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Gene Therapy – the slow road to success

November 24, 2021

G&L’s Vice President of Regulatory Affairs, Jessica Kokosinski, and Senior Director of Regulatory Affairs, Christine Piscitelli delve into the world of Gene Therapy and its benefits to healthcare.

However, with more than 900 Investigational New Drug (IND) applications pending with the U.S. Food and Drug Administration (FDA) alone for treatments that include cancer, infectious diseases and genetic disorders, gene therapy could be on the cusp of bringing revolutionary benefits to human health.

In its most basic permutation, gene therapy modifies or manipulates existing genes by altering the biological properties of living cells for therapeutic use. Not surprisingly, the process is heavily safeguarded and, to date, just four products that involve the insertion of new genetic material into a patient’s cells have been approved by the FDA.

Stringent regulations surround gene therapy and its related products and since the 1970s, in response to concerns over safety, scientific, social, and ethical issues, regulators have striven to ensure their oversight framework has been evolving in tandem with advances and breakthroughs in the field.

Indeed, due to these concerns, complex layers of federal and local oversight and regulations have been established over the last 50 years to ensure the utmost efficacy and safety in gene therapy with the first U.S. trial with humans not taking place until 1990.

Over the next decade, gene therapy research experienced an unprecedented boom. However, several high-profile incidents highlighted the underlying risks and adverse side effects of human gene therapy and public confidence in them subsequently faltered.

Responding to the wavering trust in the trials, the FDA and National Institutes of Health (NIH) took action to strengthen protections for participants with initiatives including the establishment of the Gene Therapy Clinical Trial Monitoring Plan and the Gene Transfer Safety Symposia. was also launched to provide a high level of transparency for gene therapy trials conducted by both public and private sponsors.

Additionally, the NIH and FDA jointly launched the Genetic Modification Clinical Research Information System (GeMCRIS), an information resource and analytical tool to coordinate adverse event reporting and expand public access to reports of serious events.

Confidence around gene therapy has slowly returned, and, over the last eight years, there has been a quantum leap forward with the discovery of new technologies and advancements in safer gene delivery systems. This has seen three gene therapy products being approved for use in the U.S. in 2017, and a fourth in 2019, with further products being approved in China and Europe.

As the delivery systems have evolved, so too has the regulatory framework resulting in a much more streamlined and efficient system that uses a relatively small suite of guidance documents and expedited programs to address a raft of unmet medical needs in the treatment of serious or life-threatening conditions.

A result of this is a record number of active gene therapy clinical trials aimed at treating some of the most serious illnesses and diseases such as cancer, HIV/AIDS and blood disorders with the FDA predicting the approval of 10 to 20 cell and gene therapy products a year by 2025.

Meanwhile, advances and discoveries are continuing at a rapid rate with numerous promising new approaches being developed and studied, making this a very exciting time for healthcare and gene therapies.

Of course, with these advances comes a responsibility on the regulators to ensure safety remains paramount and previous mistakes are not repeated.

If achieved, the promise of gene therapy, inclusive of cures and one-time treatments for serious and life-threatening diseases, is a welcome sight on the horizon for so many of us.

The full version of this article can be found here

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