US 2023/0407333 A1Published application

Aav capsids and compositions containing same

AssigneeThe Trustees of the University of Pennsylvania

InventorsJames M. Wilson, Kalyani Nambiar, Qiang Wang

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Abstract

Novel AAV capsids and recombinant AAV vectors comprising the same are provided.

Claims11

Dependent claims

A recombinant adeno-associated virus (rAAV) comprising a capsid and a vector genome comprising an AAV 5′ inverted terminal repeat (ITR), an expression cassette comprising a nucleic acid sequence encoding a gene product operably linked to expression control sequences, and an AAV 3′ ITR, wherein the capsid is:

(a) an AAVrh75 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
40 or a sequence at least 99% identical thereto having an Asn (N) amino acid residue at position 24 based on the numbering of SEQ ID NO:
40;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
39 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
40;
or (iii) a capsid which is heterogeneous mixture of AAVrh75 vp1, vp2 and vp3 proteins which are 95% to 100% deamidated in at least position N57, N262, N384, and/or N512 of SEQ ID NO:
40, and optionally deamidated in other positions;
(b) an AAVhu71/74 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
4;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
3 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
4;
or (iii) a capsid which is a heterogeneous mixture of AAVrh71/74 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least 4 positions of SEQ ID NO:
4, and optionally deamidated in other positions;
(c) an AAVhu79 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
6;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
5 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
6;
or (iii) a capsid which is a heterogeneous mixture of AAVhu79 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
6, and optionally deamidated in other positions;
(d) an AAVhu80 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
8;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
7 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
8;
or (iii) a capsid which is a heterogeneous mixture of AAVhu80 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
8, and optionally deamidated in other positions;
(e) an AAVhu83 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
10;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
9 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
10;
or (iii) a capsid which is a heterogeneous mixture of AAVhu83 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
10, and optionally deamidated in other positions;
(f) an AAVhu74/71 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
12;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
11 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
12;
or (iii) a capsid which is a heterogeneous mixture of AAVhu74/71 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
12, and optionally deamidated in other positions;
(g) an AAVhu77 capsid, consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
14;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
13 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
14;
or (iii) a capsid which is a heterogeneous mixture of AAVhu77 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
14, and optionally deamidated in other positions;
(h) an AAVhu78/88 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
16;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
15 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
16;
or (iii) a capsid which is a heterogeneous mixture of AAVhu78/88 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
16, and optionally deamidated in other positions;
(i) an AAVhu70 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
18;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
17 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
18;
or (iii) a capsid which is a heterogeneous mixture of AAVhu70 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
18, and optionally deamidated in other positions;
(j) an AAVhu72 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
20;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
19 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
20;
or (iii) a capsid which is a heterogeneous mixture of AAVhu72 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
20, and optionally deamidated in other positions;
(k) an AAVhu75 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
22;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
21 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
22;
or (iii) a capsid which is a heterogeneous mixture of AAVhu75 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
22, and optionally deamidated in other positions;
(l) an AAVhu76 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
24;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
23 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
24;
or (iii) a capsid which is a heterogeneous mixture of AAVhu76 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
24, and optionally deamidated in other positions;
(m) an AAVhu81 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
26;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
25 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
26;
or (iii) a capsid which is a heterogeneous mixture of AAVhu81 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
26, and optionally deamidated in other positions;
(n) an AAVhu82 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
28;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
27 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
28;
or (iii) a capsid which is a heterogeneous mixture of AAVhu82 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
28, and optionally deamidated in other positions;
(o) an AAVhu84 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
30;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
29 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
30;
or (iii) a capsid which is a heterogeneous mixture of AAVhu84 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
30, and optionally deamidated in other positions;
(p) an AAVhu86 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
32;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
31 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
32;
or (iii) a capsid which is a heterogeneous mixture of AAVhu86 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
32, and optionally deamidated in other positions;
(q) an AAVhu87 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
34;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
33 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
34;
or (iii) a capsid which is a heterogeneous mixture of AAVhu87 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
34, and optionally deamidated in other positions;
(r) an AAVhu88/78 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
36;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
35 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
36;
or (iii) a capsid which is a heterogeneous mixture of AAVhu88/78 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
36, and optionally deamidated in other positions;
(s) an AAVhu69 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
38;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
37 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
38;
or (iii) a capsid which is a heterogeneous mixture of AAVhu69 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
38, and optionally deamidated in other positions;
(t) an AAVrh76 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
42;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
41 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
42;
or (iii) a capsid which is a heterogeneous mixture of AAVhu69 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
42, and optionally deamidated in other positions;
(u) an AAVrh77 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
44;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
43 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
44;
or (iii) a capsid which is a heterogeneous mixture of AAVrh71 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
44, and optionally deamidated in other positions;
(v) an AAVrh78 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
46;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
45 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
46;
or (iii) a capsid which is a heterogeneous mixture of AAVrh78 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
46, and optionally deamidated in other positions;
(w) an AAVrh81 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
50;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
49 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
50;
or (iii) a capsid which is a heterogeneous mixture of AAVrh81 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
50, and optionally deamidated in other positions;
(x) an AAVrh89 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
52;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
51 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
52;
or (iii) a capsid which is a heterogeneous mixture of AAVrh89 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
52, and optionally deamidated in other positions;
(y) an AAVrh82 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
54;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
53 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
54;
or (iii) a capsid which is a heterogeneous mixture of AAVrh82 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
54, and optionally deamidated in other positions;
(z) an AAVrh83 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
56;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
55 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
56;
or (iii) a capsid which is a heterogeneous mixture of AAVrh83 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
56, and optionally deamidated in other positions;
(aa) an AAVrh84 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
58;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
57 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
58;
or (iii) a capsid which is a heterogeneous mixture of AAVrh84 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
58, and optionally deamidated in other positions;
(bb) an AAVrh85 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
60;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
59 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
60;
or (iii) a capsid which is a heterogeneous mixture of AAVrh85 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
60, and optionally deamidated in other positions;
(cc) an AAVrh87 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
62;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
61 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
62;
or (iii) a capsid which is a heterogeneous mixture of AAVrh87 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
62, and optionally deamidated in other positions;
or
(dd) an AAVhu73 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
74;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
73 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
74;
or (iii) a capsid which is a heterogeneous mixture of AAVrh73 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
74, and optionally deamidated in other positions.

