BioWorld International Correspondent

Providing further evidence of the ongoing revival of investor interest in gene therapy, Amsterdam Molecular Therapeutics BV raised €22 million (US$27.6 million) in a Series A funding round.

The funds will support development of its lead program, AMT-011, which is in a Phase II trial for genetic lipoprotein lipase (LPL) deficiency. The condition, a rare disorder also known as Type I hyperlipoproteinemia, arises from the absence of lipoprotein lipase, an enzyme responsible for breakdown of fat molecules. It is characterized by high levels of blood triglycerides, accelerated onset of atherosclerosis and recurrent bouts of pancreatitis, a life-threatening and highly damaging inflammation of the pancreas.

AMT, which was founded in 1998 by a group of scientists based at the Academic Medical Center (AMC) of the University of Amsterdam, aims to use the new injection of cash to expand clinical development of AMT-011 and to take several additional preclinical programs into the clinic.

The company, which is based at the AMC in Amsterdam, the Netherlands, is developing gene therapies for tackling the metabolic conditions acute intermittent porphyria and hyperoxaluria, and for combating a rare form of progressive retinal blindness.

"All are not that far from the clinic," AMT co-founder and Chief Scientific Officer Sander Van Deventer told BioWorld International.

AMT-011 has received orphan-drug designation from the London-based European Medicines Agency. The current trial, which involves just eight patients, is due to report in the first quarter of 2007. If successful, the company would rapidly expand clinical development with the aim of securing an early product launch.

"We believe we will be on the market with this product in 2008," Van Deventer said. The company aims to commercialize the product itself. The addressable market comprises around 4,000 patients worldwide. "We know who they are and where they are," he said. The therapy is delivered via several intramuscular injections that are administered in a single visit. "The process is too simple to be true," he said.

AMT also plans to develop a gene therapy for a less severe form of the condition, Type V hyperlipoproteinemia, which has an addressable market of about 100,000.

AMT met several key milestones, Van Deventer said, that enabled the company to raise cash at this point.

In preclinical studies, it obtained high levels of expression of the transferred gene over sustained periods, leading to an almost total correction of the defect. The company is using an adeno-associated vector, which, he said, the FDA has endorsed because it does not integrate into the host chromosomes. And the company has developed a method that ensures it can obtain sufficient quantities of vector for commercial use. "That has been a major, major hurdle for a long time," Van Deventer said.

ABN Amro Capital, Life Sciences, the venture capital arm of Amsterdam-based ABN Amro Bank NV, led the financing round. Other participants included London-based Advent Venture Partners; Gilde Investment Management BV, of Utrecht, the Netherlands; and Paris-based Crédit Agricole Private Equity. Sander Slootweg of ABN Amro Capital joined AMT's board.

"We believe that we are about to see a renaissance of gene therapy," Slootweg told BioWorld International. "There are a couple of companies positioning themselves for breakthrough news. We think Amsterdam Molecular Therapeutics could be one of them."