Date: 2017-11-27
Type of information: Licensing agreement
Compound: lentivirus-based autologous ex-vivo gene therapy
Company: Orchard Therapeutics (UK) the University of Manchester (UK)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: mucopolysaccharidosis type IIIA (Sanfilippo B syndrome)
Details:
- • On November 27, 2017, Orchard Therapeutics announced the extension of its collaboration with Manchester University to include Sanfilippo Syndrome type B. The company has acquired an exclusive license to develop lentivirus-based autologous ex-vivo gene therapy for Sanfilippo syndrome type B (or MPS-IIIB) from The University of Manchester. The technology has been developed in Professor Brian Bigger’s laboratory and recently published in Brain. It involves the use of a high-titre lentiviral vector to drive the expression of a codon-optimized ?-N-acetylglucosaminidase (NAGLU) gene under the control of the myeloid-specific CD11b promoter (LV.CD11b.NAGLU).
- This programme in MPS-IIIB complements the existing collaboration program between Orchard, The University of Manchester and Manchester University NHS Foundation Trust in MPS-IIIA. Autologous ex-vivo lentiviral haematopoietic stem cell gene therapy is anticipated to correct neurological manifestations through the engraftment of subpopulations of haematopoietic stem cells in the central nervous system, thereby providing supranormal and widespread enzyme expression throughout the brain. In both MPS-IIIA and MPS-IIIB, preclinical studies have produced encouraging results showing a normalization of heparan sulphate levels in the brain and peripheral organs, as well as neurological disease correction.
- Orchard’Therapeutics intends to complete preclinical activities required to enable the start of clinical studies in MPS-IIIA towards the end of 2018 and thereafter in MPS-IIIB.
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