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Reflections from Cell and Gene Meeting on the Mesa 2021

I recently had the privilege of both presenting at and attending the 2021 Alliance for Regenerative Medicine (ARM) Annual Cell and Gene Meeting on The Mesa conference. This conference is the largest of its kind in the world, bringing together over 400 member organizations and other participants with a distinct focus on advancing innovative cell and gene-based treatments and cures. Advarra is the only independent institutional biosafety committee (IBC) represented in the ARM membership; we had the honor of receiving an invitation to present at the conference. Here are my takeaways from an amazing week of discussions.

Exponential Growth

At the conference, one thing was clear: Cell and gene therapies are growing and an increasingly important part of the overall healthcare system. No longer like science fiction movies, FDA-approved gene therapies are making real differences in people’s lives today. The number of investigational new drug (IND) applications for gene therapy-based treatments have grown exponentially prior to COVID and are holding steady at a high level despite COVID.

Source: “A Changing World in Gene Therapy Research: Exciting Opportunities for Medical Advancement and Biosafety Challenges,” Applied Biosafety.

A report released at the Cell and Gene conference indicated nearly 600 companies in North America and nearly 1,200 worldwide are developing innovative treatments and using cell and gene therapy rather than traditional drug- and device-based modalities. Advarra supports the majority of these companies through our independent institutional biosafety committee (IBC) review services, and we are looking forward to supporting more innovative sponsors in the months and years to come.

Manufacturing and Distribution is a Challenge

We have delivered traditional pharmaceuticals and devices for decades. Good manufacturing practice (GMP) is an established discipline, and we understand the mass production, packaging, and distribution of pills and vials. With complex gene therapies based on an individual’s unique genome, the old paradigms of a central manufacturing plant are out the window. Reagents, viral vectors, and complex chemistry, manufacturing, and controls (CMC) are needed on-site where the participant is located. Many gene therapies involve a technique where the participant’s own cells are altered and then re-infused back into them to generate the desired resulting outcome (e.g., immune cells now know how to attack cancer in the body). This presents a logistical challenge for the industry, as cell and gene therapies are increasingly reaching beyond the halls of large academic medical centers (AMCs). How do you get the manufacturing plant to the place where the participants are?

A good portion of the collaborative discussion at the conference surrounded how to solve these supply chain and manufacturing challenges. Several companies are in the process of developing mobile cell product manufacturing labs. These mobile labs (some the size of a small car) would enable small rural community health centers to offer the same innovative and life-altering treatments as large centers in urban areas. Other innovators are working on adapting participant-derived treatments by using pluripotent stem cells (PSCs) (the ones that can turn into any other kind of cell) from healthy donors and applying genetic manipulation to those healthy PSCs at a more central location, then rapidly ship the modified cells out to the patient for infusion.

For now, participants often must travel to the manufacturing facilities. However, the take home message from the conference is the cell and gene industry knows this paradigm must change and is working rapidly to prepare for the future.

Amazing Cures

Unlike a good number of traditional drug-based “therapies” treating symptoms over a long period of time, or working to suppress but not eliminate the underlying condition, cell and gene therapies offer the possibility of a one-time treatment and cure. There were multiple examples at the conference; I’ll share one for illustration. Orchard Therapeutics presented their work in metachromatic leukodystrophy (MLD). Kids with this condition, caused by a rare genetic mutation, experience debilitating neurological and muscular issues. This results in difficulty moving, eating, and eventually leads to paralysis. Nearly half of children diagnosed with the condition die within five years. There is currently no cure – only protracted, expensive, and invasive treatments.

Orchard’s gene therapy targets and corrects the gene mutation which causes MLD. Their research has preliminarily demonstrated an ability to deliver a permanent and lasting cure. Amazingly complex science with a truly heartwarming result: The ability for a child to live a normal life. And this was just one example of many. It makes me proud to represent Advarra, knowing we are playing a direct and important role in advancing life-giving science.

It is such an exciting time to be involved with clinical research. We are on the cusp of curing many of humanity’s most devastating diseases. As cell and gene passed therapies emerge to take on a larger portion of treatment options, Advarra will continue to play an active and engaged role in optimizing clinical research for the advancement of human health.

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