This site is an educational service brought to you by BioMarin and is intended for US residents only. Many gene therapies for hemophilia A and B are being studied in people to determine if they are safe and effective. No gene therapies for hemophilia have been approved for use or determined to be safe or effective.

What is gene
therapy?

A genetic condition could potentially have a genetic solution, right? This logical thinking gave birth to the first research in gene therapy more than 50 years ago. Now being evaluated in multiple clinical trials for a range of genetic conditions to determine the possible benefits and risks, gene therapy research has the potential to bring an entirely new option to people with specific genetic conditions and those who support and care for them.

GENE THERAPY SEEKS TO ADDRESS GENETIC MUTATIONS

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Inside a cell

Simply put, gene therapy is being researched in clinical studies as a novel method that attempts to use genes to treat or prevent disease.

METHODS OF GENE THERAPY BEING EXPLORED

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Gene transfer therapy

A few different approaches to gene therapy are being explored. Gene therapy may involve attempting to repair or replace a mutated gene, disabling a mutated gene that is causing trouble, or introducing a functional copy of the gene into the body to help the body produce the affected protein.

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Ex vivo gene therapy

  • With the method of gene therapy called gene transfer, a functional gene is inserted into a cell with the intent that it will work in place of the mutated gene. Viral, chemical, and physical methods are being explored for transferring genes. The transfer of the new gene occurs inside the body (in vivo) after systemic delivery, often using an IV infusion. The original genetic material in the chromosomes is intended to be left unchanged. This means the mutated gene would still be there and can still be passed on to a person’s offspring
  • In ex vivo gene therapy, a type of cell-based therapy, the process happens outside the body. First, affected cells are removed from the body via a biopsy. In the lab, functional genetic material is introduced into the cells and are then delivered back into the patient’s body
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Gene editing

  • In gene editing, the idea is to make changes to the original DNA. This technique makes it possible to repair the original DNA or add new DNA in a specific location. Zinc finger nucleases and CRISPR (clustered regularly interspaced short palindromic repeats) are methods of gene editing being researched

50+ YEARS OF RESEARCH

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Gene therapy has been explored as a potential treatment approach for well over 50 years. In the past decade, the FDA and EMA (European Medicines Agency) have approved gene therapies for genetic conditions. Gene therapy is currently being researched in many clinical trials for various genetic disorders, including hemophilia A and B and has not been determined to be safe or effective.

To explore future, current, and past research in gene therapy, visit ClinicalTrials.gov

GENE THERAPY TIMELINE

1972 —Concept of gene therapy considered as a form of treatment in the journal Science
1984 —Dr. Gordon Vehar publishes a paper reporting successful factor VIII cloning
1990 —First gene therapy trial in humans
1999 —Lessons learned regarding risks related to potential for severe immune response in early gene therapy trial with non-adeno-associated virus (AAV) vector
2003 —The Human Genome Project is completed
2003 —China approves the first gene therapy for the treatment of head and neck cancers
2005 —First gene therapy trial in hemophilia B using AAV vector technology
2015 —First gene therapy trial in hemophilia A using AAV vector technology
2017 —The first gene therapy, for a genetic disease that causes blindness, is approved in the United States
Future —Additional gene therapies are being researched

ONGOING RESEARCH

Gene therapy is in ongoing clinical trial research to determine the potential risks and benefits of treatment.

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