Leber’s Hereditary Optic Neuropathy - Pipeline Insight, 2025
Description
DelveInsight’s, “Leber’s Hereditary Optic Neuropathy - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 4+ pipeline drugs in Leber’s Hereditary Optic Neuropathy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Leber’s Hereditary Optic Neuropathy: Overview
Mitochondrial diseases were once thought to be rare, largely due to their diverse and often subtle clinical presentations, which made diagnosis challenging. However, these conditions are increasingly recognized as a significant cause of neurological and visual dysfunction. Ophthalmic manifestations, in particular, are common in mitochondrial disorders and can lead to retinopathy, ocular motility disorders, and optic neuropathy. Among the various mitochondrial disorders, Leber hereditary optic neuropathy (LHON) is often regarded as the quintessential condition. First described by Dr. Theodore Leber in 1858, LHON was initially identified by Dr. Albrecht von Graefe in a small group of patients. It was the first mitochondrial disease recognized to be maternally inherited and linked to a point mutation in mitochondrial DNA (mtDNA). Vision loss in LHON occurs due to selective degeneration of retinal ganglion cells (RGCs), which are particularly vulnerable to mitochondrial dysfunction. The pathophysiological mechanisms underlying LHON continue to be explored, and ongoing research holds promise for potential therapeutic strategies.
LHON typically presents with painless, subacute central vision loss in one eye, followed by involvement of the second eye within weeks to months. Most patients progress to a visual acuity of 20/200 or worse, with early visual field defects manifesting as a cecocentral scotoma. Fundus examination in the acute stage may reveal optic disc hyperemia, peripapillary telangiectatic blood vessels, and retinal nerve fiber layer (RNFL) swelling, though up to 40% of patients may have a normal-appearing fundus. As the disease progresses, optic disc pallor and RNFL thinning occur. Visual evoked potentials (VEPs) and electroretinograms (ERGs) are often abnormal, reflecting optic nerve degeneration. While brain and orbital magnetic resonance imaging is typically normal, some patients show non-specific optic nerve enhancement. Interestingly, asymptomatic LHON mutation carriers may exhibit subclinical signs of disease, such as subtle fundus changes or temporal RNFL thickening.
The diagnosis of LHON involves a comprehensive neuro-ophthalmologic evaluation, with a focus on the clinical presentation of sudden, painless central vision loss, typically affecting young males in a sequential pattern. Key diagnostic tests include genetic testing for mitochondrial DNA mutations, optical coherence tomography (OCT) to assess retinal nerve fiber layer changes, and visual field testing. A pattern electroretinogram (PERG) and MRI/CT scans may be used to rule out other causes of optic neuropathy. Genetic testing is essential, often starting with blood tests to identify common LHON mutations, followed by targeted mtDNA sequencing or a multi-gene mitochondrial panel. Confirmation of the diagnosis is made by excluding alternative optic neuropathies and identifying characteristic optic nerve changes, with a family history of visual loss further supporting the diagnosis.
Treatment for LHON is primarily supportive, focusing on low vision aids and rehabilitation for central vision loss, as well as counseling to avoid environmental risk factors like tobacco and alcohol. While various nutritional supplements and medications such as idebenone and brimonidine have been explored, their efficacy remains inconclusive. Gene therapy, involving the introduction of wild-type genes to restore mitochondrial function, shows promise in early-stage clinical trials. Additionally, stem cell therapies aimed at protecting retinal ganglion cells are under investigation. However, there is currently no curative treatment, and most patients rely on supportive care.
""Leber’s Hereditary Optic Neuropathy- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leber’s Hereditary Optic Neuropathy pipeline landscape is provided which includes the disease overview and Leber’s Hereditary Optic Neuropathy treatment guidelines. The assessment part of the report embraces, in depth Leber’s Hereditary Optic Neuropathy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Leber’s Hereditary Optic Neuropathy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Leber’s Hereditary Optic Neuropathy report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Leber’s Hereditary Optic Neuropathy Emerging Drugs
Further product details are provided in the report……..
Leber’s Hereditary Optic Neuropathy: Therapeutic Assessment
This segment of the report provides insights about the different Leber’s Hereditary Optic Neuropathy drugs segregated based on following parameters that define the scope of the report, such as:
Leber’s Hereditary Optic Neuropathy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Leber’s Hereditary Optic Neuropathy therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leber’s Hereditary Optic Neuropathy drugs.
Leber’s Hereditary Optic Neuropathy Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Leber’s Hereditary Optic Neuropathy: Overview
Mitochondrial diseases were once thought to be rare, largely due to their diverse and often subtle clinical presentations, which made diagnosis challenging. However, these conditions are increasingly recognized as a significant cause of neurological and visual dysfunction. Ophthalmic manifestations, in particular, are common in mitochondrial disorders and can lead to retinopathy, ocular motility disorders, and optic neuropathy. Among the various mitochondrial disorders, Leber hereditary optic neuropathy (LHON) is often regarded as the quintessential condition. First described by Dr. Theodore Leber in 1858, LHON was initially identified by Dr. Albrecht von Graefe in a small group of patients. It was the first mitochondrial disease recognized to be maternally inherited and linked to a point mutation in mitochondrial DNA (mtDNA). Vision loss in LHON occurs due to selective degeneration of retinal ganglion cells (RGCs), which are particularly vulnerable to mitochondrial dysfunction. The pathophysiological mechanisms underlying LHON continue to be explored, and ongoing research holds promise for potential therapeutic strategies.
