Cystic Fibrosis Foundation to Give $3 Million for Novel CFTR Therapeutic

Arcturus Therapeutics, Inc. (“Arcturus” or the “Company”), a leading RNA medicines company, today announced that it has entered into a research agreement with Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation in which CFFT will pay up to $3 million to advance LUNAR-CF, a novel messenger RNA (mRNA) therapeutic formulated with Arcturus’ LUNAR™ delivery technology.

Continue reading Cystic Fibrosis Foundation to Give $3 Million for Novel CFTR Therapeutic

New License for CFTR Activators and Inhibitors

Vanda Pharmaceuticals (VNDA), UCSF Enter License Agreement for CFTR Activators and Inhibitors

https://www.streetinsider.com/

Vanda Pharmaceuticals Inc. (Vanda) (NASDAQ: VNDA) announced today that it has entered into a license agreement with UC San Francisco (UCSF), under which Vanda will acquire an exclusive Continue reading New License for CFTR Activators and Inhibitors

Concert Pharmaceuticals Initiates Phase 2 Clinical Trial

http://www.businesswire.com/news/home/20161221005037/en/Concert-Pharmaceuticals-Initiates-Phase-2-Clinical-Trial

Concert Pharmaceuticals Initiates Phase 2 Clinical Trial Evaluating CTP-656 for the Treatment of Cystic Fibrosis

Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the initiation of a U.S.-based Phase 2 clinical trial evaluating CTP-656 Continue reading Concert Pharmaceuticals Initiates Phase 2 Clinical Trial

Galapagos starts Phase 1 study with potentiator GLPG2451 for CF

http://finance.yahoo.com/news/galapagos-starts-phase-1-study-200909850.html

Galapagos NV (Euronext & NASDAQ: GLPG) announces the start of a Phase 1 study with potentiator GLPG2451 for cystic fibrosis (CF). Following GLPG1837, GLPG2451 is the second potentiator compound in Galapagos’ extended CF-portfolio to enter clinical trials.

Galapagos is conducting a randomized, double-blind, placebo-controlled Continue reading Galapagos starts Phase 1 study with potentiator GLPG2451 for CF

Laurent Pharma to Prepare Phase 2 Trial for Cystic Fibrosis on Heels of New Financing Round

Laurent Pharma to Prepare Phase 2 Trial for Cystic Fibrosis on Heels of New Financing Round

Montreal-based Laurent Pharmaceuticals recently announced the closing of a financing round, led by new investor Cystic Fibrosis Canada. The round included existing investors such as Aligo Innovation LP and Anges Quebec, and was promoted to allow the company to start preparing its upcoming Continue reading Laurent Pharma to Prepare Phase 2 Trial for Cystic Fibrosis on Heels of New Financing Round

The Paradox of Precision Medicine

http://www.scientificamerican.com/article/the-paradox-of-precision-medicine/

Precision medicine sounds like an inarguably good thing. It begins with the observation that individuals vary in their genetic makeup and that their diseases and responses to medications differ as a result. It then aims to find the right drug, for the right patient, at the Continue reading The Paradox of Precision Medicine

Galapagos starts SAPHIRA Phase 2 study with GLPG1837 in cystic fibrosis patients

Galapagos NV (Euronext & NASDAQ: GLPG) announced today the first dosing in its Phase 2 exploratory program of GLPG1837 in patients with cystic fibrosis (CF).

GLPG1837 is a candidate CFTR potentiator drug in clinical development for the treatment of Class III mutations in cystic fibrosis. The SAPHIRA Phase 2 Continue reading Galapagos starts SAPHIRA Phase 2 study with GLPG1837 in cystic fibrosis patients

Galapagos advances CF clinical development

  • Initiation of Phase 1 study with corrector GLPG2222, earning Galapagos a $10 million milestone payment from AbbVie
  • Expansion of CF portfolio with three novel candidate drugs, with aim to create the most effective combo

Mechelen, Belgium; 19 January 2016: Galapagos NV (Euronext & NASDAQ: GLPG) provides an update on progress made in the cystic fibrosis (CF) programs. Galapagos and AbbVie (NYSE: ABBV) aim to develop a triple CFTR combination therapy to address 90% of patients with CF. In order to bring a more effective therapy to patients, the companies plan to develop multiple candidates and backups for each of the three components (1st generation correctors, next generation correctors, and potentiators) with the goal of identifying a potential triple combination. In addition to our potentiator and corrector that are being tested in the clinic, a total of four additional compounds have the potential to reach clinical development in the CF program this year. Continue reading Galapagos advances CF clinical development

Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=949601

-Approximately 25,000 people with cystic fibrosis worldwide currently eligible for treatment with ORKAMBI® (lumacaftor/ivacaftor) or KALYDECO® (ivacaftor); Vertex Continue reading Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway

http://cysticfibrosisnewstoday.com/2015/12/02/cystic-fibrosis-phase-2-trial-drug-aiming-stabilize-key-cf-gene-underway/

Published originally in Cystic Fibrosis News Today
By Charles Moore

Nivalis Therapeutics, Inc., a Boulder, Colorado-based clinical stage pharmaceutical company, announced that the first patient has been dosed in the Phase 2 clinical trial of its lead investigational drug, N91115, a stabilizer Continue reading Cystic Fibrosis Phase 2 Trial of Drug Aiming to Stabilize Key CF Gene Underway