The startup develops gene therapies utilizing a proprietary chaperone platform to target and reduce abnormal proteins that aggregate in neurons, specifically addressing neurodegenerative diseases like Amyotrophic Lateral Sclerosis and Huntington's disease. This approach aims to provide patients with a permanent treatment option by mitigating the toxic effects of these protein clumps.
Funding
$25.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS), Huntington's disease (HD), and Alzheimer's disease (AD) are often caused by the accumulation of abnormally folded proteins within neurons. Current treatments do not address the underlying protein misfolding issues that contribute to disease progression. Selectively eliminating or repairing these misfolded proteins without affecting functional proteins remains a significant challenge.
Solution
SOLA Biosciences is developing gene therapies utilizing its Engineered Targeting Chaperone Platform Technology (JUMP70) to address conformational diseases. The JUMP70 platform harnesses a patient's own chaperone proteins to selectively repair or degrade disease-causing misfolded proteins. An adeno-associated virus (AAV) vector delivers the JUMP70 gene into the patient's cells, leading to the production of JUMP70 proteins that capture specific misfolded proteins. The JUMP70 protein then recruits the Hsp70 protein folding system to either refold the misfolded protein or target it for degradation, after which the JUMP70 protein is recycled to address additional misfolded proteins. This approach aims to provide disease-modifying treatments by specifically targeting the root cause of conformational diseases.
Target Audience
The primary target audience includes patients suffering from neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS) and Huntington's disease (HD), as well as medical professionals and researchers focused on developing treatments for these conditions.
Features
- Engineered Targeting Chaperone Platform Technology (JUMP70) selectively targets disease-causing misfolded proteins.
- Utilizes adeno-associated virus (AAV) vectors for gene delivery.
- Activates the patient's own Hsp70 protein folding system to repair or degrade misfolded proteins.
- Targeting domain confers binding specificity to misfolded proteins, while the Hsp70 system engages only misfolded proteins.
- Catalytic action allows JUMP70 to act on multiple misfolded proteins.
- Adaptable to virtually any intracellular target.
- Suitable for gene therapy due to the small size of JUMP70 proteins.
- Flagship candidate SOL-257 targets pathogenic TDP-43 for ALS treatment.
- SOL-175 targets polyQ stretch for Huntington's Disease (HD)