A candidate gene therapy for haemophilia B developed by scientists at University College London (UCL) was so effective in a Phase 1/2 study that Syncona LLP has decided to start a new company to commercialise it.
Following news in the UK in early November that a one-year-old baby girl with acute lymphoblastic leukaemia (ALL ) has been put into remission by UCART19, an unapproved therapy that required special permission to use, Laboratoires Servier acquired exclusive worldwide rights to the product and promptly did a deal with Pfizer Inc for the drug’s potential commercialisation.
NightstaRx Ltd, a 2014 spinout from the University of Oxford, has raised $35 million in a Series B financing round to further develop a candidate gene therapy for choroideremia, an inherited form of progressive blindness.
France-based TxCell SA plans to outsource the manufacture of its experimental cell therapies, and make new investments in process development to increase efficiencies and make way for the introduction of a second technology platform using engineered regulatory T cells.
An undisclosed amount of seed funding has been provided by two UK investors for a start-up gene therapy company that is working on a concept for preventing nerve damage in glaucoma, the leading cause of irreversible blindness worldwide.
Spark Therapeutics Inc said it plans to seek marketing approval in the US next year for a gene therapy following a Phase 3 trial in which the experimental treatment helped restore vision to patients with an inherited eye disorder.
Novadip Biosciences SA, a spinout from the University of Louvain and the Saint-Luc University Hospital, has raised €28 million in a Series A financing round to develop a therapy for bone defects that is based on adipose-derived mesenchymal stem cells.
ReNeuron Plc has outlined a three-year plan for the development of its stem-cell therapy portfolio that would, if successful, lead to regulatory applications for products in both stroke and retinitis pigmentosa. To support this development, it is proposing to raise £68.4 million in a share placement.
Juno Therapeutics Inc, which has technology for genetically engineering T cells, has teamed up with Fate Therapeutics Inc to find out whether there is a role for small molecules in improving the profile of engineered T cells as therapies. The four-year collaboration takes T cell immunotherapy one step further by potentially introducing new modulators into the mix of technologies that are now being investigated globally for a wide range of cancers. The collaboration was announced on 6 May.
Small and medium-sized enterprises (SMEs) play a special role in drug development particularly in the field of advanced therapy medicinal products (ATMPs). With small numbers of staff, many of whom are scientists, they are in a unique position to drive medical innovation.