Announced details
In the CLN1 model, the lifespan of the JUST-AAV-PPT1-treated group was nearly equivalent to that of healthy mice
In the CLN1 model, motor function at 54 weeks in the JUST-AAV-PPT1-treated group was equivalent to that of healthy mice
The survival period of the conventional AAV9-PPT1-treated group was 53–65 weeks
All animals in the disease control group and the recombinant PPT1-treated group died at 31–41 weeks after birth
Alexion AstraZeneca Rare Disease is one of JCR's partners, and the companies have partnership programs utilizing JUST-AAV technology. The Jikei University School of Medicine was the presenting entity; its capital, personnel and business relationships are Not disclosed.
The American Society of Gene and Cell Therapy (ASGCT) is a professional organization for specialists in gene and cell therapy. Researchers from The Jikei University School of Medicine presented research on GM1 gangliosidosis. The location, representative, capital, year established and business activities of Alexion AstraZeneca Rare Disease are Not disclosed.
In this announcement the company addresses none of Contract Value / Offering price / Impact on Business Performance.
American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting
| Item | Details |
|---|---|
| Names of Products / Services Covered | JUST-AAV, J-Brain Cargo® |
| Counterparty | Alexion, AstraZeneca Rare Disease, The Jikei University School of Medicine |
| Counterparty overview | The American Society of Gene and Cell Therapy (ASGCT) is a professional organization for specialists in gene and cell therapy. Researchers from The Jikei University School of Medicine presented research on GM1 gangliosidosis. The location, representative, capital, year established and business activities of Alexion AstraZeneca Rare Disease are Not disclosed. |
| Relationship with the Company | Alexion AstraZeneca Rare Disease is one of JCR's partners, and the companies have partnership programs utilizing JUST-AAV technology. The Jikei University School of Medicine was the presenting entity; its capital, personnel and business relationships are Not disclosed. |
| Applicable area | GM1 gangliosidosis, Neuronal ceroid lipofuscinosis (CLN1 and CLN2), Lysosomal diseases, Central nervous system (CNS) diseases, Gene Therapy |
| Quantitative Effects | In the CLN1 model, the lifespan of the JUST-AAV-PPT1-treated group was nearly equivalent to that of healthy mice, In the CLN1 model, motor function at 54 weeks in the JUST-AAV-PPT1-treated group was equivalent to that of healthy mice, The survival period of the conventional AAV9-PPT1-treated group was 53–65 weeks, All animals in the disease control group and the recombinant PPT1-treated group died at 31–41 weeks after birth |
| Source | American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting |
Items this document does not state (11)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.The objectives are to improve gene delivery efficiency to the central nervous system (CNS), reduce accumulation in the liver, improve the safety and efficacy of gene therapy, and address unmet medical needs, including those relating to CNS diseases.