time of delivery be prohibited under the laws of the jurisdiction to which that institution is subject; and |
• | if the securities are also being sold to underwriters acting as principals for their own account, the underwriters shall have purchased such securities not sold for delayed delivery. The underwriters and other persons acting as our agents will not have any responsibility in respect of the validity or performance of delayed delivery contracts. |
• | our Annual Report on Form 10-K for the year ended December 31, 2020 filed with the SEC on March 1, 2021; |
• | our Quarterly Reports on Form 10-Q for the quarter ended March 31, 2021 filed with the SEC on May 10, 2021 and for the quarter ended June 30, 2021 filed with the SEC on August 9, 2021; |
• | our Current Reports on Form 8-K filed with the SEC on January 7, 2021, April 27, 2021, June 21, 2021 and August 30, 2021; |
• | the description of the Company’s common stock contained in its registration statement on Form 8-A, which was filed with the SEC on February 2, 2015, and amended on January 11, 2018, including any amendment or report filed for the purpose of updating such description. |