Sepofarsen for LCA
Sepofarsen (QR-110) for CEP290 LCA
- Nature Medicine (2021) Cideciyan AV, et al., Durable vision improvement after a…
- Retina (2021) Leroy BP, et al., Leber Congenital Amaurosis Due to CEP290 Mutati…
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Plain-language summary of Retina (2021) Leroy BP, et al. paper pdf, 1.28 MB - ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second …
- AAO (2020) Russell SR, Drack AV, et al., Results of a phase 1b/2 trial of intra…
- AAO (2020) Ho A, Cideciyan AV, et al., Full field stimulus testing (FST) to ass…
- ARVO (2020) Stephen R. Russell, et al., Results of a phase 1b/2 trial of intrav…
- Nature Medicine (2018) Jacobson SG, Cideciyan AV, et al., Effect of an intravit…
- Molecular Therapy: Nucleic Acid (2018) Dulla K, et al., Splice-modulating oligo…
- Invest Ophthalmol Vis Sci. (2017) Jacobson SG, Cideciyan AV, Sumaroka A, et al…
QR-421a for RP and Usher
QR-421a for USH2A RP and Usher syndrome
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ProQR (2021) presentation of QR-421a Stellar trial results pdf, 1.8 MB -
ARVO (2019) Hester van Diepen, QR-421a antisense oligonucleotide for the treatment of RP due to USH2A exon 13 mutations pdf, 1.97 MB -
ARVO (2017) Peter Adamson, QRX-421 Antisense oligonucleotide targeting Exon 13 mutations in the USH2A gene for treatment of non- pdf, 1.71 MB -
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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ARVO (2018) Alice Davidson UCL, RNA toxicity induced by TCF4 CTG expansions is ameliorated by antisense therapeutics in a patien pdf, 1.51 MB - 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
QRX-704 for Huntington
QRX-704 for Huntington disease
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EHDN (2018) Pontus Klein et al., QRX-704, a novel antisense oligonucleotide therapy designed to prevent HD pathology while pdf, 2.55 MB - 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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OTS (2018) Julien Boudet et al., Structure-based computational approach for optimizing oligonucleotides for A-to-I editing pdf, 6.36 MB -
TIDES (2018) Antti Aalto, Axiomer technology - Therapeutic oligonucleotides for directing site-specific A-to-I editing by endoge pdf, 2.44 MB
R&D days