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| January 15, 2021

FDA Publishes Draft Guidance on Human Gene Therapy

Marina Turea

Marina is passionate about all emerging technologies in the healthcare space and love to write about all of them. Marina is passionate about all emerging technologies in the healthcare space and love to write about all of them.

The Food and Drug Administration (FDA) recently released draft guidance to producers of human gene therapy products for neurodegenerative diseases. This report was created to offer organizations, businesses, and individuals more guidance as it relates to product development, preclinical testing, as well as the clinical trial design of human gene therapies.

The draft guidance pays special attention to chemistry, manufacturing, and controls considerations, which are by far some of the most sensitive areas of concern in human gene therapy and its ensuing products. The draft guidance emphasizes that any human gene therapy products created should be designed based on informed scientific knowledge.

The field of gene therapy has advanced at a dizzying rate in the last couple of years. The increased growth of innovative research and product development of human gene therapy products has tremendously impacted the world. Thanks to human gene therapy, debilitating diseases are getting easier to manage and treat.

Most of the gene therapy technologies and products that we have today were unfathomable ten or twenty years ago. However, these days, they are no longer the stuff of fiction. They are fast becoming a therapeutic reality for a growing number of individuals all over the world that are suffering from all sorts of illnesses including rare genetic disorders, as well as autoimmune diseases.

Why is FDA guidance so important?

Because human gene therapy products are slowly becoming integrated into our daily life, the guidance issued by the FDA have never been more important. These recommendations will allow companies and businesses focusing on the creation of gene therapy products to address specific common problem areas. 

Not only is this vital for making sure that the field of human gene therapy continues to advance, but it will also make certain that any products developed to meet the FDA’s standards for safety and efficiency.

Even though human gene therapy has grown by leaps and bounds over the years. The technologies and the products being created must be regulated now that the industry is still in its infancy. The framework that’s being laid now will set the stage for the continued advancement of this trailblazing sector. 

This will then further enable the developers to design and create effective therapies for illnesses that continue to suffer from unmet homeopathic needs. Scientific development in human gene therapy is fast, complex, and poses numerous unique questions during the review stage. 

Some of the things that reviewers have to ask themselves include, how are these products going to work? How can they best be administered safely? Will they pose any health risk over long term use?

The FDA’s efforts to help improve product development in gene therapy will continue to help any products created now and in the future more efficiently. The regulatory controls are especially important where the treatment of serious or life-threatening illnesses is concerned. 

The field of gene therapy has and will continue to grow at a rapid pace partly because of the digital age that we live in. Between 1989 and 2015, approximately 2,335 clinical trials had either been finalized, were in progress, or had been given the stamp of approval to begin. By 2020, over 1,000 clinical trials were reported to be in progress and half of them were being conducted in the United States.

By 2025, it is expected that the FDA will be reviewing and approving close to 10 or even 20 cell and gene therapies every year. Because this trend is not going to slow down any time soon, FDA has pledged to keep working with product innovators, sponsors, researchers, gene therapy patients, and other shareholders to ensure that the development and review of gene therapy products are both safe and efficient. 

Unlike traditional small molecule medicines, gene therapies are one time fixes for inherited genetic defects. The sensitive nature of the field raises critical questions as to how sponsors of gene therapy should handle things. The FDA has a good track record in the field, having already approved Roche’s blindness therapy and Novartis’ muscular atrophy treatment in the past.

What’s the future of human gene therapy?

The field of human Gene therapy is definitely not what it was 5 or 10 years ago. And even though the industry has made some serious advancements within a short period, there is still a long way to go. Human Gene therapy is so sensational partly because it promises to treat and prevent disease effectively and safely.

However, even the theoretical benefits of this technology cannot be denied, so far, it’s been challenging to deliver. Clinical efficacy in most of the clinical trials that have been conducted is hard to prove, while safety concerns abound. The disparity between theory and practice is clear so it will take some time before existing technologies are converted to clinical successes.

This is not to discredit or discount the considerable progress that has been made thus far. Some of the most promising areas where gene therapy is being applied today are in monogenic diseases, in the treatment of hemophiliacs, cardiovascular diseases, as well as various multigene diseases. For other diseases such as cancer, further research and development still need to be done especially as it relates to gene delivery vectors and gene expression systems.

Final Thoughts

With gene therapy products showing more promise than ever, the sector has been waiting for these most recent recommendations that were made by the FDA. The point of the draft guidance that was recently released is to inform developers of best practices. 

The FDA has certain expectations when it comes to gene therapy and combination products, which must maintain particular manufacturing and quality standards. Before any Phase 1 clinical trials can commence, all manufacturing and quality control data and information must be at par based on the recommendations provided in the draft guidance.

The FDA is an agency housed by the Department of Health and Human Services. It is the role of the FDA to safeguard the public’s health by upholding the safety, effectiveness, and security of human and veterinary drug vaccines and any products to be consumed by humans.

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