22:15 , Jun 17, 2019 |  BC Extra  |  Company News

Trial data suggest 80% of Zynteglo patients could meet milestones in outcomes-based pricing model

The most recent long-term data for bluebird bio's Zynteglo β thalassemia gene therapy show that about 80% of patients could hit payment milestones based on details the biotech disclosed about its outcomes-based pricing model on...
23:13 , Jun 14, 2019 |  BC Extra  |  Company News

bluebird prices β thalassemia gene therapy, first to put majority of revenues at risk

bluebird bio could be the first company to test an outcomes-based pricing model for a gene therapy where the vast majority of the price is at risk. On Friday, bluebird bio Inc. (NASDAQ:BLUE) announced the...
22:59 , Jun 4, 2019 |  BC Extra  |  Company News

Celgene's β-thalassemia blockbuster luspatercept nears finish line

Celgene's luspatercept is on track for a dual approval after FDA on Tuesday accepted a BLA for the therapy to treat β-thalassemia and myelodysplastic syndrome. Luspatercept, which is partnered with Acceleron Pharma Inc. (NASDAQ:XLRN), was...
20:40 , Jun 3, 2019 |  BC Extra  |  Company News

Patients, payers await pricing plans for bluebird's first approved gene therapy

bluebird gained the first European approval of Zynteglo, a β-thalassemia gene therapy Monday, but payers and patients will have to wait until the end of next week for the company to announce pricing and commercialization...
22:42 , May 21, 2019 |  BioCentury  |  Finance

Debt and equity investor K2HV seeks ‘profit and purpose’ in $400M launch

Newly launched venture debt and equity investor K2 HealthVentures says it will redirect some of its returns to “underserved and underinvested” areas of the life sciences and healthcare sphere as it invests the first $400...
23:30 , May 13, 2019 |  BioCentury  |  Finance

How ElevateBio plans to create portfolio of cell, gene therapy newcos with $150M MPM, F2-led round

With $150 million in series A funding and an executive team led by Alexion and bluebird veterans, ElevateBio emerged from stealth on Monday with plans to accelerate cell and gene therapy discovery and development by...
00:46 , May 11, 2019 |  BioCentury  |  Product Development

Now grown up, independent biotechs bulk up their platforms with more modalities

As the incoming class of independent biotechs look to build on their clinical and commercial successes, a handful are expanding beyond the technology that got them there, bringing in new modalities to complement their platforms...
23:17 , May 10, 2019 |  BioCentury  |  Politics, Policy & Law

The unsung public policy innovators paving the path to cures

Patients with diseases like hemophilia, spinal muscular atrophy and β-thalassemia that could be cured with gene or cell therapies probably don’t know it, but their fates depend as much on the success of public policy...
21:44 , May 9, 2019 |  BC Extra  |  Company News

bluebird to rollout Zynteglo starting in Germany next half

bluebird expects to launch β-thalassemia gene therapy Zynteglo in Germany next half, followed by launches in Italy, France and the U.K. in 2020. In March, EMA's CHMP recommended conditional approval of Zynteglo to treat transfusion-dependent...
22:52 , May 2, 2019 |  BC Extra  |  Clinical News

May 2 Clinical Quick Takes: Biogen, GSK, Celgene/bluebird, Genkyotex

Biogen's ALS candidate reduces SOD1 protein levels in CSF  Biogen Inc. (NASDAQ:BIIB) will present on May 7 at the American Academy of Neurology meeting the interim Phase I/II data of tofersen that had prompted it...