Targeted Therapeutics for Auditory Regeneration

Hearing ability serves an important role in the daily communications between families, friends, and colleagues and has a big impact on quality of life. There is the public interest in the threat of hearing loss caused by exposure to unsafe levels of sound from audio device and an aged society. Increasing evidences have shown that hearing loss diminishes a person’s mode of communication leading to social isolation and is associated with several diseases in the CNS. However, there is still no curable treatment for hearing loss at this point.

Astellas is taking the approach of regaining auditory functions by leveraging its core technology in the area of regenerative medicine. It believes doing so will not only help people regain auditory functions, but will also contribute to the well-being of society.

Astellas’ Auditory Regeneration has been working on the following leading programs.

  • FX-322 (Inner ear progenitor cell activator for sensorineural hearing loss, Phase 2)
  • ASP0598 (Recombinant heparin-binding epidermal growth factor-like growth factor (HB-EGF) for regeneration of tympanic membrane, Phase 1)

In addition, Astellas has the following capabilities:

  • Unique preclinical programs and optimal animal models for translational research
  • Our original local drug delivery system technology in the ear
  • Several pipelines in clinical stage and lots of expertise in development.
  • Top-notch researchers and clinicians through partnerships

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[Assets and capabilities that Astellas expects from its partners]

  • Drug/technology for regeneration of hair cell and repair of ribbon synapse formation, Strial vascular function improvement (preferably lead molecule identified)
  • Drug delivery system that targets the inner ear with small compound/protein/nucleic acid/gene therapy, in addition to hearable devices concurrently used with drug treatment/for diagnosis
  • Animal models to predict human effectiveness in sensorineural hearing loss



Many diseases are marked by the accumulation of defective, damaged or no longer required proteins that compromise cellular function.

Autophagy is a key quality control process by which cells eliminate such aberrant proteins.

Potentiating autophagy is a direct way to address the wide range of diseases characterized by defective protein accumulation and is expected as an approach applicable to neurodegeneration, lysosomal storage disorders, renal disease, infectious disease, HIV, cancers, and even some psychiatric disorders.

Astellas utilizes its biochemistry and cell biology expertise in autophagy and aims to provide new safe and effective treatment options to patients who are suffering from various diseases without effective treatments.

Assets and capabilities that Astellas expects from its partners

  • Novel mechanism and target for regulating autophagy
  • Small molecules that can control that mechanism and target (preferably)
  • Screening technologies or novel assays for autophagy regulator



Bispecific immune cell engager is known as an approach that attracts two cells via a bispecific antibody in which two antibodies against an antigen expressed on the surface of each of the two cells of interest are bound. In particular, the bispecific T cell engager is a bispecific antibody that binds an antibody against a target antigen expressed on the surface of cancer cells and an antibody capable of activating T cells, and the T cells are placed near the target cancer cells. It is attracting attention as an approach of effectively removing cancer cells.

By utilizing these technologies, high specificity and unique control that cannot be achieved by conventional antibody technologies will be possible, and it is expected that the possibility of drug discovery will be greatly expanded even for diseases other than cancer.

Astellas aims to provide new treatment options for patients with diseases for which there is no effective treatment by combining these approaches of bispecific immune cell engager with the antibody drug discovery technology of Astellas.

Assets and capabilities that Astellas expects from its partners

  • Surface antigens that can be applied as targets for tissue-specific drug delivery and their screening techniques
  • Technology for recognizing environment-specific target antigens and proteins
  • Innovative technologies, such as multi-specific antibodies, that enable unique immune control
  • We are looking for the above technologies that can be applied to other diseases other than oncology

You can find more information on bispecific immune cell engager in oncology at the website of Primary focus Immuno-Oncology below.



Direct reprogramming is a biology that directly converts the fate of cells without passing through the pluripotent state, and the technology that enables direct reprogramming in vivo has attracted attention as a new regenerative medicine approach. Unlike conventional treatments, direct reprogramming not only removes unnecessary cells or replenishes necessary cells, but also converts unnecessary cells into necessary cells. It is expected to be highly effective against intractable diseases that have been thought to be incurable.

In addition, if agents that generate the desired cells in the body are obtained, it is expected to avoid the risk of immune elimination and tumorigenesis, which are issues in cell therapy.

By utilizing its extensive experience in the gene/cell therapy and regenerative medicine area to provide new treatment options for intractable diseases with high unmet medical needs, Astellas aims to create products in which high efficacy can be expected using the new science of direct reprogramming.

Assets and capabilities that Astellas expects from its partners

  • Factors that induce in vivo direct reprogramming to cells of interest
  • Viral, non-viral vectors and small molecules that can control the above direct reprogramming
  • Targeting and drug delivery technology that enable the cell/tissue specific direct reprogramming


Bacteriophage is a natural virus that specifically infects target bacteria. It is applied to phage therapy that aims to selectively eliminate bacteria from the living body by using bactericidal actions, the primary properties of a virus. Due to the advancement of genetic engineering technology, progress is being made in recent years in the research of enhancing the usefulness and safety of engineered bacteriophage, and it is attracting attention as the next-generation phage therapy technology.

