Delving into the Latest Updates on Cosdosiran with Synapse

Overview

Basic Info

Drug Type

siRNA

Synonyms

+ [4]

Target

caspase 2

Action

inhibitors

Mechanism

caspase 2 inhibitors(Caspase-2 inhibitors), Gene expression inhibitors, RNA interference

Therapeutic Areas

Nervous System DiseasesCardiovascular DiseasesEye Diseases+ [1]

Active Indication

Optic Neuropathy, IschemicOptic Nerve InjuriesGlaucoma, Angle-Closure+ [1]

Inactive Indication

Acute angle-closure glaucomaOptic Atrophy

Originator Organization

BioSpring

Active Organization

Suzhou Ribo Life Science Co., Ltd.

Biocon Ltd.

Quark Pharmaceuticals, Inc.

Inactive Organization

PYRAMID Laboratories, Inc.

ClinChoice Medical (TIANJIN) Co., Ltd.

License Organization

Suzhou Ribo Life Science Co., Ltd.

Quark Pharmaceuticals, Inc.

Biocon Ltd.

Drug Highest PhasePhase 3

First Approval Date-

RegulationSpecial Review Project (China)

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Structure/Sequence

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Sequence Code 29512838

Source: *****

Sequence Code 29516420

Source: *****

主要目的： 1. 评定近期发作型 NAION 受试者中 QPI-1007 玻璃体内 (IVT) 注射的安全性和耐受性。 2. 确定近期发作型 NAION 受试者中 QPI-1007 玻璃体内 (IVT) 注射对视觉功能的影响。次要目的：评价近期发作型 NAION 受试者中施用 QPI-1007 后视乳头周围视网膜神经纤维层 (pRNFL) 和神经节细胞层的结构性变化。

[Translation]

Primary Objectives: 1. To assess the safety and tolerability of intravitreal (IVT) injections of QPI-1007 in subjects with recent-onset NAION. 2. To determine the effects of intravitreal (IVT) injections of QPI-1007 on visual function in subjects with recent-onset NAION. Secondary Objectives: To evaluate structural changes in the peripapillary retinal nerve fiber layer (pRNFL) and ganglion cell layer after administration of QPI-1007 in subjects with recent-onset NAION.

Start Date09 Nov 2017

Sponsor / Collaborator

Quark Pharmaceuticals, Inc.

[+2]

EUCTR2015-003079-31-DE

/ Not yet recruitingPhase 2/3

A PHASE 2/3, RANDOMIZED, DOUBLE-MASKED, SHAMCONTROLLED TRIAL OF QPI-1007 DELIVERED BY SINGLE OR MULTI-DOSE INTRAVITREAL INJECTION(S) TO SUBJECTS WITH ACUTE NONARTERITIC ANTERIOR ISCHEMIC OPTIC NEUROPATHY (NAION)

Start Date20 Oct 2016

Sponsor / Collaborator

Quark Pharmaceuticals, Inc.

NCT02341560

/ TerminatedPhase 2/3

A Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007 Delivered By Single or Multi-Dose Intravitreal Injection(s) to Subjects With Acute Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)

This study will determine the effect of QPI-1007 on visual function in subjects with recent-onset NAION and assess the safety and tolerability of intravitreal injections of QPI-1007 in this population. This study will also evaluate the structural changes in the retina following administration of QPI-1007.

Start Date24 Feb 2016

Sponsor / Collaborator

Quark Pharmaceuticals, Inc.

100 Clinical Results associated with Cosdosiran

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100 Translational Medicine associated with Cosdosiran

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100 Patents (Medical) associated with Cosdosiran

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9

Literatures (Medical) associated with Cosdosiran

01 Jul 2021·Biochemical PharmacologyQ2 · MEDICINE

The growth of siRNA-based therapeutics: Updated clinical studies

Q2 · MEDICINE

Review

Author: Bahal, Raman ; Zhong, Xiao-Bo ; Zhang, M May ; Manautou, José E ; Rasmussen, Theodore P

