Grace Science develops small molecule inhibitors targeting the NGLY1 pathway to treat various cancers and neurodegenerative diseases. By leveraging insights from NGLY1 biology, the company aims to create effective therapies for conditions associated with NGLY1 deficiency, improving patient outcomes.
Funding
$34.3M 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
NGLY1 deficiency is an ultra-rare genetic disorder with a wide range of debilitating symptoms, including muscle weakness, speech deficiencies, and seizures. Traditional pharmaceutical companies have little incentive to develop treatments for such rare diseases, leaving affected individuals with limited options.
Solution
Grace Science is a biopharmaceutical company focused on developing therapies for NGLY1 deficiency, a rare genetic disease caused by a mutation in the NGLY1 gene. The company is pursuing both gene therapy and small molecule approaches to address the underlying cause of the disease and its associated symptoms. By leveraging insights into the NGLY1 pathway and its connection to cellular processes like protein degradation, Grace Science aims to develop treatments that can improve the lives of patients with NGLY1 deficiency and potentially address other related diseases. The company collaborates with leading scientists and clinicians to advance its research and development efforts.
Target Audience
The primary target audience is individuals diagnosed with NGLY1 deficiency and their families, as well as researchers and clinicians specializing in rare genetic diseases.
Features
- Gene therapy program focused on delivering corrected NGLY1 genes to affected cells
- Small molecule program targeting the NGLY1 pathway to modulate its activity
- Research into the role of N-glycanase in protein degradation and cellular function
- Collaboration with academic researchers to identify novel therapeutic targets
- Development of assays to screen for potential drug candidates