Gene Therapy Programs to be on the Verge of Delivering Remarkable Remedies for Diseases That Have Pr

Friday, 19 April 2019 06:11.PM

Several revolutionary gene therapies appear to be on the verge of delivering remarkable remedies for diseases that have previously been viewed as untreatable.

- Gene therapy is the next great leap forward in medicine, with several noteworthy programs underway looking to cure the incurable
- FDA expects 200 new gene and cell therapy INDs in next two years
- Genprex — and its initial product candidate Oncoprex — is pioneering a new approach to treating cancer.

Built on decades of scientific research and innovation, gene therapy is the next frontier of medicine, determined to provide remedies for previously untreatable diseases and unmet medical needs.

Disease has been a subject of both fear and fascination throughout history. With each medical discovery, the world has moved a step closer to understanding not only the complex mysteries of disease but also the intricacies of curative therapies. Over the course of centuries, medical science has developed medicines and treatments that have proved instrumental in saving and improving the quality of lives.

Now gene therapy, arguably the next great leap forward in medicine, may hold the promise of curing the incurable. Gene therapy refers to the therapeutic delivery of nucleic acid (DNA and RNA) into a patient's cells as a method to treat disease. Originally conceptualized in 1972, the first gene therapy accepted as a success in a trial took place in 1990. After 45 years of global research efforts, the first gene therapy to be approved for use was in 2012 in Europe.

In the past, gene therapies have been slow to gain government approval. However, as an indication of the efficacy and potential of gene therapy, the FDA announced in January it expects to receive some 200 new gene and cell therapy INDs in the next two years and plans to expedite the processing of these requests. Not since the advent of antibiotics in the 1920s has there been such excitement and promise in medicine.

The FDA is not the only indication that is pointing toward a tsunami of gene therapy innovations. Financial market projections tell the same tale. For comparison, the global immunotherapy drugs market was valued at $132.59 billion in 2017 and is expected to achieve growth of $385.46 billion by 2025 at a CAGR of 14.27% from 2018 to 2025. These eye-popping numbers portend explosive growth and opportunity both now and well into the future.