Krystal Biotech Announces Presentation of KB103 Data at EB2017 - The 5th World Conference of EB Research
Details of the oral poster presentation are as follows:
|Date:||Monday, September 25|
|Time:||10:30am Central European Summer Time|
|Session:||Preclinical Therapy Development II|
|Title:||HSV-1 Mediated COL7A1 (KB103) Delivery To Keratinocytes And Fibroblasts For Recessive Dystrophic Epidermolysis Bullosa (RDEB) Therapy: Preparations For Phase-I Clinical Trials|
|Presenter:||Ignacia Fuentes, Ph.D.|
|Dermatology and Cutaneous Biology|
|Thomas Jefferson University|
KB103 is Krystal’s lead product candidate, currently in preclinical development and seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin. 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.
About Dystrophic Epidermolysis Bullosa, or DEB
Dystrophic epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein 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.
About the EB2017 and EB-CLINET Conferences
The aim of EB2017 is to evaluate recent research progress, to identify opportunities and priorities for new research, and to stimulate interdisciplinary collaboration among EB research groups. EB2017 is primarily for researchers and clinicians actively engaging in fundamental, translational and clinical EB research.
EB-CLINET - Clinical Network of EB Centres and Experts', is focused on linking clinical expertise in EB worldwide. Currently the network has 84 partners from 55 countries, following their mission to further improve medical care for people with EB through exchange of knowledge.
Together, the two conferences provide an update on EB research and its translation to clinical benefit for patients – “from lab-bench to bedside”. Both the EB2017 and EB-CLINET conferences will be held at the same venue in
This press release includes certain disclosures which contain “forward-looking statements,” including, without limitation, statements regarding the anticipated timing of completion of the offering. 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