Collaboration Sparks Hope for Huntington's Disease Cure

Huntington's disease cure is coming soon. 

Gene therapy leader Spark Therapeutics has partnered with NeuExcell, another developer in the space specializing in neurodegenerative illnesses, in the hopes of finding a potential (HD) Huntington's Disease cure.

HD is a devastating genetic brain disorder for which there are no curative treatments. It is caused by a defective gene called chromosome 4 and affects various areas of the brain responsible for a person's ability to think, walk, swallow, talk and reason. Progression is gradual and, eventually, the patient will require full-time care. 

The collaboration between Spark and NeuExcell opens up access to the latter's proprietary neuro-regenerative gene therapy platform and resources. NeuExcell is searching for a Huntington's Disease cure and various other neurological diseases and central nervous system injuries through an exclusive technology that can process neural repair. The platform uses in vivo astrocyte-to-neuron conversion by bringing in neural transcription factors through adeno-associated virus-based gene treatment. 

Partnership brings Hope for Huntington's Disease Cure

The two companies are partnering under an Option License, where NeuExcell will receive around $190 million in upfront, license fees, research and development, sales milestone payments and royalties from future sales. Spark can also license the exclusive worldwide rights of NeuExcell's HD program. 

"Spark Therapeutics is always looking for ways to bring potentially groundbreaking therapies to patients, including those living with Huntington's, to challenge the inevitability of genetic diseases. We are looking forward to our collaboration with NeuExcell and to explore their unique neuroregenerative gene therapy platform," said Spark Therapeutics Chief Scientific Officer Federico Mingozzi, Ph.D. in a statement.

It has always been assumed that there can be no replacement for neurons in adult mammals, so most Huntington's Disease cure and similar illnesses are focused on delaying the disease's progression instead of curing it. NeuExcell's neural tissue regeneration technology seeks to reprogram endogenous glial cells like astrocytes into functional new neurons. While neurons cannot divide to regenerate, glial cells can generate new neurons and at quantities that may be enough to develop new therapies. 

Spark has long been looking for ways to challenge the inevitability of genetic diseases like Huntington's Disease cure. It owns an advanced proprietary AAV platform that targets CNS disorders and currently has four programs in clinical trials. Meanwhile, NeuExcell has therapies in the pipeline for stroke, amyotrophic lateral sclerosis (ALS), spinal cord injury, glioma, Alzheimer's disease, Parkinson's disease and traumatic brain injury.

Spark has also been actively collaborating with other biotech firms to find cures for various genetic disorders. Early this year, the company partnered with major gene circuit leader Senti Biosciences to use the latter's Smart Sensors technology to develop cell type and disease specific-synthetic promoters for use in eye, liver or CNS-directed treatments. Senti Bio will receive over $645 million in total payments, including any milestones achieved in the future.