RFP Open: Prime Gene Editing Strategies or Cell Therapy
We are pleased to announce a Request for Proposal (RFP) for high-impact research on prime gene editing strategies or cell therapy to address the increase in SMAD4 activity.
Proposals will be accepted for Team Science Awards, which are defined as collaborative research amongst investigators with experience in gene-editing strategies or cell therapy in or outside the Myhre syndrome field.
We expect to provide up to $450,000 over three years to undertake projects that have the clear potential to lead to gene therapies or cell therapy for Myhre syndrome patients.
Please click here for the full RFP.
Key Dates*:
July 19, 2024 - LOI submission.
July 22, 2024 - Selected LOIs are invited to submit full-length proposals.
Oct 1, 2024 – Full proposals due.
Nov 12, 2024 - Peer and organizational reviews/modifications of submissions due.
Nov 26, 2024 - Award letters/negotiate research agreements.
Dec 10, 2024 – Projects commence.
*Subject to change if an extended review period is needed.
Please submit Letters of Intent or any questions regarding the RFP to kwears@myhresyndrome.org
Additional information for collaborative RFP Letters of Intent - Areas of focus
1. Gene therapy (preferentially prime or sb gene editing, but open to other proposals).
Describe your strategy should include but not limited to:
General strategy, cell lines created or used for peg/ PE/ng RNA screening or equivalent in your strategy; new improved Cas, TAM/ PAM strategy, how your design strategy will target mut alleles vs WT SMAD4, differentiate editing frequency.
3,000 characters max
2. IPSC differentiation, characterization, and comparison treated/ healthy control.
The patient-derived IPSCs (ILe500VAl and Arg496Cys) will be provided by the Myhre Syndrome Foundation on day 1 of the project.
iPSC should be differentiated into MS-relevant cell types, including but not limited to neurons and chondrocytes.
Describe the characterization and comparison: which functional assays will be used.
1500 characters max
3. In vivo delivery strategy
Describe the delivery modality, the engineering, the capacity to produce it, the cargo localization, and loading evaluation methods.
1500 characters max
4. In vivo validation for proof of concept
Describe the animal model, the mode of injection, the assay(s) / outcome measures used for the POC.
Optional: the preliminary tropism study.
1500 characters max
Grant Overhead Policy
Myhre Syndrome Foundation will not support indirect costs, overhead costs, or other similar institutional levies in excess of 5% of the total award amount. Fringe benefits for personnel salaries are allowable.