CAMBRIDGE, Mass. — QurAlis Corporation has secured an exclusive license from UMass Chan Medical School to develop a potential first-in-class, disease-modifying therapy for Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and a leading genetic trigger for autism.
The agreement centers on a novel RNA-targeted mechanism that restores the expression of functional Fragile X Messenger Ribonucleoprotein (FMRP), the protein deficient in individuals with FXS. QurAlis will advance FMR1-217, a mis-spliced form of the FMR1 gene identified as a key therapeutic target in up to 80% of FXS patients. This collaboration builds on earlier research confirming that aberrant splicing of FMR1 mRNA disrupts protein expression, leading to the condition’s hallmark cognitive and behavioral symptoms.
QurAlis’ FlexASO® platform—an antisense oligonucleotide (ASO) technology designed for high precision in splicing correction—will be used to develop a candidate therapy. Preliminary data suggest that FMR1-217 can be reliably measured in both blood and cerebrospinal fluid, offering a potential biomarker for patient selection and treatment monitoring.
“FXS is a devastating neurodevelopmental condition with no approved disease-modifying therapies,” said Dr. Kasper Roet, CEO and co-founder of QurAlis. “Our partnership with UMass Chan confirms the therapeutic promise of correcting mis-splicing in FMR1, and we are now focused on moving toward IND-enabling studies to bring a precision medicine approach to patients.”
The foundation for this development was laid by research led by Dr. Joel Richter at UMass Chan, along with collaborators from Rush University Medical Center. Their work demonstrated that in FXS patients, the FMR1 gene still expresses RNA, but that RNA is incorrectly spliced into a truncated form—FMR1-217—that cannot produce functional FMRP. ASOs designed to inhibit this mis-splicing were able to restore proper mRNA and protein levels in patient-derived cells.
“This is a vital step in translating basic science into real treatments,” said Dr. Richter. “Our collaboration with QurAlis has validated FMR1-217 as a promising target, and we’re hopeful this will result in an effective therapy for families affected by FXS.”
Dr. Elizabeth Berry-Kravis, a renowned expert in Fragile X research at Rush University, added, “I’m thrilled that this potential therapy, grounded in genetic correction, could significantly impact the treatment landscape for FXS.”
FXS affects an estimated 87,000 people in the U.S., occurring in about 1 in 4,000 males and 1 in 6,000 females. Males are generally more severely affected. In addition to intellectual disability, patients often suffer from severe anxiety, sensory issues, attention deficits, seizures, and aggressive behavior. Despite decades of research, no approved disease-modifying treatments currently exist.
QurAlis is now working to nominate a development candidate for preclinical studies, with the aim of entering clinical trials in the near future.
