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We are transforming our innovative gene technology into potentially life-changing treatments.
Gene therapy offers the potential for life-long cures by correcting for the missing or mutated genes that underlie genetic diseases. Safe and efficacious gene therapy treatments are relatively new, however gene therapy has been investigated and pursued by scientists for the past 50 years.
The breakthrough came from our founder and CSO Professor Amit Nathwani in 2010. Professor Nathwani used a gene therapy treatment on people living with haemophilia B that produced transformative results. The techniques used in the haemophilia B treatment have been transferable, creating a new wave of gene therapy products which Freeline is developing to treat a variety of genetic diseases.
We are transforming our innovative gene technology into potentially life-changing treatments.
Gene therapy offers potential cures by correcting for the missing or mutated genes that underlie genetic diseases. Safe and efficacious gene therapy treatments are relatively new; however, gene therapy has been investigated and pursued by scientists for the past 50 years.
What is a gene?
Genes are units of DNA inherited from one’s parents that store information and instructions on how to make specific proteins. These proteins express an individual’s hereditary characteristics and control the growth and development of the body’s cells. Across the species, humans share about 99% of the same genes with only about 1% variance. While genetic variance is normal, genetic mutation can be harmful, leading to serious health impacts. When a gene is mutated, it may not produce normal protein levels, it may produce a mutated protein lacking normal function, or no protein at all.
What is a genetic disease?
A genetic disease is caused by one or more mutations in the DNA. Some diseases can be identified by a single mutated gene and others are associated with multiple gene mutations. Genetic diseases are usually inherited, but can also be acquired through genes mutating randomly or triggered by lifestyle and environmental factors.
What is gene therapy?
Current efficacious gene therapies target diseases with a single gene mutation. Many of these therapies use a specialised carrier (vector) to deliver a functioning gene to targeted cells to replace the mutated gene. Once united with the targeted cells, the replacement gene will gradually start to produce the missing or malfunctioning protein.
An animation explaining what gene therapy is, how it has evolved over 50 years and the goal of providing a long-term cure for patients who receive treatment.
Freeline gene therapy
Freeline uses gene therapy technology to target the unmet needs of genetic diseases using a vector that targets the liver. From the liver, the newly synthesized protein will be secreted to the circulation where it can reach targeted cells throughout the body. Freeline uses an in vivo approach to deliver the gene directly into the human cells of the liver to enable a high level of secretion. Freeline is currently focused on developing a liver-based gene therapy for Gaucher disease.
The drug development process
To develop a gene therapy treatment, scientists discover what gene(s) are missing, mutated or defective, and if corrected could cure disease. Next, researchers find the most effective delivery vehicle to carry and incorporate the new gene into the patient and to the target cells.
Before any gene therapy is used to treat humans, the therapy undergoes rigorous testing and must satisfy strict safety, quality, efficacy and ethical requirements.
Preclinical
During preclinical testing, the efficacy and the safety of the vector is initially tested on isolated human cells. Next, the vector is tested in live whole organisms. This is a necessary step to determine the efficacy and safety of the gene therapy in a whole organism, as it may differ from tests in isolated target cells.
Clinical
During the clinical stage, treatments are incrementally tested on humans for safety and efficacy. The first objective is to confirm product safety; if there is a notable safety concern, the trial cannot progress. In addition to safety, efficacy parameters are evaluated. Trials will start with small samples sizes and low vector dosages, and increase incrementally if safety and efficacy are confirmed throughout.
Production
To perform preclinical and clinical tasks and beyond into commercial, it is important to be able to manufacture large quantities of the gene therapy product. Living cells are used to grow the AAV vector, followed by purification steps to isolate the vector from the living production cells.
These steps are carried out under tight quality controls and in exceptionally clean facilities to ensure the purity and quality of the product. Finally, the AAV vector is formulated into a solution that makes it injectable and it is filled into sterile vials for storing.
Treatment & results
The treatment consists of a simple one-time injection that is usually performed over a short amount of time in a hospital setting.
Once the AAV has delivered the replacement gene to the patient’s targeted cells, over the course of weeks or months (depending on the individual) the targeted cells will gradually start to produce the missing or faulty protein.
Clinical data from patients receiving treatment with gene therapy have shown potential for long-lasting curative effect for various diseases. The goal of gene therapy treatment is to provide a long-term cure rather than short-term palliative treatments.
