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CombiGene AB. (12/16/21). "Press Release: CombiGene and Spark Therapeutics Plan to Expand the Clinical Development Program to Include the U.S. as Well as Europe".

Region Region EUR(WEST)+AM(NORTH)
Organisations Organisation CombiGene AB
  Organisation 2 Spark Therapeutics Inc.
  Group Roche (Group)
Products Product CG01 gene therapy (CombiGene)
  Product 2 clinical research
Index term Index term Roche–CombiGene: GC01 epilepsy gene therapy, 202110–202310 collab + excl ww license to Spark Tx $8.5m pfront + $320m milestones TERMINATED 10/23
Person Person Nilsson, Jan (CombiGene 201610– CEO before NeuroVive + Pergamum + Tripep)

The clinical development program for CombiGene’s CG01 project, which is being developed to treat drug-resistant focal epilepsy, was originally planned to be performed in Europe, CombiGenes home market. Since CombiGene and Spark Therapeutics in October this year entered an exclusive collaboration and licensing agreement for CG01, the two companies have jointly reviewed the future development of the project with the ambition to establish the best path forward.

The most significant outcome of this review is the decision to expand the clinical development program to include clinics in the U.S. as well as in Europe. The U.S. is the world’s largest pharmaceutical market and to establish a clinical presence there adds much further strength to the CG01 project.

In order to prepare CG01 to meet the needs of a global submission the remaining preclinical program will be expanded and, in some parts, complemented with additional studies. In practice, this means that the preclinical part of CG01 will take longer to finalize.

“I am extremely pleased to expand CG01’s clinical development program to include the U.S. With this decision, the project will find a natural foothold on the world’s largest pharmaceutical market, at the same time as Spark can utilize the company’s impressive resources, know-how and networks in an optimal way,” said Jan Nilsson, CombiGene’s CEO. “CombiGene and Spark will now jointly run the remaining part of the preclinical program before Spark, as per our agreement, takes over the full responsibility for the clinical development program and future global commercialization.”

About CG01

CG01 is a unique gene therapy candidate aimed at a large patient population to solve a global need in epilepsy treatment. Epilepsy is a major global medical problem with approximately 47,000 drug-resistant patients with focal epilepsy estimated to be added each year across the US, EU4, UK, Japan, and China. CG01 is in a late preclinical stage, and the production platform, jointly developed by CombiGene and its partners Cobra Biologics and Viralgen, is scalable and designed to provide material for preclinical and clinical trials to full commercial production.

About Spark Therapeutics

At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit, and follow us on Twitter and LinkedIn.

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

Record changed: 2024-01-06


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