Amarna Announces Research Collaboration for Testing MS Gene Therapy Platform

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by Patricia Inacio PhD |

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Dutch Amarna Therapeutics announced a research collaboration with Spanish Progreso y Salud Foundation (FPS) to investigate the efficacy of its gene therapy delivery technology in multiple sclerosis (MS) and diabetes.

MS is thought to be caused by immune-mediated inflammation that damages myelin — an insulating sheath around nerve cells whose loss is the hallmark of MS. The new partnership between the biotech company and the foundation’s research group aims to assess the efficacy of Amarna’s viral-based gene delivery vector in reducing the immune responses that trigger the damage and loss of myelin in animal models of MS.

The research also will assess the vehicle’s effectiveness in animal models of diabetes mellitus type 1.

“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,” Benoit Gauthier, a staff scientist at Junta de Andalucia-Consejeria de Salud y Familias, said in a press release.

The research partnership is led by Gauthier, PhD, at the FPS in Seville, Spain, together with Peter de Haan, PhD, Amarna’s chief scientific officer, and Miguel García Toscano, PhD, head of R&D of Amarna in Spain.

Preventing the immune attack against myelin in MS — a process called immune tolerance — can be achieved using gene therapy, in which myelin antigens (molecules that can trigger an immune response) are introduced into cells using a viral vector (vehicle). The aim is to desensitize the immune system’s response against these antigens.

Amarna has developed a viral vector called SVecTM as a gene delivery platform technology. This platform is based on the Simian virus 40 (SV40), which is non-immunogenic or tolerogenic to humans — meaning the virus does not trigger an immune response.

“The non-immunogenicity also named tolerogenicity in humans renders SV40 vectors highly attractive for use in gene therapy and immunotherapies,” Amarna states in its website.

Amarna will invest around 600,000€ (about $700,00) over the next two years in Gauthier’s research to conduct proof-of-principle studies using animal models of MS and diabetes mellitus type 1.

“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,” de Haan said.

“Since the quality of life for patients with [diabetes mellitus type 1] and MS is so severely impaired given the lack of cures for these … 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,” de Haan added.

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