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Nuevas terapias para la leucodistrofia metacromática (MLD)

Resumen

Tipo:
Oferta Tecnológica
Referencia:
TODE20170413001
Publicado:
12/05/2017
Caducidad:
12/05/2018
Resumen:
Una universidad alemana ha desarrollado dos enfoques complementarios para el tratamiento de la leucodistrofia metacromática (MLD). Estos enfoques superan a los tratamientos actuales, que causan importantes efectos secundarios en los pacientes. Los investigadores han generado modelos de ratón para la leucodistrofia metacromática y han validado la eficacia de sus productos terapéuticos. La universidad busca socios en los sectores de biotecnología y farmacia interesados en incorporar este know-how en sus productos y establecer acuerdos de licencia y cooperación técnica (estudios preclínicos y clínicos).


Details

Tittle:
Novel therapeutics for metachromatic leukodystrophy (MLD).
Summary:
A German university has developed two complementary approaches for the treatment of metachromatic leukodystrophy (MLD). These approaches surpass the present treatments, which cause significant side-effects for the patients. The inventors offer a license and a technical cooperation agreement.
Description:
Metachromatic leukodystrophy (MLD) is a genetic lysosomal accumulation disease caused by the deficiency of the enzymatic activity of arylsulfatase A (ASA). This enzyme hydrolyzes 3-O-sulfogalactosyl ceramide (sulfatide) to galactosylceramide and sulfate. Sulfatide is a sphingolipid of the myelin sheath of axons and thus important for nerve cell function. However, impaired lysosomal degradation of sulfatide by a reduction or loss of ASA activity leads to an accumulation of this sphingolipid, which results in progressive demyelination. Consequently, the patients show increasing neurological deficits and eventually die.

A German university developed two novel and complementary approaches for the treatment of MLD: The first approach is based on the natural products Epicoccolid B and Epipyrone, which are derived from the mold fungus Epicoccum nigrum. These compounds inhibit the cerebroside-sulfotransferase, which is an enzyme that generates sulfatide. Thereby, the sulfatide concentration is lowered.

In addition (second approach) the researchers have exchanged certain amino acids of human ASA resulting in a much higher enzymatic activity. Previous clinical studies have demonstrated the feasibility of enzyme compensation therapy for treating MLD. However, it became clear that large amounts of enzyme need to be administered thereby potentially causing significant side-effects. These problems are overcome by the present approach.

This offer addresses partners from the biotechnological and pharmaceutical sector who wish to integrate this know-how into their range of products. The university offers a license and technical cooperation agreement. This would involve preclinical and clinical studies.

Advantages and Innovations:
With regard to current treatments this invention is reducing side-effects for the patients. It is a novel and complementary approach. The researchers demonstrated proof of concept.
Stage of Development:
Field tested/evaluated
IPs:
Patent(s) applied for but not yet granted
CommeR Statunts Regarding IPR Status:
European patent priority applications have been field for both approaches.

Partner sought

Type and Role of Partner Sought:
The invented treatment is offered for license agreement as well as technical cooperation agreement for further development. This offer addresses partners from the biotechnological and pharmaceutical sector who wish to integrate this know-how into their range of products. To use this invention it has to be further developed in form of preclinical and clinical studies.

Client

Type and Size of Client:
University
Already Engaged in Trans-National Cooperation:
Si
Languages Spoken:
English
German

Keywords

Technology Keywords:
06002 Biología / biotecnología
06001012 Investigaciones médicas
06001014 Neurología, investigación cerebral
06001015 Productos farmacéuticos / medicamentos
06002003 Tecnología de enzimas