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Novartis plots 'innovative' payment models for pricey Luxturna's EU launch fiercepharma
November 27, 2018
It’s been almost a year since Spark Therapeutics won a groundbreaking gene therapy approval in the U.S. for Luxturna, and now its marketing partner Novartis has done the same across the pond.
Gene therapy firm BioMarin stages haemophilia musical pharmaphorum
November 27, 2018
BioMarin Pharmaceutical has put on a stage musical to raise awareness about haemophilia, and determine whether creative expression helps patients, as it develops an experimental cure for the condition.
Novartis/Spark’s sight gene therapy approved in EU pharmaphorum
November 27, 2018
Spark Therapeutics’ gene therapy for a rare inherited sight disorder, Luxturna has been approved in Europe, but there was still no word on price from marketing partner Novartis.
Patient-Driven Gene Therapy Company ReflectionBio Receives Orphan Drug Designation From U.S. FDA For the Treatment of Bietti's Crystalline Dystrophy (BCD) pharmafocusasia
November 26, 2018
Reflection Biotechnologies Limited ("ReflectionBio"), a gene therapy company committed to the research and development...
Mundipharma to bring world's first gene therapy for knee osteoarthritis biospectrum
November 20, 2018
Mundipharma will be responsible for the development, marketing and distribution of Invossa to medical professionals and hospitals in Japan.
apceth to Manufacture First Potential One-Time Gene Therapy for Transfusion-Dependent Beta-Thalassemia b3cnewswire
November 10, 2018
Following a successful long-term manufacturing relationship, apceth Biopharma and bluebird bio entered a commercial drug product manufacturing agreement in 2016. apceth Biopharma is bluebird bio’s clinical and commercial manufacturing partner for Europe n
Orchard Therapeutics’ war chest balloons with $200M IPO fiercebiotech
November 02, 2018
Orchard Therapeutics priced its IPO at $14 a share, raising $200 million and exceeding the $173 million goal it set earlier this month. The funds will bankroll the pivotal trials and...
FDA lifts clinical hold on gene therapy for sickle cell disease biospectrum
October 12, 2018
CRISPR and Vertex entered into a strategic research collaboration in 2015 aimed at the discovery and development of gene editing treatments using the CRISPR/Cas9 technology to correct defects in specific gene targets known to cause or contribute to partic
EU regulators to review bluebird’s gene therapy LentiGlobin pharmatimes
October 11, 2018
The European Medicines Agency will undertake an accelerated review of bluebird bio’s LentiGlobin, a gene therapy for the treatment of transfusion-dependent beta-thalassemia.
Gilead taps gene editors at Precision BioSciences for hepatitis B cure development fiercebiotech
September 14, 2018
Precision’s platform is derived from a natural enzyme that can target specific gene locations and insert or delete DNA, disrupting sequences.

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