uniQure-JPM-202401.pdf

1、Leadership in Gene TherapyCorporate PresentationJanuary 2024Ashley,Huntingtons disease patient advocateLEADERSHIP IN GENE THERAPY|2JANUARY 2024DisclaimerDisclaimerThis presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933,as amended,and Sect

2、ion 21E of the Securities Exchange Act of 1934,as amended.All statements other than statements of historical fact are forward-looking statements,which are often indicated by terms such as“anticipate,”“believe,”“could,”“estimate,”“expect,”“goal,”“intend,”“look forward to,”“may,”“plan,”“potential,”“pr

3、edict,”“project,”“should,”will,”“would”and similar expressions and the negatives of those terms.Forward-looking statements are based on managements beliefs and assumptions and on information available to management only as of the date of this presentation.Examples of these forward-looking statements

4、 include,but are not limited to,statements concerning:the potential clinical and functional effects of AMT-130,including as an effective treatment option for patients with Huntingtons disease;the utility of CSF mHTT or NfL as an effective biomarkers of target engagement with respect to AMT-130;the d

5、esign and engineering of AMT-130 to maximize clinical and functional benefit;the potential that volumetric data yielded from the AMT-130 clinical trial will be clinically significant;our use of a natural history cohort as a basis for comparison with respect to the efficacy of AMT-130;the initiation

6、of the third cohort in our ongoing Phase I/II clinical trial of AMT-130 and the timing and release of additional clinical data;and our plans to request regulatory interactions with the relevant authorities in the U.S.and Europe and the potential for accelerated regulatory pathways.Because these stat