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Journal: Gene Editing for CEP290-Associated Retinal Degeneration. (Pubmed Central) - May 6, 2024
P1/2
The safety profile and improvements in photoreceptor function after EDIT-101 treatment in this small phase 1-2 study support further research of in vivo CRISPR-Cas9 gene editing to treat inherited retinal degenerations due to the IVS26 variant of CEP290 and other genetic causes. (Funded by Editas Medicine and others; BRILLIANCE ClinicalTrials.gov number, NCT03872479.).
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Journal: Prime Editing for the Installation and Correction of Mutations Causing Inherited Retinal Disease: A Brief Methodology. (Pubmed Central) - Dec 10, 2022
Prime editing is a novel double-strand break (DSB) independent CRISPR/Cas system which has the potential to correct all 12 possible transition and transversion mutations in addition to small deletions and insertions. Here, as a proof-of-concept study, we describe a methodology using prime editing for the in vitro installation and correction of the classical Pde6b c.1678C > T (p.Arg560Cys) mutation which causes autosomal recessive retinitis pigmentosa (RP) in mice.
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Enrollment closed, Trial completion date, Trial primary completion date: Single Ascending Dose Study in Participants With LCA10 (clinicaltrials.gov) - Dec 5, 2022
P1/2, N=34, Active, not recruiting,
Here, as a proof-of-concept study, we describe a methodology using prime editing for the in vitro installation and correction of the classical Pde6b c.1678C > T (p.Arg560Cys) mutation which causes autosomal recessive retinitis pigmentosa (RP) in mice. Recruiting --> Active, not recruiting | Trial completion date: Mar 2024 --> May 2025 | Trial primary completion date: Mar 2024 --> May 2025
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Using CRISPR to treat inherited retinal degenerations (1EF Mile High Blrm) - Apr 29, 2022 - Abstract #ARVO2022ARVO_1560;
Our data suggests that EDIT-101 has a favorable immunogenic profile and no adverse events have been attributable to immunogenic responses in subjects treated in the BRILLIANCE trial. Presentation Description:Results from the Phase 1/2 Brilliance using Edit101 to treat patients with CEP290-related retinopathy will be presented.
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Enrollment change: Single Ascending Dose Study in Participants With LCA10 (clinicaltrials.gov) - Apr 20, 2022
P1/2, N=34, Recruiting,
Our data suggests observed EDIT-101 shedding is transient and at low levels of detection, with no risk of systemic viral persistence. N=18 --> 34
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Journal: Genome editing strategies for treating human retinal degenerations. (Pubmed Central) - Feb 1, 2022
Progress in this rapidly evolving field has been highlighted by recent Food and Drug Administration clinical trial approval for EDIT-101 (Editas Medicine, Inc., Cambridge, MA), which has demonstrated efficacious genome editing in a mouse model of CEP290-associated Leber congenital amaurosis and safety in nonhuman primates...In particular, the heterogeneity of IRD-causing mutations in more than 200 known genes and long-term safety concerns relating to Cas9 expression in vivo. This review highlights (i) the technological advances in gene editing technology, (ii) major safety concerns associated retinal genome editing, and (iii) potential strategies for overcoming these challenges to develop clinical therapies.
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Review, Journal: Prime Editing for Inherited Retinal Diseases. (Pubmed Central) - Dec 14, 2021
However, there is currently only one FDA-approved gene augmentation therapy, Luxturna (voretigene neparvovec-rzyl), available to patients with RPE65-mediated retinitis pigmentosa (RP)...EDIT-101, a CRISPR-based therapy that is currently in clinical trials, uses double-strand breaks and nonhomologous end joining to remove the IVS26 mutation in the CEP290 gene...Instead, PE uses reverse transcriptase and Cas9 nickase to repair mutations in the genome. While this technique is still developing, with several challenges yet to be addressed, it offers promising implications for the future of IRD treatment.
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Genetic variations in a Natural History Study for Leber CEP290-Associated Retinal Degeneration (Exhibit Hall: Posterboard# A0066) - Mar 9, 2020 - Abstract #ARVO2020ARVO_2961;
P=N/A
The purpose is to determine the genetic variation and explore phenotypic differences among patients enrolled in the Allergan/Editas sponsored Natural History Study of CEP290 associated retinal degeneration (NCT03396042), which will inform a therapeutic study for AGN-151587, a CRISPR/Cas9 gene editing therapy designed to remove CEP290 c.2991+1655A>G mutation.Methods Genetic testing using a vision panel test modified for this study consisting of 254 genes associated with IRDs was performed by Molecular Vision Laboratory...Homozygous patients, along with patients in Category 4 and 5, had the worst visual acuity, while patients in Category 2 and 3 had better vision.Conclusions This study demonstrates the spectrum of mutations present with the c.2991+1655A>G mutation in CEP290. Further study of the phenotypes of the patients in the CEP290 Natural History Study will help define potential genotype–phenotype correlations as well as potential for therapeutic interventions.
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Enrollment open: Single Ascending Dose Study in Participants With LCA10 (clinicaltrials.gov) - Jul 23, 2019
P1/2, N=18, Recruiting,
Further study of the phenotypes of the patients in the CEP290 Natural History Study will help define potential genotype–phenotype correlations as well as potential for therapeutic interventions. Not yet recruiting --> Recruiting
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