More than two decades after the natural gene-silencing mechanism of RNA interference was elucidated, small interfering RNA (siRNA)-based therapeutics have finally broken into the pharmaceutical market. With three agents already approved and many others in advanced stages of the drug development pipeline, siRNA drugs are on their way to becoming a standard modality of pharmacotherapy. The majority of late-stage candidates are indicated for rare or orphan diseases, whose patients have an urgent need for novel and effective therapies. Additionally, there are agents that have the potential to meet the need of a broader population. Inclisiran, for instance, is being developed for hypercholesterolemia and has shown benefit in patients who are uncontrolled even after maximal statin therapy. This review provides a brief overview of mechanisms of siRNA action, physiological barriers to its delivery and activity, and the most common chemical modifications and delivery platforms used to overcome these barriers. Furthermore, this review presents comprehensive profiles of the three approved siRNA drugs (patisiran, givosiran, and lumasiran) and the seven other siRNA candidates in Phase 3 clinical trials (vutrisiran, nedosiran, inclisiran, fitusiran, teprasiran, cosdosiran, and tivanisiran), summarizing their modifications and delivery strategies, disease-specific mechanisms of action, updated clinical trial status, and future outlooks.

01 Feb 2021·Fundamental & Clinical PharmacologyQ3 · MEDICINE

Progress on ocular siRNA gene‐silencing therapy and drug delivery systems

Q3 · MEDICINE

Review

Author: Jiang, Jinjin ; Li, Shuhan ; Tang, Yue ; Zhang, Xinru ; Chen, Jing

AbstractAge‐related macular degeneration (AMD) and glaucoma are global ocular diseases with high blindness rate. RNA interference (RNAi) is being increasingly used in the treatment of these disorders with siRNA drugs, bevasiranib, AGN211745 and PF‐04523655 for AMD, and SYL040012 and QPI‐1007 for glaucoma. Administration routes and vectors of gene drugs affect their therapeutic effect. Compared with the non‐viral vectors, viral vectors have limited payload capacity and potential immunogenicity. This review summarizes the progress of the ocular siRNA gene‐silencing therapy by focusing on siRNA drugs for AMD and glaucoma already used in clinical research, the main routes of drug delivery and the non‐viral vectors for siRNA drugs.

01 Feb 2020·British Journal of OphthalmologyQ2 · MEDICINE

Controversies on neuroprotection therapy in non-arteritic anterior ischaemic optic neuropathy

Q2 · MEDICINE

Review

Author: Hayreh, Sohan Singh

ObjectiveThere has long been a great interest in neuroprotection therapy for ischaemic stroke and various types of optic neuropathies. In view of that, I reviewed the literature on the role of neuroprotection for non-arteritic anterior ischaemic optic neuropathy (NA-AION).MethodsThe review is based on a PubMed search of literature about the use of neuroprotectors in stroke and optic neuropathies and about current clinical trials of RPh201 and QPI-1007 in NA-AION.ResultsSeveral neuroprotection agents for ischaemic stroke and various types of optic neuropathies have been evaluated extensively in experimental studies in animals and benefits claimed. However, translation of therapeutic strategies for neuroprotection from experimental research to humans has invariably been fraught with failure. Two currently ongoing studies dealing with neuroprotection by RPh201 and QPI-1007 in NA-AION may have limitations in their rationale and study designs.ConclusionsUnfortunately, in spite of all the experimental and clinical research on neuroprotection agents in NA-AION so far, we have no scientifically proven evidence of neuroprotection agents showing any benefit in the human clinical studies so far.

100 Deals associated with Cosdosiran

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Optic Nerve Injuries	Phase 3	-	Quark Pharmaceuticals, Inc.	-
Optic Nerve Injuries	Phase 3	-	Biocon Ltd.	-
Optic Neuropathy, Ischemic	Phase 1	India	PYRAMID Laboratories, Inc.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	Singapore	Quark Pharmaceuticals, Inc.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	Australia	Quark Pharmaceuticals, Inc.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	United States	PYRAMID Laboratories, Inc.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	Singapore	PYRAMID Laboratories, Inc.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	Germany	ClinChoice Medical (TIANJIN) Co., Ltd.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	Singapore	ClinChoice Medical (TIANJIN) Co., Ltd.	24 Feb 2016
Optic Neuropathy, Ischemic	Phase 1	United States	ClinChoice Medical (TIANJIN) Co., Ltd.	24 Feb 2016