Batten Disease (BD) Drug Discovery Service

Creative Biolabs is well-known in the field of neuroscience and has completed many innovative projects in this field. Now we provide one-stop research services against Batten disease (BD) for our clients all over the world.

Introduction to Batten Disease (BD)

Batten disease (BD), also known as neuronal ceroid lipofuscinoses, refers to a family of progressive neuro-paediatric disorders. Typical clinical features of this disease include seizures, decreased motor capacity, decreased vision and cognition, and eventually premature death. Studies have shown that BD is caused by mutations in 1 of 13 different genes.

Fig.1 Gene therapy and enzyme replacement therapy strategies in Batten disease. (Johnson, 2019)

Animal Models of Batten Disease (BD)

To better understand the pathology and disease hallmarks of Batten disease, Creative Biolabs has developed a series of robust animal models to accelerate therapeutic development. These mouse models were designed to match individual and uncommon mutations from patients to ensure the reliability of research results. At present, there are corresponding animal models for almost all forms of Barton disease. However, it is important to note that there are differences in neurodevelopment, neuroanatomy, etc., between mice and humans. Several clinical trials have shown that these differences lead to certain therapies that showed promise in mouse models but lacked efficacy in clinical patients.

Therapeutic Approaches of Batten Disease (BD)

Because the functions of many disease-related proteins have not been clarified, there are currently no effective treatments against BD.

• Enzyme Replacement Therapy (ERT)

ERT is a therapeutic strategy to address enzyme deficiency by introducing purified recombinant enzymes intravenously, intraventricularly, or intrathecally. ERT has been successfully applied to four of the Batten disease subtypes, including CLN1 (PPT1), CLN2 (TPP1), CLN10 (CTSD), and CLN13 (cathepsin F (CTSF)). It is important to note that successful treatment of patients with Barton disease requires efficient targeting of ERT to the central nervous system (CNS) by bypassing the blood-brain barrier (BBB).

• Gene Therapy

Adeno-associated virus (AAV)-mediated gene therapy presents great potential for the treatment of lysosomal storage disorders and neurodegenerative diseases. Various AAV serotypes have been used in different forms of Batten disease. For example, intracranial injection of AAV2-hPPT1 increased palmitoyl-protein thioesterase 1 (PPT1) enzymatic activity and alleviated many of the classic Batten disease pathological features.

• Stem Cell Therapy

Stem cell-based therapy has been studied to address visual deficits associated with the progression of Barton disease. Human CNS-derived stem cells (HuCNS-SCs) secreting endogenous TPP1 (Tripeptidyl Peptidase 1) and PTT1 are being tested.

• Small-molecule Therapy

In the past decades, a variety of pharmaceutical and biological agents have been tested in various models of Batten disease. These small molecules always act as immunomodulators or neuroprotectants.

Services for Batten Disease (BD)

• In Vitro Services
• In Vivo Services
• Ex Vivo Services
• Discovery Services
• Development Services

Batten Disease (BD) Drug Discovery Service

Equipped with world-leading platforms and extensive experience, Creative Biolabs provides one-stop drug discovery service for our clients. From the basic target identification to the final IND-enabling, we can meet all the needs of customers.

Creative Biolabs is a leading service provider that focuses on neuroscience research. We assist you in designing the best research outline customized to meet the requirements of clients' programs. If you are interested in our services and products, please do not hesitate to contact us for more details.

Reference

1. Johnson, T.B.; et al. Therapeutic landscape for Batten disease: current treatments and future prospects. Nature Reviews Neurology. 2019, 15(3): 161-178.