Guest Column | February 10, 2020

Cell & Gene's 2020 Outlook: Enzyvant, Spark Therapeutics, And Orchard Therapeutics

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Hopefully you’ve been following Cell & Gene’s 2020 Outlook series, in which many of our Editorial Advisory Board members have provided their data-driven insights for the coming year — and beyond — for the cell and gene sector based on their professional role. Bruce Levine, Ph.D. detailed 7 trends to watch in cell and gene sector, and Bruce Bunnell, Ph.D. shared valuable data on three intense areas of stem cell research.

Here, Rachelle Jaques, CEO of Enzyvant, Jamie Ring, Head of Patient Advocacy at Spark Therapeutics, and Mark Rothera, President and CEO of Orchard Therapeutics discuss continued momentum in regenerative medicines, demystifying gene therapies, and treatment of disorders of the central nervous system (CNS) respectively.

Rachelle Jacques, CEO, Enzyvant on the 2020 Outlook for cell and gene therapy.

Rachelle Jaques, CEO, Enzyvant
The outlook is strong for cell, gene, and tissue-based regenerative therapies, and there is great anticipation from patients battling rare and ultra-rare diseases. Amidst all this excitement, it is important to acknowledge the sector is in the earliest stages of maturation. While this brings great opportunity, these technologies also are challenging basic industry frameworks and motivating sweeping changes.

Regenerative medicines are different than anything that has come before. Regulators recognized the importance of these therapies with accelerated approval pathways that have advanced the sector. However, these priority pathways primarily address safety and efficacy, not other BLA requirements such as Chemical and Manufacturing Controls (CMC). The gaps are especially problematic for one-patient-one-lot regenerative medicines.

Some off-the-shelf good manufacturing practices (GMP) for established biopharmaceuticals simply do not apply to many regenerative technologies. The industry, with consensus among key stakeholders, must redefine what good looks like in GMP for unique regenerative technologies to build the overall knowledge base and clear pathways for many more needed therapies.   

Regenerative medicines also are changing commercial capabilities. Traditionally, biopharmaceutical companies are manufacturers and commercial organizations as much if not more than R&D organizations. In regenerative medicine, centers of excellence are central to a commercial model that is multidisciplinary inside and outside the clinic. As the sector matures, future company commercial roles may evolve to become integrators and facilitators more like today’s alliance management functions than global brand or franchise roles.

Significant momentum will continue for regenerative medicines because speed matters to patients in desperate need. A call to action for the industry is to prioritize creation and consensus with regulators on transitional bridges, such as conditional approvals. This will help speed emerging technologies into global markets so they can provide benefit to patients and create real-world experience that will inform future standards.  

The industry will make significant progress advancing regenerative therapies in 2020 and beyond. We will create relevant frameworks and clear pathways for successive successful approvals because we must. Patients and their families are counting on us.

Jamie Ring, Head of Patient Advocacy, Spark Therapeutics on the Outlook for Patient Advocacy in 2020 for the Gene Therapy Space.

Jamie Ring, Head of Patient Advocacy, Spark Therapeutics
2020 looks to be another exciting year of growth for Cell and Gene Therapies.  Product approvals continue to increase, new research programs are being initiated regularly, and rare diseases that long have had no therapeutic intervention are seeing their first clinical trials using gene therapy.  In the advocacy space, I expect to see two trends — collaborative education and more discussion about the Gene Therapy experience.  Gene therapy education will continue to be an imperative for physicians, patients and caregivers, and advocacy groups alike.  With a shared interest to help ensure that families have the most accurate and empowering information, increases in collaborative educational efforts will be at the forefront.  We are starting to see more group effort in creating innovative resources for community members where multiple patient groups, industry partners and trade groups all align on empowering disease communities to understand what gene therapy is and the decision-making process that goes into it.

Additionally, as we now have several cumulative years of commercial access to gene therapies, helping patient communities understand how to prepare for the reality of a gene therapy by hearing from families who have successfully had treatment is critical.  Both from a process, and an experience standpoint, I expect that gene therapy users will begin to share their stories more readily via patient organization communications, family conferences, and medical conferences to help demystify what gene therapy actually looks like in practice. 

Mark Rothera, President and CEO, Orchard Therapeutics on the next major scientific advancement in gene therapy in 2020.

Mark Rothera, President and CEO, Orchard Therapeutics
Thanks to gene therapy, we are now living in a bold new era of medicine. Today, we have the potential to cure devastating and sometimes fatal conditions through a one-time treatment with the hope of providing a lifetime of benefit to people with rare, inherited diseases around the globe.

But despite this progress, there is plenty of work still to do, particularly in the treatment of disorders of the central nervous system (CNS).

For decades, many CNS disorders have been difficult or impossible to treat. In order to achieve a therapeutic effect, treatments that are administered systemically must first move from the bloodstream into the brain. However, crossing this “blood-brain barrier” has historically proven challenging. As a result, some current clinical approaches involve more invasive medical procedures to administer treatments directly into the brain.

Having dedicated my career to developing and commercializing novel therapies for rare diseases, I have seen these challenges firsthand. We must continue to build on the scientific innovation to date if we are to make the promise of gene therapy a reality for people with CNS disorders.

Recent advances in the field are providing reason for optimism. Preclinical studies have demonstrated that ex vivo autologous hematopoietic stem cell-based therapies, which are infused intravenously, can deliver gene-corrected blood stem cells. Once the cells engraft in the bone marrow, they begin to self-renew and produce healthy blood cells of all types. A subset of these cells is able to cross the blood-brain barrier through a natural process and potentially achieve a therapeutic effect in the brain.

More research and clinical investigation must to be done in this area, but I am hopeful that we will soon live in a world where gene therapies can effectively target the brain as well as other tissues. We have a responsibility to all those affected by rare disorders to continue developing and advancing innovative gene therapies to transform the lives of people with CNS disorders and other rare neurometabolic diseases.