US-based synthetic biology companies Agilis Biotherapeutics and Intrexon have entered into an exclusive channel collaboration (ECC) agreement to develop DNA-based therapeutics for Friedreich’s ataxia (FRDA), a rare genetic neurodegenerative disease.

Under the agreement, the companies will develop DNA-based therapeutics to repair or replace the ‘broken’ gene in FRDA and enable increased production of the frataxin protein to alleviate the downstream effects of frataxin deficiency.

In addition, Agilis has an option to expand its relationship with Intrexon by adding another rare genetic disease to the collaboration.

The combination of gene correction with additional therapeutic modulators in a multigenic approach has the potential to create a potent treatment.

This treatment is expected to further improve cardiovascular and neurological function by targeting the underlying causes of the disease.

At present, FRDA therapies are primarily focused on supportive care and symptom relief and currently there are no FDA-approved treatment options to address the cause of FRDA.

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Agilis Biotherapeutics CEO George S Zorich said: "We believe Agilis is on the forefront of one of the most promising treatment breakthroughs for Friedreich’s ataxia. I am personally excited to collaborate with the Intrexon team and look forward to developing new transformative therapies together."

"We will make use of our UltraVector multi-gene engineering and other platforms to develop therapeutic candidates designed to treat both the neurological and the cardiovascular pathologies of FRDA."

Agilis will use Intrexon’s UltraVector platform and RheoSwitch Therapeutic System (RTS) to develop gene therapies and genetically modified cell therapies for the treatment of FRDA.

The RTS is a clinically validated inducible gene switch technology that regulates the expression of therapeutic proteins or bioactive RNA in a dose-dependent manner.

Intrexon founder and chief science officer Dr Thomas Reed said that Intrexon is equipped to pursue several different therapeutic approaches for treating the complexities associated with FRDA.

"As an inventor and integrator of technology platforms, we will make use of our UltraVector multi-gene engineering, RTS gene switch, advanced protein engineering, and other platforms to develop therapeutic candidates designed to treat both the neurological and the cardiovascular pathologies of FRDA," Dr Reed said.

FRDA is an inherited disease caused by a gene mutation that reduces the expression of frataxin, a protein localised in the ‘power centre’ of cells known as the mitochondria, and results in a physically debilitating, life-shortening condition.

It is the most common hereditary ataxia with an estimated 5,000 to 10,000 patients in the US.

Image:Reduced expression of frataxin is the cause of Friedreich’s ataxia. Photo: courtesy of Emw.