New License for CFTR Activators and Inhibitors

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

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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

New cystic fibrosis drug (Vertex)

https://www.ft.com/content/aa944954-13f4-11e7-b0c1-37e417ee6c76

Combination treatment offers safer option for patients suffering from deadly illness

Vertex Pharmaceuticals said that a combination of two of its medicines had successfully treated cystic fibrosis in a closely watched clinical trial, paving the way for regulators to approve a Continue reading New cystic fibrosis drug (Vertex)

Phase 2a CF corrector study

Galapagos doses first patient with novel CF corrector GLPG2222

Galapagos NV (Euronext & NASDAQ: GLPG) announces dosing of the first patient with cystic fibrosis (CF) Class III (F508del and a gating mutation like G551D) with novel CF corrector GLPG2222 as an add-on to Kalydeco®[1] in a Phase 2a study. Galapagos further announced the opening of an Investigational New Drug (IND) file with the US Food & Drug Administration for GLPG2222, triggering a $10 million milestone payment.
Continue reading Phase 2a CF corrector study

Nanoparticle-delivered CRISPR tools could treat cystic fibrosis

Nanoparticle-delivered CRISPR tools could treat hemophilia, cystic fibrosis and muscular dystrophy

More and more scientists are using the powerful new gene-editing tool known as CRISPR/Cas9, a technology isolated from bacteria, that holds promise for new treatment of such genetic diseases as cystic Continue reading Nanoparticle-delivered CRISPR tools could treat cystic fibrosis

Spyryx Biosciences Announces Successful Completion of Phase 1 Clinical Trial

Spyryx Biosciences Announces Successful Completion of Phase 1 Clinical Trial for SPX-101 and Planned Phase 2 Initiation in Cystic Fibrosis During 2017

Spyryx Biosciences, Inc., a clinical stage biopharmaceutical company developing innovative therapeutics to address severe lung diseases, today announced the successful completion of a Phase 1 study for its Continue reading Spyryx Biosciences Announces Successful Completion of Phase 1 Clinical Trial

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016

Midphase Verona CF trial

http://www.fiercebiotech.com/biotech/verona-secures-cash-for-mid-phase-cystic-fibrosis-trial

Verona secures cash for midphase cystic fibrosis trial

Verona Pharma (LON:VRP) has secured funding for a Phase IIa trial of its PDE3/PDE4 inhibitor RPL554 in cystic fibrosis patients. The funding, which comes Continue reading Midphase Verona CF trial

Spyryx Biosciences to Present New Data at NACFC

http://www.prnewswire.com/news-releases/spyryx-biosciences-to-present-safety-stability–effectiveness-data-for-spx-101-at-the-north-american-cystic-fibrosis-conference-october-27-29-2016-orlando-fl-300350083.html

Spyryx Biosciences to Present Safety, Stability & Effectiveness Data for SPX-101 at the North American Cystic Fibrosis Conference October 27-29, 2016 Orlando, FL

Spyryx Biosciences, a clinical-stage biopharmaceutical company developing first-in-class innovative therapeutics to address severe lung diseases, will present Continue reading Spyryx Biosciences to Present New Data at NACFC

Vertex to test new class of CF drugs

Vertex to test new class of cystic fibrosis drugs in patients

https://www.bostonglobe.com/business/2016/10/25/vertex-test-new-class-cystic-fibrosis-drugs-patients/IcwjZhIPc2YXyguss2OGSM/story.html

Vertex Pharmaceuticals Inc. said Tuesday it will begin clinical trials for a new class of “triple combination” drug candidates that could nearly double the Continue reading Vertex to test new class of CF drugs

Gene Therapy for Treating CF

http://www.news-medical.net/news/20160920/Gene-therapy-may-be-viable-approach-for-treating-CF-lung-problems.aspx

Gene therapy may be viable approach for treating CF lung problems

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis Continue reading Gene Therapy for Treating CF