Drs. Graham Dempsey, Noelle Germain, and James Fink of Quiver Biosciences

Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome

FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.

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Fragile X Research Update: A Turning Point for Treatments and Curative Approaches

Fragile X research is at a turning point. FRAXA is funding ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.

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Dr. Joel Richter and Dr. Sneha Shah in their UMass Chan Medical School lab researching ASO therapy for Fragile X syndrome.

ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits

A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein.

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2025 FRAXA Research Grants Webinar with Dr. Michael Tranfaglia and Katie Clapp

Fragile X Syndrome Research in 2025: FRAXA Announces New Grants & Promising Leads

Fragile X syndrome research is advancing in 2025 with new FRAXA-funded grants supporting breakthrough science, therapy platforms, and clinical tools.

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UMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment

UMass Chan Medical School licenses RNA-based Fragile X treatment approach using ASOs to QurAlis, moving gene-targeted therapy closer to clinical trials.

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FRAXA Funded Research

Current Research Grants (44)