Blog | HemaCare

HemaCare Aids Strategy for Boosting Gene Therapy Efficiency

May 21, 2019 10:06:00 AM / by Nancy Andon, MSc posted in gene therapy, bone marrow stem cells, CD34, stem cells, mobilized peripheral blood

0 Comments

An independent publication cites the use of bone marrow and mobilized peripheral blood sourced from HemaCare in a study aimed at improving the efficiency of resource consumption in gene therapy.

The publication, published in the journal Molecular Therapy, focuses on the use of lentiviral vector (LV) to introduce corrected versions of defective genes into stem cells. The goal of gene therapy is to transplant these stem cells into people suffering from genetic disease, in hopes of restoring the function of the faulty gene. One of the main challenges in this clinical strategy is the cost; substantial amounts of LV are needed to modify a therapeutically effective dose of stem cells.

Read More

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, cell therapy starting material

0 Comments

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.

Read More

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, GMP, cell therapy, gmp-compliant

0 Comments

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:

Read More

Improving the Efficiency of Clinical Gene Therapy

Jul 23, 2018 10:09:00 AM / by Nancy Andon, MSc posted in engraftment, gene therapy, gene editing, hematopoietic stem cells, progenitor cells

0 Comments

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.

Read More

Gene Therapy Tackles Sickle-Cell Disease

Jan 8, 2018 10:25:00 AM / by Stacy Matthews Branch, DVM, PhD posted in gene therapy, sickle cell disease

0 Comments

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.

Read More

Subscribe Here!

Posts by Topic

see all

Recent Posts