Dec 11, 2023
InsightAce Analytic | Clinical Trials , virtual clinical trials , remote clinical trials , market , industry , business , forecast , consulting ,
Gene Therapy Industry Outlook.
Gene therapy is a type of medical treatment that involves introducing or altering genetic material in an individual's cells in order to treat or prevent a disease. It has the potential to revolutionize the way we treat diseases by addressing the underlying genetic causes of conditions, rather than simply treating the symptoms.
Since the launch of the first gene therapy trial in 1990, when scientists at the NIH used gene therapy to treat a severe combined immunodeficiency (SCID); numerous gene therapy trials have been conducted for a variety of different genetic diseases, including cancer, cystic fibrosis, hemophilia, cardiovascular disease, and neurological disorders. In 2017, the US FDA approved the first gene therapy called Luxturna for use in the United States. This therapy is being used to treat a rare genetic disorder that causes vision loss in children and adults.
While gene therapy has shown promise as a treatment for many genetic diseases, it is still considered as a new and experimental form of the treatment. To fully understand its potential risks and benefits more research is being conducted with the new technologies. Rapidly changing technologies, ongoing clinical trials, and regulatory changes, are making this industry more dynamic.
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Some of the major trends in the gene therapy industry that are worth noting:
One trend in the gene therapy industry is the increasing use of CRISPR technology. This technology allows for precise and efficient editing of the genome, enabling researchers to target specific genetic mutations that cause diseases.
The future of CRISPR looks bright, as it has already been shown to be effective in various clinical trials. In 2020, the first CRISPR-based gene therapy, called Zynteglo, was approved by the European Medicines Agency for the treatment of a rare genetic blood disorder called beta thalassemia. The therapy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, has also been granted breakthrough therapy designation by the U.S. Food and Drug Administration.
Several other CRISPR-based therapies are currently in clinical trials, including treatments for sickle cell disease, Duchenne muscular dystrophy, and cancer. In addition, researchers are working on developing CRISPR therapies for a wide range of diseases, including neurological disorders, cardiovascular diseases, and autoimmune conditions.
It is expected that more CRISPR-based therapies will be approved and launched in the coming years, as the technology continues to advance and more clinical trials reach completion. In the long term, it is possible that CRISPR could become a standard treatment for many genetic diseases.
Some potential future applications of CRISPR technology include:
1. Precision medicine: CRISPR technology can be used to tailor therapies to the specific genetic makeup of individual patients, which could improve the effectiveness of treatments and reduce the risk of side effects.
2. Gene editing: CRISPR technology can be used to edit specific genetic mutations that cause diseases, including genetic disorders and cancer.
3. Gene therapy: CRISPR technology can be used to insert functional genes into cells to replace or repair damaged or mutated genes. This could be used to treat a wide range of diseases, including genetic disorders, cancer, and cardiovascular diseases.
4. Agricultural applications: CRISPR technology could be used to improve crop yields, increase resistance to pests and diseases, and reduce the need for fertilizers and pesticides.
5. Environmental applications: CRISPR technology could be used to remove harmful genetic mutations from species, such as invasive species or species that are at risk of extinction.
Overall, the potential applications of CRISPR technology are vast and varied, and it is likely that we will see it used in many different fields in the future.
Gene Therapy in Rare Diseases:
Rare diseases, also known as orphan diseases, are medical conditions that affect a small percentage of the population (1 or less in 2000 people). These conditions are often genetic in nature and can be caused by a mutation or alteration in a single gene. Many rare diseases are severe or life-threatening, and many do not have effective treatments or cures.
Till date, there are around 7000 rare diseases such as cystic fibrosis, muscular dystrophy, sickle cell anemia, etc. have been identified but only few hundreds have treatments approved. The rarity of these conditions can make it difficult for patients to find doctors with experience in treating the specific disease, and it can also make it challenging for pharmaceutical companies to justify the investment in developing treatments for such small patient populations.
Advocacy groups and government initiatives, such as the Orphan Drug Act in the United States, have been established to support research and development of treatments for rare diseases and to provide resources and support for individuals and families affected by rare diseases.
Gene therapy is an important option for treating rare diseases, as the majority of rare diseases are caused by a single defective gene. Unlike traditional medications, which only help manage symptoms, gene therapy has the potential to cure the disease by replacing the defective gene.
