CombiGene signs agreement to evaluate the leading gene therapy candidate within the lipodystrophy project CGT2

STOCKHOLM, January 25, 2022 — CombiGene AB (“CombiGene”, “the Company”) today announced that the Company has signed an important agreement with Professor Ormond MacDougald at the University of Michigan Medical School. The agreement comprises one pilot study and one main study in which CombiGene’s most promising gene therapy candidate within the lipodystrophy project CGT2 will be evaluated.
Professor Ormond MacDougald’s new experimental model, which was published in the journal Diabetes in June 2021 has several characteristics that are similar to partial lipodystrophy in humans, the disorder that CombiGene is targeting with their CGT2 therapy.
“We observed a striking loss of white adipose tissue in adult lipodystrophic mice, along with increased fat deposition in the liver, elevated blood glucose levels, and increased insulin levels compared to the controls,” said Professor MacDougald.
Professor MacDougald and his clinical collaborator, Dr. Elif Oral, have an entire team working within the lipodystrophy field and their extensive knowledge and technical expertise will benefit CombiGene’s CGT2 project. The MacDougald Lab is a leading discovery lab for adipocyte biology. University of Michigan Metabolism, Diabetes and Endocrinology Division has emerged as a leader in the treatment of lipodystrophy syndromes due to Dr. Oral’s longstanding clinical interests in this condition.
“I’m very happy that CombiGene has signed this agreement with Professor MacDougald,” said Annika Ericsson, Preclinical Project Manager at CombiGene. “Professor MacDougald and his team have exactly the knowhow and experimental model that we need to evaluate our leading candidate in the lipodystrophy project.”
Reference to the article
Adipocyte-Specific Deletion of Lamin A/C Largely Models Human Familial Partial Lipodystrophy Type 2
Callie A.S. Corsa, Carolyn M. Walsh, Devika P. Bagchi, Maria C. Foss Freitas, Ziru Li, Julie Hardij, Katrina Granger, Hiroyuki Mori, Rebecca L. Schill, Kenneth T. Lewis, Jessica N. Maung, Ruth D. Azaria, Amy E. Rothberg, Elif A. Oral, Ormond A. MacDougald
Journal: Diabetes
Diabetes 2021;70(9):1970–1984
Published: 04 June 2021
About CGT2
The goal of the CGT2 project is to develop a gene therapy treatment for partial lipodystrophy, a rare disease characterized by altered fat distribution on the body. In the absence of normal body fat, various organs begin to accumulate fat, leading on to serious metabolic complications, including extreme insulin resistance, hypertriglyceridemia (elevated values of blood fat triglyceride) and liver steatosis (fatty liver). There are currently a few symptom-relieving treatments for lipodystrophy, but no therapy that targets the root cause of the disease. For patients suffering from partial lipodystrophy, there are currently no treatments at all.

Eurostars Programme 
CombiGene’s project CGT2 is supported by the Eurostars Programme.
Project ID: 114714

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