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Novartis says $4m price is reasonable for SMA gene therapy pharmaphorum
December 14, 2018
Novartis says it thinks its one-off gene therapy for spinal muscular atrophy (SMA) could deliver value for money even if its price was set upwards of $4 million – which would set a record for a pharmaceutical product.
Orchard Therapeutics Announces the Build-out of New Gene Therapy Manufacturing Facility in California b3cnewswire
December 14, 2018
Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening....
bluebird bio Presents New Data for LentiGlobin Gene Therapy in Sickle Cell Disease at 60th Annual Meeting of the American Society of Hematology firstwordpharma
December 05, 2018
SCD is a serious, progressively debilitating and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis.
bluebird bio Presents New Data for LentiGlobin Gene Therapy in Sickle Cell Disease at 60th Annual Meeting of the American Society of Hematology firstwordpharma
December 05, 2018
SCD is a serious, progressively debilitating and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis.
Gene Therapy for Sickle Cell Takes Another Step Forward drugs
December 05, 2018
A new gene therapy shows early promise against sickle cell anemia, researchers say.The therapy targets the genetic flaw that causes sickle cell.......
FDA grants priority review to Novartis' filing seeking clearance of gene therapy for spinal muscular atrophy firstwordpharma
December 03, 2018
Novartis said Monday that the FDA granted priority review to an application seeking approval of Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) Type 1, with the agency setting a target data in May next year.
US scientists reveal new gene therapy for Parkinson’s disease pharmaceutical-technology
December 03, 2018
Scientists from Feinstein Institute for Medical Research in the US have revealed that a gene therapy called AAV2-GAD for Parkinson’s disease will create new brain circuits for motor function......
New brain circuits formed for motor function after Parkinson’s therapy europeanpharmaceuticalreview
November 30, 2018
A gene therapy called AAV2-GAD has been associated with improved motor function, leading to researchers looking at its use in an upcoming Phase III trial…
Gene Therapy Forms New Polysynaptic Pathways in Parkinson's drugs
November 29, 2018
For patients with Parkinson's disease (PD) undergoing gene therapy, new polysynaptic functional pathways develop in the brain, according to a study published in the Nov. 28 issue of Science Translational Medicine.
Novartis scores EU nod for gene therapy Luxturna fiercepharma
November 28, 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.

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