SOL-257 / SOLA Biosci - LARVOL DELTA

Novel chaperone-based gene therapy for ALS (Neuroscience 2024) - "This in vivo proof of concept study demonstrated that SOL-257 gene therapy effectively prevents TDP-43-related toxicity in a mouse model of ALS. These results strongly support the continued preclinical development of SOL-257 for ALS treatment. Moreover, the adaptability of this gene therapy platform, which expresses engineered co-chaperones with tailored target specificities, holds promise for treating a range of protein misfolding diseases."

Gene therapy • CNS Disorders • Proteinopathy • TARDBP

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 (Businesswire) - "As a pioneer in innovative chaperone technology, SOLA Biosciences Inc. will present its compelling preclinical proof-of-concept data for SOL-257, a targeted gene therapy for misfolded TDP-43 in Amyotrophic Lateral Sclerosis (ALS), at Neuroscience 2023 in Washington, D.C. on November 15 and the ALS One Symposium on November 17....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."

Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Preclinical proof of concept of SOL-257, a gene therapy targeting misfolded TDP-43 in ALS (Neuroscience 2023) - "This in vivo proof of concept study demonstrated that SOL-257 gene therapy can effectively prevent TDP-43-related toxicity in the rNLS mouse model of ALS. These results provide strong support for the ongoing preclinical development of SOL-257 for the treatment of ALS. Additionally, this versatile gene therapy platform, capable of expressing engineered co-chaperones with different target specificities, holds promising potential for the treatment of protein misfolding diseases in general."

Gene therapy • Preclinical • CNS Disorders • Proteinopathy • NEFH • TARDBP

SOLA Biosciences Shares Preclinical Proof-of-Concept Data for SOL-257, a Gene Therapy Targeting Misfolded TDP-43 in ALS at 22nd Annual NEALS Meeting (Businesswire) - "SOLA Biosciences...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....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."

Preclinical