Recent Advances in Ocular Nucleic Acid-Based Therapies: The Silent Era

Pañeda, Covadonga; Martínez, Tamara; Wright, Natalia; Jiménez, Ana I.

doi:10.5772/48454

Cited by 5 publications

(8 citation statements)

References 127 publications

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“…The ongoing phase II/III trial for this compound analyzes the potential of multiple IVT doses to improve visual acuity in patients suffering NAION [87,88]. Bamosiran is a canonical-designed naked siRNA-targeting β2-adrenergic receptor (ADRB2) under development for the treatment of increased IOP associated to glaucoma [64,[89][90][91]. Glaucoma is a degenerative, chronic disease of the optic nerve that can lead to blindness if left untreated [92].…”

Section: Programs In Development and Future Aheadmentioning

confidence: 99%

“…SYL1001 has shown to be safe when administered in eye drops to animals and humans and to have analgesic effect in the capsaicin-induced eye pain model. The compound is currently undergoing a phase I/II for the treatment of ocular pain associated to dry eye disease, a condition for which no specific treatment currently exists [89].…”

Section: Programs In Development and Future Aheadmentioning

confidence: 99%

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Preclinical Development of RNAi-Inducing Oligonucleotide Therapeutics for Eye Diseases

Martínez¹,

González²,

Vargas³

et al. 2016

RNA Interference

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RNA interference (RNAi) is a posttranscriptional mechanism of gene regulation present in eukaryotic cells. Inducers of RNAi are small molecules of RNA that act in the cytoplasm where they are able to impair translation of a specific mRNA to protein, hence modifying gene expression. The discovery of this mechanism in mammals led to the development of a new class of therapeutics with the aim of exploiting this endogenous mechanism of action. In the last decade, great efforts have been put into understanding RNAi and translating this accumulated knowledge into the design of modern therapeutics. With several compounds in phase III clinical development, the field is getting closer to its first market authorization. Here we make a thorough overview of the field of RNAi therapeutics in ophthalmology, one of the fields in which RNAi has been most successful.

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Section: Programs In Development and Future Aheadmentioning

confidence: 99%

Section: Programs In Development and Future Aheadmentioning

confidence: 99%

Preclinical Development of RNAi-Inducing Oligonucleotide Therapeutics for Eye Diseases

Martínez¹,

González²,

Vargas³

et al. 2016

RNA Interference

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“…Oligonucleotides can be designed to target specific genes or RNAs with the aim of altering gene expression or even exert a direct interaction by binding to molecules. The main classes of oligonucleotides that are currently being developed as therapeutic tools are aptamers, ASOs, siRNAs and microRNAs (miRNAs) [48].…”

Section: Oligonucleotide-based Compoundsmentioning

confidence: 99%

“…In addition, as other oligonucleotides, ASOs are chemically synthetized, with all the advantages this entails. On the other hand, ASOs are labile products that require chemical modifications to increase their stability; these modifications can potentially increase their toxicity [48,56].…”

Section: Antisense Oligonucleotides (Asos)mentioning

confidence: 99%

Managing Intraocular Pressure: Innovation in Glaucoma Management

Bleau¹,

Vargas²,

Jiménez³

et al. 2016

Glaucoma - Intraocular Pressure and Aqueous Dynamics

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Primary open-angle glaucoma is a progressive ocular neuropathy that if left untreated may lead to blindness. The main risk factor for developing glaucoma is increased intraocular pressure. Intraocular pressure is regulated by the balance of aqueous humour synthesis and secretion into the eye and outflow from the eye; therefore, most therapies for glaucoma seek lowering intraocular pressure to avoid disease progression. There are several types of drugs in the market for the treatment of glaucoma, but there are still unmet needs to be overcome; therefore, significant effort has been put in the last few years to develop new medicines with innovative mechanisms of action as well as devices to improve quality of life in glaucoma patients. The present review offers a thorough revision of the latest advances in the glaucoma therapy field, focusing on innovative approaches, new targets and new mechanisms of action.

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“…The eye offers several advantages as a target organ for RNAi therapeutics. It has a relatively low content in RNases, and it is quite isolated from the rest of the body, limiting the access of the compound administered into this organ to systemic circulation (Pañeda, 2012[ 103 ]). These advantages made the eye one of the first targets of RNAi therapeutics.…”

Section: Programs In Clinical Developmentmentioning

confidence: 99%