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Gene Therapy Milestones

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GENE THERAPY

Gene Therapy Milestones

2023
Nov 11

Gene Interference via ASO Qalsodyâ„¢/ tofersen

Nov 11

Aptamer Izervayâ„¢/avacincaptad pegol

Nov 11

Gene Interference via siRNA Rivflorzaâ„¢/ nedosiran

Nov 11

Gene Interference via ASO Wainuaâ„¢/eplontersen

Nov 11

ddPCR

2022
Jul 20

Gene Interference via siRNA Amvuttra®/vutrisiran

Feb 24

ddPCR

2021
Sep 04

Gene Interference via ASO Amondys 45â„¢/Casimersen

Sep 04

Gene Interference via siRNA Leqvio®/inclisiran

2020
Nov 04

Gene Interference via ASO Viltepso®/viltolarsen

Oct 04

Gene Interference via siRNA Oxlumoâ„¢/lumisiran

Sep 04

Ex vivo Gene Insertion via CAR-T Tecartusâ„¢/brexucabtagene autoleucel

2019
Nov 05

Gene Interference via siRNA Givlaari®/givosiran

Nov 04

Ex vivo Gene Insertion via LVV Zynteglo®/betibeglogene autotemcel

Nov 04

Gene Interference via ASO Waylivra®/volanesorsen

Nov 03

In vivo Gene Insertion via AAV9 Zolgensma®/onasemnogene abeparvovec-xioi

2018
Nov 05

Gene Interference via siRNA Onpattro®/patisiran

Nov 04

Gene Interference via ASO Tegsedi®/inotersen

2017
Nov 05

Ex vivo Gene Insertion via CAR-T Yescarta®/axicabtagene ciloleucel

Nov 04

Ex vivo Gene Insertion via CAR-T Kymriah®/tisagenlecleucel

Nov 03

In vivo Gene Insertion via AAV2 Luxturna®/voretigene neparvovec

2016
Nov 04

mRNA ADA

Nov 04

Vector ADA

Nov 04

Gene Interference via polydisperse natural oligonucleotidesDefitelio®/defibrotide

Nov 04

Oligo in vitro Drug Transport

Nov 04

Gene Interference via ASO Spinraza®/nusinersen

Nov 04

Gene Interference via ASO Exondys 51®/eteplirsen

Nov 04

Ex vivo Gene Insertion via GammaretrovirusStrimvelis®/autologous CD34+ enriched cell fraction

2015
Nov 04

Oligo in vitro Reaction Phenotyping

Nov 04

In vivo Gene Insertion via HSV-1 Imlygic®/talimogene laherparepvec

2014
Nov 04

Oligo PK via LC-HRMS

Nov 04

Oligo ADA

Nov 04

Oligo in vitro Protein Binding

Nov 04

mRNA PK/ Biodistribution/Shedding via QPCR

Nov 04

Vector PK/Biodistribution/Shedding via QPCR

2013
Nov 04

Oligo in vitro CYP Induction

Nov 04

Gene Interference via ASO Kynamro®/mipomersen

2012
Nov 04

In vivo Gene Insertion via AAV1 Glybera/alipogene tiparvovec

Nov 04

Oligo Biodistribution via Microautoradiography

2011
Nov 04

Oligo Biodistribution via QWBA

Nov 04

Oligo Mass Balance/Excretion

2010
Nov 04

Oligo Biodistribution via Autoradiography

Nov 04

Oligo in vitro Metabolic Stability

Nov 04

Oligo Metabolite Identification

Nov 04

Oligo in vitro CYP Inhibition

2009
Nov 04

Oligo PK via hybridization-LC-FLD

Nov 04

Oligo PK via LC-UV

2007
Nov 04

Oligo PK via hybridization-ELISA

2006
Nov 04

CLIA Assay – Inclusion/Exclusion Criteria

Nov 04

Biomarkers – Primary End-point

2004
Nov 04

Gene Interference via Aptamer Macugen®/pegaptanib

2003
Nov 04

Oligo PK via LC-MS/MS

1998
Nov 04

Gene Interference via ASO Vitravene/fomivirsen

Key
Bioanalysis
ADME
Gene Therapy Approval
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