Juvenile Macular Degeneration (JMD) Pipeline Analysis Report 2025

According to the National Institute of Health, in boys, the leading inherited cause of macular degeneration is X-linked juvenile retinoschisis with an approximate prevalence of 1 in 5,000-25,000. It accounts for approximately 5% of all childhood-onset, inherited retinal dystrophies. There is no specific treatment to stop the disorder but there are several gene therapy trials underway to correct abnormal genes in the retina. These emerging drugs are expected to slow down the progression of disease and prevent vision loss in the coming years.

Report Coverage

The Juvenile Macular Degeneration (JMD) Drug Pipeline Insight Report by the publisher gives comprehensive insights into juvenile macular degeneration (JMD) drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for juvenile macular degeneration (JMD). The report includes the analysis of over 20+ pipeline drugs and 10+ companies . The juvenile macular degeneration (JMD) pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Juvenile Macular Degeneration (JMD).

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing product development activities related to juvenile macular degeneration (JMD).

Juvenile Macular Degeneration (JMD) Drug Pipeline Outlook

Juvenile macular degeneration refers to several inherited and rare disorders which affect children and young adults. These disorders include Best disease, Stargardt's disease, and juvenile retinoschisis. These disorders cause central vision loss which often starts in childhood or sometimes in young adulthood. Juvenile macular degeneration damages the macula, which is the tissue in the center of the retina at the back of the eye. This is the region which provides sharp central vision and allows us to do things, see colors and recognize faces. These conditions are passed down in families from gene changes. Stargardt’s disease is only inherited if the two parents are affected by the gene. It can be inherited even if one of the parents is affected in the case of Best’s vitelliform macular dystrophy and in the case of juvenile retinoschisis affects males as it is linked to the X chromosome, but it is transmitted from mother to child.

The symptoms of juvenile macular degeneration include the display of gray and black spots, photophobia, blurred vision with dark areas, impairment of color perception, and yellowish flecks around the macula among others. There is no treatment available for juvenile macular degeneration. Visual aids, adaptive training, and some other tools can help people with vision loss remain active. Sunglasses with ultraviolet protection and to perform mobility exercises for patients are recommended. Several studies and research continue to find ways to prevent and manage JMD. Pharma companies and research institutes are developing innovative gene therapies. Alkeus Pharmaceuticals for Regenerative Medicine announced promising results from its TEASE-3 clinical trial for the drug ALK-001. The increasing clinical trials and changing dynamics are impacting the juvenile macular degeneration (JMD) clinical trial landscape significantly.

Juvenile Macular Degeneration (JMD) - Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of Juvenile Macular Degeneration (JMD) based on several segmentations including:

By Phase

• Late-Stage Products (Phase 3 and Phase 4)
• Mid-Stage Products (Phase 2)
• Early-Stage Products (Phase I)
• Preclinical and Discovery Stage Products

By Drug Class

• Recombinant Fusion Proteins
• Small Molecule
• Monoclonal Antibody
• Peptide
• Polymer
• Gene Therapy

By Route of Administration

• Oral
• Parenteral
• Others

Juvenile Macular Degeneration (JMD) - Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase I and II covers a major share of the total clinical trials for juvenile macular degeneration (JMD) as there are not many drugs present in late-stage clinical trials.

Juvenile Macular Degeneration (JMD) - Pipeline Assessment Segmentation, By Drug Classes

The drug molecules categories covered under juvenile macular degeneration (JMD) pipeline analysis include recombinant fusion proteins, small molecules, monoclonal antibodies, peptides, polymers, and gene therapy. Astellas Institute is conducting a phase I/II trial for Biological: MA09-hRPE. The trial is assessing the subretinal injection of human embryonic stem cell-derived retinal pigment epithelium cells in patients suffering from Stargardt's macular dystrophy. Gene therapies are also being under several trials to prevent and treat JMD. The therapy works by correcting the abnormal gene in the retina. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for juvenile macular degeneration (JMD).

Juvenile Macular Degeneration (JMD) Clinical Trials Therapeutic Assessment - Competitive Dynamics

The report for the juvenile macular degeneration (JMD) pipeline covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in juvenile macular degeneration (JMD) clinical trials:

• Astellas Institute for Regenerative Medicine
• Ascidian Therapeutics, Inc.
• Others

Juvenile Macular Degeneration (JMD) - Emerging Drugs Profile

Leading drugs in the pipeline are as follows:

Biological: MA09-hRPE

The trial is designed to assess the tolerability and safety of RPE cellular therapy in patients with Stargardt's macular dystrophy. The trial is sponsored by Astellas Institute for Regenerative Medicine and is currently under phase I/II.

Drug: ACDN-01

The objective of the study is to assess the safety and efficacy of ACDN-01 following a single subretinal injection of ACDN-01. This is the first human clinical trial in which ACDN-01 will be evaluated, sponsored by Ascidian Therapeutics, Inc. and is currently under phase I/II.

Reasons To Buy This Report

The Juvenile Macular Degeneration (JMD) Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for juvenile macular degeneration (JMD). It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within juvenile macular degeneration (JMD) pipeline insights.

Key Questions Answered in the Juvenile Macular Degeneration (JMD) - Pipeline Insight Report

• What is the current landscape of juvenile macular degeneration (JMD) pipeline drugs?
• How many companies are developing juvenile macular degeneration (JMD) drugs?
• How many phase III and phase IV drugs are currently present in juvenile macular degeneration (JMD) pipeline drugs?
• Which companies/institutions are leading the juvenile macular degeneration (JMD) development?
• What is the efficacy and safety profile of juvenile macular degeneration (JMD) pipeline drugs?
• What are the opportunities and challenges present in the juvenile macular degeneration (JMD) pipeline landscape?
• Which company is conducting major trials for juvenile macular degeneration (JMD)?
• What geographies are covered for juvenile macular degeneration (JMD) clinical trials?
• What are emerging trends in juvenile macular degeneration (JMD) clinical trials?

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