New Gene Therapy Collaboration Targets Huntington Disease


NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.

NeuExcell Therapeutics and Spark Therapeutics are collaborating to develop gene therapy for the treatment of Huntington disease (HD).1

The collaboration plans to combine NeuExcell’s neuroregenerative gene therapy platform with Spark’s adeno-associated virus (AAV) vector platform, with researchers from both companies working together to develop an AAV-based gene therapy.

"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, making them an ideal partner to accelerate our HD program," said Ronald HW Lorijn, MD, PhD, chief executive officer, NeuExcell Therapeutics, in a statement.1 

NeuExcell’s regenerative gene therapy platform uses transcription factor-based trans-differentiation technology designed to reprogram endogenous glial cells. They are developing AAV-based gene therapies to target neurodegenerative sites and regenerate functional new neurons in their place. 

READ MORE: FDA Lifts Hold on VY-HTT01 IND for Huntington Disease

"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," Joseph La Barge, chief business officer, Spark Therapeutics, added.1 "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 Disease."

NeuExcell is also targeting stroke, Alzheimer disease, amyotrophic lateral sclerosis, Parkinson disease, spinal cord injury, and traumatic brain injury with their proprietary platform, although all programs are in preclinical or animal studies. They aim to file an investigational new drug application for their stroke program in late 2021 and for the HD program in early 2022.

Meanwhile, Spark Therapeutics has gene therapy programs closer to market, such as SPK-7001 for choroideremia in phase 1/2 trials, other neurodegenerative programs in early studies, and a number of therapies in phase 1/2 studies for hemophilia A and Pompe disease. Fidanacogene elaparvovec (SPK-9001), developed in collaboration with Pfizer, is in phase 3 studies and previously received breakthrough therapy and orphan drug designations for the potential treatment of hemophilia B by the FDA.

One of Spark’s investigational therapies for hemophilia A, SPK-8011, previously demonstrated positive data at the International Society of Thrombosis and Hemostasis 2021 Virtual Congress, July 17-21.2 Spark announced data from the phase 1/2 trial (NCT03003533) that showed sustained Factor VIII (FVIII) expression in 16 of 18 participants with up to 4 years of follow-up. Additionally, there was a 91.2% reduction in annual bleeding rate and a 97% reduction in annual infusion rate across all dose groups.

“We are encouraged by the results from the phase 1/2 trial for investigational SPK-8011, which has been evaluated in the largest phase 1/2 gene therapy trial in this disease to date, and demonstrates continued response over time, a critical measure of a therapy’s potential to transform lives for people living with this chronic condition,” said Gallia Levy, MD, PhD, chief medical officer, Spark Therapeutics, in a statement at that time.2 “We remain focused on optimizing the dose and immunomodulatory regimen in the phase 1/2 study and look forward to continuing our evaluation of this therapy in a Phase 3 study.”

1. NeuExcell Therapeutics and Spark Therapeutics announce research collaboration agreement to develop a novel gene therapy for Huntington's Disease. News release. NeuExcell Therapeutics. September 7, 2021.
2. Spark Therapeutics’ SPK-8011 suggests stable and durable Factor VIII expression in largest phase 1/2 gene therapy study in Hemophilia A to date. News release. Spark Therapeutics. July 21, 2021.
Related Videos
Carlos Moraes, PhD, on Understanding Mitochondrial Mutations for Neurodegenerative Diseases
Frederick “Eric” Arnold, PhD
Jeffrey Chamberlain, PhD
Michael Kelly, PhD
George Tachas, PhD
Jeffrey Chamberlain, PhD
Carrie Miceli, PhD, a professor of microbiology, immunology, and molecular genetics at University of California, Los Angeles (UCLA)
PJ Brooks, PhD
Carlos Moraes, PhD
Evan Mizerak
© 2024 MJH Life Sciences

All rights reserved.