Read highlights from the latest installment of Dusko Ilic’s industry updates, 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 news of note. Read the full update for February 2020 in Regenerative Medicine here>>
What happened this month that you were expecting?
The UK’s innovation agency Innovate UK (Swindon, UK) has continued backing Plasticell (Stevenage, UK) in developing its lead hematopoietic stem cell program for the fourth time. That should tell us that the first-in-human clinical study using ex vivo expanded cord blood stem cells will happen relatively soon.
What happened that surprised you this month?
Could induced pluripotent stem cell technology reveal the secrets of aging? AgeX Therapeutics (CA, USA) thinks so. A biotechnology company, focused on developing therapeutics for human aging and regeneration, has published an article describing the conversion of cells from one of the longest-lived people in history — a deceased 114-year-old American woman — into a young pluripotent state.
If we only read about one story this month, what should it be?
Being born with a rare genetic disorder can be a lifelong ordeal, potentially even fatal, but this is starting to change. AVROBIO (MA, USA) is tackling rare diseases with their investigational lentivirus-based gene therapy utilizing the patient’s own stem cells. Their first target was Fabry disease, a rare genetic condition that results in a deficiency of alpha-galactosidase A enzyme.
Symptoms of Fabry disease affect nearly all organ systems and the patients often die at an early age. The first patient treated for Fabry disease has a stable viral copy number 32 months after the therapy, and Phase II patients are still producing the missing enzyme 18 months after treatment. The next disease to be tackled is cystinosis, a fatal condition that leads to a lifespan of approximately 10 years. The first patient has already been treated and the initial data are encouraging.
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