So far, the US Food and Drug Administration (FDA) has approved two genetic therapies for rare diseases: Zolgensma for spinal muscular atrophy and Luxturna for leber congenital amaurosis. Both of these therapies have been designated as orphan drugs, which are drugs developed specifically for rare diseases. In addition to these two therapies, the European Medicines Agency (EMA) has also approved and given orphan medicine status to Strimvelis for the treatment of severe combined immunodeficiency (ADA-SCID) and Zynteglo for beta-thalassemia.
However, there are more than 10 drugs in the clinical trials for rare disease treatment and there has been a significant increase in investment in research on gene therapy for rare disease treatment in recent years. Further, a partnership between the National Institutes of Health, the U.S. Food and Drug Administration, 10 pharmaceutical companies, and five non-profit organizations has been formed to speed up the development of gene therapies.
Gene therapy has the potential to revolutionize the treatment of rare diseases by offering a one-time treatment for a rare disease, rather than ongoing treatment with medications or other therapies. This can be particularly beneficial for rare diseases that affect children, as it can potentially improve their quality of life and reduce the burden of lifelong treatment.Gene therapy also has the potential to be personalized to the specific genetic mutation that is causing a rare disease. This allows for the development of targeted therapies that are more likely to be effective for a particular patient, which can be particularly beneficial for rare diseases where there are few treatment options available.
In addition, gene therapy has the potential to be more cost-effective in the long term compared to traditional therapies, as it may only require a one-time treatment rather than ongoing treatment with medications or other therapies.
Overall, gene therapy has the potential to revolutionize the treatment of rare diseases by offering targeted, effective therapies that have the potential to improve the quality of life for patients and their families.
Gene Therapy for Retinal Diseases:
Retinal disease refers to a group of conditions that affect the retina, the light-sensitive tissue at the back of the eye and retinal diseases can cause vision loss or blindness. According to WHO, worldwide around 1 billion people are affected with some sort of retinal disease and of those at least 400 million people are affected with the conditions which currently do not have cure. For instance,
1. Age-related macular degeneration (AMD): AMD is a leading cause of vision loss in people over the age of 50. It is estimated to affect over 196 million people worldwide.
2. Diabetic retinopathy: Diabetic retinopathy is a complication of diabetes that affects the retina. It is estimated to affect over 93 million people worldwide.
3. Glaucoma: Glaucoma is a group of eye conditions that damage the optic nerve and can lead to vision loss. It is estimated to affect over 76 million people worldwide.
4. Cataracts: Cataracts are cloudy areas in the lens of the eye that can cause vision loss. They are the most common cause of vision loss in the world, and it is estimated that they affect over 20 million people worldwide.
5. Retinitis pigmentosa (RP): RP is a genetic disorder that causes progressive vision loss due to the degeneration of cells in the retina. It is estimated to affect approximately 1.5 million people worldwide.
Currently, there is no cure for these diseases, but treatment options are available to slow the progression of the disease and improve vision. Amongst these, age-related macular degeneration (AMD), and retinitis pigmentosa (RP) are caused by mutations in specific genes that affect the function of the retina. And gene therapy is being explored as a potential treatment for these and a number of similar retinal diseases. As of December 2022, 36 clinical trials being conducted where gene therapy-based treatment for retinal disease is being studied and 6 of these are already in phase 3.
In the future, gene therapy for retinal diseases is likely to continue to evolve and advance as researchers work to develop more effective approaches and treatments. Currently, the majority of gene therapy trials are focused on gene augmentation strategies, which aim to provide functional proteins by expressing the defective gene. However, in disorders that are caused by gain-of-function genetic errors, it will be necessary to develop strategies for gene inhibition in order to effectively treat these conditions. The CRISPR/CAS9 gene editing technique has the potential to be effective in achieving this goal.
In addition, the development of gene therapies for polygenic diseases, such as AMD or retinopathy of prematurity, may require the use of multiple approaches, such as targeting neurotrophic factors or inhibiting degenerative pathways.
AAVs are currently the most commonly used vectors in ophthalmic gene therapy due to their low immunogenicity and reduced rate of side effects, but their capacity for genetic data transport is limited to 5 kb DNA. Numerous important genes that encode functional and structural proteins are larger; thus, a different vector system will be required. However, due to complex and delicate nature of retinal tissue it can be difficult to deliver the gene to the right cells without damaging the surrounding tissue. Therefore, researchers are likely to continue to explore and evaluate the use of different vectors and delivery methods including viral vectors, non-viral vectors, and gene editing technologies in order to optimize the safety and effectiveness of gene therapies for retinal diseases.
