Two significant steps towards the first human studies for CG01
This is an article from Ingenious number three.
CombiGene has recently taken two major steps towards the first studies in humans, so-called clinical studies. The first step has been to identify a suitable CDMO (Contract Development and Manufacturing Organization) and the other has been selection of a
CRO (Contract Research Organization).
Finding the most suitable organizations has been a very long and painstaking process. In total, 20 companies have been assessed; 13 CDMOs and seven CROs, and our efforts to find the right partners began in late 2018.
Selection of CDMO
The process of finding the right CDMO partner has been managed by Karin Agerman, CombiGene’s Chief Research and Development Officer. ”The final choice was a company called Cobra Biologics, which we feel is the very best partner for CombiGene,” says Karin. ”The choice of a CMO marks a significant milestone in the CG01 project and an important step towards clinical studies. Cobra meets all of our selection criteria and has shown itself to be a company that responds quickly and is easy to collaborate with. Cobra’s
roots can be traced back as far as 1963 and the company has an excellent
reputation, with operations in both the UK and Sweden. Our collaboration will now begin in earnest and we see potential for it to continue for many years..”
How does it feel, now that a CDMO has been chosen, and what happens next?
”It is extremely satisfying to have concluded a very intensive and lengthy selection process and to have laid an important foundation for the continuation of CG01. Even though we have selected a CDMO, the project’s pace of progress will not diminish; on the contrary. Cobra is well under way with production of the plasmids required for production of CG01. Concurrently, they will implement a broad transfer of technology and methodology from CGT Catapult, CombiGene’s partner in the development of a manufacturing method for CG01. The next important stage is a so-called Engineering Run, whereby the entire production process is tested. Material from this Engineering Run will be used in the toxicity and biodistribution tests.”
What has been the most difficult part of finding the right CDMO?
Karin laughs. ”Finding the right CDMO has been a fantastic and stimulating learning experience. The most difficult part of the process had nothing to do with the work itself, but mostly with inconveniences related to travel and hotel accommodation. I’ll never forget how Jan Nilsson (CombiGene’s CEO) and I ran like mad through Frankfurt Airport to make it on time for our flight to Stockholm, only to arrive at a closed gate. That was probably the most intensive cardio workout I’ve had all year. Another time, we struggled to find hotel rooms in Hamburg. The only available rooms in the whole city were priced at nearly 35,000 kronor per night. I can reassure all of our shareholders that we didn’t take these rooms. Instead, we managed to find considerably cheaper accommodation far outside the city limits.”
Selection of CRO
The process of identifying the right CRO partner is in many ways much like that of finding the right CDMO. This very extensive and painstaking endeavour has been led by Annika Ericsson, Senior Project Manager at CombiGene. CombiGene has chosen to work with Northern Biomedical Research (NBR), a company based in Michigan, USA. ”I’m really looking forward to working with NBR,” says Annika. ”Among other things, our CRO partner must be able to introduce the candidate drug, CG01, into the hippocampus, i.e., the part of the brain in which the epileptic focus starts, they must have experience of gene therapy and AAV vectors, and they have to comply with GLP, Good Laboratory Practice. NBR meets all of our requirements perfectly.”
What is the next step in the collaboration with NBR?
”NBR and CombiGene are now going to conduct a small pilot study. This step is necessary for providing material to our other partners, who will develop analysis methods for assessing the
efficacy of CG01. The full-scale biodistribution and toxicity studies will be carried out with material from the Engineering Run that is to be conducted by Cobra.” Annika’s reflections on the task of selecting a contract organization are very much like Karin’s. ”It has been incredibly stimulating and instructive. Unfortunately, I never got a chance to dash through Frankfurt Airport with Jan Nilsson. That would have been fun,” says Annika, who is one of the world’s top-ranked swimrun competitors. ”On the other hand, I won’t forget endless trips on the Paris metro, where passengers stood packed like sardines in the summer heat. But I remember a wonderful car journey along Lake Michigan, from Chicago to Spring Lake, where NBR’s head office is located.”
CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282
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 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 firstname.lastname@example.org.