Title:
Selection and validation of the targeted population and clinical biomarkers to support Phase I/II trials and long-term monitoring of patients treated with the LPLD gene therapy
Agreement Value:
$215,193.00
Agreement Date:
Jul 21, 2022 - Dec 31, 2025
Description:
The current project aims at obtaining relevant data to select the targeted population and clinical biomarkers for the long-term monitoring of efficacy of LPLD gene therapy to support Phase I/II clinical trial design to be proposed to Health Canada at a pre-CTA meeting Definition and validation of the study population and of the main inclusion and exclusion criteria will be performed using data obtained from discussions with Canadian/international experts and from the documentation of the natural history of LPL deficiency, including epigenetic, gene expression and sequencing analyses. The natural history of LPL deficiency, including a panel of critical biomarkers, will become a ‘baseline’ against which to benchmark long-term efficacy of LPLD gene therapy. The analysis and selection of clinical biomarkers will leverage the ongoing research program on the natural history of severe hypertriglyceridemia (SMASH: Systems and Molecular Approaches of Severe Hyperlipidemias) (Table 1). In parallel, the same panel of biomarkers will be monitored in preclinical long-term safety and efficacy study of the lead LPLD gene therapy construct in LPL-/- transgenic mice conducted by UBC. This will provide pre-clinical baseline of potential effect of gene therapy treatment on selected biomarkers representative of the ‘natural’ history of disease in humans. ( Figure 1). Studies above will allow to identify safety and efficacy assessment criteria that will be used in the clinical trial, including the definition of the primary and secondary objectives, as well as to identify all potential concurrent and companion treatments. Finally, socioeconomic study of chylomicronemia and pharmacoeconomic analyses will be performed to position this new gene therapy in relation to the competitive environment and the burden of LPL deficiency
Organization:
National Research Council Canada
Expected Results:
In the short term, anticipated outcomes will be strengthened collaborations across industry, academia, and government to support research excellence. In the medium term, anticipated outcomes will be the development of new and potentially disruptive technologies with collaborators. In the long term, find collaborative solutions to public policy challenges and create stronger innovation systems.
Location:
Saguenay, Quebec, CA G7H 7K9
Reference Number:
172-2022-2023-Q2-992766
Report Type:
Grants and Contributions
Recipient Business Number:
896148004
Recipient Type:
Not-for-profit organization or charity
Additional Information:
This agreement has been amended 1 time(s) the total amended value is $215,193.The end date of this agreement has been modified by 975 days.
Amendment Date
Mar 3, 2024
Recipient's Legal Name:
Ecogene 21
Federal Riding Name:
Chicoutimi–Le Fjord
Federal Riding Number:
24022
Program:
Collaborative Science, Technology and Innovation Program - Collaborative R&D Initiatives
Program Purpose:
Collaborate on multiparty research and development programs to catalyze transformative, high-risk, high-reward research with the potential for game-changing scientific discoveries and technological breakthroughs in priority areas.
NAICS Code:
541710 - R&D in the physical, engineering and life sciences