Cambridge University scientists giving hope to lung disease patients

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15/05/2017

Cambridge University spin-out Z Factor hopes its treatment for lung disease will have the, er, X factor after raising £7m to help get new drugs to needy patients.

The company, which was founded Jim Huntington, Professor of Molecular Haemostasis at the Cambridge Institute for Medical Research, has raised the cash from existing investor Medicxi, which led the round, along with Cambridge Enterprise and Cambridge Innovation Capital.

Proceeds from the Series A funding round will be used to help take the company’s drug candidate, which can be used to treat Alpha-1-Antitrypsin Deficiency (AATD), to clinic.

For those unfamiliar with AATD, Professor Huntingdon explained: “AATD, which is a significant cause of liver and lung disease, results from a defect in gene encoding.

“Individuals with two defective copies of the gene, making up around one in 2000 of the Western population, typically develop emphysema starting in their 30s. They are also at an increased risk of developing liver diseases such as cirrhosis and cancer. Around two per cent of people have one defective copy of this gene, and are at five-fold increased risk of developing chronic obstructive pulmonary disease (COPD) as they age.”

The most common mutation causing AATD is called the Z mutation (the company name makes sense now, doesn’t it?), which disrupts the normal folding of the protein. Professor Huntington and his team at the university obtained the crystallographic structure of this mutatation, which shows how its atoms are arranged. Apparently this allowed the rational design of drugs that could correct folding and prevent the development of associated diseases. Clever stuff, I’m sure you’ll agree.

“These small molecule drugs act like molecular chaperones for the defective protein, accelerating folding to the correct native state” said Nigel Ramsden at another drug discovery firm RxCelerate, who together with David Fox, is leading the drug discovery effort for Z Factor.

Cambridge Enterprise helped in Z Factor’s formation in 2015, licensing key intellectual property to the company. It has already identified dozens of molecules that can correct the folding defect caused by the Z mutation, and shown that some of these drug candidates can increase Alpha-1-antitrypsin levels in cell models. The company is now working to select the best molecules for use as a drug in human trials, which it hopes to begin in 2019.

“The existing molecules clearly work well in model systems” said Fox. “The next challenge is to create molecules that will work safely in human clinical trials.”

Christine Martin from Cambridge Enterprise also sits on the Z Factor board, and added: “We are delighted to be working alongside Medicxi and Jim in support of Z Factor. Jim is a leading academic innovator and Z Factor is dedicated to developing a therapy that will address a serious unmet medical need.”

Z Factor is based at Babraham Research Campus.


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