First in human study within reach

May 25, 2021

This is an article from Ingeneious number 2 2021.
Read the whole Ingeneious here >>

A lot has happened at CombiGene during the first months of 2021 and when Ingeneious gets the opportunity to talk to CEO Jan Nilsson and his colleagues Annika Ericsson, Pernilla Fagergren and Martin Linhult, there is no shortage of questions. Jan, how would you describe CombiGene’s position right now?
“I know I’ve said it before, but the work at CombiGene is becoming increasingly exciting and interesting with each day. The future has never been closer, as it were. During the second half of 2020 and the beginning of 2021, we have worked intensely to create the financial resources we need to run the epilepsy project CG01 and the lipodystrophy project CGT2 at full force. I would like to take this opportunity to sincerely thank all our shareholders. It is you who enable CombiGene to continue the development of our promising therapies.”

How do you plan to use the capital you have now raised?
“A rough split looks like this. About 70 percent will be used to drive the epilepsy project forward, 10 percent will go to our lipodystrophy project and 20 percent will go to the day-to-day running of the company. The fact that such a large part of the money goes to the epilepsy project is explained by the fact that this project is now in a cost-intensive phase with large investments, including in the production of the GMP material that will be used in the first clinical study.”

“I know I’ve said it before, but the work at CombiGene is becoming increasingly exciting and interesting with each day. The future has never been closer, as it were.”
Jan Nilsson, CEO, Combigene

“Thanks to the latest share issues, we have also been able to strengthen CombiGene with additional expertise and capacity, which is a prerequisite for us to be able to run our projects so intensely as we do. Over the course of a few months, we recruited Pernilla Fagergren to the position of Clinical Project Manager, Martin Linhult to the position of Project Manager CMC and Esbjörn Melin as Industrial Post Doc.”  

Can you give an overall picture of the current state of the epilepsy project?
”Absolutely. Generally speaking, it can be said that we work along three tracks that all have the same end station – the Clinical Trial Application. The three tracks are the preclinical studies, the production of GMP materials, and the preparations for the clinical studies.”

Annika, let’s start with the preclinical studies that are your area of responsibility, what is the situation there?
”The preclinical work is very intense right now. Currently, we are conducting a toxicology and biodistribution study in small animals. In parallel, we are preparing selection of the injection device we are going to use in the first clinical study. Together with our UK partner Neurochase, we will also develop an optimized administration method for CG01. The administration itself is extremely important since we need to reach a very carefully defined area of the brain with a very precise dose of our drug. When the work in these areas is completed, it is time for the toxicology and biodistribution study in large animals, the last and final preclinical study.”

Martin, if we look at the production part of CG01, what’s the situation there?
“In many ways, it is similar to the preclinical work. We have come a long way through previous choice of production method and choice of suppliers. The majority of the production process is in place and the material used in the two studies in toxicology and biodistribution has been delivered. We are also focusing on producing the material that will be used in the first in human study. We are currently performing the final post-production quality assays of the GMP plasmids included in CG01 – the starting material for production – and plan to produce GMP-quality materials for the first clinical study later this year. This material will then be analysed and quality assured before being released for human use.” 

If we then look at the third and final “track”, the preparations for the first clinical study, what is the situation, Pernilla?
“We are currently working on the two most central parts: the selection of CRO partner and the selection of the clinics where we will conduct our first in human study. CRO stands for ‘Contract Research Organization’ and is thus the company with which we will work to carry out the study itself. We will work out the final study design in collaboration with our CRO partner, our scientific study committee and the clinics included in the study. Before submitting our clinical trial application, we will present the entire study plan to the relevant Regulatory Agencies.”

Jan, it feels like the path to the first study in humans is clearly mapped out.
“That’s really the way it is. In all development work, there is a great deal of uncertainty and no guarantees can be issued. This applies not least to our epilepsy project where we have sometimes and in several ways traveled through unknown territories. We have come so far in the project that many of the uncertainties and challenges in the preclinical work are now behind us. It is not impossible that new challenges will emerge on the project’s way forward, but right now everything is looking really good. Our ambition is to complete the work on the final preclinical studies, finalize the production of GMP material and land the preparation work for the clinical studies with the ambition to submit a clinical trial application sometime in 2022.”

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


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