19:01 , Dec 8, 2017 |  BC Week In Review  |  Clinical News

bluebird bio starts Phase III for LentiGlobin BB305 gene therapy

bluebird bio Inc. (NASDAQ:BLUE) began the U.S. Phase III Northstar-3 (HGB-212) trial to evaluate LentiGlobin BB305 gene therapy to treat 15 patients with transfusion-dependent beta thalassemia of the beta-0/beta-0 genotype. The single-arm, open-label study's primary endpoint...
00:53 , Dec 31, 2016 |  BioCentury  |  Finance

Delivering takeouts

  BioCentury’s 25th annual Buyside View finds biotech investors focusing on mid-cap names with late-stage or marketed products that could become M&A targets. “The main theme playing at the moment is investors like us are looking more...
07:00 , Aug 5, 2013 |  BC Week In Review  |  Clinical News

Autologous CD34+ cells: Phase I/II data

The open-label, Italian Phase I/II TIGET-WAS trial showed that all 3 evaluable patients who received IV infusions of autologous CD34+ cells transfected with a lentiviral vector encoding functional Wiskott-Aldrich syndrome protein showed stable engraftment of...
07:00 , Aug 5, 2013 |  BC Week In Review  |  Clinical News

Autologous CD34+ cells: Phase I/II data

The open-label, Italian Phase I/II TIGET-MLD trial in 3 evaluable pre-symptomatic patients with evidence of late infantile MLD showed that IV infusions of autologous CD34+ cells transfected with a lentiviral vector encoding functional ARSA protein...
07:00 , Jul 2, 2012 |  BC Week In Review  |  Clinical News

Autologous CD34+ cells containing the Wiskott-Aldrich syndrome protein gene regulatory update

The European Commission granted Orphan Drug designation for Fondazione Telethon's autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene to treat Wiskott-Aldrich syndrome. The cells are in Phase I/II testing for...
07:00 , Aug 9, 2010 |  BC Week In Review  |  Clinical News

Gamma-c gene therapy hematology data

Researchers at the hospital and colleagues reported long-term follow-up data from 9 boys treated with ex vivo gamma-c gene therapy showing that 8 patients were alive at a median follow-up of 9 years. Four patients...
07:00 , Mar 19, 2007 |  BC Week In Review  |  Clinical News

Autologous CD34+ cells regulatory update

EMEA's Committee for Orphan Medicinal Products issued a positive opinion to grant Orphan Drug designation for autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA to treat metachromatic leukodystrophy. Fondazione Telethon,...