Bluebird Bio Launches First Gene Therapy for Beta Thalassemia in Germany

Labiotech | January 15, 2020

The US company bluebird bio has launched the first gene therapy for the blood disorder transfusion-dependent beta thalassemia in the EU, with a hospital in Germany becoming its first qualified treatment center. Bluebird bio’s gene therapy, branded as Zynteglo, was given conditional market approval by the EU in June 2019. It was the first gene therapy approved to treat people over 12 years of age with all but the most severe form of transfusion-dependent beta thalassemia, a condition that normally requires the patient to have a lifetime of blood transfusions to stay alive. To be eligible for treatment with the therapy, the patients must have no matching donor available for a stem cell transplant, one of the current methods for treating the condition.

Spotlight

If you’ve been diagnosed with heart failure, your doctor has likely ordered a test of your heart's ejection fraction (EF). The EF is a measure of how efficiently your heart is pumping blood out to the rest of the body. When measuring your EF, your healthcare provider takes measurements of how much blood is in your heart and what percentage of that blood is pumped out to the body.

Spotlight

If you’ve been diagnosed with heart failure, your doctor has likely ordered a test of your heart's ejection fraction (EF). The EF is a measure of how efficiently your heart is pumping blood out to the rest of the body. When measuring your EF, your healthcare provider takes measurements of how much blood is in your heart and what percentage of that blood is pumped out to the body.

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RESEARCH

Vipergen Collaborates with Anavo Therapeutics to Support Phosphatase-Targeted Discovery Programs

Vipergen ApS, Anavo Therapeutics | May 18, 2021

Vipergen, a leading supplier of small-molecule drug discovery services focused on DNA-encoded library (DEL) technologies, has signed a drug discovery service agreement with Anavo Therapeutics. Vipergen will use its patented high-fidelity DNA-encoded library (DEL) technology platforms to provide novel small-molecule drug leads against selected Anavo discovery targets, according to the terms of the agreement. The agreement's financial terms were not disclosed. "Vipergen's novel solutions to DNA-encoded libraries are useful components for ramping up our patented drug development activities against a chosen portfolio of innovative goals," said Anavo CEO Birgit Zech, Ph.D. "We are excited to collaborate with Vipergen to kick-start Anavo's proprietary drug development activities as we strive to create a comprehensive and secure platform for phosphatase drug discovery against all valuable targets in the space." "We are delighted to have been chosen by Anavo, a new company focusing on understanding the full therapeutic potential of human phosphatase biology in the development of new cancer therapies," said Nils Hansen, Ph.D., Chief Executive Officer of Vipergen. "Phosphatases are a rich target space that has been thought to be impenetrable for decades. We are excited to use our DNA-encoded library technology to quickly produce high-quality hits and leads as part of Anavo's comprehensive approach to finding and improving small-molecule phosphatase activity modulators." About Vipergen ApS Vipergen is the world's largest supplier of small-molecule drug discovery services focused on DNA-encoded library (DEL) technology, as well as the first and only organization capable of screening DELs within a living cell. Vipergen's patented portfolio of cutting-edge DEL technologies is made accessible by funded discovery collaborations with leading pharmaceutical and biotechnology firms, including top pharmaceutical companies in the United States, Europe, and Japan.

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CELL AND GENE THERAPY

Tevogen Bio™ Further Strengthens IP Portfolio With Additional Patent for Method of Preparing Its Investigational SARS-CoV-2 Specific T cell Therapy

Tevogen Bio | January 13, 2022

Tevogen Bio, a clinical stage biotechnology company specializing in cell and gene therapies in oncology and viral infections, announced that the U.S. Patent and Trademark Office has granted a new patent for the method of preparing COVID-19 peptide specific cytotoxic T cells for the treatment of COVID-19 infection. The patent further reinforces the biotech pioneer’s expanding IP portfolio. “Omicron’s extensive mutations and its subsequent ability to evade antibodies highlights the importance of Killer T cells, which can still recognize and eliminate virus-infected cells. Millions among us suffer from inadequate T cell response for various reasons. After two years of the pandemic and witnessing the challenges posed by the ever-evolving virus, we must explore scientific options beyond our current approaches. CTL therapeutics for large patient populations is not an easy undertaking, but Tevogen’s ability to manufacture hundreds of doses from a single donor gives me hope,” Tevogen CEO Ryan Saadi, M.D., M.P.H The granted patent covers Tevogen’s investigational allogeneic SARS-CoV-2 specific Cytotoxic CD8+ T Lymphocytes therapy, TVGN-489, for high-risk COVID-19 patients. The product is currently undergoing a proof of concept clinical trial at Jefferson University Hospitals in Philadelphia. Trial details are available at Clinical Trials - Tevogen. About TVGN-489 TVGN-489 is a highly purified, SARS-CoV-2-specific cytotoxic CD8+ T lymphocyte product, which detects targets spread across the entire viral genome. These targeted CTLs are expected to recognize and kill off virally infected cells, allowing the body to replace them with healthy, uninfected cells. TVGN-489 demonstrated strong antiviral activity toward SARS-CoV-2 targets in preclinical studies. About Tevogen Bio Tevogen Bio is driven by a team of distinguished scientists and highly experienced biopharmaceutical leaders who have successfully developed and commercialized multiple franchises. Tevogen’s leadership believes that accessible personalized immunotherapies are the next frontier of medicine, and that disruptive business models are required to sustain medical innovation in the post-pandemic world.

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MEDTECH

Halo Labs Launches the Aura GT™ for Gene Therapy Product Quality

Halo Labs | October 05, 2021

Halo Labs, a life science instrumentation company developing tools for biologics researchers, today announced it has launched its latest product, Aura GT. Aura GT is part of the next generation in Halo Labs' popular family of instruments. Using Backgrounded Membrane Imaging (BMI) and Fluorescence Membrane Microscopy (FMM), Aura GT can distinguish aggregated capsids from contaminants, carrier proteins, and other particles that might adversely harm patients. Unique to Aura GT is the fact that it can also identify DNA leakage in gene therapy products so the mechanism of aggregation can be determined and corrected. Aura GT is going to completely revolutionize gene therapy product development. Gene therapy products are inherently unstable, which is a huge challenge. Up until now, there has been no effective way to correlate capsid aggregation with DNA leakage or empty and full capsids, In one assay, Aura GT can quantify capsid aggregates, distinguish them from protein aggregates, and determine if the aggregation is exacerbated by DNA leaking. - Bernardo Cordovez, Chief Science Officer at Halo Labs. We are very excited to be launching our second instrument this year specifically developed for the cell and gene therapy market, Halo Labs has quickly become a leader in gene therapy analytics because we can answer many questions with very little volume. While other subvisible particle analyzers only do one thing, Aura systems offer a complete multi-assay platform that can be used to fully characterize gene therapy products from early development through lot release. - Rick Gordon, CEO of Halo Labs. About Halo Labs Halo Labs in Burlingame, CA is a venture-backed scientific instrumentation and consumable company that commercializes high throughput biopharmaceutical formulation, stability, and product quality control tools for aggregate and subvisible particle analysis using state-of-the-art custom optics and image processing techniques. Biopharmaceutical companies around the globe depend on Halo Labs for unprecedented insights into their drug products.

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