Central Nervous System Targeted Protein Degraders
Diseases of the central nervous system, which once occupied a large component of the pharmaceutical industry research and development portfolio, have for many years played a smaller part in major pharma pipelines—primarily due to the well cited challenges in target validation, valid translational mo...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-07-01
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| Series: | Biomolecules |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2218-273X/13/8/1164 |
| _version_ | 1797585395089670144 |
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| author | Bedwyr ab Ion Thomas H. Lois Lewis D. Heulyn Jones Simon E. Ward |
| author_facet | Bedwyr ab Ion Thomas H. Lois Lewis D. Heulyn Jones Simon E. Ward |
| author_sort | Bedwyr ab Ion Thomas |
| collection | DOAJ |
| description | Diseases of the central nervous system, which once occupied a large component of the pharmaceutical industry research and development portfolio, have for many years played a smaller part in major pharma pipelines—primarily due to the well cited challenges in target validation, valid translational models, and clinical trial design. Unfortunately, this decline in research and development interest has occurred in tandem with an increase in the medical need—in part driven by the success in treating other chronic diseases, which then results in a greater overall longevity along with a higher prevalence of diseases associated with ageing. The lead modality for drug agents targeting the brain remains the traditionally small molecule, despite potential in gene-based therapies and antibodies, particularly in the hugely anticipated anti-amyloid field, clearly driven by the additional challenge of effective distribution to the relevant brain compartments. However, in recognition of the growing disease burden, advanced therapies are being developed in tandem with improved delivery options. Hence, methodologies which were initially restricted to systemic indications are now being actively explored for a range of CNS diseases—an important class of which include the protein degradation technologies. |
| first_indexed | 2024-03-11T00:05:31Z |
| format | Article |
| id | doaj.art-85bb439cc9d247d9b862d23a047fe66d |
| institution | Directory Open Access Journal |
| issn | 2218-273X |
| language | English |
| last_indexed | 2024-03-11T00:05:31Z |
| publishDate | 2023-07-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomolecules |
| spelling | doaj.art-85bb439cc9d247d9b862d23a047fe66d2023-11-19T00:23:07ZengMDPI AGBiomolecules2218-273X2023-07-01138116410.3390/biom13081164Central Nervous System Targeted Protein DegradersBedwyr ab Ion Thomas0H. Lois Lewis1D. Heulyn Jones2Simon E. Ward3Medicines Discovery Institute, Cardiff University, Cardiff CF10 3AT, UKMedicines Discovery Institute, Cardiff University, Cardiff CF10 3AT, UKMedicines Discovery Institute, Cardiff University, Cardiff CF10 3AT, UKMedicines Discovery Institute, Cardiff University, Cardiff CF10 3AT, UKDiseases of the central nervous system, which once occupied a large component of the pharmaceutical industry research and development portfolio, have for many years played a smaller part in major pharma pipelines—primarily due to the well cited challenges in target validation, valid translational models, and clinical trial design. Unfortunately, this decline in research and development interest has occurred in tandem with an increase in the medical need—in part driven by the success in treating other chronic diseases, which then results in a greater overall longevity along with a higher prevalence of diseases associated with ageing. The lead modality for drug agents targeting the brain remains the traditionally small molecule, despite potential in gene-based therapies and antibodies, particularly in the hugely anticipated anti-amyloid field, clearly driven by the additional challenge of effective distribution to the relevant brain compartments. However, in recognition of the growing disease burden, advanced therapies are being developed in tandem with improved delivery options. Hence, methodologies which were initially restricted to systemic indications are now being actively explored for a range of CNS diseases—an important class of which include the protein degradation technologies.https://www.mdpi.com/2218-273X/13/8/1164PROTACstargeted protein degradersCNS |
| spellingShingle | Bedwyr ab Ion Thomas H. Lois Lewis D. Heulyn Jones Simon E. Ward Central Nervous System Targeted Protein Degraders Biomolecules PROTACs targeted protein degraders CNS |
| title | Central Nervous System Targeted Protein Degraders |
| title_full | Central Nervous System Targeted Protein Degraders |
| title_fullStr | Central Nervous System Targeted Protein Degraders |
| title_full_unstemmed | Central Nervous System Targeted Protein Degraders |
| title_short | Central Nervous System Targeted Protein Degraders |
| title_sort | central nervous system targeted protein degraders |
| topic | PROTACs targeted protein degraders CNS |
| url | https://www.mdpi.com/2218-273X/13/8/1164 |
| work_keys_str_mv | AT bedwyrabionthomas centralnervoussystemtargetedproteindegraders AT hloislewis centralnervoussystemtargetedproteindegraders AT dheulynjones centralnervoussystemtargetedproteindegraders AT simoneward centralnervoussystemtargetedproteindegraders |