A genetic mutation has caused around 6800 rare diseases, and the majority of them do not have any approved treatment. A few biopharmaceutical companies are developing possible cell and gene therapies to meet the clinical requirements. These cell and gene therapies are licensed for managing rare diseases and can be used to administer closed systems like the brain or eye, where the immune system protects the viral vector delivery vehicles. The cell and gene therapy holds incredible clinical potential for neurological disorders, heart diseases, eye diseases, and hemophilia A and B. The rapid penetration of cell and gene therapy in global clinical trials is a testimony to its great potential and growing milestones. Some pharmaceutical companies that offer cell and gene therapies are blooming considerably. Type 1 diabetes, osteoarthritis, Alzheimer’s disease, amyotrophic lateral sclerosis, Parkinson’s disease, and spinal cord injuries, are some key diseases that are attracting contract research organizations.
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Support by Government Helps Market Players Overcome Challenges
The cell and gene therapy clinical trials market is driven by increased patient demand for therapies, the growing interest in better cancer treatment, R&D funding, and a positive regulatory environment. While fighting against deadly diseases, the therapy presents a new innovating wave in the life sciences industry. It directly targets disease drivers, thus works efficiently.
But the products used for cell and gene therapy clinical trials are costly. This factor curbs the development in the industry. The clinical trials for cell and gene therapy are relatively less elaborate and need less time but complex compared to the traditional biotherapeutics process. Multiple dysfunctional genes’ diseases such as Alzheimer’s and diabetes require cell and gene therapies to target multiple genes.
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Despite challenges, venture capitalists eagerly spend dollars in cell and gene therapy companies. Due to COVID – 19, there is a delay in clinical trials, thereby impeding companies to raise finance and investors for their programs. To overcome this, the governments are taking initiatives to strengthen the cell and gene therapy market.