Dermatology indications are a promising area of focus for future gene therapy in dermatology. The following list gives a brief detail of some of these pharmaceutical companies, along with their lead products.
Abeona Therapeutics
Abeona Therapeutics is the leader of the rapidly advancing field of genetic medicine. It is a fully integrated gene and cell therapy company. Its competence across discovering novel cell and gene therapies, manufacture, and R&D has led to the production of new drugs for patients in need.
Patients suffering from RDEB, a rare connective tissue disorder that has no valid treatment, face severe epidermal wounds that impact their longevity. Abeone’s lead asset EB-101 is a gene-corrected, autologous cell therapy that addresses RDEB.
Castle Creek Biosciences
Rare skin diseases lack available treatment options for suffering patients. Castle Creek Biosciences, Inc. — a clinical-stage cell and gene therapy company, works on developing and commercializing disease-modifying treatment options for this segment. The company uses technology that is compatible with the unique biology of each patient. It does so by extracting fibroblast cells from a patient’s skin and then creating localized treatments.
The company has two lead therapies undergoing clinical development.
Therapy 1 includes FCX-013 that is to be used as an autologous cell-based, disease-modifying gene therapy. It utilizes lentiviral vectors to deliver functional matrix metalloproteinase 1 genes (MMP-1) to patients with moderate to severe localized scleroderma.
Therapy 2 includes D-Fi (dabocemagene autoficel, formerly known as FCX-007) developed as an autologous cell-based, disease-modifying gene therapy. It addresses functional type VII collagen protein (COL7) deficiency in patients that have autosomally recessive or dominant dystrophic epidermolysis bullosa (DEB).
Krystal Biotech
Krystal Biotech, Inc. is a company that is in the clinical stage, working to develop effective and innovative treatments for skin diseases. It has been working on expanding the use of its proprietary Skin TARgeted Delivery (STAR-D) platform to target and treat many skin conditions. It has also been developing topical and intradermal “off-the-shelf” novel therapies for treating rare dermatological indications.
Beremagene geperpavec (“B-VEC,” previously “KB103”) is Krystal’s lead therapy prospect, currently in Phase III stage of development, that uses gene therapy for treating all forms of dystrophic epidermolysis bullosa.
Conclusion
Apart from the above-mentioned companies, there are many more Key Companies In Gene Therapy For Dermatology working continuously to satisfy the unmet demands of treatment options for rare conditions in skin diseases. The future of gene therapy in dermatology looks promising since many biotech and pharmaceutical companies are developing novel gene therapies products for various skin diseases. Thus, we may see many more advancements in this subject shortly.