Another challenge is the potential for immune responses to the therapeutic gene or the vector used to deliver it. The immune system may recognize the therapeutic gene or vector as foreign and mount an immune response against it, which could limit the effectiveness of the treatment. Researchers are working to develop strategies to minimize the risk of immune responses, such as using vectors that are less likely to trigger an immune response or pre-treating patients with immune-suppressing drugs.
Overall, the future outlook for gene therapy for retinal diseases is promising, but there are still many challenges to be addressed before it can be widely adopted as a clinical treatment. Researchers are actively working to overcome these challenges and advance the field of gene therapy, and it is likely that we will see continued progress in the coming years.
Gene Therapy for Blood Disorders:
A blood disorder is a medical condition that affects the production, circulation, or function of blood cells or components of the blood. These disorders can range from mild to severe and can cause symptoms such as fatigue, weakness, shortness of breath, bruising or bleeding easily, or anemia. Some examples of blood disorders include anemia, leukemias, thalassemia, sickle cell anemia, lymphomas, bleeding disorders, and clotting disorders.
Amongst these, Sickle Cell Anemia has affected 300 million, Thalassemia affected 50 million, Lukemia affected 4.3 million, lymphoma affected 1.9 million and hemophilia A affected 0.4 million. These disorders/conditions currently do not have a cure but a treatment that can help to manage symptoms, and prevent complications. However, gene therapy is a promising approach in treating many of these blood disorders that are caused by genetic mutations.
Currently the most common blood disorders where gene therapy is being used are hemophilia A, and sickle cell anemia. Gene therapy for thalassemia is another area of research that has shown promise. Gene therapy is also being researched as a potential treatment for other blood disorders, including beta thalassemia, dysfibrinogenemia, and lysosomal storage disorders.
Overall, gene therapy has shown promise as a potential treatment for a variety of blood disorders. However, there are still many challenges and limitations to overcome in order to make gene therapy a more widely available and effective treatment option for these disorders.
Gene therapy is a rapidly evolving field with the potential to revolutionize the treatment of blood disorders. In the future, gene therapy could provide a permanent cure for a wide range of blood disorders that are currently treated with medications, blood transfusions, and bone marrow transplants. This could significantly improve the quality of life for patients with these disorders and reduce the burden on the healthcare system.
One of the most promising areas of gene therapy research for blood disorders is the treatment of hemophilia A. In the future, gene therapy could provide a one-time, permanent cure for hemophilia A by delivering a functional copy of the Factor VIII gene to the patient's cells, thereby correcting the deficiency and allowing the body to produce its own clotting protein. This could significantly reduce the need for frequent infusions of Factor VIII concentrates and the associated costs and risks. In addition, gene therapy could potentially improve the quality of life for patients with hemophilia A by reducing the frequency and severity of bleeding episodes and the risk of bleeding-related complications.
Gene therapy could also be used to treat other bleeding disorders, such as von Willebrand disease and Factor XIII deficiency, which are currently treated with medications and blood transfusions. In the future, gene therapy could potentially provide a permanent cure for these disorders by correcting the underlying genetic defects and allowing the body to produce its own clotting proteins. This could significantly improve the quality of life for patients with these disorders and reduce the burden on the healthcare system.
Another area of promise for gene therapy in blood disorders is the treatment of sickle cell anemia. In the future, gene therapy could provide a one-time, permanent cure for sickle cell anemia by correcting the mutation in the HBB gene and allowing the body to produce normal hemoglobin. This could significantly reduce the need for frequent blood transfusions and bone marrow transplants and the associated costs and risks. In addition, gene therapy could potentially improve the quality of life for patients with sickle cell anemia by reducing the frequency and severity of anemia and the risk of complications such as infections and pain crises.
Gene therapy could also be used to treat other blood disorders that are caused by a deficiency in the production of hemoglobin, such as thalassemia and beta thalassemia. In the future, gene therapy could potentially provide a permanent cure for these disorders by correcting the underlying genetic defects and allowing the body to produce normal hemoglobin. This could significantly improve the quality of life for patients with these disorders and reduce the burden on the healthcare system.
In addition to treating blood disorders, gene therapy could also be used to treat other conditions that are related to blood disorders, such as cardiovascular disease and stroke. In the future, gene therapy could potentially be used to modify the genes that are associated with these conditions and prevent or reverse the development of these conditions. This could significantly improve the quality of life for patients with these conditions and reduce the burden on the healthcare system.
Overall, gene therapy has the potential to revolutionize the treatment of blood disorders and related conditions in the future. While there are still many challenges and obstacles to overcome, the progress that has been made so far is very promising and suggests that gene therapy could become a viable treatment option for a wide range of blood disorders in the near future.