IP Library Patent Application 19025457
Patent Application
App. No. 19/025,457

Adeno-Associated Virus Factor VIII Vectors

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Patent No.
US None
App. No.
19/025,457
Abstract

The invention provides completely packaged adeno-associated virus (AAV) Factor VIII (FVIII) vectors. This invention also provides completely packaged AAV FVIII vectors with high expression activity.

Claims (55)

1 . (canceled)

2 . An adeno-associated virus (AAV) Factor VIII (FVIII) comprising a nucleotide sequence selected from the group consisting of:

i. the Proto 1 sequence set forth in SEQ ID NO: 1,

ii. the Proto 1S sequence set forth in SEQ ID NO: 2,

iii. the Proto 2S sequence set forth in SEQ ID NO: 3,

iv. the Proto 3S sequence set forth in SEQ ID NO: 4,

v. the Proto 4 sequence set forth in SEQ ID NO: 5,

vi. the Proto 5 sequence set forth in SEQ ID NO: 6,

vii. the Proto 6 sequence set forth in SEQ ID NO: 7,

viii. the Proto 7 sequence set forth in SEQ ID NO: 8,

ix. the Construct 100ATG sequence set forth in SEQ ID NO: 9,

x. the Construct 100ATG bGH poly A sequence set forth in SEQ ID NO: 10,

xi. the Construct 100ATG short bGH polyA sequence set forth in SEQ ID NO: 11,

xii. the Construct 103ATG sequence set forth in SEQ ID NO: 12,

xiii. the Construct 103ATG short bGH poly A sequence set forth in SEQ ID NO: 13,

xiv. the Construct 105ATG bGH poly A sequence set forth in SEQ ID NO: 14,

xv. the Construct DC172ATG FVIII sequence set forth in SEQ ID NO: 15,

xvi. the Construct DC172 ATG FVIII hAAT sequence set forth in SEQ ID NO: 16,

xvii. the Construct DC172 2×HCR ATG FVIII sequence set forth in SEQ ID NO: 17,

xviii. the Construct DC172 2×HCR ATG FVIII hAAT sequence set forth in SEQ ID NO: 18,

xix. the Construct 2× SerpinA hAAT ATG FVIII sequence set forth in SEQ ID NO: 19,

xx. the Construct 2× SerpinA hAAT ATG FVIII 2× μ-globulin enhancer sequence set forth in SEQ ID NO: 20,

xxi. the Construct 100ATG short polyA 2× μ-globulin enhancer sequence set forth in SEQ ID NO: 21,

xxii. the Construct Factor VIII-BMN001 sequence set forth in SEQ ID NO: 22,

xxiii. the Construct Factor VIII-BMN002 sequence set forth in SEQ ID NO: 23,

xxiv. the Construct 99 sequence set forth in SEQ ID NO: 24,

xxv. the Construct 100 sequence set forth in SEQ ID NO: 25,

xxvi. the Construct 100 reverse orientation sequence set forth in SEQ ID NO: 26,

xxvii. the Construct 100AT sequence set forth in SEQ ID NO: 27,

xxviii. the Construct 100AT 2× MG sequence set forth in SEQ ID NO: 28,

xxix. the Construct 100AT 2× MG polyA sequence set forth in SEQ ID NO: 29,

xxx. the Construct 100AT 2× MG (reverse) bGH polyA sequence set forth in SEQ ID NO: 30,

xxxi. the Construct 100 bGH polyA sequence set forth in SEQ ID NO: 31,

xxxii. the Construct 100-400 sequence set forth in SEQ ID NO: 32,

xxxiii. the Construct 101 sequence set forth in SEQ ID NO: 33,

xxxiv. the Construct 102 sequence set forth in SEQ ID NO: 34,

xxxv. the Construct 103 sequence set forth in SEQ ID NO: 35,

xxxvi. the Construct 103 reverse orientation sequence set forth in SEQ ID NO: 36,

xxxvii. the Construct 103AT sequence set forth in SEQ ID NO: 37,

xxxviii. the Construct 103AT 2× MG sequence set forth in SEQ ID NO: 38,

xxxix. the Construct 103AT 2× MG polyA sequence set forth in SEQ ID NO: 39,

xl. the Construct 103 bGH polyA sequence set forth in SEQ ID NO: 40,

xli. the Construct 104 sequence set forth in SEQ ID NO: 41,

xlii. the Construct 105 sequence set forth in SEQ ID NO: 42,

xliii the Construct 106 sequence set forth in SEQ ID NO: 43,

xliv. the Construct 106AT sequence set forth in SEQ ID NO: 44, and

xlv. the Construct 2× Serpin A hAAT sequence set forth in SEQ ID NO: 45.

3 . A method of producing a recombinant adeno-associated virus (AAV) particle comprising

A) culturing a cell that has been transfected with a AAV vector of claim 2 ; and

B) recovering recombinant AAV particle from the supernatant of the transfected cell.

4 . A viral particle comprising the viral vector of claim 2 .

5 . A cell comprising the viral vector of claim 2 .

6 . A method of treating a patient suffering from hemophilia A comprising administering to the patient an effective amount of an AAV FVIII vector of claim 2 .

7 . (canceled)

8 . A composition comprising an AAV FVIII vector of claim 2 .

Assignments (1)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →