PITTSBURGH, PA, USA I October 19, 2021 IKrystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced the presentation of pre-clinical data describing the nonclinical pharmacology of nebulized KB407, a redosable gene therapy candidate for the treatment of cystic fibrosis, in a poster at the North American Cystic Fibrosis Conference (NACFC) taking place virtually November 2 – 5, 2021. The data include more detailed results from the IND-enabling good laboratory practice (GLP) toxicology study of KB407 from which top line results were previously announced. The company was recently granted approval to conduct a Phase 1 clinical study of inhaled KB407 in patients with cystic fibrosis (CF) in Australia by the Bellberry Human Research Ethics Committee (HREC) and plans to initiate a clinical trial in the United States in 2022.
Abstract Information:
e-Poster Title: Nonclinical Pharmacology of Nebulized KB407 for the Treatment of
Cystic Fibrosis
Poster Number: 541
Full text of accepted abstracts is now available on the conference website at https://www.nacfconference.org/WorkArea/DownloadAsset.aspx?id=1073.
About KB407
KB407 is a redosable gene therapy designed to correct the underlying cause of CF by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebulizer. By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation.
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in the United States, affecting more than 80,000 patients worldwide. CF is caused by genetic mutations that result in dysfunctional or absent CFTR protein. Lack of functional CFTR in secretory airway epithelial cells causes dehydrated mucus buildup in the lungs, pancreas and other organs. This mucus buildup in the lungs leads to recurrent chest infections, increased airway secretions, and eventually, respiratory failure. According to the US Cystic Fibrosis Foundation (“CFF”), the median age at death for patients with CF in the United States was 30.8 years in 2018.
CFTR modulators are effective in a subset of patients with certain CFTR mutations, though because these therapies work to improve the function of mutated protein, patients still experience pulmonary exacerbations and a progressive decline in lung function, which represents a significant unmet need. In addition, approximately 10% of CF patients harbor genetic mutations that are not expected to be responsive to current therapies, and currently have no available disease-modifying treatment options.
About Krystal Biotech
Krystal Biotech, Inc. (NASDAQ: KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.
SOURCE: Krystal Biotech
Post Views: 70
PITTSBURGH, PA, USA I October 19, 2021 IKrystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced the presentation of pre-clinical data describing the nonclinical pharmacology of nebulized KB407, a redosable gene therapy candidate for the treatment of cystic fibrosis, in a poster at the North American Cystic Fibrosis Conference (NACFC) taking place virtually November 2 – 5, 2021. The data include more detailed results from the IND-enabling good laboratory practice (GLP) toxicology study of KB407 from which top line results were previously announced. The company was recently granted approval to conduct a Phase 1 clinical study of inhaled KB407 in patients with cystic fibrosis (CF) in Australia by the Bellberry Human Research Ethics Committee (HREC) and plans to initiate a clinical trial in the United States in 2022.
Abstract Information:
e-Poster Title: Nonclinical Pharmacology of Nebulized KB407 for the Treatment of
Cystic Fibrosis
Poster Number: 541
Full text of accepted abstracts is now available on the conference website at https://www.nacfconference.org/WorkArea/DownloadAsset.aspx?id=1073.
About KB407
KB407 is a redosable gene therapy designed to correct the underlying cause of CF by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebulizer. By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation.
About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in the United States, affecting more than 80,000 patients worldwide. CF is caused by genetic mutations that result in dysfunctional or absent CFTR protein. Lack of functional CFTR in secretory airway epithelial cells causes dehydrated mucus buildup in the lungs, pancreas and other organs. This mucus buildup in the lungs leads to recurrent chest infections, increased airway secretions, and eventually, respiratory failure. According to the US Cystic Fibrosis Foundation (“CFF”), the median age at death for patients with CF in the United States was 30.8 years in 2018.
CFTR modulators are effective in a subset of patients with certain CFTR mutations, though because these therapies work to improve the function of mutated protein, patients still experience pulmonary exacerbations and a progressive decline in lung function, which represents a significant unmet need. In addition, approximately 10% of CF patients harbor genetic mutations that are not expected to be responsive to current therapies, and currently have no available disease-modifying treatment options.
About Krystal Biotech
Krystal Biotech, Inc. (NASDAQ: KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.
SOURCE: Krystal Biotech
Post Views: 70
