Condensate Therapeutics for Neurodegenerative Diseases

CD BioSciences is dedicated to developing disease-targeted condensate therapeutics to advance the field of refractory diseases. Here, we offer condensate therapeutics for neurodegenerative diseases, offering new hope for the treatment of neurodegenerative diseases.

Condensate Research Offers New Hope for the Treatment of Neurodegenerative Diseases

Neurodegenerative diseases (NDD) are a heterogeneous group of incurable and debilitating disorders, including Alzheimer's disease (AD), Parkinson's disease (PD), frontotemporal lobar degeneration (FTLD), and amyotrophic lateral sclerosis (ALS). Studies have shown that biomolecular condensates are closely related to the pathogenesis of these NDD. In addition to the classical idea that nucleation-aggregation processes trigger protein aggregation from misfolded seeds, pathological transformations of biomolecular condensates can also promote protein aggregation found in NDD deposits. Thus, understanding protein misfolding and abnormal accumulation in the nervous system is key to unraveling the pathogenesis of NDD and helping develop new therapeutic approaches for these still incurable NDD.

Fig. 1. Drug discovery avenues for targeting aberrant phase transitions associated with neurodegeneration. (Hurtle BT, et al, 2023)

Our Solutions

We offer professional services to analyze abnormal protein phase transitions in neurodegenerative diseases, including TDP-43, FUS, Tau, and α-Synuclein, etc. The characterization of these abnormal phase change biomolecular condensates in NDD helps us to develop new therapeutic approaches. CD BioSciences is committed to developing condensate therapeutics for NDD.

We aim to develop targeted therapeutics that modulate or solubilize these disease-causing condensates, thereby restoring cellular homeostasis and mitigating disease progression. We offer several potential condensate-based therapeutic strategies for NDD as follows:

• Targeting RNA. we reduce the cellular accumulation of proteins associated with NDD by targeting RNA to reduce, an approach that bypasses questions about the toxicity of specific protein conformations, modifications and polymer assemblies, and effectively prevents downstream toxicity.
• Small molecule modulators. Our experts can design small molecule modulators that interact specifically with dysregulated condensates. These modulators act by disrupting aberrant molecular interactions, promoting the solubilization of pathological condensates, and preventing the segregation of essential cellular components.
• Immunotherapy. We offer peptide-based therapies to target dysfunctional condensates in NDD. Peptides are short-chain amino acids that have the advantage of precise, selective binding of specific target condensates. We aim to provide effective therapeutic interventions by designing peptides that disrupt the assembly or stabilize the solubilization of pathological condensates.
• PTM modification therapies. Based on extensive studies of the complex interactions between individual PTMs and combinatorial PTMs and how PTMs alter biomolecular condensates interaction partners, resulting in phase behavior, and subsequent neurotoxicity, we can modulate pathological protein PTMs.
• Biomolecular engineering. With its expertise in biomolecular engineering, our experts utilize state-of-the-art technologies to optimize the properties of condensate targeted therapeutics. Through careful design and modification, we work to enhance drug delivery, stability, and selectivity, ultimately maximizing the therapeutic potential of these innovative therapies.

Through its cutting-edge research and development efforts, CD BioSciences is advancing the field of NDD therapeutics by targeting dysfunctional cellular condensates. With a focus on small molecule modulators, RNA therapeutics, peptide therapeutics, and biomolecular engineering, we are poised to revolutionize the way we fight these diseases. If you are interested in our solutions, please contact us for more information.