Category GENE THERAPY RARE DISEASES

Sangamo and Pfizer report data for haemophilia A gene therapy

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

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How will we pay for the coming generation of potentially curative gene therapies? Bill Cassidy US Senator proposals

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

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Gene Therapies clinical trials see 17% jump year-over-year, with two approvals near and pipelines expanding

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

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Gene Therapy: Commercial Challenges And Strategic Choices

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

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bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

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)

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Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I

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

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ICER Comments on the FDA Approval of Zolgensma for the Treatment of Spinal Muscular Atrophy

In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma®, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, the Institute for Clinical and Economic Review (ICER) today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA). Since the publication of ICER’s Final Evidence Report on

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