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Preliminary results in 18 patients undergoing retinal gene therapy for X-linked retinitis pigmentosa with codon-optimized AAV8-RPGR

Preliminary results in 18 patients undergoing retinal gene therapy for X-linked retinitis pigmentosa with codon-optimized AAV8-RPGR
Preliminary results in 18 patients undergoing retinal gene therapy for X-linked retinitis pigmentosa with codon-optimized AAV8-RPGR

Purpose: To investigate the safety and efficacy of adeno-associated viral vector (AAV8) encoding retinitis pigmentosa GTPase regulator (RPGR) for X-linked retinitis pigmentosa (XLRP).

Methods: This study (ClinicalTrials. gov ID NCT03116113) is being conducted in 2 Parts: Part I is a Phase 1 dose-escalation study to identify the maximum tolerated dose (MTD); Part II is a Phase 2/3, assessor-masked, dose-expansion study. In Part I, subjects with genetically confirmed RPGR-associated XLRP were administered a sub-retinal dose of AAV8-RPGR. The vector used a rhodopsin kinase promoter to drive expression of a codon-optimized sequence that generated the correct full length RPGR protein. A 3+ 3 escalation scheme was used, with 3 subjects/dose (5× 10 9, 1× 10 10, 5× 10 10, 1× 10 11, 2.5× 10 11, and 5× 10 11 gp). Part I endpoints were safety (adverse events [AEs], dose-limiting toxicities [DLTs], ophthalmic …
0146-0404
1329
MacLaren, Robert E.
c7a2f458-a7b1-4b96-b88e-d376c90ec059
Davis, Janet L.
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Gregori, Ninel
94b2d498-8417-4656-bfbe-121ac9ecf635
Cehajic-Kapetanovic;, Jasmina
58f9cc83-5a9c-419b-9532-709367cc319e
Lam, Byron L.
3ee2e9ee-4ec6-4bab-ba4b-255c5284197b
Lotery, Andrew
5ecc2d2d-d0b4-468f-ad2c-df7156f8e514
Martinez-Fernandez dela Camara, Cristina
21a152c0-5f72-4d74-8260-59718a0fe789
Nanda, Anika
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Salvetti, Anna
f82e95da-8cdc-41b4-9982-a816d2a11389
Stanga, Paulo
b666b207-ad55-4c71-9bd1-f1fdad667c6f
Xue, Kanmin
161b105d-40c3-41b7-8db9-f5af21a52335
MacLaren, Robert E.
c7a2f458-a7b1-4b96-b88e-d376c90ec059
Davis, Janet L.
1cdb4a09-5a03-4d18-a907-01c29bb9417c
Gregori, Ninel
94b2d498-8417-4656-bfbe-121ac9ecf635
Cehajic-Kapetanovic;, Jasmina
58f9cc83-5a9c-419b-9532-709367cc319e
Lam, Byron L.
3ee2e9ee-4ec6-4bab-ba4b-255c5284197b
Lotery, Andrew
5ecc2d2d-d0b4-468f-ad2c-df7156f8e514
Martinez-Fernandez dela Camara, Cristina
21a152c0-5f72-4d74-8260-59718a0fe789
Nanda, Anika
5366dbf2-d4b0-4b9f-9026-7ed20601a396
Salvetti, Anna
f82e95da-8cdc-41b4-9982-a816d2a11389
Stanga, Paulo
b666b207-ad55-4c71-9bd1-f1fdad667c6f
Xue, Kanmin
161b105d-40c3-41b7-8db9-f5af21a52335

MacLaren, Robert E., Davis, Janet L., Gregori, Ninel, Cehajic-Kapetanovic;, Jasmina, Lam, Byron L., Lotery, Andrew, Martinez-Fernandez dela Camara, Cristina, Nanda, Anika, Salvetti, Anna, Stanga, Paulo and Xue, Kanmin (2019) Preliminary results in 18 patients undergoing retinal gene therapy for X-linked retinitis pigmentosa with codon-optimized AAV8-RPGR. Investigative Ophthalmology & Visual Science, 60 (9), 1329.

Record type: Meeting abstract

Abstract


Purpose: To investigate the safety and efficacy of adeno-associated viral vector (AAV8) encoding retinitis pigmentosa GTPase regulator (RPGR) for X-linked retinitis pigmentosa (XLRP).

Methods: This study (ClinicalTrials. gov ID NCT03116113) is being conducted in 2 Parts: Part I is a Phase 1 dose-escalation study to identify the maximum tolerated dose (MTD); Part II is a Phase 2/3, assessor-masked, dose-expansion study. In Part I, subjects with genetically confirmed RPGR-associated XLRP were administered a sub-retinal dose of AAV8-RPGR. The vector used a rhodopsin kinase promoter to drive expression of a codon-optimized sequence that generated the correct full length RPGR protein. A 3+ 3 escalation scheme was used, with 3 subjects/dose (5× 10 9, 1× 10 10, 5× 10 10, 1× 10 11, 2.5× 10 11, and 5× 10 11 gp). Part I endpoints were safety (adverse events [AEs], dose-limiting toxicities [DLTs], ophthalmic …

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More information

Published date: 22 July 2019

Identifiers

Local EPrints ID: 433973
URI: http://eprints.soton.ac.uk/id/eprint/433973
ISSN: 0146-0404
PURE UUID: 593d3f9c-ad48-477c-babd-da9bd889c17e
ORCID for Andrew Lotery: ORCID iD orcid.org/0000-0001-5541-4305

Catalogue record

Date deposited: 09 Sep 2019 16:30
Last modified: 10 Nov 2021 03:08

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Contributors

Author: Robert E. MacLaren
Author: Janet L. Davis
Author: Ninel Gregori
Author: Jasmina Cehajic-Kapetanovic;
Author: Byron L. Lam
Author: Andrew Lotery ORCID iD
Author: Cristina Martinez-Fernandez dela Camara
Author: Anika Nanda
Author: Anna Salvetti
Author: Paulo Stanga
Author: Kanmin Xue

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