NeuExcell, Spark to Develop Neuroregenerative Gene Therapy

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by Margarida Maia |

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NeuExcell Therapeutics has entered into a research deal with Spark Therapeutics to advance the development of a safe and effective neuroregenerative gene therapy for Huntington’s disease.

Under the terms of the agreement, Spark, a member of the Roche Group, will have access to NeuExcell’s gene therapy platform and hold an option to license the exclusive global rights over its Huntington’s disease program.

In exchange, NeuExcell will be eligible to receive an upfront payment, license fees, and additional payments for sales, and research and development milestones totaling up to about $190 million, plus product royalties.

“We are excited to collaborate with Spark Therapeutics,” Ronald HW Lorijn, PhD, CEO of NeuExcell, said in a press release. “Their in-house know-how and capabilities to develop gene therapies that may have the potential to slow, halt or cure neurological diseases.”

Huntington’s disease is a genetic neurodegenerative disease. Research is currently ongoing to develop experimental therapies that might be able to prevent or delay the progression of the disease and better manage its symptoms.

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One new therapeutic approach involves the use of gene therapy to regenerate — or regrow to replace — nervous tissue, which is damaged or lost in Huntington’s disease.

Nervous tissue has two main types of cells: neurons and glial cells. Glial cells surround neurons and become highly reactive when these are injured or die. Contrary to neurons, glial cells can divide to regenerate and thus provide a renewable source of new cells at the site of neurodegeneration.

NeuExcell’s neuroregenerative gene therapy platform is built around a technology that converts those reactive glial cells into new nerve cells by manipulating their genes.

The conversion can be induced by delivering the genetic material containing the instructions to make the transcription factor NeuroD1, a protein that turns specific genes on or off, to the nervous tissue. The delivery is operated through a harmless, adeno-associated virus (AAV) capsule.

The newly generated nerve cells mature and become functional in a few weeks to integrate ultimately into the existing network of cells.

“At Spark, we understand that in order to break down barriers for people and families affected by genetic diseases, we need to work with like-minded partners that can integrate innovative technologies with our advanced proprietary AAV vector platform,” said Joseph La Barge, chief business officer of Spark.

NeuExcell expects to file an investigational new drug application to bring its Huntington’s disease program into clinical testing in early 2022.

“We are looking forward to our collaboration with NeuExcell,” said Federico Mingozzi, PhD, chief scientific officer of Spark, “and to explore their unique neuroregenerative gene therapy platform.”