Rare Daily Staff
Perlara entered a collaboration with Vivan Therapeutics to identify personalized therapeutics for rare genetic diseases by screening FDA approved drugs and drug libraries against model systems designed to serve as avatars for patients.
“This collaboration is our first outside of oncology and will enable us to utilize our platform technology to potentially identify therapeutics for diseases for which there are no treatments,” said Laura Towart, CEO of Vivan Therapeutics.
Under this agreement, Perlara will refer patients, families, and foundations seeking to embark on a discovery journey for a rare genetic disease to Vivan Therapeutics. Vivan’s technology known as the Personal Discovery Process (PDP), enables the design and generation of personalized fruit fly avatars. Using robotics, thousands of FDA approved drugs and other drug libraries are screened in combinations to identify potential therapeutics.
“Our fruit fly in vivo models are specifically designed to identify novel and unlikely therapies for diseases with clear genetic components, said Vivan Therapeutics Chief Scientific Officer Nahuel Villegas. “In this exciting collaboration with Perlara, we will employ our unique platform to run massive drug screenings to define therapeutic options for diseases with no current standard of care treatments.”
The first two diseases to be modelled are MAN1B1-CDG (congenital disorder of glycosylation) and ECHS1 deficiency. The ECHS1 gene and MAN1B1 gene are very well conserved in fruit flies.
Individuals with MAN1B1-CDG have two deficient copies of the MAN1B1 gene. Currently, there are no treatments for this disease. Individuals with MAN1B1-CDG typically develop signs and symptoms of the condition during infancy, including a broad array of phenotypic abnormalities such as developmental disability, decreased muscle tone, delayed motor skills, and behavioral problems among others.
ECHS1 deficiency is a rare congenital metabolic disorder caused by biallelic mutations in the ECHS1 gene. Individuals with this mitochondrial condition typically have signs and symptoms of developmental delay, dystonia, seizures, and brain abnormalities.
Photo: Laura Towart, CEO of Vivan Therapeutics