Therapeutic Approaches: Gene and Cell Therapy Strategies for Myhre Syndrome

The objective of this RFP is to identify promising gene and cell therapies that have the potential to dramatically reduce pathologic burden in MS patients. Potential areas of interest include:

• Gene Editing

MS-identified pathogenic variants are limited to missense single nucleotide mutations SMAD4 I500V/T/M/L and R496C. MS is autosomal dominant, usually de novo, with a gain of function. Haploinsufficiency and loss of function of SMAD4 are highly pathogenic and push for a gene-editing therapeutic approach. Proposals using prime gene editing strategies would be preferred.

• Fibrosis Cell/Gene therapy

Fibroproliferative reactivity is one of the threats and burdens of MS patients, and efficient therapeutic options in anti-fibrosis remain a major challenge for the treatment of MS. Proposals aimed at developing innovative therapeutics, other than the use of small molecules capable of meeting these challenges are of significant interest.

Funding: $450,000 total 
Duration: 3 years 

Research Authority due date: 24.6.24

LOI (required) due date: 1.7.24

Full proposal due date: 2.9.24