Johns Hopkins University mRNA Amplifier Research Grant

Award: $130,000 grant to Johns Hopkins University School of Medicine

Funder: SynGAP Research Fund (SRF), Wandoan Campdraft, Soley for SYNGAP1, and Leon & Friends (Austrian SYNGAP1 patient group)

Principal Investigator: Jeff Coller, PhD, Bloomberg Distinguished Professor of RNA Biology and Therapeutics at Johns Hopkins University School of Medicine

Project Details

Technology: mRNA Amplifier - a novel therapeutic candidate for SYNGAP1 haploinsufficiency

Mechanism: Enhances expression of the normal, unaffected SYNGAP1 gene by increasing its messenger RNA's potency. The mRNA Amplifier binds to SYNGAP1 mRNA to enhance the normal transcript's translation, demonstrating ability to double protein output in preliminary studies.

Key Characteristics:

• Mutation agnostic (could help patients regardless of their SYNGAP1 variant)
• Potentially applicable to other haploinsufficiency disorders
• Based on messenger RNA biology

Project Timeline and Activities

Year 1: Critical in vitro development of the mRNA Amplifier in treatment of SYNGAP1 haploinsufficiency, including cytotoxicity assessments

Year 2: Proof-of-concept experiments using animal models

Background

In early vitro studies, the technology demonstrated the ability to enhance expression of the normal, unaffected gene by increasing its messenger RNA's potency. Preliminary work shows the mRNA Amplifier can double protein output.