Our JUMP70 platform activates the body’s natural protein-folding
machinery to selectively fix or remove misfolded proteins
treating diseases at their molecular roots.
Proteins are the workhorses of our cells, performing essential tasks that sustain life. To function properly, each protein must fold into a precise three-dimensional shape. However, when this folding process goes awry, proteins can misfold and lose their function or worse, accumulate and form toxic aggregates that damage cells. This misfolding lies at the heart of a group of illnesses known as conformational diseases.
Among the most devastating conformational diseases are neurodegenerative disorders, which primarily affect the brain and nervous system. These include Amyotrophic Lateral Sclerosis (ALS), Huntington’s Disease (HD), and Alzheimer’s Disease (AD). In each case, the buildup of misfolded proteins overwhelms the brain’s natural quality control systems, leading to progressive neuronal damage, loss of function, and ultimately, severe cognitive or motor decline.
Despite decades of scientific progress, cures for neurodegenerative diseases remain elusive, and existing treatments offer only limited relief. At SOLA Biosciences, we believe the key to truly transformative therapies lies in restoring the body’s natural ability to manage protein folding, a fundamental process at the core of cellular health.
Our Approach
At SOLA, we are committed to developing gene therapies that address the root cause of conformational diseases, and to restoring hope where options have long been limited.
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that causes progressive muscle weakness and paralysis due to the loss of motor neurons in the brain and spinal cord. With no cure and only limited treatment options, ALS remains a critical area of unmet medical need.
In approximately 97% of ALS cases, the accumulation of misfolded TDP-43 protein is a key driver of disease. These toxic protein aggregates impair cellular function and contribute to motor neuron degeneration.
SOL-257 is an innovative gene therapy candidate developed through our proprietary JUMP70 platform. It is designed to enhance the body’s natural protein-folding system to selectively refold or eliminate toxic TDP-43 proteins, restoring cellular balance and function. With a single-dose administration, SOL-257 aims to provide a first-in-class, disease-modifying therapy that targets ALS at its molecular root.