NeuExcell and Spark Developing Treatment for Huntington’s Disease

11/02/2021

Happy Valley-based early-stage gene technology company NeuExcell Therapeutics is on the cusp of a life-changing breakthrough treatment for patients suffering from Huntington’s Disease, an incurable, hereditary brain disorder that affects different parts of the brain, impacting movement, behavior and cognition. In September, the company announced a gene therapy collaboration with Spark Therapeutics aimed at developing a safe and effective treatment for Huntington's Disease patients — an agreement which makes NeuExcell eligible for up to $190 million in payments plus product royalties.

“We are excited to collaborate with Spark Therapeutics. Their 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 HW Lorijn, CEO of NeuExcell Therapeutics.

There are currently no treatments available to cure Huntington’s Disease or to slow its progression, but with the advancements in genetic therapies like those made possible via the collaboration between NeuExcell and Spark, these patients now have hope. 

“We are excited to collaborate with Spark Therapeutics. Their 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.”

Progress in this gene therapy work has earned NeuExcell the confidence of investors. In August, NeuExcell Therapeutics announced a $10+ million Series Pre-A financing round. The round was led by Co-Win Ventures, joined by other institutional investors YuanBio, Oriza Seed, Tsingyuan and InnoAngel. The financing will allow NeuExcell to leverage their proprietary neuroregenerative gene therapy platform across multiple neurodegenerative indications.

Happy Valley Support Makes Breakthrough Therapies Possible

NeuExcell has seen a large amount of Happy Valley support. Early funding from the Skip Smith family made a huge impact on the study. In 2018, a group of co-founder Dr. Gong Chen’s Chinese friends in Happy Valley became aware of his groundbreaking efforts and came together to make a $1 million investment in NeuExcell. In 2020 and 2021, some of them added more funds to continue their support.

"There are currently no treatments available to cure Huntington’s Disease or to slow its progression, but with the advancements in genetic therapies like those made possible via the collaboration between NeuExcell and Spark, these patients now have hope."

“With the support of industry-leading partners like Spark and first-class investors, together we can progress the potential of breakthrough therapies for millions of patients suffering many devastating neurodegenerative diseases and injuries,” said NeuExcell’s co-founder Dr. Gong Chen.

Collaborating for a Cure

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 progress. NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue, however. The company's neuroregenerative gene therapy 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. 

“With the support of industry-leading partners like Spark and first-class investors, together we can progress the potential of breakthrough therapies for millions of patients suffering many devastating neurodegenerative diseases and injuries.”

Spark Therapeutic's advanced proprietary adeno-associated virus vector platform targeted to the central nervous system offers the Huntington’s Disease research and development program at NeuExcell a major advantage. Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities, while NeuExcell's research team will collaborate closely with Spark Therapeutics to advance the program. NeuExcell is eligible to receive upfront, license fees, research and development and sales milestone payments up to approximately $190 million plus product royalties. Under this agreement, Spark Therapeutics has the option to license the exclusive worldwide rights of the NeuExcell's Huntington’s Disease program.

"NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue."

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