SOLA Biosciences Presents Preclinical Proof-of-Concept Data for SOL-257, a Gene Therapy Targeting Misfolded TDP-43 in ALS, at Neuroscience 2023 and ALS One Symposium

TDP-43 (TAR DNA-binding protein 43) is a multifunctional protein primarily localized in the cell nucleus. It plays a crucial role in regulating RNA processes, including transcription, splicing, and stabilization. However, in 97% of ALS cases, TDP-43 becomes mislocalized to the cytoplasm. Both the loss of nuclear TDP-43 function and the toxic gain of function caused by TDP-43 aggregates are believed to contribute to the underlying pathophysiology of neurodegeneration in ALS.

SOL-257 is an AAV gene therapy that expresses a therapeutic fusion protein. This fusion protein serves as a co-chaperone, working in conjunction with HSP70, a crucial cellular chaperone. The fusion protein’s unique design enables it to effectively present misfolded TDP-43 to HSP70, promoting either the re-folding of TDP-43 into its correct conformation or facilitating its degradation.

In vivo proof-of-concept studies have showcased the effectiveness of SOL-257 gene therapy in alleviating TDP-43-related toxicity in both the TDP-43-based mouse model and the C9orf72-based ALS mouse model. The compelling results from a range of behavioral tests provide strong support for the ongoing preclinical development of SOL-257 as a potential ALS therapeutic.

“Our findings underscore SOL-257 as a highly promising translational therapy for ALS,” stated Dr. Akinori Hishiya, Chief Scientific Officer at SOLA. “Demonstrating efficacy in two distinct genetic mouse models of ALS supports our confidence that by targeting misfolded TDP-43, SOL-257 holds the potential to treat a vast majority of ALS patients.”

“As SOLA Biosciences’ CEO, I’m thrilled to announce SOL-257, a breakthrough gene therapy poised to tackle ALS by targeting TDP-43 pathology,” Dr. Keizo Koya added, “Our compelling preclinical data highlights a significant step toward harnessing the body’s cellular defenses. Committed to excellence, we are advancing this promising therapy, striving to bring hope to the ALS community.”

About SOLA Biosciences, Inc.:

Leading the frontier in chaperone technology, SOLA Biosciences is the developer of the revolutionary JUMP70 platform technology, designed to selectively eliminate disease-causing misfolded proteins using the patients’ own chaperones. SOLA’s portfolio boasts nine gene therapy programs utilizing JUMP70, aimed at tackling formidable conformational diseases like ALS, Huntington’s, Parkinson’s, and Alzheimer’s diseases.

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