Abeona zegt goede vorderingen te maken. Dit lijkt me de benchmark voor ProQR. Opmerkelijk is dat het percentage patienten dat een verbetering van meer dan 50% ervaart in 2 jaar afneemt van 100% naar 81%. Dit verwacht je niet bij een dna therapie. Is na 5 jaar een herhaling van de behandeling noodzakelijk (en mogelijk)?
On May 17, 2018, we announced updated clinical data during the ASGCT (American Society for Gene and Cell Therapy) 21st Annual Meeting in Chicago, IL. EB-101 (LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]) are gene-corrected autologous keratinocyte grafts transduced with a retroviral vector containing the COL7A1 gene for patients with severe Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is an ultra-rare, catastrophic genetic skin disorder and unmet medical need. RDEB is caused by loss of function mutations in COL7A1, the gene coding for type VII collagen. RDEB is characterized by generalized cutaneous and mucosal blistering and scarring associated with severe deformities and major extracutaneous involvement. These wounds are also extremely painful and uncomfortable. There is no approved therapy for this disease, and standard of care remains simple dressing changes and palliative care.
At ASGCT, we reported Phase 1/2 results for EB-101 treated subjects (n=7 patients, N=42 total treatments) with non-healing chronic wounds with an average time open prior to treatment of 8.5 years. Wound healing defined as >50% healing compared over baseline has been observed in 100% (42/42 grafts) at 3 months, 90% (38/42) at 6 months, 63% (24/38) at 12 months, and 81% (21/26) at 24 months. By comparison, untreated control wounds healing >50% have been observed in 20%
The EB-101 program has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (the "FDA") and granted Orphan Drug Designation from the European Medicines Agency (the "EMA").