Cystic Fibrosis

Cystic Fibrosis Foundation Licenses Experimental Compound to AbbVie

Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

Chicago-based AbbVie announced plans for a strategic collaboration to develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Cystic Fibrosis Foundation.

October 24, 2019/by BioSpace

Vertex Triple Combination CF Drugs Hit the Mark in Phase III Trials

Cystic Fibrosis

A Vertex triple-combination treatment for cystic fibrosis hit the mark in two Phase III trials, setting the stage for the company to secure approval for another treatment for the life-shortening genetic disease that affects approximately 75,000 people in North America, Europe and Australia.

March 6, 2019/by BioSpace

Top 10 Pipelines To Watch: 2019 Annual Report

Analysts, Ankylosing Spondylitis (AS), Annual Reports, Antiretroviral Drugs, Atopic Dermatitis (Eczema), Autoimmune Diseases, B Cells, BRCA Gene, BRCA mutation, Big Pharma, Biologics, Biopharma, Biotechnology, Blockbusters, Breakthrough Therapy Designation, Business, CAR-T Therapy, Cancer, Cells, Chemotherapy, Clinical Data, Clinical Trials, Collaborations, Crohn's Disease, Cystic Fibrosis, Drug Discovery, EU, European Medicines Agency (EMA), FDA, FDA/Regulatory, Fast Track Designation, February 2019, Future Blockbusters, Gene Editing, Generalized Myasthenia Gravis (gMG), Genomics, HIV, Human Genome, Immune Cells, Immune System, Immune-Mediated Diseases, Immunotherapy, Inflammatory Diseases, Injectables, Innovation, Integrase strand transfer inhibitors, Issue Archives, Japan, M&A, Med Ad News, Monoclonal Antibodies, Multiple myeloma, Neoantigens, Neurology, Neuromyelitis Optica Spectrum Disorder (NMOSD), New Drug Applications, Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), Non-Small Cell Lung Cancer (NSCLC), Nonalcoholic Steatohepatitis (NASH), Nuclear Medicine, Oncology, Orphan Drug Designation, Paroxysmal Nocturnal Hemoglobinuria (PNH), Precision Medicine, Prescription Drug User Fee Act (PDUFA), Priority Medicines (PRIME) Designation, Priority Review Voucher, Product Pipelines, Proteins, Psoriatic Arthritis, R&D, RNA Interference (RNAi), Radioligands, Rare Disorders, Research, Rheumatoid Arthritis, Sickle Cell Disease, Small Molecules, Special Reports, T-Cells, Top 10 Pipelines, Top 10 Pipelines To Watch, Tumors, Ulcerative Colitis, Ultra Rare Diseases

The return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.

February 13, 2019/by Andrew Humphreys

AbbVie Takes Over Galapagos Cystic Fibrosis Collaboration Instead of Walking Away

Cystic Fibrosis

Chicago-based AbbVie decided to buy up researcher partner Galapagos’ cystic fibrosis portfolio.

October 25, 2018/by BioSpace

Proteostasis Therapeutics’ Double and Triple-Combo CF Drugs Possible Threat to Vertex Dominance

Analysts, Business, Clinical Trials, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), Investors, R&D, Shares

Boston-based Proteostasis Therapeutics announced positive preliminary data from the ongoing Phase I clinical trial of PTI-808 and PTI-801 in cystic fibrosis. Despite being an early-stage trial and preliminary data, company shares exploded upward by 301 percent, with more than 44 million shares trading hands.

October 19, 2018/by BioSpace

ProQR childhood blindness drug succeeds in early study

Analysts, Blindness, Children, Clinical Trials, Cystic Fibrosis, Dystrophic Epidermolysis Bullosa, Gene Editing, Genetics, Leber’s Congenital Amaurosis 10, Mutations, R&D, RNA, Shares, Skin Disorders, Stargardt’s Disease, Usher Syndrome, Vision

An interim analysis of an early-stage trial showed that ProQR Therapeutics NV’s experimental treatment for a rare form of childhood blindness improved vision.

September 5, 2018/by Reuters Health

New Partnership Formed to Develop First-in-Class Gene Therapy for Cystic Fibrosis

Business, Collaborations, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), Drug Discovery, Gene Therapy, Respiratory Disease

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium – consisting of Imperial College London and the Universities of Oxford and Edinburgh – Imperial Innovations, and Oxford BioMedica announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis.

August 6, 2018/by Business Wire

Celtaxsys cystic fibrosis drug reduces key symptom in mid-stage study

Cystic Fibrosis

Privately held Celtaxsys Inc. said a mid-stage trial testing the company’s experimental cystic fibrosis treatment was successful in reducing a key symptom of the genetic lung disease, but did not improve lung function.

August 2, 2018/by Reuters Health

Mixed CF Trial Results for Galapagos

Cystic Fibrosis

Shares of Galapagos NV fell after the Belgium-based company said AbbVie opted to walk away from its cystic fibrosis collaboration.

June 29, 2018/by BioSpace

Vertex Tops EvaluatePharma World Preview With Phase III Cystic Fibrosis Drug

Analysts, Cystic Fibrosis, FDA, JAK1 Inhibitors, Mutations, Potential Blockbusters

An experimental triple combination treatment for cystic fibrosis landed Vertex Pharmaceuticals at the top of EvaluatePharma’s “World Preview 2018” list.

June 26, 2018/by BioSpace