Gene therapy is a highly promising new field of medicine in which normal genes are introduced into the body to treat, and potentially cure, genetic diseases (ie diseases caused by defective genes in the body). A large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4D products. Our products are unlocking the full potential of gene therapy.
Humans are afflicted by thousands of genetic diseases. Most genetic diseases do not have safe and effective treatments available. Hundreds of thousands of people, mainly children, suffer greatly; their disease symptoms are determined by which of the body’s ~25,000 genes is defective. Some lose their sight, others bleed constantly, and others lose the ability to simply walk or breath normally. Many of these patients even die at a young age. In a few diseases, children can be injected frequently with replacement proteins that can partially stem the tide; nevertheless, the disease progresses steadily and the child’s quality of life is progressively degraded.
For most of the children with these diseases, no treatment is available; the disease rages on until the pursuit of happiness, and even life, are lost. We need effective therapies for these kids. We need cures for these kids. This is what 4D fights for every day.
What are “genes”, and why does it matter when they are defective in a genetic disease?
Genes are blueprints for the manufacturing of proteins by cells in the body; humans have about 25,000 genes for the same number of proteins. Each one is essential for a normal life. While every cell in the body has all of these genes, it only expresses (ie turns on) a small subset required for its specialized function in the body. These genes are the cells’ “software”, coding for how each cell should function through the action of proteins. The proteins that genes encode for are the “hardware” that carry out almost every function in the body, from structures (eg bone, cartilage) to enzymes (eg that digest food) to messengers (eg hormones).
Genetic diseases (disease of a specific gene):
When a gene is defective, either missing or “mutated”, the result is that the corresponding normal functional protein will not be present in the cells where it should be. Since a normal functional protein is missing, or at too low a level, the affected person suffers. If the gene for a blood clotting factor is defective, the person lacks a normal blood clotting protein and they will have problems with bleeding (eg hemophilia A and B). If the gene for a muscle factor is defective, the person lacks a normal muscle strength protein and they will have problems with muscle wasting and even with normal activities (eg muscular dystrophy). If the gene for a specific lung factor is defective, the person lacks a normal lung maintenace protein and they will have problems with breathing and lung infections (eg cystic fibrosis).
What is the good news? We are close to cures.
There is good news and reason for hope:
- The human genome revolution:
First, we are in the midst of an unprecedented revolution in our understanding of human genes. The entire human genome (all genes in our DNA) has now been sequenced. The time and cost of sequencing a person’s genes (their DNA) has dropped dramatically in just the last decade. These technological advances will continue to accelerate our understanding of genes and genetic diseases. Every person with a genetic disease will have the opportunity to have their genes sequenced to identify the defective gene causing disease.
- The genetic engineering revolution:
Scientists and doctors in the gene therapy community have discovered and perfect many clever ways to insert a gene into a delivery vehicle (vector) and to have it activated in the right cells in the body. We can cure almost any genetic defect now in a petri dish of cells in the lab. If we could do the same in the human body we could cure people with genetic diseases.
- 4D is delivering the final piece of the puzzle that is necessary for cures:
Given our understanding of genetic diseases now, and our gene therapy technology, when will cures become a reality. Simply put, once we have highly efficient and customized delivery vehicles (vectors) to deliver the normal genes to the diseased cells in the body, we can turn the hope for cures into a reality. This is the problem that 4D is working to solve every day. We are working to create new customized delivery vehicles (vectors) to shuttle genes into the cells in any organ in the body. This 4D “library” of vectors will soon include several for the liver, several for the brain, several for the heart, the eye, muscle and so on. Doctors will be able to hand pick a vector for any patient based on the organ (a collection of cells that has a function) that is diseased. These 4D vectors will be markedly superior to the current first-generation vectors because they will be selected form a much greater vector pool (see Therapeutic Vector Evolution). We are working to create industry-leading delivery vectors every single day.