Customer Stories

Expanding access to cell and gene therapies

Global pharmaceutical services network drives access to life-saving therapies for patients who need them most

By Kathy Lang
Senior Manager, Clinical Research
Thermo Fisher Scientific

After several years of battling an aggressive leukemia, with multiple rounds of chemotherapy and a bone marrow transplant, a 33-year-old professional gardener waits in a hospital room for what doctors hope is the last medical intervention to improve his quality of life. He’s surrounded by a team of caregivers who watch intently as a tiny bag containing about 6 million of his own cells, which have been modified to fight the disease, is slowly infused into his circulatory system through a needle in his arm.

 

“The process seemed almost too simple, even anticlimactic,” the patient says. “However, I am beyond grateful for the chance to live and, even more importantly, live life to the fullest.”

 

On the surface, getting an infusion of potentially life-saving cells may seem straightforward. But what happens behind the scenes to develop the contents of that tiny bag is far from trivial. It’s the end product of an intense process that involves not only technological and scientific innovation, but a strong commitment to collaboration among cell and gene therapy developers, clinicians, partners such as Thermo Fisher Scientific and, ultimately, patients. 

Cell and gene therapy, one of the latest advancements in medicine, could not get any more personal. In autologous cell therapy, T-cells are harvested from a patient and modified to turn them into supercharged, cancer-killing cells before they are infused back into that same patient. Gene therapy is a technique that modifies a person’s genes to treat or cure disease. This is done by replacing a disease-causing gene with a healthy copy, inactivating a disease-causing gene that is not functioning properly or introducing a new or modified gene into the body.

 

While such treatment is usually implemented as a last resort for those who have relapsed or have become resistant to traditional standard of care, it has proved to be disease-modifying and, in some instances, even curative in a growing number of cases worldwide. These results are a testament to advancements in CGT research and clinical trials decades in the making. 

A Field Ripe with Opportunity

The cell and gene therapy field holds great promise for patients and represents a significant growth area for biopharma. Scientists and clinicians have identified more than 7,000 molecularly based diseases, but only 500 have an existing treatment.1 Additionally, more than 2,000 CGT trials are active globally,2 and the number of developers worldwide (around 1,400) has increased by more than 10% from 2021 to 2022, according to the Alliance for Regenerative Medicine.3

 

The market for cell therapy was estimated at $4.77 billion in 2022 and is projected to grow at a compound annual rate of 16.5% between 2023 and 2030.4 To support this next chapter in personalized medicine, Thermo Fisher has built its global pharmaceutical services network, which now includes a new cGMP manufacturing facility adjacent to the University of California, San Francisco (UCSF) Medical Center.

 

Working in collaboration with UCSF clinical researchers and closer to patients, Thermo Fisher scientists and product specialists aim to expedite development of breakthrough treatments for conditions such as sickle cell disease and diabetes, as well as cancers such as glioblastoma and multiple myeloma, using updated approaches to chimeric antigen receptor (CAR) T-cell therapy and gene-editing technologies, including clustered regularly interspaced short palindromic repeats (CRISPR). 

 

Thermo Fisher’s global network consists of more than 15 sites providing cell and gene therapy development, manufacturing and supply chain services. Major product manufacturing sites include Grand Island, N.Y. (media and reagents); Bedford, Mass. (resins); Vilnius, Lithuania (Dynabeads and reagents); Singapore (instruments and software); and R&D locations in Carlsbad, Calif., and Frederick, Md.

“Our aspiration is to increase the accessibility of life-saving cell and gene therapies to patients, and we're doing that by accelerating the adoption of our best-in-class technology, products and services through close collaboration with our customers,” says Betty Woo, vice president, cell, gene and advanced therapies at Thermo Fisher Scientific.

“When we collaborate with our customers, we like to think that it's a ‘virtuous cycle’,” Woo says. “They get early access to our technologies, our knowledge and our insights by working directly with our scientists, so we have a sharing of ideas that provides them a competitive advantage. That's the payoff for them.”

Betty Woo, vice president, cell, gene and advanced therapies, Thermo Fisher Scientific

Leveraging Broad Expertise to Meet Customer Needs

The result of strategic investments, Thermo Fisher’s global pharmaceutical services network is designed to support established and emerging developers across the cell and gene therapy continuum. The company leverages its deep product portfolio and expertise in contract development and manufacturing and clinical research to help customers navigate the chemistry, manufacturing, controls and regulatory landscape.

 

 

“We meet the customers where they are and have a strong understanding of their needs,” says Rupa Pike, senior director of technical affairs, advanced therapies at Thermo Fisher Scientific.

“Our partners vary from small startups to established biopharmaceutical companies. Some of them are moving from an R&D space and want to conduct their very first clinical trial, while others know everything about GMP manufacturing and clinical trials but want to take their first step into cell and gene therapy.”

Collaborating with customers at an early stage helps reduce the overall “vein-to-vein” time – meaning the time it takes to harvest cells from patients and reinfuse the modified versions back into them.

This is accomplished through many best practices in supply chain and cold chain logistics, streamlining of the manufacturing process and well-defined analytical assays that meet regulatory requirements. For example, Thermo Fisher is currently developing assays designed to reduce sample analysis turnaround time from 28 days to as little as three.

Rupa Pike, senior director of technical affairs, advanced therapies, