CombiGene’s Epilepsy Project

Collaboration and licensing agreement with Spark Therapeutics
On October 12, 2021, CombiGene and Spark Therapeutics entered a collaboration and licensing agreement, which gives Spark the exclusive global right to develop, manufacture and commercialize CG01. CombiGene and Spark are now working together to complete the remaining parts of the preclinical program. Once the preclinical phase is finalized, Spark will take over full responsibility for CG01.

About CG01

CG01 is a groundbreaking AAV-based gene therapy with the potential to improve the quality of life for patients with drug-resistant focal epilepsy for whom there is currently no effective treatment. The therapy candidate CG01 uses a gene therapy vector to administer a combination of neuropeptide Y (NPY) and its receptor Y2 directly to the part of the brain which triggers the epileptic seizures.

CombiGene has in recent years conducted a number of preclinical studies which step by step have moved CG01 forward and the project is now in the final phase of preclinical development where the next step is to complete the mandatory studies to support start of the first in human study.

Continuous progress in the preclinical program

Since 2017, CombiGene has conducted a number of successful preclinical studies:

  • Dose response study. In 2017, a preclinical dose response study was conducted that gave the company valuable knowledge of the drug candidate’s dose-dependent properties.
  • Proof-of-concept study. After completing the dose response study in 2017, CombiGene initiated a concept verification study of CG01 in a model of chronic epilepsy. The results of the study show that CG01 has clear antiepileptic effects.
  • Human expression study. In 2017, CombiGene also conducted a study on brain tissue donated in conjunction with resective epilepsy surgery from patients with confirmed pharmacoresistance. Data from the study show that CG01 can be expressed in human epileptic brain tissue and thus confirms that the technique of administering genes using CG01 works as intended.
  • Pharmacokinetics study. The preclinical pharmacokinetics study concluded in 2020 provides further evidence that an injection into brain tissue can create long-term expression of the active substances NPY and Y2.
  • Learning and memory study. NPY is associated with a number of physiological processes, including memory and learning ability, but the learning and memory study shows that CG01 has no adverse impact on either learning or memory.
  • Cell tropism study. CG01 is intended to reach the neurons in hippocampus that trigger the epileptic seizures. CombiGene’s tropism study shows that CG01 is taken up into the neurons of the hippocampus.


Scalable production platform

In the autumn of 2020, CombiGene completed the extensive work to establish a production platform for CG01 in collaboration with its CDMO-partners CGT Catapult, Cobra Biologics, and Viralgen. The platform consists of three parts: production of quality assured starting material (plasmids), quality control methods and a scalable suspension-based production process which makes it possible to produce both limited volumes for preclinical and clinical studies as well as large-scale volumes for future commercial needs.


Preparation for biodistribution and safety studies

In mid-2021, CombiGene produced its first GMP batch of CG01 for use in the large animal toxicology and biodistribution studies. The material is currently undergoing customary quality control.

Collaboration and licensing agreement with Spark Therapeutics
Under the terms of agreement, CombiGene is eligible to receive up to $328.5 million excluding royalties, with $8.5 million upon signing, up to $50 million at preclinical and clinical milestones, and payments based on net sales milestones. CombiGene will also be reimbursed for certain authorized R&D expenses.

About Spark Therapeutics
Spark Therapeutics works to discover, develop, and deliver gene therapies. Spark Therapeutics, founded in March 2013, is based on the technology and know-how that accumulated over two decades at the Children’s Hospital of Philadelphia (CHOP). Spark is a member of the Roche-Group.

CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282

About CombiGene

CombiGene’s vision is to provide patients affected by severe life-altering diseases with the prospect of a better life through novel gene therapies. CombiGene’s business concept is to develop effective gene therapies for severe life-altering diseases where adequate treatment is currently lacking. Development assets are sourced from an external research network and developed to achieve clinical proof of concept. Drug candidates for common diseases will be co-developed and commercialized through strategic partnerships, while the company may manage this process on its own for drugs targeting niched patient populations.

The Company has an exclusive collaboration and licensing agreement for the CG01 project with Spark Therapeutics.

The company is public and listed on the Swedish marketplace Nasdaq First North Growth Market and the company’s Certified Advisor is FNCA Sweden AB, +46 (0)852 80 03 99



CombiGene AB, Agavägen 52A, SE-181 55 Lidingö


Linked In     Twitter      Facebook