Read highlights from the latest installment of Dusko Ilic’s industry news, which discuss the latest developments and news in regenerative medicine and stem cell research, and are published every month in Regenerative Medicine.
Every month, Dusko comments on regenerative medicine industry news of note. Read the full update for September 2020 in Regenerative Medicine here>>
What happened this month that you were expecting?
There have been big steps forward in treatment of orphan diseases.
The European Commission (EC) has granted to AVROBIO (MA, USA) an orphan drug designation for AVR-RD-02, the company’s investigational lentiviral gene therapy for the treatment of Gaucher disease. Although Gaucher disease is relatively rare, affecting 1 in 44,000, it is very debilitating and the current standard of care, enzyme replacement therapy, does not actually provide symptom-free life. The patients still have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life.
The European Medicines Agency (EMA) granted eligibility to the Priority Medicines (PRIME) program to bluebird bio (MA, USA) and its investigational gene therapy for sickle cell disease, LentiGlobin™ (bb1111). SCD affects approximately 1 in 10,000 people in the EU.
US FDA has granted Orphan Drug Designation to MB-207, Mustang Bio’s (MA, USA) ex vivo lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, both newly diagnosed and those that were previously treated with a hematopoietic stem cell transplantation and for whom re-treatment is indicated. XSCID is a rare genetic disorder that occurs in approximately 1 per 225,000 births.
What happened that surprised you this month?
Allergan Aesthetics, an AbbVie Company (IL, USA), and Fortunis Capital (UK) are investing US$7.5 million in Stemson Therapeutics (CA, USA) to advance development of Stemson’s therapeutic solution to cure hair loss. Restoring human hair growth using the patient’s own cells to generate new hair follicles? This I can’t wait to see!
If we only read about one story this month, what should it be?
Cynata Therapeutics (Australia) has published the data from the Phase 1 clinical trial of CYP-001 in patients with graft-versus-host disease (GvHD). CYP-001, Cynata’s lead induced pluripotent stem cells (iPSC)-derived mesenchymal stromal/stem (MSC) product candidate broke ground by being the first MSC therapy to be produced at scale without the limitation of multiple donors, through Cynata’s novel Cymerus™ technology. The published trial results detail the world’s first clinical trial of an allogeneic hiPSC-derived product.
Dusko Ilic is a Senior Lecturer in stem cell science, coordinator of the cross-divisional postgraduate program in stem cells and regenerative medicine, and Head of the Induced Pluripotent Stem Cell Core Facility at King’s College London (London, UK). He is also Head of the Assisted Conception Unit’s Human Embryonic Laboratories at Guy’s Hospital (London, UK). He is also a member of the editorial board of the journal Regenerative Medicine, where he writes the Industry Report, a regular feature compiling information from non-academic institutions in the field of stem cells and regenerative medicine.