209 more elements

Dependencies are inferred from the claim text.

Facts

Application: 18/249,842Filed 29 Oct 2021
Priority: 29 Oct 2020
Published: 21 Dec 2023
Term ends: Not estimatedThe source records don’t support an estimate.

Classification10 codes

C12N 15/86
For animal cells: viral vectors
C07K 14/005
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof: from viruses
C12N 7/00
Viruses
C12N 2750/14121
Dependovirus, e.g. adenoassociated viruses: viruses as such, e.g. new isolates, mutants or their genomic sequences

Family

Application63/107,030Us provisional application · filed 29 Oct 2020
Application63/214,530Us provisional application · filed 24 Jun 2021
US 2023/0407333 A1This patent

From the US filing history. Foreign family members aren’t listed.

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(a) an AAVrh75 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
40 or a sequence at least 99% identical thereto having an Asn (N) amino acid residue at position 24 based on the numbering of SEQ ID NO:
40;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
39 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
40;
or (iii) a capsid which is heterogeneous mixture of AAVrh75 vp1, vp2 and vp3 proteins which are 95% to 100% deamidated in at least position N57, N262, N384, and/or N512 of SEQ ID NO:
40, and optionally deamidated in other positions;
(b) an AAVhu71/74 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
4;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
3 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
4;
or (iii) a capsid which is a heterogeneous mixture of AAVrh71/74 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least 4 positions of SEQ ID NO:
4, and optionally deamidated in other positions;
(c) an AAVhu79 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
6;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
5 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
6;
or (iii) a capsid which is a heterogeneous mixture of AAVhu79 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
6, and optionally deamidated in other positions;
(d) an AAVhu80 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
8;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
7 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
8;
or (iii) a capsid which is a heterogeneous mixture of AAVhu80 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
8, and optionally deamidated in other positions;
(e) an AAVhu83 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
10;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
9 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
10;
or (iii) a capsid which is a heterogeneous mixture of AAVhu83 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
10, and optionally deamidated in other positions;
(f) an AAVhu74/71 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
12;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
11 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
12;
or (iii) a capsid which is a heterogeneous mixture of AAVhu74/71 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
12, and optionally deamidated in other positions;
(g) an AAVhu77 capsid, consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
14;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
13 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
14;
or (iii) a capsid which is a heterogeneous mixture of AAVhu77 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
14, and optionally deamidated in other positions;
(h) an AAVhu78/88 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
16;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
15 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
16;
or (iii) a capsid which is a heterogeneous mixture of AAVhu78/88 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
16, and optionally deamidated in other positions;
(i) an AAVhu70 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
18;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
17 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
18;
or (iii) a capsid which is a heterogeneous mixture of AAVhu70 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
18, and optionally deamidated in other positions;
(j) an AAVhu72 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
20;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
19 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
20;
or (iii) a capsid which is a heterogeneous mixture of AAVhu72 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
20, and optionally deamidated in other positions;
(k) an AAVhu75 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
22;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
21 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
22;
or (iii) a capsid which is a heterogeneous mixture of AAVhu75 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
22, and optionally deamidated in other positions;
(l) an AAVhu76 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
24;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
23 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
24;
or (iii) a capsid which is a heterogeneous mixture of AAVhu76 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
24, and optionally deamidated in other positions;
(m) an AAVhu81 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
26;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
25 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
26;
or (iii) a capsid which is a heterogeneous mixture of AAVhu81 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
26, and optionally deamidated in other positions;
(n) an AAVhu82 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
28;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
27 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
28;
or (iii) a capsid which is a heterogeneous mixture of AAVhu82 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
28, and optionally deamidated in other positions;
(o) an AAVhu84 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
30;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
