Specialty Pharma Journal Specialty, Precision & Technology
BOSTON & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Moderna Therapeutics today announced that the two companies have entered into an exclusive research collaboration and licensing agreement aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics™ for the treatment of cystic fibrosis (CF). The three-year collaboration …
Tag Archives: cystic fibrosis
July, 2016
June, 2016
-
8 June
Study Sheds Light on Uncategorized Genetic Mutations in Cystic Fibrosis
LOS ANGELES–(BUSINESS WIRE)–When it comes to cystic fibrosis (CF), more than 2,000 different genetic mutations have been reported. However, only 200 of them have been categorized – leaving a genetic soup of 1,800 others for scientists and genetic counselors to figure out. A new study led by researchers at Children’s …
May, 2016
-
12 May
Scientists Develop New Treatment to Prolong Life of Cystic Fibrosis Patients
Scientists at Queen’s University Belfast have discovered a new molecule which has the potential to prolong the life of individuals with cystic fibrosis (CF). The molecule represents a possible future treatment and works by altering cellular ion channels resulting in improved airway hydration and significantly increased mucous clearance. For individuals …
February, 2016
-
5 February
FDA Rejects Expanded Use of Vertex’s Kalydeco in Cystic Fibrosis
BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for the use of KALYDECO® (ivacaftor) in people with cystic fibrosis (CF) ages 2 and older who have one of …
October, 2015
-
9 October
Quest Diagnostics Presents Study Demonstrating Clinical Value in Genetic Tests for Predicting Disease
MADISON, N.J., Oct. 8, 2015 /PRNewswire/ — Genetic testing for mutations beyond those currently recommended in medical guidelines may aid the identification of more couples at risk of having a child with cystic fibrosis, according to a study presented today at the 2015 American Society of Human Genetics (ASHG) Annual Meeting, which runs …
August, 2015
-
21 August
Raptor Acquires Cystic Fibrosis Drug in $418 Million Deal
NOVATO, Calif., Aug. 20, 2015 (GLOBE NEWSWIRE) — Raptor Pharmaceutical Corp. (Nasdaq:RPTP) announced today that it has signed a definitive agreement with Tripex Pharmaceuticals to acquire Quinsair, the first inhaled fluoroquinolone approved for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with cystic fibrosis, expanding its …
June, 2015
-
8 June
Vertex and Parion Sciences Sign Up to $1.17 Billion Cystic Fibrosis Deal
Vertex Pharmaceuticals and Parion Sciences have teamed up to develop new drugs for treatment of cystic fibrosis (CF) and other pulmonary diseases. The companies have initiated a collaboration worth up to $1.17 billion, under which Vertex and Parion will work together to develop investigational epithelial sodium channel (ENaC) inhibitors for …
May, 2015
-
12 May
FDA Panel Recommends Approval of Vertex’s Cystic Fibrosis Combo
A US Food and Drug Administration (FDA) advisory committee has recommended approval of Vertex Pharmaceuticals’ new combination therapy for treatment of cystic fibrosis (CF). The agency’s Pulmonary-Allergy Drugs Advisory Committee voted 12 to 1 in favor of approving the drug, which consists of Vertex’s already approved drug Kalydeco (ivacaftor) and …
-
8 May
FDA Staff Questions Benefit of Vertex’s Combo Cystic Fibrosis Drug
US health regulators released briefing documents Friday morning questioning the benefit of Vertex Pharmaceuticals’ combination therapy for cystic fibrosis patients. Ahead of an advisory panel review, scheduled for Tuesday, May 12, US Food and Drug Administration (FDA) staff reviewers questioned one of the two drugs in the combination cystic fibrosis …
-
1 May
Yale Researchers Successfully Correct Most Common Cystic Fibrosis Mutation
Researchers at Yale University have used gene editing to successfully correct the most common mutation in the gene that causes cystic fibrosis (CF). A multidisciplinary team of Yale researchers developed a novel approach to edit the genetic defect. In order to correct the mutation, the team used synthetic molecules similar …