|View printer-friendly version|
|FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich’s Ataxia|
Catabasis is developing CAT-4001 as a potential treatment for neurodegenerative diseases such as FA. CAT-4001 is designed to activate Nrf2 and inhibit NF-kB, both of which are implicated in FA pathophysiology. Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB pathways in both cellular assays and animal models.
“We are excited to support this research as it brings a potential new
candidate to the FA treatment pipeline and the research development team
advancing the work is an example of FARA’s private public partnership
strategy. We believe that we make the most meaningful research
“We greatly appreciate the support of the Friedreich’s
Friedreich’s ataxia is a rare, degenerative, life-shortening
neuro-muscular disorder that affects children and adults, and involves
the loss of strength and coordination usually leading to wheelchair use;
diminished vision, hearing and speech; scoliosis (curvature of the
spine); increased risk of diabetes; and a life-threatening heart
condition. There are no
Catabasis is developing CAT-4001 as a potential treatment for neurodegenerative diseases such as Friedreich’s ataxia (FA) and amyotrophic lateral sclerosis (ALS). CAT-4001 is a small molecule that activates Nrf2 and inhibits NF-kB, two pathways that have been implicated in FA and ALS. Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB pathways in both cellular assays and animal models.
Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics using its proprietary Safely Metabolized And Rationally Targeted, or SMART, linker technology platform. The Company's SMART linker technology platform is based on the concept of treating diseases by simultaneously modulating multiple targets in one or more related disease pathways. The Company engineers bi-functional product candidates that are conjugates of two molecules, or bioactives, each with known pharmacological activity, joined by one of its proprietary SMART linkers. The SMART linker conjugates are designed for enhanced efficacy and improved safety and tolerability. The Company’s focus is on treatments for rare diseases. The Company is also developing other product candidates for the treatment of serious lipid disorders. For more information on the Company's technology and pipeline of drug candidates, please visit www.catabasis.com.