NeuExcell, Spark Therapeutics to Develop Gene Therapy for HD
Spark Therapeutics will receive access to NeuExcell’s neuroregenerative gene therapy platform and capabilities to advance treatment options for Huntington’s Disease.
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By - NeuExcell Therapeutics and Spark Therapeutics recently announced a gene therapy collaboration to develop a safe and effective treatment for patients with Huntington’s Disease (HD).
Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell’s neuroregenerative gene therapy platform and capabilities. The companies will collaborate closely to advance the program.
“We are excited to collaborate with Spark Therapeutics. Their 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, making them an ideal partner to accelerate our HD program,” Ronald HW Lorijn, CEO of NeuExcell Therapeutics, said in the announcement.
HD is an incurable, hereditary brain disorder caused by a single defective gene on chromosome 4. An infected individual will need full-time care, and the complications associated with HD are usually fatal.
Currently, NeuExcell is developing adeno-associated viruses (AAVs)-based neuroregenerative gene therapy to regenerate new neurons at the site of the neurodegeneration.
The collaboration will leverage NeuExcell’s gene therapy platform to develop a treatment option for HD patients. The platform is built around transcription factor-based trans-differentiation technology and seeks to reprogram endogenous glial cells.
Glial cells are renewable sources for generating new neurons at the injury site, which is crucial for successful HD therapy impact, a spokesperson said in the announcement.
And Spark Therapeutics’ has been at the forefront of gene therapy research for more than two decades. The company’s advanced AAV vector platform, which targets the central nervous system, offers the HD research and development program a notable advantage.
“We are looking forward to our collaboration with NeuExcell and to explore their unique neuroregenerative gene therapy platform,” said Federico Mingozzi, PhD, chief scientific officer of Spark Therapeutics.
NeuExcell is eligible to receive upfront payments of up to nearly $190 million plus product royalties while Spark Therapeutics has the option to license the exclusive worldwide rights of NeuExcell’s HD program.
