Chiesi Farmaceutici S.p.a.

Shire Pharmaceuticals Ireland Limited

Zymenex A/s

Hemebiotech A/s

Jens Fogh

Claes Andersson

Cecilia Weigelt

Pia Hydén

Stefan Nilsson

Helena Reuterwall

Christer Moller

Pär Gellerfors

Kerstin Lundell

Kurt Von Figura

Christer MØLLER

Meher Irani

Diego Prieto Roces

Christer Môller

Pia Ringholm Gulstad

Pia Hyden

Magnus Hjertman

The present invention relates to a process for purification of recombinant alpha-mannosidase, a process for production of alpha-mannosidase, a composition comprising alpha-mannosidase, use of the composition as a medicament, use as a medicament for the treatment of alpha-mannosidosis and a method of treating alpha-mannosidosis and/or alleviating the symptoms of alpha-mannosidosis.

Process for production and purification of recombinant lysosomal alpha-mannosidase

The present invention relates to an animal model for infantile globoid cell leucodystrophy, and use of said animal model for screening and/or validation of agents which may be useful as a medicament for treatment of globoid cell leukodystrophy.

Animal model of Krabbe's disease

The present invention pertains to a process for production of recombinant arylsulfatase A in a cell culture system, the process comprising culturing a mammalian cell capable of producing rASA in liquid medium in a system comprising one or more bio-reactors; and concentrating, purifying and formulating the rASA by a purification process comprising one or more steps of chromatography. Other aspects of the invention provides a pharmaceutical composition comprising rASA, which is efficiently endocytosed via the mannose-6-phosphate receptor pathway in vivo as well as a rhASA a medicament and use of a rhASA for the manufacture of a medicament for reducing the galactosyl sulphatide levels within target cells in the peripheral nervous system and/or within the central nervous system in a subject. A final aspect of the invention provides a method of treating a subject in need thereof, said method comprising administering to said subject a pharmaceutical composition comprising a rhASA and thereby obtaining a reduction in the galactosyl sulphatide levels in target cells within said subject.

Production and purification of recombinant arylsulfatase A

Production and purification of recombinant arylsulftase

A process for purification of recombinant porphobilinogen deaminase (rhPBGD) on an industrial scale by starting from a rhPBGD containing extract obtained from a fermentation of a recombinant cell capable of expressing the rhPBGD and the use of the purified product for the preparation of a medicament.

Process for purification of recombinant porphobilinogen deaminase

A method for treatment or prophylaxis of disease caused by deficiency, in a subject, of an enzyme belonging to the heme biosynthetic pathway, the method comprising administering, to the subject, an effective amount of a catalyst which is an enzyme or an enzymatically equivalent part or analogue thereof. The disease is selected from the group consisting of acute intermittent porphyria (AIP), ALA deficiency porphyria (ADP), Porphyria cutanea tarda (PCT), Hereditary coproporphyria (HCP), Harderoporphyria (HDP), Variegata prophyria (VP), Congenital erthropoetic porphyria (CEP), Erythropoietic protoporphyria (EPP), and Hepatoerythropoietic porphyria (HEP). The catalyst is one or more enzymes selected from the group consisting of delta-aminolevulininic acid synthetase, delta-aminolevulinic acid dehydratase (ALAD), porphobilinogen deaminase (PBGD), uroporphyrinogen III cosythetase, uroporphyrinogen decarboxylase, coproporphyrinogen oxidase, protoporphyrinogen oxidase, and ferrochelatase, or an enzymatically equivalent part or analogue thereof. In addition the invention relates to the use of PBGD, to human recombinant PBGD and to a method of gene therapy. The invention also relates to an expression plasmid pExp1-M2-BB (Seq. ID No. 1) and to use of a DNA fragment, the EcoRI-Hind III linear fragment (seq. ID No. 2), used for transformation in the hemC disruption strategy for production of rhPBGD expressed in

Human porphobilinogen deaminase sequences

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Lynge, Denmark

Jens Fogh