NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Disease

NeuExcell Therapeutics and Spark Therapeutics, a member of the Roche Group announced a gene therapy collaboration aimed at developing a safe and effective treatment for patients suffering from Huntington's Disease (HD).

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 Therapeutics 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. Under this Agreement, Spark Therapeutics has the option to license the exclusive worldwide rights of the 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 progress. 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 (AAVs)-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 neuro-regenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington's Disease."

Spark Therapeutic's advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development (R&D) program a major advantage. At the forefront of gene therapy research for more than two decades, Spark Therapeutics has extensive knowledge and capabilities in this field that it will use to bring the HD program forward.

About 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.

About NeuExcell Therapeutics.
NeuExcell is a privately held early-stage gene therapy company headquartered in Pennsylvania, USA. Its mission is to improve the lives of patients suffering from neuro-degenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen (Co-Founder and Chief Scientific Advisor), the Company has developed a potentially disruptive neural repair technology through in vivo astrocyte-to-neuron conversion by introducing neural transcription factors through adeno-associated virus (AAV)-based gene therapy. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer's Disease, Parkinson's Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma.

About Spark Therapeutics
At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease.