Blog | HemaCare

Minimizing Risk from the Start: HemaCare Publication Spotlights Starting Material Logistics

May 14, 2019 9:21:09 AM / by Nancy Andon, MSc posted in Gene Therapy, Cell Therapy, quality control

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As the industry and regulatory landscape continues to evolve, it’s vital that as an industry, we take a lifecycle approach to risk management –to ensure that we mitigate as much risk as possible all the way from discovery through to commercialization.” - Dr. Dominic Clarke, Global Head of Cell Therapy, HemaCare

Cell and Gene Therapy Insights has just published HemaCare’s white paper on how to manage starting material quality and stability to maximum effect during cell therapy manufacturing. [1] Quality cell therapies can only be created from quality starting materials, yet paradoxically, starting materials are the single greatest source of variability in the cell therapy manufacturing process. Mitigating risk requires minimizing that variability, and also managing shelf-life limitations and biopreservation logistics.

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FDA Policy Changes will Accelerate Cell and Gene Therapy Approvals

Feb 21, 2019 10:02:00 AM / by Nancy Andon, MSc posted in FDA, Gene Therapy, Cell Therapy, GMP Cellular Therapeutics

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The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies. [1] The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years:

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Improving the Efficiency of Clinical Gene Therapy

Jul 23, 2018 10:09:00 AM / by Nancy Andon, MSc posted in Gene Therapy, Bone Marrow, Cord Blood, Stem Cells

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An independent study [1] cites using HemaCare-sourced human cord blood, bone marrow, and mobilized peripheral blood as the starting material in a new strategy for more efficient genetic modification of human stem cells.

The study is based at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Researchers there have been looking into ways to make gene transduction, which is used to introduce an edited copy of a human gene into stem cells, more efficient. The new strategy is part of their goal to improve the potency and long-term engraftment of stem cells used for gene therapy in the clinic.

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Gene Therapy Tackles Sickle-Cell Disease

Jan 8, 2018 10:25:00 AM / by Stacy Matthews Branch, DVM, PhD posted in Gene Therapy, Blood Disorders

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Sickle cell disease (SCD) is a genetic disorder that affects red blood cells. This disease affects people of color more commonly and is due to the effect of abnormally shaped red blood cells (crescent or sickle-shaped instead of disc shaped). The misshapen red blood cells contain a form of hemoglobin, hemoglobin S, resulting from mutation of the beta-globin gene. Hemoglobin S does not hold oxygen efficiently, and the abnormal shape of the red blood cells prevents normal flow through the blood vessels, leading to vessel blockage. This leads to the pain and tissue damage seen in patients with SCD.

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Autologous Hematopoietic Stem Cells Slow Progression of Cerebral ALD

Jun 1, 2016 1:00:51 PM / by Steffen Porwollik posted in Gene Therapy, Bone Marrow, Stem Cell Therapy, Stem Cells

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A clinical trial evaluates the utility of genetically altered autologous hematopoietic stem cells for the treatment of childhood cerebral adrenoleukodystrophy, a deadly disease portrayed in the movie Lorenzo’s oil. The interim results are encouraging.

In 1984, at the age of six, Lorenzo Odone was a happy, bright, precocious kid, who was fluent in English, French and Italian and loved Greek mythology. However, suddenly that year he appeared to become hard of hearing, started to stumble a lot, and displayed mood swings. Concerned, his parents took him to a doctor who quickly ordered a brain scan. The result was devastating – Lorenzo was diagnosed with childhood cerebral adrenoleukodystrophy (CCALD), a disease that is rare, fatal, and untreatable. Doctors gave him two more years to live. The courageous struggle of his parents to find a cure was portrayed in the 1992 movie Lorenzo’s oil. The real life Lorenzo beat the odds by remaining alive until 2008. However, his disease never relented.

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