Sepofarsen for LCA
Sepofarsen (QR-110) for CEP290 LCA
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Download link FFB IS (2022) B Leroy, Efficacy and safety of sepofarsen: A randomized, double-masked, sham-controlled, Ph2/3 trial pdf, 473.59 KB -
Download link ARVO (2022) SR Russell et al., Long-term safety and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in LCA10 pdf, 391.97 KB -
Download link ARVO (2022) B Leroy et al., Efficacy and safety of sepofarsen: A randomized, double-masked, sham-controlled, Ph2/3 study pdf, 790.29 KB - External link Nature Medicine (2022) Russell SR, et al., Intravitreal antisense oligonucleoti…
- External link Ophthalmology Science (2022) Cideciyan AV, et al., Restoration of Cone Sensitiv…
- External link Nature Medicine (2021) Cideciyan AV, et al., Durable vision improvement after a…
- External link Retina (2021) Leroy BP, et al., Leber Congenital Amaurosis Due to CEP290 Mutati…
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Download link Plain-language summary of Retina (2021) Leroy BP, et al. paper pdf, 1.28 MB - Internal link ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second …
- Internal link AAO (2020) Russell SR, Drack AV, et al., Results of a phase 1b/2 trial of intra…
- Internal link AAO (2020) Ho A, Cideciyan AV, et al., Full field stimulus testing (FST) to ass…
- Internal link ARVO (2020) Stephen R. Russell, et al., Results of a phase 1b/2 trial of intrav…
- External link Nature Medicine (2018) Jacobson SG, Cideciyan AV, et al., Effect of an intravit…
- External link Molecular Therapy: Nucleic Acid (2018) Dulla K, et al., Splice-modulating oligo…
- External link Invest Ophthalmol Vis Sci. (2017) Jacobson SG, Cideciyan AV, Sumaroka A, et al…
Ultevursen for RP and Usher
Ultevursen (QR-421a) for USH2A RP and Usher syndrome
- External link Molecular Therapy (2021) Dulla K, et al., Antisense oligonucleotide-based treat…
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Download link Euretina (2021) DG Birch. Phase 1b/2 interim results of QR-421a RNA therapy in retinitis pigmentosa due to mutations in USH2A pdf, 618.04 KB -
Download link ProQR (2021) presentation of QR-421a Stellar trial results pdf, 1.8 MB -
Download link ARVO (2019) Hester van Diepen, QR-421a antisense oligonucleotide for the treatment of RP due to USH2A exon 13 mutations pdf, 1.97 MB -
Download link ARVO (2017) Peter Adamson, QRX-421 Antisense oligonucleotide targeting Exon 13 mutations in the USH2A gene for treatment of non- pdf, 1.71 MB -
Download link Preclinical evidence. Hester van Diepen et al., QR-421a an antisense oligonucleotide for the treatment of RP due to USH2A pdf, 1.12 MB
QR-1123 for RP
QR-1123 for RHO RP
QR-504 for FECD
QR-504 for TCF4 FECD
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Download link ARVO (2018) Alice Davidson UCL, RNA toxicity induced by TCF4 CTG expansions is ameliorated by antisense therapeutics in a patien pdf, 1.51 MB - External link Am. J. Hum. Genet. (2018) Zarouchlioti et al., Antisense Therapy for a Common C…
QR-411 for RP and Usher
QR-411 for USH2A RP and Usher syndrome
QR-1011 for Stargardt
QR-1011 for Stargardt disease
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Download link ARVO (2022) M Kaltak etal., QR-1011 Corrects Splicing In The Stargardt Disease Type 1-causing Variant ABCA4 c.5461-10T>C pdf, 935.9 KB - External link OTS (2021) M Kaltak et al., QR-1011 corrects splicing in the Stargardt disease …
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Download link ARVO (2018) Kalyan Dulla et al., Oligonucleotide-Based Splice Correction of the ABCA4 c.5461-10T>C Mutation in Stargardt Disease pdf, 758.4 KB
QRX-704 for Huntington
QRX-704 for Huntington disease
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Download link EHDN (2018) Pontus Klein et al., QRX-704, a novel antisense oligonucleotide therapy designed to prevent HD pathology while pdf, 2.55 MB - External link Cell (2006) Graham et al., Cleavage at the Capspase-6 Site Is Required for Neur…
Axiomer technology
Axiomer RNA editing technology
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Download link TIDES USA (2022) Gerard Platenburg, Development of RNA Base Editing Technologies for Precision Medicines pdf, 1.46 MB -
Download link Axiomer Technology Overview pdf, 2.25 MB -
Download link OTS (2018) Julien Boudet et al., Structure-based computational approach for optimizing oligonucleotides for A-to-I editing pdf, 6.36 MB -
Download link TIDES (2018) Antti Aalto, Axiomer technology - Therapeutic oligonucleotides for directing site-specific A-to-I editing by endoge pdf, 2.44 MB
