The World's First Peptide Protein Degrading Agent PDR-001 Officially Launches IIT Clinical Research

Proteins are the executors of life activities, but when certain proteins accumulate abnormally or become dysfunctional, it can lead to the occurrence of diseases. Traditional drugs mainly adopt a "occupancy driven" mode, regulating their function by binding to target proteins.
This model has obvious limitations: many pathogenic proteins lack suitable binding pockets; Drug occupancy may not be sufficient to completely inhibit protein function; Easy to develop drug resistance.
The protein degradation targeted chimeric technology has opened up a new "event driven" mode, directly labeling pathogenic proteins and delivering them to the cell's protein degradation system - proteasome - for clearance.
PROTAC technology can enable the development of drugs targeting traditional "untreatable" targets, improve selectivity, reduce drug resistance, and bring breakthrough treatment opportunities for difficult to treat diseases such as cancer and neurodegenerative diseases.

What is PDR-001?

The biggest innovation that sets PDR-001 apart from traditional small molecule protein degrading agents is its peptide structure design. Peptides are short chains composed of amino acids, located in the middle zone between small molecule drugs and biomacromolecule drugs.
Compared with small molecule degraders based on PROTAC technology, PDR-001 adopts a peptide structure, which gives it higher target selectivity and binding affinity. Peptides can form a wider range of interactions than small molecules, thereby targeting protein targets that are traditionally considered "untreatable".
PDR-001 utilizes the natural ubiquitin proteasome system in cells. The two ends of its molecule are designed as a "target head" that binds to the target protein and a "ligand" that binds to E3 ubiquitin ligase, respectively, and are connected in the middle by a linker of appropriate length.
When PDR-001 binds to both the target protein and E3 ubiquitin ligase, it will label the ubiquitin chain on the target protein. The labeled protein will be recognized and degraded by the proteasome of the cell. Importantly, PDR-001 can be reused after completing the degradation task, achieving catalytic degradation.
According to researchers, the first clinical study of PDR-001 will primarily target solid tumor patients, especially those who have exhausted standard treatment options or developed resistance to existing treatments in advanced stages.
The first phase clinical trial mainly evaluates safety, tolerability, pharmacokinetics, and preliminary efficacy, using a "3+3" dose escalation design to determine the maximum tolerated dose and the recommended dose for the second phase.
Protein degradation therapy, due to its unique mechanism of action, has the potential to overcome drug resistance issues in various cancers. For example, in the treatment of cancer types driven by specific mutations but prone to drug resistance, directly degrading pathogenic proteins may be more durable and effective than inhibiting their function.
In addition to the field of cancer, the peptide protein degradation technology platform represented by PDR-001 powder can also be extended to the field of neurodegenerative diseases. Alzheimer's disease, Parkinson's disease, etc. are closely related to the accumulation of abnormal proteins.

What are its advantages?

PDR-001 API powder is the world's first Peptide based Degrader to enter the human clinical stage, utilizing an innovative three-stage peptide degradation modality design. The drug efficiently crosses the blood-brain barrier and cell membrane through the TAT transmembrane region, targeting the peptide region to achieve highly specific recognition of alpha synuclein. The degradation region is designed with a Degron signaling peptide based on the N/C-end rule pathway, directly targeting and clearing the most critical pathogenic protein alpha synuclein in the pathogenesis of Parkinson's disease.Our email address is: sales6@faithfulbio.com.Welcome to our store to inquire about other products of our company.
Unlike traditional Parkinson's disease drugs that mainly improve motor symptoms by supplementing dopamine, the design of PDR-001 targets the pathological core - abnormally aggregated alpha synuclein. By using AAV vectors with specific peptide sequences through local application, infected cells become "cell factories" that continuously express peptides, solving the problem of short half-life of peptide drugs and achieving long-term expression with a single use. Local use of doses 1-2 orders of magnitude lower than systemic administration can significantly reduce AAV related liver toxicity and immunogenicity risks.

What is the focus of this study?

This study aims to investigate the safety, tolerability, and efficacy of PDR-001 stereotactic bilateral basal ganglia clearance of alpha synuclein. The first patient enrollment has been successfully completed. At the kickoff meeting, the project team representatives had a systematic discussion with the pharmaceutical company on key elements such as drug mechanism, inclusion and exclusion criteria, trial process, surgical pathway, and subject protection, laying a solid foundation for high-quality promotion of the trial. Currently, patient recruitment work has been successfully launched in Ruijin.

Why choose our company?

Xi'an Faithful BioTech Co., Ltd are a biotechnology enterprise dedicated to exploring new mechanisms of protein degradation and innovating drug forms. The company has established the Prodegre Atlas innovative protein degradation/interaction platform and a deeply integrated translational medical biotechnology development system. Starting from the underlying degradation mechanism, with target driven as the core, differentiated protein degradation agents are developed for the central nervous system and inflammation fields. Despite the long road ahead, the launch of PDR-001 clinical research undoubtedly marks a solid step forward for Chinese innovative pharmaceutical companies towards the ultimate goal of "treating the cause", bringing new hope to Parkinson's disease patients worldwide. If you wish to proceed further, please contact us via email.

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