Rare Skin Diseases get new Gene based Therapy

Krystal Biotech, a biopharmaceutical company that develops Gene based therapeutics for rare diseases, recently published encouraging topline results from a Phase III trial for a potential treatment for dystrophic Epidermolysis Bullosa (dystrophic EB). The GEM-3 trial on beremagene geperpavec (B-VEC), also known as VYJUVEK, found statistical significance in its capacity to induce complete wound healing in six months compared to a placebo. VYJUVEK is the first topical, non-invasive, and redosable gene currently under development, according to reports.

It’s also the sole Gene based treatment for dystrophic EB, according to researchers. The GEM-3 trial is an intra-patient, randomized, double-blind research that assesses the drug’s safety and efficacy. Thirty-one patients, ranging in age from one to 44 years old, were enrolled in the study at three different locations. Some wounds in each patient received VYJUVEK topically, while others received a placebo.

Once a week, the wounds were dosed and monitored until they healed. The injuries were examined for complete wound healing at the end of six months and complete wound healing at three-month timepoints and mean change in pain severity, as defined by either a FLACC-R scale or a visual analog scale (VAS). When VYJUVEK gets into touch with DEB wounds, it delivers two copies of the COL7A1 gene, functioning at the molecular level, unlike the current standard of care.

If the results stay favorable, it could be the first topical medicine to target the disease’s fundamental cause. The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have given VYJUVEK orphan drug classification for DEB as of this writing. The FDA has also given it a fast-track designation as well as a rare pediatric type.