Dutch Amarna Therapeutics enters research collaboration with Spanish FPS, examining the efficacy of its SV40-based SVecTM gene delivery vector platform technology in diabetes mellitus type 1 and multiple sclerosis

• Focus on effect of SVecTM on downregulation of pathological immune responses underlying the destruction of own cells in DM1 and in MS patients

• Investment of €0.6 million (US$0.7 million) in animal proof-of-principle studies

LEIDEN, The Netherlands and SEVILLE, Spain I September 25, 2020 I Amarna Therapeutics, a Dutch privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform transforming gene-replacement and immunotherapy across many disease areas. The company today announced it has entered into a collaboration with scientists from the Progreso y Salud Foundation (FPS) at Cabimer in Seville, to jointly examine the efficacy of Amarna’s SVecTM gene delivery vector to develop effective immunotherapies for diabetes mellitus type 1 (DM1) and multiple sclerosis (MS).

The collaboration is a joint effort between the research group of the FPS at research institute Cabimer, led by Dr. Benoit Gauthier, and Amarna Therapeutics, represented by Dr. Peter de Haan (CSO) and Miguel García Toscano (Head of Laboratory in Spain).

To date, the symptoms of DM1 and MS can be managed, but patients cannot be cured from both autoimmune diseases. The aim of this joint effort is to study the efficacy of Amarna’s SV40-based gene delivery vector platform, denoted SVecTM, for downregulation of pathological immune responses that underlie the destruction of own cells in DM1 and in MS patients.

The research will focus on the induction of SVecTM-mediated immune tolerance to the primary self-antigens of both diseases. The studies will use advanced animal models of both autoimmune diseases, that have been established by the collaboration partners. Amarna will invest some €0.6 million over the next two years in the Gauthier research group to conduct the animal proof-of-principle studies for these two indications, for which at present there are no cures available.

Benoit Gauthier, Staff Scientist at Junta de Andalucia-Consejeria de Salud y Familias, comments:

“We are thrilled to start this new venture with Amarna Therapeutics, a world leader in viral gene therapy and we anticipate the studies to generate exciting results”

Peter de Haan, Amarna Therapeutic’s Chief Scientific Officer, adds:

“We are delighted entering this collaboration with such a renowned academic partner like FPS and we look very much forward to initiate the planned studies. Since the quality of life for patients with DM1 and MS is so severely impaired given the lack of cures for these invalidating diseases, the more efficiently we can develop our groundbreaking SV40-based gene delivery vector based therapies, the sooner patients will experience the positive impact of our solution on their lives.”

About FPS

For more than ten years, Benoit Gauthier's research group has focused on the field of diabetes and recently other autoimmune diseases. Its basic quality research has generated important new knowledge which enable the development of new therapies for this disease cluster. An important finding of the group was the mandatory association of immune responses to pancreatic beta cells with their capacity to regenerate in patients with type 1 diabetes. In addition, the discovery of the PAX8 gene, and the relationship between type 2 diabetes (T2D) and increased risk of pancreatic cancer, led to international recognition and generated numerous publications in peer-reviewed scientific journals.

The group is funded by different national and international public and private institutions, as well as from diabetes patient associations and supported by the Andalusian Government.

Amarna Therapeutics

Amarna Therapeutics is a privately held Biotech company founded in 2008. Its head office is located in Leiden (The Netherlands), and it also holds a research facility in Seville (Spain). The company has developed a proprietary production and gene therapy delivery platform in its SuperVeroTM cell line and SVecTM vector for the development of safe and efficient therapies. The company’s pipeline targets several major indications as well as orphan diseases within the field of degenerative, inflammatory and autoimmune diseases. The company plans to take the first candidate from its pipeline into clinical development in 2021.

In October 2019, Amarna secured €10 million in new equity, with the aim of bringing the first product into clinical studies. The financing round was led by the Swedish Flerie Invest AB, together with existing shareholders and an innovation credit from the “Netherlands Enterprise Agency” (RVO.nl).

SOURCE: Amarna Therapeutics

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