Gene therapy company NeuExcell Therapeutics revealed on Tuesday that it plans to develop a safe and effective treatment for patients suffering from Huntington's Disease (HD) under a gene therapy collaboration with Spark Therapeutics, a member of the Roche Group (SIX:RO) (OTCQX:RHHBY).
HD is an incurable, hereditary brain disorder caused by a single defective gene on chromosome . As the disease affects different parts of the brain, it impacts movement, behavior, and cognition. The complications associated with HD are usually fatal.
The agreement provides Spark Therapeutics with 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 programme.
Pursuant to the option license, NeuExcell is eligible to receive upfront, license fees, R&D and Sales milestone payments up to approximately USD190m plus product royalties. Spark has the option to license the exclusive worldwide rights of the NeuExcell's HD programme.
Spark Therapeutic's advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development (R&D) programme a major advantage as well as has extensive knowledge and expertise to bring the HD programme forward.
Nuvalent commences NVL-655 phase 2 clinical trial in subjects with NSCLC and other solid tumours
GC Biopharma's GC1130A receives EMA Orphan Drug Designation
Guangzhou Fermion Technology's FZ008-145 IND application receives Chinese regulatory approval
Newron enrols 290 patients in schizophrenia study
Vistagen granted European patent for AV-101 to treat neuropathic pain
GenSight Biologics reports business and financial update
Alora Pharmaceuticals announces Relexxii commercial launch
IGC Pharma granted patent for Alzheimer's drug formulation
NRx Pharmaceuticals signs data agreement with Columbia University for IV Ketamine trials