Leber's Hereditary Optic Neuropathy - Pipeline Insight, 2025

DelveInsight’s, “Leber’s hereditary optic neuropathy - Pipeline Insight, 2025,” report provides comprehensive insights about 6+ companies and 7+ 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

• Global coverage

Leber’s hereditary optic neuropathy Understanding

Leber’s hereditary optic neuropathy: Overview

Leber hereditary optic neuropathy (LHON) is often characterized by bilateral, painless subacute loss of central vision during young adult life. In most cases, symptoms begin with one eye first, followed a few weeks later by visual failure in the other eye. Extremely rarely there may be neurologic abnormalities, such as peripheral neuropathy, postural tremor, nonspecific myopathy, and movement disorders. LHON is caused by mutations in mitochondrial DNA and it is strictly transmitted by maternal inheritance.

Individuals with LHON typically display symptoms in their young adult years. If vision is lost, then it usually occurs before 40 years of age. LHON is caused by genetic mutations in the mitochondrial DNA (mtDNA). Some mothers with a LHON gene mutation do not show symptoms, but family history often reveals female relatives with visual loss at an early age.

Affected individuals should receive supportive management and treatment through the usage of visual aids, occupational rehabilitation, and local social services. Small studies have shown that therapies involving ubiquinone and idebenone may provide possible benefits during the acute and chronic phases of the disorder. Affected individuals should avoid smoking and excessive alcohol consumption, which generate reactive oxygen species (ROS) producing mitochondrial impairments.

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 for Leber’s hereditary optic neuropathy.

Leber’s hereditary optic neuropathy Emerging Drugs Chapters

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

• GS 010: GenSight Biologics

LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. “LUMEVOQ” was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ is currently in the review phase of its registration process in Europe, and in Phase III to move forward to a BLA filing in the U.S.

• NFS-02: Neurophth Therapeutics

Neurophth is currently developing NFS-01 (NR082, rAAV-ND4) to treat LHON diseases caused by G11778A (ND4) site mutation and G3460A (ND1) site mutation, respectively. The mechanism of action is to leverage gene therapy strategy and use recombinant adeno-associated virus as the vector to deliver the correct genes to the patients' damaged optic ganglion cells through introvitinreous injection, thus repair the mitochondrial biological respiratory chain, and restore the vitality and visual function of optic ganglion cells.

NFS-01 (NR082, rAAV-ND4) was designated as orphan drug by the U.S. FDA in September 2020, which laid a foundation for accelerating international development of this product.

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 working on Leber’s hereditary optic neuropathy

There are approx. 6+ key companies which are developing the Leber’s hereditary optic neuropathy. The companies which have their Leber’s hereditary optic neuropathy drug candidates in the most advanced stage, i.e. Preregistration include, GenSight Biologics.

• Phases

DelveInsight’s report covers around 7+ products under different phases of clinical development like

• Late-stage products (Phase III and
• Mid-stage products (Phase II and
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Leber’s hereditary optic neuropathy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

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

Leber’s hereditary optic neuropathy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current 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 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?

Key Players

• GenSight Biologics
• Neurophth Therapeutics
• Mitotech
• Stealth BioTherapeutics
• Fortify Therapeutics
• Neuroptika

Key Products

• GS 010
• NFS-02
• NFS-01
• Visomitin
• Elamipretide
• Research programme: Leber's hereditary optic neuropathy therapeutics
• NRO-1

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