SiSaf has initiated the process to obtain an Orphan Drug Designation from the US FDA for its siRNA therapeutic, SIS-101-ADO, to treat rare genetic skeletal disorders.
The company is seeking regulatory approval for SIS-101-ADO to treat Autosomal Dominant Osteopetrosis Type 2 (ADO2) patients.
CSSi LifeSciences will be responsible for coordinating the regulatory process that SIS-101-ADO needs to undergo.
A systemic Bio-Courier siRNA, SIS-101-ADO has been designed for blocking the CLCN7 gene’s dominant-negative mutations and rescue the ADO2’s phenotype.
It combines an siRNA which suppress the CLCN7 expression, using the company’s Bio-Courier next generation silicon stabilised hybrid lipid nanoparticles (sshLNPTM) technology.
By combining lipid nanoparticle technology with inorganic bioabsorbable silicon, the sshLNPTM technology addresses the limitations of other RNA delivery technologies.
SiSaf founder and CEO Dr Suzanne Saffie-Siebert said: “In recent years, there has been an explosion of interest in RNA therapeutics for a wide range of medical concerns.
“Initiating the regulatory process to have our ADO2 therapeutic obtain Orphan Drug Designation will move this revolutionary treatment closer to the goal of alleviating the pain and suffering that this disease inflicts on people.
“Provided successful, SIS-101-ADO and other Bio-Courier formulated drugs will not only be able to treat rare skeletal disorders but can clear the way for therapeutics for other rare diseases once thought impossible to treat.”
According to the company, genetic skeletal disorders, including ADO2 account for 5% of all birth defects across the world.
The company’s Bio-Courier technology has been designed for accelerating the technical advances and application of promising RNA therapeutics.