QurAlis Announces Exclusive License for FMR1-217 to Target Fragile X Syndrome
Deal News | May 15, 2025 | SV Health Investors, LLC
QurAlis Corporation, a biotechnology company specializing in neurodegenerative and neurological diseases, has finalized an exclusive license agreement with UMass Chan Medical School for a pioneering RNA-targeted mechanism that promises to restore functional protein in Fragile X Syndrome (FXS). The agreement stems from a collaboration initiated in 2024, exploring antisense oligonucleotide (ASO)-mediated correction for FXS by targeting mis-spliced FMR1 mRNA, specifically the variant FMR1-217. QurAlis has leveraged its expertise and the FlexASO platform to advance the development of a precision medicine targeting FMR1, with preliminary data suggesting the feasibility of detecting FMR1 mis-splicing through biomarkers. The collaboration with UMass Chan, led by esteemed neuroscientists, has reached a significant milestone, underscoring the potential of RNA-targeted therapies in addressing the underlying genetic causes of FXS, a condition currently lacking effective disease-modifying treatments.
Sectors
- Biotechnology
- Healthcare
Geography
- United States – QurAlis is based in Cambridge, Massachusetts, and the collaboration involves UMass Chan Medical School and Rush University Medical Center, all located in the United States.
Industry
- Biotechnology – The article discusses a biotechnology company, QurAlis Corporation, involved in the development of precision medicines for neurodegenerative and neurological diseases, specifically targeting Fragile X Syndrome.
- Healthcare – The focus on developing potential therapies for Fragile X Syndrome—a neurodevelopmental disorder—places this article within the sphere of healthcare innovations.
Financials
- – No financial terms of the exclusive license agreement were disclosed in the article.
Participants
| Name | Role | Type | Description |
|---|---|---|---|
| QurAlis Corporation | Biotechnology Company | Company | A clinical-stage biotechnology company focused on developing precision medicines for neurodegenerative and neurological diseases. |
| UMass Chan Medical School | Research Partner | Institution | A medical school affiliated with the University of Massachusetts that partnered with QurAlis to explore FXS therapies. |
| SV Health Investors, LLC | Private Equity Firm | Company | A private equity firm supporting QurAlis in their advancements in RNA-targeted therapies. |
| Kasper Roet, Ph.D. | CEO and Co-founder | Person | Chief Executive Officer of QurAlis Corporation, overseeing the development of RNA-targeted therapies for FXS. |
| Joel Richter, Ph.D. | Researcher | Person | Professor of molecular medicine at UMass Chan involved in validating FMR1 as a target for FXS therapy. |
| Elizabeth Berry-Kravis, M.D., Ph.D. | Researcher | Person | Researcher at Rush University Medical Center known for her work in FXS therapy development. |