Thank you for your interest in Spark's Competitive Research Grant Program in Pompe disease. The deadline for submissions was Monday, May 31st, 2021. The window for submissions is closed and applications are being reviewed.
In 2022, Spark plans to continue the Competitive Research Program in Pompe disease encompassing both basic science and clinical research. In December 2021, information will be updated here and submissions enabled.
Requirements for the 2021 program are reproduced below for your information and planning. However, these are subject to change.
All grant proposals must directly relate to one of the following areas of research focus to advance basic and
clinical science in Pompe disease:
- Mechanisms and enhancement of acid alpha-glucosidase (GAA)
uptake and lysosome trafficking
- Novel clinical biomarkers of Pompe disease (plasma or tissue markers of disease/treatment
efficacy, proteomics, next-generation sequencing, cell-free DNA, microRNA, etc.)
- Understanding central nervous system
involvement in Pompe disease
- Evaluation of novel biomarkers in early and/or mid-stage Pompe disease to inform (a) patient
selection and/or (b) clinical efficacy endpoints (eg- clinical neuromuscular testing, muscle biopsy, advanced imaging of glycogen
in vivo, advanced magnetic resonance [MR] imaging techniques, diaphragmatic ultrasound, MR spectroscopy, etc.)
- Role of autophagy, mitophagy, and mitochondrial function in Pompe disease
- Patient-reported outcomes and quality-of-life evaluations in Pompe disease
- Potential correlation between enzyme replacement therapy (ERT) dose and frequency, GAA level, and anti-GAA antibody level with
immune-related adverse events over time
- Potential mechanisms to determine durability for ERT and/or gene therapy research
- Next-generation clinical therapy concepts
An external panel of Pompe
disease experts will score each completed application based on scientific merit. Projects will be funded considering panel ranking
until funds are exhausted.
In 2020, Spark funded two 2-year research projects:
Multi-omics Biomarker Discovery and Liquid Biopsy in Pompe Disease. Principal Investigator: Antionetta Tarallo, MD
Pompe Disease: From Pathophysiology to New Therapeutic Targets.
Principal Investigator: Giancaarlo Parenti, MD
- This is a global program
- Grants must be 1-2 years in length
- Grant amounts may be up to $125,000/year INCLUSIVE of
overhead costs capped at 25%
- Funding may include support for salaries and direct costs related to the research project
- Funding MAY NOT include:
- Support for a research project already being funded through a different grant program
- Capital equipment, durable medical/scientific equipment
- Travel to professional society meetings to present research
- Fellowship funding or sponsorship
- CME events
- Expenses not associated with the study
- Expenses not specified in the budget submitted as part of the application
- If grant funds remain unspent at the conclusion of the research, they must be returned to Spark Therapeutics
- Proposals will be accepted beginning Friday, 8 January 2021, 09:00 hr EDT
- To register for a portal account to fill out and submit your application:
1. Click the 'Register'
button in the navigation bar at the top of this page
2. Complete the new user registration process
3. Follow the prompts to submit a new proposal.
4. For technical support related to the site, please e-mail SteepRock Client Services Support
APPLICATION SUBMISSION DEADLINE
- Proposals must be received by Monday, 31 May 2021, 23:59 hr EDT
Please contact the Spark Competitive Grants Research Program Pompe Disease at SparkCompetitiveGrantsPompe@sparktx.com with any additional questions.