Rare Daily Staff

NeuExcell Therapeutics said it entered into a research collaboration with Roche subsidiary Spark Therapeutics to develop a gene therapy for Huntington’s disease, a rare, genetic, neurodegenerative condition.

Huntington’s disease (HD) is an incurable, hereditary brain disorder caused by a single defective gene on chromosome 4. As the disease affects different parts of the brain, it impacts movement, behavior, and cognition. It becomes harder to walk, think, reason, swallow, and talk. Eventually, the person will need full-time care. The complications associated with HD are usually fatal.

Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell’s proprietary neuro-regenerative gene therapy platform and capabilities. NeuExcell’s research team will collaborate closely with Spark to advance the program.

Under the option license, NeuExcell is eligible to receive upfront, license fees, R&D and sales milestone payments up to approximately $190 million plus product royalties, and Spark has the option to license the exclusive worldwide rights to NeuExcell’s HD program.

The prevailing assumption has been that mammalian adult neurons cannot be replaced, and so therapeutic approaches for brain diseases tend to focus on slowing disease progression. NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue.

The company’s neuroregenerative gene therapy platform is built around transcription factor-based trans-differentiation technology. The platform seeks to reprogram endogenous glial cells like astrocytes, which surround neurons and are often reactive after neurons are injured or die, into functional new neurons. While neurons cannot divide to regenerate themselves, glial cells are a renewable source for generating new neurons at the site of injury, and at the scale needed to have a meaningful therapeutic impact. NeuExcell is developing adeno-associated viruses-based neuroregenerative gene therapy to regenerate functional new neurons at the site of the neurodegeneration.

“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 Therapeutics. “Using our existing expertise in gene therapy development and NeuExcell’s neuroregenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington’s disease.”

At the forefront of gene therapy research for more than two decades, Spark has extensive knowledge and capabilities in this field to bring the HD program forward. Its advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development program a major advantage.

“[Spark’s] in-house know-how and capabilities to develop gene therapies that may have the potential to slow, halt or cure neurological diseases and seek to enhance the industry standard for AAV engineering, make them an ideal partner to accelerate our HD program,” said Ronald Lorijn, CEO of NeuExcell Therapeutics.

Photo: Joseph La Barge, chief business officer of Spark Therapeutics

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