Gene Therapy, the Cure and the Reality

January 29, 2019 Becky Hurt

Authored By: Kimberley Buytaert-Hoefen, Principal Consultant

Today is the day when the cure is possible.  With advances in basic and clinical science and biotechnology, gene therapy is no longer a concept but holds promise for treating a wide range of diseases.  In 2017, the United States, Food and Drug Administration (FDA) has approved approvals of the first gene therapy products , including chimeric antigen receptor (CAR)–T cells to treat B cell malignancies and AAV vector-mediated gene therapy for treatment of RPE65 mutation-related retinal dystrophy.  PAREXEL is here and we are already guiding companies to successful gene therapy development from discovery to the market.

The challenge is taking the concept that you have discovered and developing it into manufacturing for Good Laboratory Practices (GLPs), Good Clinical Practices (GCPs) and Good Manufacturing Practices (GMPs).  All of these terms may be overwhelming to you, but rest-assured, the frontier has been paved and the guidelines have been established. 

Despite the progress made in the recent years, significant challenges endure.  How do you navigate the gene therapy rush to market?  It is moving too fast for a classically trained Quality by Design (QbD) mindset.  Yes of course we need quality by design, but how can this be obtained when rare diseases are treated, small batches are required and the cost of validations in the true pharmaceutical definition are not able to be performed due to time and financial limitations?  This is an understood struggle and the regulatory agencies are willing to listen.  The benefit lies in curing patients in need.  This era is dependent on using all available knowledge of the manufacturing of a viral vector to facilitate quality by design attributes for the control of the manufacturing of these products.  This is essential in that quality cannot be forced into a product but needs to be built into the design plan.  Failing to comply with such a plan may result in company failure due to lost profits in product recalls and safety issues for patients. 

A difficulty for current gene therapy companies is the sterile filling process.  This is an art and science of its own.  Yes, you may have developed a lifesaving gene therapy product and even successfully manufacture and administer it, but can you sterile fill it to assure patient safety?  Taking this on without prior experience is a ticket for failure.  Aseptic filing is one of the greatest challenges for pharmaceutical manufacturing.  Gene therapies offer a unique hurdle due to small batches and fast tracking to market.  Resources for small batch, closed systems for aseptically filled manufacturing are needed.  Rudimentary filling processes are currently used to bridge the gap between research and development to clinical and commercially manufactured product.  While the industry is moving so fast, these lines are often blurred, but need to be addressed and optimized.

PAREXEL has a team of experts who have extensive experiences in clinical trial design and regulatory knowledge.  Our dedicated staff is well-established in the areas of FDA’s expedited programs (e.g., Fast Track, RMAT designation, Breakthrough Designation), INTERACT, Investigation New Drug (IND) applications and Biologic License Applications (BLAs).  With team work, PAREXEL can help you in facilitating your product from discovery to a commercially manufactured product.  Our expertise can save you time and money by successfully guiding your company in bringing your product to the market: readily available to patients in need.

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