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SektorHälsovård
IndustriBioteknik
CombiGene är ett genterapibolag som fokuserar på att utveckla nya behandlingar avsedda för sjukdomar relaterade till det centrala nervsystemet och metabola sjukdomar med fokus på läkemedelsresistent fokal epilepsi respektive partiell lipodystrofi. CombiGene samarbetar med en rad aktörer inom såväl akademi som industri. Bolaget har sitt huvudkontor i Lidingö.

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2021-07-28 13:00:00

CombiGene's gene therapy candidate CG01, which is being developed for the treatment of drug-resistant focal epilepsy, has received patent protection in the U.S., the world’s largest pharmaceutical market as well as in Russia. The first application for patent protection for CG01 was filed in 2016, an international PCT application was filed in 2017 and the now granted U.S. and Russian patents last until 2037. The approvals are the first national approvals of the applications completed from the PCT application.
 
Every year, a large number of new patients with drug-resistant focal epilepsy are diagnosed and it is this group of patients that CG01 is intended for. CombiGene currently estimates that the annual global sales potential is somewhere between USD 750 million and USD 1500 million.
 
The epilepsy project CG01 is in late preclinical phase and CombiGene plans to start the clinical program in the second half of 2022.
 
“The fact that CG01 has now obtained patent protection in the U.S., the world's largest pharmaceutical market, is extremely important from a future commercial perspective”, says Jan Nilsson, CEO of CombiGene. “The patent also verifies the level of innovation in CG01, which has the potential to become the first approved gene therapy treatment for epilepsy.”
 
 
About CG01
Unlike many gene therapies, which are developed for the treatment of rare diseases, CG01 caters to a large population of patients. Epilepsy is a major global problem. Every year, approximately 47,000 drug-resistant patients with focal epilepsy are estimated to be added in the US, EU4, UK, Japan and China. CombiGene believes that it is realistic that 10-20% of these patients could be treated with the drug candidate CG01. Assuming, for example, that the therapy cost per patient is somewhere between $134,000 and $200,000 (which compared to approved gene therapy drugs is low), it provides sales between $750-$1,500 million annually.