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Drug Discovery for Retinal Neurodegenerative Diseases

Table of Contents

Retinal and optic nerve diseases are serious conditions that cause visual impairment. In particular, glaucoma and retinitis pigmentosa (RP) are progressive and have limited effective treatments. Drug discovery for these diseases is critical to restoring visual function. Among past examples, technology for generating optic nerve cells using human induced pluripotent stem cells (iPSCs) has attracted attention. This technology has supported the development of drug candidates for retinal and optic nerve diseases.

Below, specific examples of treatments for retinal and optic nerve diseases, and the nonclinical studies for each are presented.

Development Case Study: National Center for Child Health and Development (NCCHD)

NCCHD succeeded in generating optic nerve cells—retinal ganglion cells (RGCs)—from human iPSCs. This achievement has advanced research on the causes and pathophysiology of optic nerve diseases that lead to severe visual impairment, as well as research on diagnosis and treatment. In addition, technology to assess drug effects has been developed, enabling the development of new therapeutic agents for optic nerve diseases.

Nonclinical Studies

NCCHD generates optic nerve cells from human iPSCs as well as from mouse iPS and embryonic stem (ES) cells. This work contributes to elucidating disease mechanisms that impair the optic nerve, researching diagnostic technologies, discovering treatments, clinical research such as optic nerve transplantation and regenerative medicine, optic nerve development, pathfinding mechanisms in nerve fiber growth, and the molecular mechanisms of visual formation—collectively supporting treatment advances for retinal and optic nerve diseases.

Reference: National Center for Child Health and Development official websitehttps://www.ncchd.go.jp/press/2017/20171130.html

Development Case Study: The Japanese Pharmacological Society

The Japanese Pharmacological Society conducts research on retinal degenerative diseases and visual information transmission to elucidate mechanisms, pathophysiology, and novel therapeutic strategies. For example, the roles of microRNAs in glaucoma and RP are being clarified, and neuroprotective agents targeting these genes are under development. In addition, clinical trials of human iPSC-derived photoreceptor cell sheet transplantation for the treatment of RP have begun in Japan.

Nonclinical Studies

The Japanese Pharmacological Society reports basic studies using a mouse model of RP to evaluate a novel therapeutic strategy involving transplantation of human iPSC-derived photoreceptor cell sheets. These studies have demonstrated the potential of this approach to address visual dysfunction.

Reference: The Japanese Pharmacological Society official website [PDF].https://www.jstage.jst.go.jp/article/fpj/155/2/155_20002/_pdf/-char/ja

Summary

Retinal and optic nerve diseases are an increasingly important focus in drug discovery due to the limited availability of effective treatments. As shown by the examples from NCCHD and the Japanese Pharmacological Society, innovative technologies and research are progressing and are expected to lead to new therapeutic agents for optic nerve diseases. Further research and development are anticipated as pharmaceutical companies and research institutes utilize drug-evaluation and testing services.

3 Recommended Contract Research Organizations for Non-Clinical Studies
— by Target goal and Expertise

In drug discovery, the quality and efficiency of non-clinical studies have a direct impact on clinical success rates, development costs, and overall length of time required in R&D. In recent years, there has been more demand for clinically relevant data, globally accepted reliability, and accurate early-stage screening. Thus, it is more important than ever to select the right CRO (Contract Research Organization) for strategic approach.
In this article, we highlight three CROs with proven technical capabilities, expertise, and long standing track records. These are our TOP 3 choices based on their capabilities and the specific target goals of the researchers for their non-clinical studies.

Pharmacology (Efficacy) Studies
Replicate unknown pathological models and
Discovery to clinically oriented drug evaluation

SMC Laboratories, Inc.

Reference: SMC Laboratories, Inc. official website (https://www.smccro-lab.com/jp/)

SMC Laboratories, Inc. has established a disease models using patented mouse technologies. The company has established proprietary pathological models—particularly in liver disease and fibrosis—and continues to expand their approach across a wide range of models in cancer, inflammation, and metabolic diseases.
From exploratory research to clinically oriented efficacy evaluation, SMC offers customized study designs, dosing strategies, and evaluation analysis tailored to each project. Their collaborative approachallows researchers to discuss and refine study plans together with SMC’s expert scientists.
With flexible small-scale study options and strong technical support, SMC Laboratories, Inc. is an ideal partner for start-ups, biotech ventures, and academic institutions alike.

Consult study design and disease model development
with SMC Laboratories, Inc.

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Safety Studies
Comprehensive Safety Evaluation for FIH Applications

Labcorp Holdings Inc.
(Labcorp Drug Development)

Reference: Labcorp Holdings Inc. official website (https://jp.labcorp.com/)

Labcorp Holdings Inc. provides a fully integrated GLP testing system aligned with international regulatory standards, including FDA, EMA, and PMDA requirements. All studies are conducted under ICH-compliant quality assurance, making it ready for data submission.
The company has extensive expertise in long-term toxicity studies such as Segment I–III reproductive and carcinogenicity studies, as well as 2-year chronic toxicity assessments.
Labcorp Holdings Inc.’s comprehensive approach enables sponsors to efficiently outsource the entire preclinical package from toxicology, toxicokinetic (TK), and safety pharmacology study design to execution. This accelerates a path to First-in-Human (FIH) trials. For most of the global drug developers, this all-in-one service structure minimizes cost, risk, and expedite the time before advancing to clinical phase.

Consult Labcorp Holdings Inc.
Discuss GLP/ICH-compliant study design for your FIH submission.

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Pharmacokinetic (PK/PD) Studies
High-Precision Bioanalysis for Clinically Predictive PK/PD Evaluation

PhoenixBio Co., Ltd.

Source: PhoenixBio Co., Ltd. Official Website (https://phoenixbio.co.jp/)

PhoenixBio Co., Ltd.offers pharmacokinetic and hepatic metabolism studies using their proprietary PXB-mouse®, a humanized-liver chimeric mouse model. This platform enables the acquisition of data with high clinical correlation in ADME, drug–to-drug interaction studies, bridging the gap between preclinical and clinical stages.
With advanced LC-MS/MS-based bioanalysis, PhoenixBio Co., Ltd. provides aseamless workflow from plasma concentration measurement and metabolite identification to quantitative validation.
The company offersan integrated evaluation analysiscovering pharmacokinetics, hepatotoxicity, and safety with flexibility to accommodate complex modalities such as oligonucleotide and middle-molecule therapeutics. For compounds where hepatic metabolism is a development bottleneck—or where quantitative, reproducible exposure data are critical—PhoenixBio Co., Ltd. delivers unmatched analytical precision and consistency.

Consult Phoenix Bio for PK/PD analysis with a clinical focus

Phoenix Bio.
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