Batten disease is a rare and deadly hereditary neurological illness that is caused by mutations in CLN genes, which are in charge of breaking down cellular waste. It mainly affects children. Seizures, visual loss, cognitive decline, motor skill impairment, and behavioral disorders are among the progressive symptoms that result from the buildup of lipopigments in brain cells. Usually starting between the ages of 5 and 10, the symptoms get worse over time, leading to blindness, paralysis, and early death by the late teens or early twenties. Only symptomatic measures to enhance quality of life are available; there is no cure. The Batten disease pipeline analysis by Expert Market Research focuses on various treatment options for this disease.
Major companies involved in the Batten disease pipeline analysis include Amicus Therapeutics, and Neurogene Inc., among others.
Leading drugs currently in the pipeline include ALIS, Bedaquiline, and others.
Increased investment in research and development, along with regulatory support, is accelerating clinical trials and new treatment approvals.
The Batten Disease Pipeline Analysis Report by Expert Market Research gives comprehensive insights into Batten disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Batten disease. The Batten disease report assessment includes the analysis of over 15 pipeline drugs and 10+ companies. The Batten disease 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 the condition, and alignment with Batten disease treatment guidelines to ensure optimal care practices.
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 Batten disease.
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Defective lysosomal enzymes or proteins necessary for the digestion of cellular waste result from mutations in one of multiple CLN genes, which causes Batten disease. Because lysosomes, the cell's "recycling centers," are unable to break down and eliminate waste, lipopigments build up toxically in brain cells. Progressive neurodegeneration, visual loss, seizures, and motor decline are the results of this accumulation, which impairs normal cell function, particularly in neurons. The disease progresses as a result of the compromised waste clearance, which eventually causes organ dysfunction and cell death.
Despite the lack of a cure, Batten disease is generally treated with palliative and supportive measures to control symptoms and enhance quality of life. Drugs manage behavioral problems, muscle spasms, and seizures. Mobility, everyday functioning, and communication are all maintained with the aid of physical, occupational, and speech treatments. Pain is reduced with palliative treatment and nutritional support. Certain Batten forms can be treated with approved enzyme replacement treatment, such as cerliponase alfa, which slows the disease's course. Research is being done on experimental gene and stem cell therapies to address the root problems.
The frequency of Batten disease, a rare neurological condition, is roughly 1 in 100,000 live births globally. Between two and four children out of every 100,000 are affected in the United States. In other communities, such those in Northern Europe and Newfoundland, Canada, it is more prevalent. Mutations in the CLN3 gene are the most common cause of juvenile Batten disease. There are approximately 14,000 children with the illness worldwide.
This section of the report covers the analysis of Batten disease drug candidates based on several segmentations, including:
By Phase
By Drug Class
By Route of Administration
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 EMR analysis, phase I covers a major share of the total Batten disease clinical trials.
The drug molecule categories covered under the Batten disease pipeline analysis include monoclonal antibody, polymers, peptides, small molecule and gene therapy. The Batten disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Batten disease.
The EMR report for the Batten Disease pipeline analysis 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 Batten disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Batten disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Batten disease drug candidates.
Amicus Therapeutics created the experimental gene therapy AT-GTX-502 to treat CLN3 Batten disease. It provides a functional copy of the CLN3 gene with a single intrathecal injection using an AAV9 vector. Early results from a Phase 1/2 trial indicated that the medication was well tolerated and might slow the progression of the disease. Over a 12-month period, there were only minor changes in physical impairment scores as compared to individuals who did not get treatment.
The experimental gene therapy NGN-101, created by Neurogene Inc., targets CLN5 Batten disease, a rare and deadly neurological condition in children. It delivers a functional CLN5 gene through intravitreal and intracerebroventricular injections using an AAV9 vector. Its safety and effectiveness were evaluated in a Phase 1/2 experiment. However, after the FDA rejected RMAT designation in November 2024, citing difficulties in aligning on a streamlined regulatory process, Neurogene put a halt to future development.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
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