Genetics company 23andMe announced the launch of a new consumer genetic test service on Wednesday that will show whether an individual carries genes associated with 36 different disorders, such as cystic fibrosis. The launch is a major step for the company, which in 2013 was ordered by the Food and Drug Administration to stop selling […]

Boston-based Third Rock Ventures announced today it had raised $52 million to launch a company called Decibel Therapeutics to work on hearing loss.   Decibel Therapeutics will be headquartered at 215 First Street in Cambridge, Mass. near Kendall Square. The company has already started to hire, planning on having 50 to 60 scientists working by […]

Raptor Pharmaceutical Corp. (RPTP) is beefing up its rare disease portfolio with the $453 million acquisition of Quinsair, first inhaled fluoroquinolone approved for the management of chronic pulmonary infections in adults with cystic fibrosis, the company announced this morning. Quinsair is a twice-per-day treatment that contains levofloxacin, an antimicrobial active against a wide range of […]

Recently, Vertex received approval for a new two-drug therapy called Orkambi, designed to treat 8,500 cystic fibrosis patients over the age of 12 who have the most common mutation for this disease. Vertex has priced this drug at $259,000 per patient annually and, not surprisingly, many are outraged. As reported by the Boston Globe’s Robert […]

The U.S. Food and Drug Administration approved Vertex Pharmaceutical Inc.’s cystic-fibrosis drug Orkambi, which could treat as many as 8,500 patients in the U. S. — but at a whopping annual wholesale cost of $259,000 per patient. The drug, a twice-daily oral tablet for patients 12 years and older, expands the company’s reach in the […]

One U.S. biotech company is celebrating Independence Day weekend with a fresh $1 billion in cash from a corporate partner, which it was able to secure without giving up its corporate independence. For more, read on: Juno Secures $1B in Cancer Deal With Celgene Seattle-based Juno Therapeutics was the talk of the industry this week. […]

Scientists conducting a major trial of a therapy that replaces the faulty gene responsible for cystic fibrosis say the treatment has showed significant benefit for the first time in patients’ lung function. The technique, developed with the technology commercialization firm Imperial Innovations, replaces the defective gene behind the inherited lung disease by using inhaled molecules […]

Vertex Pharmaceuticals Inc’s unique combination therapy for a rare lung disorder was given the green light by U.S. health regulators, extending the company’s reach to the most common form of genetic mutation responsible for cystic fibrosis. Orkambi, a combination of Vertex’s approved drug Kalydeco and a new compound called lumacaftor, is intended for patients aged […]

June 30, 2015By Alex Keown and Riley McDermid, BioSpace.com Breaking News Staff BOSTON – Vertex Pharmaceuticals (VRTX) is anxiously waiting for regulatory approval of its newest cystic fibrosis medication Orkambi, a treatment which is widely expected to have a broad impact on the cystic fibrosis market – and a treatment widely expected to sharply boost […]

June 5, 2015By Riley McDermid, BioSpace.com Breaking News Sr. Editor Boston-based Vertex Pharmaceuticals (VRTX) has added another weapon to its arsenal of cystic fibrosis fighting drugs, announcing Friday it $80 million for the rights to two promising drugs from tiny Durham, N.C. biotech Parion Sciences. The drugs, dubbed P-1037 and P-1055, are epithelial sodium channel […]