Designed with your teams in mind, HemDifferently created the following checklists as tools in supporting their understanding of gene therapy research. Each individual resource guide can benefit your team by highlighting gene therapy research and how it applies to different team structures.
This checklist is your tool in understanding how multidisciplinary teams can be formed, and the practical knowledge they may need on gene therapy.
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Developed for all nurse coordinators and program managers, this checklist helps teams prep and consider how gene therapies processes could affect institutions.
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This pharmacist centric checklist covers potential biosafety considerations and potential infrastructure setup, from procedure policies to tasks on infusion day.
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The prescriber’s checklist will highlight information to consider such as identifying who will take the lead and what requirements are necessary, defining SOPs, and pre infusion through to post infusion follow up.
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Not planning to infuse at your institution? This checklist covers what to consider for referring your patients including identifying an institution that provides gene therapy and what to consider for both coordinating care and follow up.
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This checklist identifies information to consider as your institution designates responsibility for access and reimbursement.
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A simple one page diagram helping patients and caregivers to understand the 5 steps involved in gene transfer research.
A comprehensive brochure for patients and caregivers that goes into the science and research behind gene therapy.
Annette von Drygalski, MD, PharmD, RMSK
Director, Hemophilia and Thrombosis Treatment Center at University of California San Diego Health
Professor of Clinical Medicine, UC San Diego, California
With 2,500 studies completed, ongoing, or initiated worldwide, gene therapy is being evaluated to determine the benefits and risks for a range of genetic conditions, including hemophilia A and B. Some gene therapies have already been approved for treatment of certain conditions, including a genetic eye disease and spinal muscular atrophy. No gene therapies for hemophilia A or B have been approved for use or determined to be safe or effective.
There are three different types of gene therapy being researched, including gene editing, cell therapy, and gene transfer. At HemDifferently, we’re particularly interested in the method of gene transfer.
In gene transfer research, a working copy of a mutated gene is designed in a laboratory to provide instructions for the body to make the proteins it needs. That working gene is placed in a protective viral shell and delivered through one-time infusion. Once inside the body, the functional gene is designed to provide instructions for the body to produce the missing or mutated gene. Ongoing research monitors clinical trial participants to gather data to better understand short as well as long term safety and efficacy.
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