21:57 , Dec 15, 2017 |  BC Week In Review  |  Clinical News

bluebird reports additional Phase II/III data for Lenti-D gene therapy in childhood CALD

In October, bluebird bio Inc. (NASDAQ:BLUE) reported additional data from 17 boys with cerebral adrenoleukodystrophy (CALD) in the Phase II/III Starbeam (ALD-102) trial showing that Lenti-D led to no major functional disabilities at 24 months...
07:00 , May 2, 2016 |  BC Week In Review  |  Clinical News

Lenti-D gene therapy: Additional Phase II/III data

Additional data from 17 patients ages <=17 with childhood CALD in the open-label, international Phase II/III Starbeam (ALD-102) trial showed that bluebird bio’s Lenti-D gene therapy did not lead to any major functional disabilities in...
07:00 , Mar 14, 2016 |  BC Week In Review  |  Clinical News

Lenti-D gene therapy: Interim Phase II/III data

Interim data from 17 patients with childhood CALD in the open-label Phase II/III Starbeam (ALD-102) trial showed that bluebird bio’s Lenti-D gene therapy led to ALD protein expression in leukocytes of all patients. The company...
07:00 , Sep 7, 2015 |  BioCentury  |  Strategy

Back to School: Changing the subject

No longer content with a walk-on role as research subjects, and empowered by access to more information and the necessity of shouldering a bigger share of the healthcare bill, patients are demanding a speaking role...
00:14 , Jun 25, 2015 |  BC Extra  |  Financial News

bluebird raises $500M follow-on

bluebird bio Inc. (NASDAQ:BLUE) dipped $4.21 to $170.10 after it raised $500 million through the sale of 2.9 million shares at $170 in a follow-on underwritten by BofA Merrill Lynch; Morgan Stanley; Cowen; SunTrust Robinson...
08:00 , Dec 8, 2014 |  BioCentury  |  Strategy

Pricing gene therapies

The launch of the first gene therapy, Glybera alipogene tiparvovec from uniQure N.V. and partner Chiesi Farmaceutici S.p.A. , provides at least one lesson for companies thinking about annuity pricing models: disease outcomes must be...
07:00 , Apr 21, 2014 |  BioCentury  |  Emerging Company Profile

Minoryx: Mediating rare metabolism

Minoryx Therapeutics S.L. has two complementary strategies for developing therapies to treat inherited metabolic diseases involving enzyme deficiencies: identifying small molecule chaperones that can restore mutant enzyme function, and repurposing existing drugs to treat the...
08:00 , Feb 10, 2014 |  BioCentury  |  Finance

uniQure completes comeback

uniQure completes comeback Dutch gene therapy play uniQure N.V. (NASDAQ:QURE) is back on the public markets after a two-year hiatus. The company has taken advantage of renewed interest in gene therapy as well as the...
08:00 , Nov 11, 2013 |  BC Week In Review  |  Clinical News

Lenti-D gene therapy: Phase II/III started

bluebird began the open-label Phase II/III Starbeam trial to evaluate Lenti-D gene therapy in up to 15 male patients ages <=17 years. The product has Orphan Drug designation in the U.S. and EU to treat...
07:00 , Jul 30, 2012 |  BioCentury  |  Finance

Blue skies for bluebird

Blue skies for bluebird Deerfield Partners made a rare investment in a private company last week when the hedge fund participated in gene therapy company bluebird bio Inc. 's $60 million series D round. Deerfield...