Source Human Gene Therapy Clinical Development Background: Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for the treatment and prevention of diseases that until recently were only managed chronically. The objective of this study was to examine the characteristics
Sangamo Therapeutics and Pfizer have reported additional findings from the Phase I/II Alta clinical trial of investigational gene therapy SB-525 to treat patients with severe haemophilia A. SB-525 is made up of a recombinant adeno-associated virus serotype 6 vector (AAV6) that encodes the complementary deoxyribonucleic acid for B domain deleted human Factor VIII (FVIII). During
We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such
Bluebird bio Inc on Friday set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe this month to treat a rare genetic blood disorder. The company proposed an installment plan, with 315,000 euros paid up front and four additional annual payments due
Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned. ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were
Harvard University breakthrough research shows that stem cell genes can be edited in living systems New research led by Harvard scientist Amy Wagers has demonstrated that gene-editing machinery can be delivered straight to stem cells where they live, rather than in a dish. Published in Cell Reports, the findings have major implications for biotechnology research and
Source BIOPROCESS ONLINE Innovation in gene therapy brings the potential for transforming patient care and obviating the need for chronic therapy through single-dose cures. Despite the potential long-term benefits of this new therapeutic modality, gene therapy companies face a number of underappreciated challenges. While there have been recent curative achievements in hepatitis C virus, curative
ZYNTEGLO is the first gene therapy approved for transfusion-dependent β-thalassemia (TDT) European marketing authorization for ZYNTEGLO follows the fastest assessment of an advanced therapy medicinal product (ATMP) as part of the European Medicines Agency’s Priority Medicines (PRIME) program ZYNTEGLO is bluebird bio’s first gene therapy to gain regulatory approval bluebird bio, Inc. announced today that the European Commission (EC)
Source: Nature Study of almost half a million people links mutation that protects against HIV infection to an earlier death. The scientist who edited the genomes of twin girls in an attempt to make them resistant to HIV might have inadvertently shortened their life expectancy. People with two disabled copies of the CCR5 gene — the version
Encouraging Clinical Data Generated from Ongoing Proof-of-Concept Study Conducted by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget); Enrollment Expected to Complete in the First Half of 2020 Leverages Orchard’s Expertise in Treating Neurometabolic Diseases Using its Investigational Autologous Hematopoietic Stem Cell Gene Therapy Builds Upon Orchard / SR-Tiget Partnership to Develop Novel Gene Therapy Candidates Orchard Therapeutics, Fondazione
