Amarna Raises Over $5M to Advance Gene Therapy for Hemophilia B

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by Marisa Wexler MS |

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Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B.

The goal is to advance the company’s hemophilia B program toward a first-in-human clinical study, Amarna said in a press release.

The new funds are from existing backers C4 holding BV, Flerie Invest AB, and RVO, the Netherlands Enterprise Agency, which together invested €10 million (about $11.3 million) in Amarna in 2019.

According to Amarna, the new investment reflects the progress that the company has made toward bringing AMA005 to the clinic. The company is currently finishing studies to support applications to begin clinical trials of the investigational therapy.

“As Amarna is entering an exciting next phase, we would like to thank both our existing investors and RVO for their continued support, which enables us to advance our lead gene therapy towards the clinic,” said Steen Klysner, PhD, Amarna’s CEO.

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Klysner added that this investment also will help Amarna to “progress our broader R&D pipeline for the treatment of autoimmune and chronic inflammation towards pre-clinical proof-of-principle.”

Hemophilia B is caused by mutations in the gene that encodes a clotting protein called factor IX (FIX). The overarching aim of gene therapy for this condition is to deliver a healthy version of the gene to patient cells in order to restore the production of functional FIX.

AMA005 uses a viral vector called simian virus 40, or SV40, which is a virus that normally infects certain species of macaques, a monkey typically found in Asia. Modified viruses are often employed in gene therapy because they are highly effective at inserting genetic material into cells, since that is the mechanism by which they normally infect cells.

As the company moves toward clinical testing of AMA005, Amarna has partnered with Halix, which will be manufacturing the product for use in clinical trials. The partners are currently working to move Amarna’s production process to Halix’s manufacturing facility. Both companies are headquartered in Leiden, in the Netherlands.

“We are pleased to have partnered with renowned Dutch CDMO [contract development and manufacturing organization] HALIX for the production of our first clinical batches, providing an optimal setting for process transfer and collaboration, due to its location in our close proximity in Leiden,” Klysner said.