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

Spyryx Biosciences Partners with Cystic Fibrosis Canada, Serves as Presenting Sponsor of the 16th Broken Arrow Conference May 4-6, Toronto, Ontario

Company continues its outreach to the CF community in support of SPX-101’s Phase 2 clinical program

DURHAM, N.C., May 4, 2017 – Spyryx Biosciences, Inc., a clinical-stage biopharmaceutical company developing innovative therapeutics to address severe lung disease, announced today that it will serve as the presenting sponsor Continue reading Spyryx Biosciences Partners with Cystic Fibrosis Canada, Serves as Presenting Sponsor of the 16th Broken Arrow Conference May 4-6, Toronto, Ontario

Scientists turn human induced pluripotent stem cells into lung cells: ‘Bronchospheres’ may pave way for personalized cystic fibrosis treatments — ScienceDaily

https://www.sciencedaily.com/releases/2017/05/170503131922.htm

For years, scientists who study lung diseases like cystic fibrosis have tried to track this process in detail, from start to finish, in the hope that understanding how lungs form normally may help explain how things go wrong. Now, scientists at Boston University’s Center for Regenerative Medicine (CReM) have announced Continue reading Scientists turn human induced pluripotent stem cells into lung cells: ‘Bronchospheres’ may pave way for personalized cystic fibrosis treatments — ScienceDaily

Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences

Spyryx Biosciences Presents Data and Corporate Update at Two Recent Conferences in Support of Clinical Candidate for Cystic Fibrosis

– Provides corporate update at Needham Healthcare Conference

– Highlights positive data & mechanism of action of SPX-101 at
ECFS Basic Science Conference Continue reading Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences

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

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)

Cystic fibrosis and sickle-cell anemia could be corrected in embryos

https://www.geneticliteracyproject.org/2017/03/08/cystic-fibrosis-sickle-cell-anemia-could-be-corrected-in-embryos-with-new-crispr-variant/

Cystic fibrosis, sickle-cell anemia could be corrected in embryos with new CRISPR variant

Since the discovery of the genome-editing tool CRISPR/Cas9, scientists have been looking to utilize the technology to make a significant impact on correcting genetic diseases. Technical challenges have made it Continue reading Cystic fibrosis and sickle-cell anemia could be corrected in embryos

News about Concert Pharmaceuticals’ cystic fibrosis drug

http://cen.acs.org/articles/95/web/2017/03/Vertex-buys-Concerts-cystic-fibrosis-drug.htm

Vertex buys Concert’s cystic fibrosis drug
Vertex Pharmaceuticals will pay up to $250 million for Concert Pharmaceuticals’ cystic fibrosis drug CTP-656. Currently in Phase II clinical trials, the drug is a deuterated version of Vertex’s own Continue reading News about Concert Pharmaceuticals’ cystic fibrosis drug

Hopeful new clinical trails for nonsense mutations

Translarna’s Turn In Cystic Fibrosis And Brodalumab’s Approval Tardiness

http://seekingalpha.com/article/4043273-translarnas-turn-cystic-fibrosis-brodalumabs-approval-tardiness

Welcome to your weekly digest of approaching regulatory and clinical readouts. PTC Therapeutics (NASDAQ:PTC) is awaiting data in the first quarter with its transcription modulator Translarna in nonsense Continue reading Hopeful new clinical trails for nonsense mutations

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