Krystal Biotech’s KB103 Granted Orphan Drug Designation by the FDA to Treat Patients With Dystrophic Epidermolysis Bullosa
“Receiving orphan drug designation is an important step forward in our efforts to bring hope to DEB patients and their families," said
About Dystrophic Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7, a major component of the anchoring fibrils which anchor the epidermis to the underlying dermis, and provide structural adhesion in a normal individual. The lack of COL7 in DEB patients causes blisters to occur in the dermis as a result of separation from the epidermis. This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is progressive and incredibly painful.
The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene. DEB also occurs in the form of dominant DEB, or DDEB, which is considered to be a milder form of DEB. There are no known treatments which affect the outcome of either form of the disease and the current standard of care for DEB patients is limited to palliative treatments.
KB103 is Krystal’s lead product candidate, currently in preclinical development and seeks to use gene therapy to treat DEB. KB103 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.
This press release includes certain disclosures which contain “forward-looking statements,” including, without limitation, statements regarding the anticipated timing for Krystal’s filing of an IND, prospects for the development and potential efficacy of KB103, and the benefits of receiving the “rare pediatric disease” indication. You can identify forward-looking statements because they contain words such as “believes” and “expects.” Forward-looking statements are based on Krystal’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Krystal’s filings with the
Source: Krystal Biotech, Inc.