Table 3 AAVs used for IRD clinical applications

AAV2

CHM

RPE1

CAG

subretinal

Photoreceptors and RPE

Phase 2

NCT02407678

AAV2

AMD

CFI complement factor

CMV

subretinal

Photoreceptors and RPE

Phase 1/2

NCT03846193

AAV2

RP

MCO

CMV

Intravitreal

Inner nuclear layer

(INL)

Phase 1/2a

NCT04919473

AAV2

RP

MCO-010

N/A

Intravitreal

Bipolar cells

Phase 2

NCT04945772

AAV2

RP

hMERTK

VMD2

subretinal

RPE

Phase 1

NCT01482195

AAV2

LCA

RPE65v2

CBA

subretinal

RPE

Phase 3

NCT00999609

AAV2

LCA

RPE65

CMV

subretinal

RPE

Phase 1

NCT00481546

AAV2

CHM

CHM

CAG

subretinal

RPE

Phase 2

NCT03507686

AAV2

RP

ChrimsonR-tdTomato

CAG

Intravitreal

RGCs

Phase 1/2a

NCT03326336

AAV2

LCA

RPE65

CMV

subretinal

RPE

Phase 1

NCT00821340

AAV2

CHM

RPE1

CAG

subretinal

Photoreceptors & RPE

Phase 2

NCT02077361

AAV2

CHM

RPE1

CAG

subretinal

Photoreceptors & RPE

Phase 2

NCT02553135

AAV2

LHON

ND4

CMV

Intravitreal

Macular RGCs

Phase 3

NCT03293524

AAV2

AMD

sFLT-1

CMV

subretinal

Anti-angiogenesis

Phase 1/2

NCT01494805

AAV2

CHM

REP1

CAG

subretinal

Photoreceptors & RPE

Phase 2

NCT02671539

AAV2

CHM

REP1

CAG

subretinal

Photoreceptors & RPE

Phase 1

NCT01461213

AAV2

RP

rhodopsin

GRM6

Intravitreal

RGCs

Phase 1/2

NCT02556736

AAV2

CHM

CHM

CAG

subretinal

Photoreceptors & RPE

Phase 1/2

NCT02341807

AAV2

LHON

ND4

CMV

Intravitreal

RGC

—

NCT03672968

AAV2

AMD

VEGF-neutralizing proteins FLT01

CAG

Intravitreal

Anti-angiogenesis

Phase 1

NCT01024998

AAV2

CHM

REP1

CAG

subretinal

Photoreceptors & RPE

Phase 3

NCT03496012

AAV2

LCA

RPE65

CB^o

subretinal

RPE

Phase 1/2

NCT00749957

AAV4

LCA

RPE65

hRPE65

subretinal

RPE

Phase 1/2

NCT01496040

AAV5

LCA

RPE65

NA65p

subretinal

RPE

Phase 1/2

NCT02781480

AAV5

LCA

CEP290

hGRK1

subretinal

Photoreceptors

Phase 2

NCT03872479

AAV5

RP

RPGR

RK

subretinal

Photoreceptors

Phase 3

NCT04671433

AAV5

RP

RPGR

RK

subretinal

Photoreceptors

Phase 3

NCT04794101

AAV5

RP

RPGR

RK

subretinal

Photoreceptors

Phase 3

NCT03252847

AAV5

RP

PDE6B

RK

subretinal

Photoreceptors

Phase 1/2

NCT03328130

AAV5

RP

RPGR

GRK

subretinal

Photoreceptors

Phase 1/2

NCT03252847

AAV8

RS

RS1

hRS

Intravitreal

—

Early Phase 1

NCT06289452

AAV8

STGD

ABCA4

—

subretinal

—

Phase 1/2

NCT06467344

AAV8

RP

PDE6A

RHO

subretinal

Photoreceptors

Phase 1/2a

NCT04611503

AAV8

LCA

hopt- LCA5

CAG

subretinal

Photoreceptors

Phase 1/2

NCT05616793

AAV8

BCD

CYP4v2

CAG

subretinal

RPE

Early Phase 1

NCT05399069

AAV8

RP

RPGR

RK

subretinal

Photoreceptors

Phase 1/2

NCT03116113

AAV8

RP

RLBP1

CPK850

subretinal

Photoreceptors

Phase 1

NCT03374657

AAV8

BCD

CYP4V2

CAG

subretinal

RPE

Phase 1

NCT04722107

AAV2tYF

RP

hRPGRco

GRK1

subretinal

Photoreceptors

Phase 1/2

NCT06275620

AAV2tYF

RP

RPGR

GRK1

subretinal

Photoreceptors

Phase 1/2

NCT06333249

AAV2tYF

RP

RPGR

GRK1

subretinal

Photoreceptors

Phase 1/2

NCT03316560

AAV2tYF

RS

RS1

CAG

Intravitreal

Bipolarcells and PR

Phase 1/2

NCT02416622

AAV2tYF

ACH

CNGA3

PR1.7

subretinal

ConePR

Phase 1/2

NCT02935517

AAV2tYF

ACH

CNGB3

PR1.7

subretinal

ConePR

Phase 1/2

NCT02599922

AAV2-7m8

AMD

Aflibercept

CMV

Intravitreal

Anti-angiogenesis

—

NCT04645212

AAV2-7m8

AMD

Aflibercept

CMV

Intravitreal

Anti-angiogenesis

Phase 1

NCT03748784

AAV2-7m8

AMD

Aflibercept

CMV

Intravitreal

Anti-angiogenesis

Phase 2

NCT04418427

AAV2-7m8

RP

ChrimsonR-tdTomato

CAG

Intravitreal

RGCs

Phase 1/2a

NCT03326336

4D-R100

RP

RPGR

—

Intravitreal

Photoreceptors

Phase 1/2

NCT04517149

4D-R100

CHM

CHM

—

Intravitreal

Photoreceptors & RPE

Phase 1

NCT04483440

OCU400

—

—

—

—

—

—

NCT06574997

FT-002

—

—

—

—

—

—

NCT05874310

AAV2.NN

RP

CNGA1

—

Intravitreal

Photoreceptors

Phase 1

NCT06291935