29 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
30;
or (iii) a capsid which is a heterogeneous mixture of AAVhu84 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
30, and optionally deamidated in other positions;
(p) an AAVhu86 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
32;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
31 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
32;
or (iii) a capsid which is a heterogeneous mixture of AAVhu86 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
32, and optionally deamidated in other positions;
(q) an AAVhu87 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
34;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
33 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
34;
or (iii) a capsid which is a heterogeneous mixture of AAVhu87 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
34, and optionally deamidated in other positions;
(r) an AAVhu88/78 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
36;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
35 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
36;
or (iii) a capsid which is a heterogeneous mixture of AAVhu88/78 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
36, and optionally deamidated in other positions;
(s) an AAVhu69 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
38;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
37 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
38;
or (iii) a capsid which is a heterogeneous mixture of AAVhu69 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
38, and optionally deamidated in other positions;
(t) an AAVrh76 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
42;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
41 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
42;
or (iii) a capsid which is a heterogeneous mixture of AAVhu69 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
42, and optionally deamidated in other positions;
(u) an AAVrh77 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
44;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
43 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
44;
or (iii) a capsid which is a heterogeneous mixture of AAVrh71 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
44, and optionally deamidated in other positions;
(v) an AAVrh78 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
46;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
45 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
46;
or (iii) a capsid which is a heterogeneous mixture of AAVrh78 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
46, and optionally deamidated in other positions;
(w) an AAVrh81 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
50;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
49 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
50;
or (iii) a capsid which is a heterogeneous mixture of AAVrh81 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
50, and optionally deamidated in other positions;
(x) an AAVrh89 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
52;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
51 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
52;
or (iii) a capsid which is a heterogeneous mixture of AAVrh89 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
52, and optionally deamidated in other positions;
(y) an AAVrh82 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
54;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
53 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
54;
or (iii) a capsid which is a heterogeneous mixture of AAVrh82 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
54, and optionally deamidated in other positions;
(z) an AAVrh83 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
56;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
55 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
56;
or (iii) a capsid which is a heterogeneous mixture of AAVrh83 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
56, and optionally deamidated in other positions;
(aa) an AAVrh84 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
58;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
57 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
58;
or (iii) a capsid which is a heterogeneous mixture of AAVrh84 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
58, and optionally deamidated in other positions;
(bb) an AAVrh85 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
60;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
59 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
60;
or (iii) a capsid which is a heterogeneous mixture of AAVrh85 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
60, and optionally deamidated in other positions;
(cc) an AAVrh87 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
62;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
61 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
62;
or (iii) a capsid which is a heterogeneous mixture of AAVrh87 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
62, and optionally deamidated in other positions;
or
(dd) an AAVhu73 capsid consisting of (i) a capsid produced from a nucleic acid sequence encoding SEQ ID NO:
74;
(ii) a capsid produced from a nucleic acid sequence of SEQ ID NO:
73 of a sequence or a sequence at least 95% identical thereto encoding SEQ ID NO:
74;
or (iii) a capsid which is a heterogeneous mixture of AAVrh73 vp1, vp2, and vp3 proteins which are 95% to 100% deamidated in at least four positions of SEQ ID NO:
74, and optionally deamidated in other positions.

209 more elements

Facts

Application

18/249,842Filed 29 Oct 2021

Priority

29 Oct 2020

Published

21 Dec 2023

Term ends

Not estimatedThe source records don’t support an estimate.

Classification10 codes

C12N 15/86
For animal cells: viral vectors
C07K 14/005
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof: from viruses
C12N 7/00
Viruses
C12N 2750/14121
Dependovirus, e.g. adenoassociated viruses: viruses as such, e.g. new isolates, mutants or their genomic sequences