According to a story from andreeger.com, gene therapy, a type of medical treatment in which genes are modified in order to treat disease, has become a hot button topic in the medical field as of late. These therapies have the potential to offer effective treatment or cures for many disorder and diseases that are currently lacking in treatment options, including many rare diseases.
A Rising Star in Medicine
The potential of gene therapy has quickly made it a popular route of drug development for many drug companies, and dozens of startups have begun to form with the goal of developing a gene therapy product. There are over 800 gene therapy research initiatives happening right now, and Dr. Peter Marks, who is currently overseeing gene therapy at the US Food and Drug Administration (FDA), says that the agency has seen a major increase in interest. Over 200 gene therapy trial applications were submitted to the FDA in 2018.
With the increasing popularity surrounding gene therapy Dr. Marks, who spoke at the Gene Therapy for Rare Disorders Conference, offered a number of suggestions for companies and startups looking to develop gene therapy products.
- Understand possible incentives programs offered by the FDA. Many gene therapies could potentially qualify for Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation, particularly for therapies that are intended to treat rare diseases. These designation can speed development and facilitate greater collaboration from the FDA.
- Stay up to date on FDA guidances related to gene therapy. These guidances are unlikely to remain the same for very long. As the science and technology surrounding gene therapy continues to advance, the guidelines must be correspondingly updated.
- Studies and trials need to be modeled and structured very thoroughly. Patient populations are likely to be small so studies with modified endpoints and controls may be necessary. Trial and study design are another are where the FDA can work with drug developers.
- Try to develop ways to streamline the process of gene therapy development. Scaling up gene therapy development and production is still a tricky process at times and new standards should be implemented as gene therapies become more commonplace.
- Small startups should be aware that they can get help from the FDA. The agency is open to answering questions and clear and concise communication can go a long way to developing therapies that are successful and effective.
