characteristic of Netherton Syndrome. Ultimately, those afflicted by Netherton Syndrome often experience chronic skin inflammation, severe dehydration, and stunted growth.
KB407
We are
developing KB407 as a non-invasive inhaled gene therapy product for the treatment of cystic fibrosis, or CF, and are currently in the pre-clinical phase with plans to
file an IND for KB407 in 2021.
CF, the most common inherited genetic disorder in the United States, is caused by mutations in the gene
encoding cystic fibrosis transmembrane conductance regulator, or CFTR. Lack of functional CFTR in secretory airway epithelia results in defective Cl-, bicarbonate, and thiocyanate secretion, coupled with
enhanced Na+ absorption and mucus production, leading to dehydration and acidification of the airway surface liquid. CF is characterized by recurrent chest infections, increased airway secretions, and eventually, respiratory failure. While CF
comprises a multiorgan pathology affecting the upper and lower airways, gastrointestinal and reproductive tracts, and the endocrine system, the primary cause of morbidity and mortality in CF is due to progressive lung destruction. According to the
US Cystic Fibrosis Foundation, or CFF, the median age at death for patients with CF in the United States was 30.8 years in 2018. Currently approved CFTR modulating therapies are limited to patients with specific genetic mutations and there is a
significant unmet medical need for patients with CF who have genetic mutations non-amenable to currently approved CFTR small molecule modulators. According to the CFF, approximately 30,000 patients
in the United States and more than 70,000 patients worldwide are living with CF, and approximately 850 new cases of CF were diagnosed in 2018.
Patents
In January 2020, Krystal diversified its patent estate through the issuance of US Patent and Trademark Office, or USPTO, USPTO)
patent number 10,525,090 covering our second product candidate, KB105, as well as medical applications of this product for treating ARCI. We believe the speed in which the patent prosecution was successfully concluded for this application is
indicative of Krystals pioneering work in HSV-based gene therapies in the field of dermatology.
In December 2019, Krystal strengthened its international patent portfolio covering our lead product candidate,
B-VEC, when the European Patent Office issued an intent to grant European patent application number 16826873 directed, in part, to pharmaceutical compositions comprising
B-VEC, as well as uses thereof.
In October 2019, the USPTO granted the Company US
patent number 10,441,614 covering its fully integrated vector platform, STAR-D, for skin-targeted therapeutics, as well as methods of its use for delivering any effector of interest to the skin. This new
patent provides further validation of the Companys novel work in the field of skin diseases leveraging its HSV-1-based gene therapy technologies.
In September 2019, the Australian patent office granted the Company its first foreign patent (Application No. 2016401692) in Australia
for its lead product candidate B-VEC. This patent covers pharmaceutical compositions comprising B-VEC, as well as medical uses such as the treatment of wounds,
disorders, or diseases of the skin, particularly those found in epidermolysis bullosa patients.
On December 18, 2018, the USPTO
granted US Patent No. 10,155,016 which covers compositions containing B-VEC, formulated for alternate routes of administration.
On January 16, 2018, we announced that the USPTO had granted US Patent No. 9,877,990, which covers compositions comprising HSV
vectors encoding certain effectors and methods of using the same for providing prophylactic, palliative or therapeutic relief of a wound, disorder or disease of the skin. A corresponding