
Batten Disease- Pipeline Insight, 2024
Description
Batten Disease- Pipeline Insight, 2024
DelveInsight’s, “Batten Disease- Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Batten 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.
Geography Covered
- Global coverage
Batten Disease: Overview
Batten disease (also known as neuronal ceroid lipofuscinosis, NCL) is the name for a group of inherited nervous system disorders that most often begin in childhood. They interfere with a cell's ability to recycle a cellular residue called lipofuscin. Batten is the term commonly used to describe the many forms of the disease, which is formally called neuronal ceroid lipofuscinosis. The many forms of the disease are classified by the gene that causes the disorder, with each gene being called ""ceroid lipofucinosis, neuronal"" (CLN) and given a different number as its subtype. Because of the different gene mutations, signs and symptoms range in severity and progress at different rates. Symptoms generally include progressive vision loss leading to blindness, seizures, movement disorder, and dementia. Developmental skills such as standing, walking, and talking may not be achieved or are gradually lost. Other symptoms that continue to worsen over time include learning difficulties, poor concentration, and progressive loss of language skills and speech. Most children become bedridden and unable to communicate. Some children develop problems sleeping. Currently, most diagnosis of Batten disease are made by genetic testing.
Pathologically, Batten disease is characterized by lysosomal accumulation of autofluorescent storage material, glial reactivity and neuronal loss. Substantial progress has been made towards the development of effective therapies and treatments for the multiple forms of Batten disease. In 2017, cerliponase alfa (Brineura), a tripeptidyl peptidase enzyme replacement therapy, became the first globally approved treatment for CLN2 Batten disease. Here, we provide an overview of the promising therapeutic avenues for Batten disease, highlighting current FDA-approved clinical trials and prospective future treatments.
""Batten Disease- Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Batten Disease pipeline landscape is provided which includes the disease overview and Batten Disease treatment guidelines. The assessment part of the report embraces, in depth Batten 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, Batten 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 Batten Disease R&D. The therapies under development are focused on novel approaches to treat/improve Batten Disease.
This segment of the Batten 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.
Batten Disease Emerging Drugs
- PLX-200: Polaryx Therapeutics
- NGN-101: Neurogene Inc
Further product details are provided in the report……..
Batten Disease: Therapeutic Assessment
This segment of the report provides insights about the different Batten Disease drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Batten Disease
- There are approx. 10+ key companies which are developing the therapies for Batten Disease. The companies which have their Batten Disease drug candidates in the most advanced stage, i.e. phase III include, Polaryx Therapeutics.
- 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
Batten Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Batten 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 Batten Disease drugs.
Batten Disease Report Insights
- Batten Disease 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 Batten Disease drugs?
- How many Batten Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Batten Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Batten 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 Batten Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- Amicus Therapeutics
- REGENXBIO
- Polaryx Therapeutics
- Neurogene Inc
- Theranexus
- M6P Therapeutics
- AT-GTX-502
- RGX-381
- PLX-200
- NGN-101
- BBDF-101
- M-082
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Batten 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
- Batten Disease– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- PLX-200: Polaryx Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- NGN-101: Neurogene Inc
- 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
- Batten Disease Key Companies
- Batten Disease Key Products
- Batten Disease- Unmet Needs
- Batten Disease- Market Drivers and Barriers
- Batten Disease- Future Perspectives and Conclusion
- Batten Disease Analyst Views
- Batten Disease Key Companies
- Appendix
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