LHON typically presents with painless, subacute central vision loss in one eye, followed by involvement of the second eye within weeks to months. Most patients progress to a visual acuity of 20/200 or worse, with early visual field defects manifesting as a cecocentral scotoma. Fundus examination in the acute stage may reveal optic disc hyperemia, peripapillary telangiectatic blood vessels, and retinal nerve fiber layer (RNFL) swelling, though up to 40% of patients may have a normal-appearing fundus. As the disease progresses, optic disc pallor and RNFL thinning occur. Visual evoked potentials (VEPs) and electroretinograms (ERGs) are often abnormal, reflecting optic nerve degeneration. While brain and orbital magnetic resonance imaging is typically normal, some patients show non-specific optic nerve enhancement. Interestingly, asymptomatic LHON mutation carriers may exhibit subclinical signs of disease, such as subtle fundus changes or temporal RNFL thickening.
The diagnosis of LHON involves a comprehensive neuro-ophthalmologic evaluation, with a focus on the clinical presentation of sudden, painless central vision loss, typically affecting young males in a sequential pattern. Key diagnostic tests include genetic testing for mitochondrial DNA mutations, optical coherence tomography (OCT) to assess retinal nerve fiber layer changes, and visual field testing. A pattern electroretinogram (PERG) and MRI/CT scans may be used to rule out other causes of optic neuropathy. Genetic testing is essential, often starting with blood tests to identify common LHON mutations, followed by targeted mtDNA sequencing or a multi-gene mitochondrial panel. Confirmation of the diagnosis is made by excluding alternative optic neuropathies and identifying characteristic optic nerve changes, with a family history of visual loss further supporting the diagnosis.
Treatment for LHON is primarily supportive, focusing on low vision aids and rehabilitation for central vision loss, as well as counseling to avoid environmental risk factors like tobacco and alcohol. While various nutritional supplements and medications such as idebenone and brimonidine have been explored, their efficacy remains inconclusive. Gene therapy, involving the introduction of wild-type genes to restore mitochondrial function, shows promise in early-stage clinical trials. Additionally, stem cell therapies aimed at protecting retinal ganglion cells are under investigation. However, there is currently no curative treatment, and most patients rely on supportive care.
""Leber’s Hereditary Optic Neuropathy- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leber’s Hereditary Optic Neuropathy pipeline landscape is provided which includes the disease overview and Leber’s Hereditary Optic Neuropathy treatment guidelines. The assessment part of the report embraces, in depth Leber’s Hereditary Optic Neuropathy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Leber’s Hereditary Optic Neuropathy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Leber’s Hereditary Optic Neuropathy R&D. The therapies under development are focused on novel approaches to treat/improve Leber’s Hereditary Optic Neuropathy.
This segment of the Leber’s Hereditary Optic Neuropathy report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Leber’s Hereditary Optic Neuropathy Emerging Drugs
- GS010: GenSight Biologics
- Visomitin: Mitotech
Further product details are provided in the report……..
Leber’s Hereditary Optic Neuropathy: Therapeutic Assessment
This segment of the report provides insights about the different Leber’s Hereditary Optic Neuropathy drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Leber’s Hereditary Optic Neuropathy
- There are approx. 3+ key companies which are developing the therapies for Leber’s Hereditary Optic Neuropathy. The companies which have their Leber’s Hereditary Optic Neuropathy drug candidates in the most advanced stage, i.e. Phase II include, GenSight Biologics.
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Leber’s Hereditary Optic Neuropathy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Leber’s Hereditary Optic Neuropathy therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leber’s Hereditary Optic Neuropathy drugs.
Leber’s Hereditary Optic Neuropathy Report Insights
- Leber’s Hereditary Optic Neuropathy Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Leber’s Hereditary Optic Neuropathy drugs?
- How many Leber’s Hereditary Optic Neuropathy drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Leber’s Hereditary Optic Neuropathy?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Leber’s Hereditary Optic Neuropathy therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Leber’s Hereditary Optic Neuropathy and their status?
- What are the key designations that have been granted to the emerging drugs?
- GenSight Biologics
- Mitotech
- Biovista
- Neurophth Therapeutics
- GS010
- Visomitin
- BVA203
- NFS-01
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Leber’s Hereditary Optic Neuropathy: Overview
- Introduction
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Treatment
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Leber’s Hereditary Optic Neuropathy– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- GS010: GenSight Biologics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Visomitin: Mitotech
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Leber’s Hereditary Optic Neuropathy Key Companies
- Leber’s Hereditary Optic Neuropathy Key Products
- Leber’s Hereditary Optic Neuropathy- Unmet Needs
- Leber’s Hereditary Optic Neuropathy- Market Drivers and Barriers
- Leber’s Hereditary Optic Neuropathy- Future Perspectives and Conclusion
- Leber’s Hereditary Optic Neuropathy Analyst Views
- Leber’s Hereditary Optic Neuropathy Key Companies
- Appendix
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