close

Agreements

Date: 2017-12-15

Type of information: Licensing agreement

Compound:

  • G1XCGD lentiviral vector (autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene)
 

Company: Genethon (France) Orchard Therapeutics (UK) Yposkesi (France)

Therapeutic area: Rare diseases - Genetic diseases

Type agreement: research - development - licensing

Action mechanism:

  • gene therapy. X-linked chronic granulomatous disease is a primary immune deficiency affecting mainly boys. X-CGD accounts for two thirds of the CGD patient population, with an estimated incidence of about 1 in 100,000 live births and a prevalent population of thousands of patients worldwide. The underlying defect lies in a gene which makes up a critical part of the NADPH-oxidase complex (the catalytic subunit; gp91-phox protein). Patients suffering from this disease are susceptible to severe life-threatening bacterial and fungal infections and excessive inflammation characterized by granuloma formation in any organ, for instance, the gastrointestinal and genitourinary tract. Repeated episodes of infection and inflammation severely reduce the life expectancy of individuals.
  • The gene therapy is made up of immature haematopoietic cells that are taken from the patient. These cells are able to develop into different types of blood cell. In this gene therapy, the cells are modified by a virus that contains the gene for the gp91(phox) protein, so that this gene is carried into the cells. When these modified cells are transplanted back into the patient, they are expected to populate the bone marrow and produce healthy phagocytes with the gp91(phox) protein, which is lacking in patients with X-linked CGD. The type of virus used in this medicine (lentivirus) is modified in order not to cause disease in humans.

Disease: X-linked chronic granulomatous disease (X-CGD)

Details:

  • • On December 15, 2017, Orchard Therapeutics announced a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (X-CGD). Généthon is a non-profit research and development organization whose mission is to develop gene therapies for orphan genetic diseases from research to clinical validation.
  • Under the terms of the agreement, Orchard has obtained an exclusive option to license Généthon’s rights and know-how related to the G1XCGD lentiviral vector (autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene), including rights to the data generated under ongoing clinical trials supported by Généthon in the US and in Europe to assess the safety and efficacy of CD34+ autologous stem cells transduced with G1XCGD for the treatment of X-CGD.
  • The program has already received an Orphan Drug Designation in Europe where the trial has been supported by the FP7 Health program of the European Commission through the Net4CGD collaborative project. As part of the agreement, Yposkesi, the industrial platform for gene and cell therapy created by Généthon, AFM-Telethon and the Sociétés de Projets Industriels  from BPI-France (Banque Publique d’Investissement) will manufacture a number of G1XCGD lentiviral batches for Orchard.
  • In Europe, two clinical studies are ongoing: G1XCGD.01 study (NCT01855685) and G1XCGD.02 study (NCT02757911). Another study is also ongoing in USA (NCT02234934).

Financial terms:

Latest news:

Is general: Yes