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Gene therapy research grant information

Calliope Joy Foundation

Request for Proposal

August 2017

Background

Leukodystrophies are a group of disorders affecting the brain’s white matter (or myelin). The word comes from "leuko" (Greek for white) and "dystrophy" meaning "imperfect growth." Therefore, leukodystrophies are characterized by imperfect development of the brain's white matter. Children with leukodystrophy appear healthy and achieve their milestones but then lose the ability to crawl, walk, swallow, speak, eat, smile, and laugh. There are more than 30 forms of the disease, and 1 in 7400 live births are affected.

In most cases, once children develop symptoms they cannot be treated since the damage to the brain and central nervous system are irreversible. Newborn screening will be a crucial tool for finding children with leukodystrophy at birth since treatments can only work before the symptoms appear. Revolutionary gene therapies being pioneered by researchers in France and Italy will do more than cure children, they will ensure children never become sick.

Lysosomal Storage Diseases are a group of approximately 50 rare inherited metabolic disorders that are caused by defects in the function of the lysosome. Usually the defects in lysosomal function are caused by mutations in single enzymes that are responsible for the metabolism of lipids, glycoproteins, or mucopolysaccharides. Lysosomal Storage Diseases affect mostly children as those individuals affected usually die at young ages. Innovative approaches to treating these diseases include enzyme replacement therapy and gene therapy.

Request for Proposal: Gene therapy approaches to leukodystrophy or lysosomal storage disease treatment

The Calliope Joy Foundation seeks to support research focused on developing gene therapy approaches to treat leukodystrophies and lysosomal storage diseases.

Proposal Details

Those eligible to submit proposals include investigators from biotechnology/pharmaceutical companies, other for-profit entities, public and private nonprofit universities, colleges, hospitals, laboratories, and government agencies, irrespective of the country of origin.

A Letter of Intent (LOI) is required for all grant applications and must be submitted as one PDF file by November 1, 2017 containing the following information:

1) A brief description of the objectives and rationale for the project (one page); 2) Key preliminary data (up to 4 pages); 3) An estimate of the budget; 4) CV/biosketch for Principal Investigator (including contact information). Submit this PDF file via email to grants@thecalliopejoyfoundation.org. The subject line of the email should read as follows, LOI Calliope Joy Foundation RFP. The LOIs will be reviewed and, based on the outcome of this review, the submitting investigators will be either invited to submit a full proposal or informed that the LOI has been declined. This notification should be made within six weeks of the LOI deadline.