BOSTON--(BUSINESS WIRE)--SOLA Biosciences, a pioneer in innovative chaperone technology, will present its encouraging preclinical proof-of-concept data for SOL-257, a targeted gene therapy for misfolded TDP-43 in ALS, at the 22nd Annual Northeast ALS Consortium (NEALS) Meeting held in Florida on October 5.
TDP-43 is a nuclear protein that plays a vital role in DNA and RNA processing. 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 cytoplasmic 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 acts as a co-chaperone alongside HSP70, a vital cellular chaperone. The uniquely designed fusion protein efficiently presents misfolded TDP-43 to HSP70, promoting either the re-folding of TDP-43 into its correct conformation or facilitating its degradation.
The in vivo proof-of-concept studies demonstrated the effectiveness of SOL-257 gene therapy in mitigating TDP-43-related toxicity in both the TDP-43-based mouse model and the C9orf72-based ALS mouse model. The compelling results from various behavioral tests provide strong support for the continued preclinical development of SOL-257 as a potential ALS therapeutic.
"Our findings underscore SOL-257 as a promising transformational therapy in ALS," expressed Dr. Akinori Hishiya, Chief Scientific Officer at SOLA. “Demonstrating efficacy in two different genetic mouse models of ALS supports our belief that by targeting misfolded TDP-43, SOL-257 has the potential to treat nearly all patients with ALS.”
Dr. Gerry Cox, Chief Medical Officer at SOLA, added, "The robust in vivo proof-of-concept data makes me excited to complete the preclinical studies necessary to take SOL-257 into ALS clinical trials. The unmet medical need in ALS remains high, despite the current therapeutic landscape, and the prospect of a broadly applicable, one-time therapy brings new hope to the ALS community."
About SOLA Biosciences, LLC:
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.
