Amarna Therapeutics & Phenocell: Moving towards a Potential Breakthrough in Dry Age-Related Macular Degeneration Treatment

Amarna Therapeutics (NL) and Phenocell’s (FR) new Eurostars project deals with the development of the first cure for dry age-related macular degeneration (dAMD) using a breakthrough gene therapy platform. In the recent March round, they were awarded a €500K subsidy by Eurostars to bring this 3-year project to life. Learn more below, and read what Peter de Haan, Amarna’s Chief Scientific Officer & Co-Founder, and Dr. Brigitte Onteniente, R&D and Site Leader, Phenocell SAS, had to say about the project and their collaboration with Catalyze in achieving the successful submission.

Addressing dry age-related macular degeneration

AMD is an incurable eye disease and the leading cause of blindness in the over 50s affecting 196 million people worldwide. AMD destroys the macula (a small section in the center of the retina), resulting in central vision loss needed for critical tasks such as reading, driving, and recognizing faces. AMD has three stages of progression: dAMD, geographic atrophy (GA) and wet AMD (wAMD). The first stage, dAMD, is characterised by inflammation and destruction of the macula. Current AMD treatments are limited to slowing the progression of wAMD, but do not stop it from advancing to later stages..

Amarna Therapeutics’ HALT-AMD Eurostars project

In their project, HALT-AMD, Amarna Therapeutics will generate a Proof-of-Principle (PoP) data package, including in vitro and in vivo PoP studies, and safety & efficacy studies, for their groundbreaking AMA004 gene therapy for dAMD. Amarna’s gene therapy platform uses a proprietary SV40-based gene delivery vector (Nimvec™) that is non-immunogenic and tolerogenic in humans. The gene therapy will stop inflammation and promote regeneration of the retina, thereby restoring the macular functionality, with the potential to cure treated patients from the disease.

Peter remarks on the importance of this funding:

“The Eurostars funding is pivotal in advancing our research by enabling us to thoroughly assess the safety profile and therapeutic potential of our Nimvec-based treatment. This support is crucial in determining the feasibility of our therapy to treat a large number of patients suffering from dry AMD.”

Joining Amarna in the project is Phenocell, a French research-based biotechnology company, now part of Axol Biosciences family, who specialize in the creation, production, and commercialization of cellular models & bioassays for research and drug discovery.

Dr. Brigitte Onteniente, R&D and Site Leader, Phenocell SAS commented: “We are  happy to partner with Amarna Therapeutics to tackle such an urgent unmet clinical need. Our iPSC-derived retinal cells and advanced co-culture methodologies offer unprecedented potential to create physiologically relevant disease models. This Eurostars grant is a testament to the transformative impact this project could have on the field.”

Collaborating with Catalyze on the successful proposal

Eurostars is a highly competitive funding programme. In an effective collaboration, the Catalyze team supported Amarna to achieve successful submission of the proposal.

Reflecting on the collaboration with Catalyze, Peter comments:

“Collaborating with Catalyze on the HALT-AMD project proposal has been an enlightening experience for the Amarna team. It allowed us to fully grasp the immense potential impact of our treatment on the daily lives of AMD patients.

The expertise and professionalism of the Catalyze team have been instrumental in securing the Eurostars funding, which is essential for advancing our AMD treatment to clinical trials focused on safety and efficacy.”

About Amarna Therapeutics

Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases.

The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyomavirus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, enables the production of Nimvec™ vectors suitable for clinical use.

The company’s fully-integrated Nimvec™gene therapy platform is unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product in humans.

Leveraging this ‘natural’ non-immunogenicity feature of Nimvec™, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic, autoimmune and chronic inflammatory diseases.

Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.

About Phenocell

Phenocell, part of the Axol Bioscience family, is a leading biotechnology company specializing in human iPSC-derived models for life sciences research. Located in Grasse, France, Phenocell works on a mission to provide physiologically relevant tools to support drug discovery and research.

The Catalyze team is proud to have supported Amarna Therapeutics towards their momentous Eurostars success.

Learn more about Amarna Therapeutics

Learn more about Phenocell