Amarna Therapeutics, a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, today announced that it has secured a further €4 million from existing investors C4 holding BV and Flerie Invest AB, as well as € 1 million of additional innovation credit funding from RVO, the Netherlands Enterprise Agency. The funds will enable Amarna to advance its lead gene therapy for the treatment of hemophilia B towards a first-in-human clinical trial, and to progress its R&D pipeline in selected autoimmune diseases and chronic inflammation.
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This new capital complements a €10 million funding from the same investors and RVO in October 2019, recognizing the progress the company has made in advancing its lead project towards the clinic, and demonstrating the strong potential of Amarna’s platform in other highly prevalent disease areas. The latter includes the generation of promising in vitro and in vivo results, showing the potential to induce immune tolerance in autoimmune diseases, as well as to downregulate infection in chronic inflammatory indications.
Broadening pipeline through SVec gene delivery platform
Amarna Therapeutics is uniquely positioned to develop a broad pipeline of safe effective and durable gene therapies by leveraging the intrinsic non-immunogenicity features of its proprietary in vivo SV40-mediated gene delivery platform, called SVec. The company’s lead product,
AMA005, aims to restore the blood clotting process in hemophilia B patients. The company is currently completing IND enabling studies and has initiated manufacturing with the aim of starting a Phase 1/2a trial in approximately two years. For this, Amarna has recently engaged HALIX, a leading biopharmaceutical contract development and manufacturing organization (CDMO), to produce the clinical trial material. Technology transfer of Amarna’s production process to HALIX’s GMP manufacturing facility in the Leiden Bio Science Park in the Netherlands, is currently ongoing.
In parallel with AMA005, Amarna is progressing its R&D pipeline of selected autoimmune and chronic inflammation programs towards proof-of-principle in order to maximize the therapeutic potential of its SVec gene delivery platform beyond gene replacement