SOLA Biosciences Presented Positive ALS Animal Efficacy Data of SOL-257 at the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular Disease

April 29, 2021 |

2 min read

SOLA Biosciences, LLC, today announced positive preclinical data in mice from a study designed to evaluate the efficacy of SOL-257, an innovative gene therapy candidate selectively targeting pathogenic TDP-43, which is found in approximately 97% of ALS patients.

BOSTON--(BUSINESS WIRE)-- SOLA Biosciences LLC, today announced positive preclinical data in mice from a study designed to evaluate the efficacy of SOL-257, an innovative gene therapy candidate selectively targeting pathogenic TDP-43, which is found in approximately 97% of ALS patients. The data was presented during the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular Disease on April 29, 2021, by Akinori Hishiya, Ph.D., Founder/CSO of SOLA.

SOLA has developed an innovative chaperone technology (JUMP70) to selectively eliminate disease-causing misfolded proteins using the patients’ own chaperones. SOLA has developed nine gene therapy candidates incorporating JUMP70 to address conformational diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington’s disease, Parkinson’s disease, and Alzheimer’s disease. These candidates demonstrated preclinical activities and safety to prove the mechanism of the JUMP70 technology. SOL-257 is a flagship compound to selectively target misfolded and neurotoxic TDP-43 proteins without affecting healthy TDP-43.

At the conference, SOLA presented unprecedented in vivo efficacy data using the NEFH-hTDP-43ΔNLS mouse model, which expresses a doxycycline-repressible form of human TDP-43 lacking a nuclear localization signal (NLS). The transgenic mice accumulate cytoplasmic insoluble TDP-43 in neurons of the brain and spinal cord soon after pathogenic TDP-43 proteins are expressed. The mice develop many features reminiscent of ALS, including motor deficits, denervation of neuromuscular junctions, motor neuron loss, and rapid loss in viability. The mice treated with control gene therapy (placebo) started dying 3 weeks after pathogenic TDP-43 expression, and by 5 weeks, 100% of male mice died. In contrast, all male mice treated with SOL-257 gene therapy survived.

“Now we know many diseases such as ALS are caused with protein folding issues. Repairing or removing the misfolded disease-causing proteins can be a fundamental treatment for the patients, although the treatment has not been realized yet. We learned the natural protein quality control system and carefully designed our novel therapeutics,” says Dr. Akinori Hishiya. “Our designed therapeutic enables highly specific intervention, only misfolded disease-causing proteins can be targeted and removed.”

“SOL-257 targeting only pathogenic TDP-43 to repair the specific problem has a significant potential for a broadly applicable ALS treatment,” says Keizo Koya, Ph.D., Founder/CEO of SOLA. “I hope this groundbreaking animal data of SOL-257 will bring new hope for patients with ALS.”