Argobio Partners With GeneCode to Develop GDNF Mimetics

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Argobio and GeneCode are teaming up to develop small molecules mimicking glial cell-derived neurotrophic factor (GDNF), a protein that helps nerve cells survive, for the treatment of Parkinson’s and other diseases.

The collaboration builds on previous work by researchers at GeneCode, who together with Mart Saarma, PhD, professor and research director of HiLIFE’s Institute of Biotechnology at the University of Helsinki in Finland, identified and patented a number of small molecules activating a protein receptor for GDNF called RET.

Now, the company will be incorporated by Argobio, which will provide up to €3 million (about $3.4 million) in funding to develop the work further. Additional support is expected to be provided by venture capital investors after incubation.

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“Our combined expertise will catapult this technology beyond what we would have been able to achieve alone,” Paavo Pilv, CEO of GeneCode, said in a press release. “Applications of compounds developed by GeneCode are not limited only to Parkinson’s disease but can be applied to treat other medical conditions.”

GDNF helps nerve cells in the brain survive by playing a role in their growth, maturation (how they become cells with specialized functions), and maintenance. However, GDNF is too large a molecule to be able to cross the blood-brain barrier,  a semipermeable membrane that protects the brain and spinal cord against the external environment.

The molecules identified at GeneCode reproduce some of the features of GDNF — such as binding to the RET receptor — but are smaller in size.

One such small molecule, called BT13, was found to increase the levels of dopamine, the neurotransmitter whose levels are low in the brain of people with Parkinson’s, in lab-grown cells and mice. It also prevented the dopamine-secreting nerve cells from dying. A neurotransmitter is a chemical that allows nerve cells to communicate.

“Argobio’s investment and participation in the development of effective GDNF mimetics is extremely encouraging news. GDNF mimetics can overcome the limitations of the GDNF protein and can open up a completely new page in the treatment of Parkinson’s disease,” said Saarma, whose team established the therapeutic potential of BDNF mimetics.

“We have ambitious goals for the next two years,” Yves Ribeill, PhD, entrepreneur in residence at Argobio, said. “The whole is greater than the sum of the parts with the combined, diverse expertise of GeneCode, Argobio and Professor Saarma. I am confident that we will be able to demonstrate that the unique business model of funded partnerships initiated by Argobio in Europe will give new therapeutics a boost to succeed.”

With €50 million (about $56 million) in capital, Argobio said it hopes to advance this and other projects to Series A funding.

“The launch of our first collaboration is a great achievement for Argobio. Argobio intends to incubate until series A [financing] at least five projects in the fields of rare diseases, neurosciences, and oncology over the next three years,” said Thierry Laugel, chairman of Argobio.

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