This technology is expected to be useful not only for the treatment of common bacterial infections, but also as a new treatment for difficult-to-treat bacterial infections and for solving the problem of antimicrobial resistance, a serious global threat to public health.

With the intention of providing new treatment options, Astellas aims to create products in which high efficacy against infectious diseases and microbiome-related diseases—diseases with high unmet medical needs—can be expected by utilizing its expertise in the field of infectious diseases and the new technology of next generation phage therapy.

Assets and capabilities that Astellas expects from its partners

  • Information on pathogenic bacteria as new targets of drug discovery for the treatment of microbiome-related diseases
  • Animal model for microbiome-related diseases
  • Genome editing and modification technology in bacteriophage
  • Unique library of bacteriophage applicable to phage therapy


Innate Immunity (Innate Lymphoid Cells)

Innate Lymphoid Cells (ILCs) are newly discovered cells of the immune system. Compared to other immune cells, ILCs have superior effector functions and can produce large amounts of cytokines to induce inflammation. On the other hand, they are also known to induce immune tolerance.

If agents that regulate the function of ILCs are found, they are expected to lead to the avoidance of side effects associated with the use of immunosuppressants in addition to the effects that could not be achieved by conventional immunoregulation targeting known immune cells.

With the intention of providing new treatment options, Astellas aims to create products in which high efficacy against autoimmune, allergic diseases, cancer, etc.—diseases with high unmet medical needs—can be expected by utilizing its extensive experience in the field of immunity and the new biology of ILCs.

Assets and capabilities that Astellas expects from its partners

  • Novel mechanisms and target molecules to regulate ILCs, including selective activation/inhibition, tolerance induction through ILCs, and control of differentiation/plasticity of ILCs
  • Antibodies and small molecules etc. that can selectively modulate and control such targets and ILCs



Microglia are the only immunocompetent cells in the brain and spinal cord that help keep the brain functioning normally by removing dead cells and waste and protecting neural circuits. Excessive activation of microglia by neuropathy, stress, insomnia, infection and aging, etc. induce inflammation in the brain, causing the onset and exacerbation of various neuroimmune diseases such as neurodegenerative diseases, psychiatric disorders, and autoimmune diseases.

In recent years, the role of innate immune cells in central and peripheral nerve functions has attracted attention, and the regulation of microglia function, in particular, is expected to have a new therapeutic effect on a wide range of neuroinflammation-related diseases from neurodegenerative diseases to autoimmune diseases.

If agents that control the function of microglia are found, it is expected to avoid the side effects commonly seen with drugs that act directly on the nerves, in addition to the effects that could not be achieved by conventional regulation of nerve function targeting neuron directly.

With the intention of providing new treatment options, Astellas aims to create products in which high efficacy against neurodegenerative diseases, psychiatric diseases, autoimmune diseases, etc.—diseases with high unmet medical needs—can be expected by utilizing its expertise in microglia and its extensive experience in the field of central nervous system and immunoinflammatory areas.

Assets and capabilities that Astellas expects from its partners

  • Novel mechanisms and target molecules to regulate function and phenotype in disease specific microglia
  • Small molecules and antibodies that modulate such targets/mechanisms and biomarkers that monitor the effects


Human Genetics

The decrease in the probability of drug discovery success and the increase of R & D-related costs have become major issues for the pharmaceutical industry, and appropriate solutions are strongly demanded. In recent years, it has been found that the probability of success can be improved by advancing drug discovery with human genetic evidence, and the importance of human genetics has been reaffirmed. With the tremendous progress of human genome sequence analysis technology represented by next-generation sequencers, the spread of electronic medical records, and innovations in information technology, it is believed that future drug discovery will be changed significantly due to the great improvement of our understanding of human diseases such as the relationship between gene function and human diseases, causes of diseases, and useful factors for prevention.

For example, in monogenic diseases, more causal genes have been identified, and new therapeutic approaches such as gene therapy are being actively researched. In addition, in the case of more general diseases, so-called "common diseases," the understanding of the mechanism of disease and identification of drug discovery target molecules are being accelerated. Furthermore, human genetics is expected to contribute not only to the research and development of therapeutics, but also to preventive medicine aimed to suppress the onset of diseases.

Astellas is promoting the identification of causal genes of monogenetic disorders through collaboration with domestic and overseas medical institutions / research institutes that promote cutting-edge human genetics research and it is also conducting research that will lead to the treatment of common diseases by making effective use of collaboration with biobanks in the relative countries. Astellas aims to provide the value of a new treatment option to many patients by integrating these findings and utilizing its extensive experience, know-how, and technology in drug discovery.

Assets and capabilities that Astellas expects from its partners

  • Target molecules that are identified by leveraging human genetics
  • Drug candidates that regulate such a target


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