Shanghai, Philadelphia, November 7, 2023 - Genemagic Biosciences, is pleased to announce the selection of its first pre-clinical candidate, GM101-PD, marking its official entry into the clinical trial preparation phase. This compound, having undergone successful third-party CRO independent replication and two-photon validation, represents a groundbreaking AAV-based gene therapy designed to deliver a reprogramming factor, utilizing a non-genome editing approach, to striatal astrocytes and directly convert them into dopamine neurons in situ, with the aim of achieving a reversal of Parkinson's disease.


GM101-PD in situ cell reprogramming effect has been successfully independently validated by a third-party CRO


The selection of this candidate compound was based on multiple validation approaches, involving third-party CRO-conducted blinded, multi-dose efficacy testing in a Parkinson's animal model, as well as in-house multi-dose and multi-time-point testing. The third-party results revealed a significant presence of dopamine neurons in the striatum of the experimental group mice, while no such signals were observed in the control group model (it is well-known that there are almost no dopamine neurons in the normal striatum).


GM101-PD in situ cell reprogramming effect has been successfully demonstrated by Two-Photon imaging


Furthermore, the company's founding team recently used high-resolution two-photon microscopy for lineage tracing in mice with highly specific labeling of astrocytes, successfully confirming the efficacy of GM101-PD. The team was able to observe, in real-time and with exceptional clarity, the complete process of clusters of astrocytes directly transdifferentiating into neurons. This result stands as the most critical evidence internationally recognized to substantiate the transformation of astrocytes into neurons in mice. The figure below illustrates the gradual morphological changes in astrocytes under the influence of GM101-PD, ultimately culminating in their transformation into neurons (the axons of newly formed neurons are clearly visible). The relevant experimental results are expected to be submitted for peer review in scientific journals in the second half of 2024.


PTBP1 in situ cell reprogramming effect has been successfully independently validated by a third-party CRO


Finally, Genemagic also announced that a third-party CRO, commissioned by the company, has successfully replicated the experiment conducted in the striatum, targeting PTBP1, to induce the transformation of astrocytes into dopamine neurons in a Parkinson's mouse model. Members of Genemagic's founding team published a paper in the journal "Cell" in 2020, highlighting the potential effectiveness of reprogramming astrocytes into dopamine neurons as a treatment for Parkinson's disease. Due to the complexity of this experiment, some laboratories have faced challenges in replicating the results. To address the issue of reproducibility concerning PTBP1, Genemagic took the proactive step of engaging a third-party CRO to conduct independent replication experiments, which yielded successful independent replication results.


History teaches us that skepticism towards groundbreaking scientific discoveries is inevitable and constitutes an essential component of scientific progress and refinement. We encourage responsible questioning while discouraging hasty dismissal. Genemagic Biosciences upholds a profound respect for science and a sense of responsibility towards patients. We allocate substantial resources for multifaceted validation efforts, with the overarching goal of introducing disruptive therapeutic approaches for profoundly challenging neurodegenerative diseases like Parkinson's.


About Genemagic Biosciences


Genemagic Biosciences is dedicated to the development of regenerative therapies for neurodegenerative and ophthalmic diseases with the aim of providing patients with a one-time treatment capable of reversing their conditions. The company's core technologies encompass in vivo cellular reprogramming and neuroprotection techniques, designed not only to regenerate new neurons to compensate for lost cells but also to safeguard both existing and newly formed neurons for maximum therapeutic effectiveness. The company's most advanced project at present focuses on a regenerative therapy for Parkinson's disease, based on in vivo cellular reprogramming technology, and is currently in the clinical trial preparation phase.


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Genemagic has selected its preclinical candidate for Parkinson’s program and enters into clinical preparation phase

2023-11-06 23:02