Category ADVANCED THERAPY MEDICINAL PRODUCT

PIER MARIA FORNASARI
2022-02-19

Gene Therapies Developers slowly emerge from a pandemic: a new ebook published by BioProcess Insider

This eBook gauges shifting expectations for the gene therapy industry amid the COVID-related uncertainties and clinical setbacks of the past couple years. BioProcess Insider founding editor Dan Stanton reports on the January 2022 Phacilitate Advanced Therapies Week event, specifically a standing presentation on the 10 most important industry drivers from the past year. Since 2017, advancements in

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PIER MARIA FORNASARI
2022-02-06

Gene therapy in transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides

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PIER MARIA FORNASARI
2022-01-25

Landing therapeutic genes safely in the human genome

Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient, and predictable gene and cell therapies. Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and

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PIER MARIA FORNASARI
2022-01-11

Engineered Mesenchymal Stem Cells as a Biotherapy Platform for Targeted Photodynamic Immunotherapy of Breast Cancer

Interleukin-12 (IL12) is a pleiotropic cytokine with promising prospects for cancer immunotherapy. Though IL12 gene-based therapy could overcome the fatal hurdle of severe systemic toxicity, targeted delivery and tumor-located expression of IL12 gene remain the challenging issues yet to be solved. Photo-immunotherapy emerging as a novel and precise therapeutic strategy, which elaborately combines immune-activating agents

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PIER MARIA FORNASARI
2021-12-03

Robotic high-throughput biomanufacturing and functional differentiation of human pluripotent stem cells using the CompacT SelecT (CTST) platform

The CompacT SelecT (CTST) platform is a modular robotic system that integrates a full range of cell culture proceduresunder sterile conditions that mimic the manual cell culture process. These procedures include automated handling of different cell culture vessels, pipetting large and small volumes at adjustable speeds, cell counting, cell viability analysis, cell density assessment, microscopic

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No Comments InADVANCED THERAPY MEDICINAL PRODUCT REGENERATIVE MEDICINE
PIER MARIA FORNASARI
2021-12-01

A new CRB Life Sciences Report Highlights rapid advances in the development of cell and gene therapies

Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.” That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and

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No Comments InADVANCED THERAPY MEDICINAL PRODUCT REGENERATIVE MEDICINE
PIER MARIA FORNASARI
2021-11-17

Monash University study uncovers a new understanding of how mesenchymal stromal cells benefit patients in cell therapy

The therapeutic benefit to patients receiving mesenchymal stromal cell (MSC) therapy is not because the injected cells remain viable, but because of cell death, researchers at the Monash Biomedicine Discovery Institute (BDI) have found. In recent years, significant efforts have been made to develop stem cell-based therapies for difficult-to-treat diseases. MSC therapy is regenerative cell-based

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PIER MARIA FORNASARI
2021-11-10

Cell therapies, Gene therapy and CAR-T cells highlighted in EFPIA Pipeline Review 2021 Update

The new EFPIA Pipeline Review reveals that infectious, inflammatory, cancer, Alzheimer’s and rare disesases areas are among the top priorities of researchers running clinical trials today to investigate new medicines and vaccines. The comprehensive report found that in 2020 alone, around 5,000 clinical trials were launched – despite the disruption caused by the COVID-19 pandemic. In fact, the volume of trials has

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PIER MARIA FORNASARI
2021-07-16

EMA PRAC Concludes There is No Link Between Zynteglo and AML

EMA’s safety committee, PRAC, has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). The European Medicines Agency’s (EMA’s) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known

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PIER MARIA FORNASARI
2021-04-15

Risdiplam improves motor function and survival in infants with SMA

PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial of Evrysdi (risdiplam), which demonstrated that infants with type 1 spinal muscular atrophy (SMA) continued to show improvements in survival and key motor milestones after two years of treatment. PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial

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