This “Lafora disease - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Lafora disease 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.
Lafora disease Understanding
Lafora disease (LD) is a rare, progressive, autosomal recessive neurodegenerative disorder characterized by intractable seizures, inexorable neurological deterioration, cognitive decline, dementia, and death within 10 years of onset. It is caused by loss-of-function mutations in either the laforin gene (EPM2A) or malin gene (NHLRC1) and is associated with gradual accumulation of Lafora bodies, aggregates of poorly branched, hyperphosphorylated, insoluble glycogen also known as polyglusan. LD usually begins in late childhood or adolescence. Animal studies have shown that reducing the production of glycogen in neurons can prevent the disorder. Valerion's goal is to enzymatically degrade neuronal glycogen, preventing its aggregation and rescuing the neurons from degeneration.
"Lafora disease - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Lafora disease pipeline landscape is provided which includes the disease overview and Lafora disease treatment guidelines. The assessment part of the report embraces, in depth Lafora disease 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, Lafora disease 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 Lafora disease R&D. The therapies under development are focused on novel approaches to treat/improve Lafora disease.
This segment of the Lafora disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.
TGTX 111-LAFORIN is a preclinical stage gene therapy being developed by Taysha Gene Therapies to treat Lafora disease. TSHA-111-LAFORIN has achieved effective knockdown of GYS1 expression and insoluble glycogen and decreased Lafora body formation in laforin and malin mouse models. Taysha Gene therapies plans IND/CTA submission for Lafora disease in 2021.
In preclinical proof-of-concept studies, Valerion and collaborators at the University of Kentucky demonstrated in vitro and in vivo, the ability to deliver VAL-0417 to the skeletal muscle and brain with retention of up to 24 hours following intracerebroventricular (ICV) injection in Lafora mouse models. These early but encouraging data demonstrate preclinical proof-of-concept for VAL-0417 and further validate the Valerion platform technology for use in CNS disease.
This segment of the report provides insights about the different Lafora disease drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 3+ key companies which are developing the therapies for Lafora disease. The companies which have their Lafora disease drug candidates in the most advanced stage, i.e. preclinical include, Valerion Therapeutics.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Lafora disease therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Lafora disease drugs.
Current Treatment Scenario and Emerging Therapies:
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Geography Covered
- Global coverage
Lafora disease Understanding
Lafora disease: Overview
Lafora disease (LD) is a rare, progressive, autosomal recessive neurodegenerative disorder characterized by intractable seizures, inexorable neurological deterioration, cognitive decline, dementia, and death within 10 years of onset. It is caused by loss-of-function mutations in either the laforin gene (EPM2A) or malin gene (NHLRC1) and is associated with gradual accumulation of Lafora bodies, aggregates of poorly branched, hyperphosphorylated, insoluble glycogen also known as polyglusan. LD usually begins in late childhood or adolescence. Animal studies have shown that reducing the production of glycogen in neurons can prevent the disorder. Valerion's goal is to enzymatically degrade neuronal glycogen, preventing its aggregation and rescuing the neurons from degeneration.
"Lafora disease - Pipeline Insight, 2021" report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Lafora disease pipeline landscape is provided which includes the disease overview and Lafora disease treatment guidelines. The assessment part of the report embraces, in depth Lafora disease 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, Lafora disease 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 Lafora disease R&D. The therapies under development are focused on novel approaches to treat/improve Lafora disease.
Lafora disease Emerging Drugs Chapters
This segment of the Lafora disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.
Lafora disease Emerging Drugs
TSHA-111-LAFORIN: Taysha Gene Therapies
TGTX 111-LAFORIN is a preclinical stage gene therapy being developed by Taysha Gene Therapies to treat Lafora disease. TSHA-111-LAFORIN has achieved effective knockdown of GYS1 expression and insoluble glycogen and decreased Lafora body formation in laforin and malin mouse models. Taysha Gene therapies plans IND/CTA submission for Lafora disease in 2021.
VAL-0417: Valerion Therapeutics
In preclinical proof-of-concept studies, Valerion and collaborators at the University of Kentucky demonstrated in vitro and in vivo, the ability to deliver VAL-0417 to the skeletal muscle and brain with retention of up to 24 hours following intracerebroventricular (ICV) injection in Lafora mouse models. These early but encouraging data demonstrate preclinical proof-of-concept for VAL-0417 and further validate the Valerion platform technology for use in CNS disease.
Lafora disease: Therapeutic Assessment
This segment of the report provides insights about the different Lafora disease drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Lafora disease
There are approx. 3+ key companies which are developing the therapies for Lafora disease. The companies which have their Lafora disease drug candidates in the most advanced stage, i.e. preclinical include, Valerion Therapeutics.
Phases
This report covers around 3+ products under different phases of clinical development like
- 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
Lafora disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- intravenous
- Subcutaneous
- Topical.
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Lafora disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Lafora disease 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 Lafora disease drugs.
Lafora disease Report Insights
- Lafora disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Lafora disease Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Lafora disease drugs?
- How many Lafora disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Lafora disease?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Lafora disease 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 Lafora disease and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Taysha GTx
- Valerion Therapeutics
- Ionis Pharmaceuticals
- LQT Therapeutics
Key Products
- TSHA-111-MALIN
- TSHA-111-LAFORIN
- ION 283
- VAL-0417
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Table of Contents
IntroductionExecutive Summary
Lafora disease: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
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
Lafora disease - Analytical Perspective
Early Stage Products
- Comparative Analysis
Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
VAL-0417: Valerion Therapeutics
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Lafora disease Key Companies
Lafora disease Key Products
Lafora disease- Unmet Needs
Lafora disease- Market Drivers and Barriers
Lafora disease- Future Perspectives and Conclusion
Lafora disease Analyst Views
Lafora disease Key Companies
AppendixList of Tables
Table 1 Total Products for Lafora disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Lafora disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Taysha GTx
- Valerion Therapeutics
- Ionis Pharmaceuticals
- LQT Therapeutics
