Castle Creek Biosciences raised US $112.8 million in its latest round of preferred stock financing. The financing is expected to advance Castle Creek’s cell and gene therapy pipeline, including funding the completion of a Phase III study of D-Fi, a cell-based gene therapy delivered by local...
Tetra Bio-Pharma's PPP004, a cannabinoid-derived cream, has been granted Orphan Drug Designation by the European Medicines Agency (EMA). EMA Orphan Drug Designation supports the development of investigational products that are intended to treat rare diseases affecting fewer than 5 in 10,000 people. Read the press release from...
BridgeBio Pharma and Phoenix Tissue Repair reported positive results from a phase II trial of PTR-01, an intravenously-administered recombinant collagen VII protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB). In the trial, PTR-01 was administered once per week for four weeks and then...
In collaboration with Great Ormond Street Hospital and Children's Charity (GOSH) and the NIHR we are so pleased that Mission EB has started, the first large-scale stem cell clinical trial for children with EB. Researchers from GOSH have begun the largest ever stem cell clinical trial for...
The first global congress on Epidermolysis Bullosa took place on 19-23 January, in London. ...
Krystal Biotech have announced that the first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB). The Phase 1/2 trial at Stanford University is a single-centre, open-label, placebo-controlled study conducting an intra-subject comparison...
In 2016, together with two other US based medical research charities, we funded the EB iPS Cell Consortium, which includes researchers from leading universities in the US. The results of its early work were so promising that it leveraged a major grant of $3.8 million,...
Sharmila Nikapota, the mother of a child with a rare genetic disorder, has high hopes for gene editing...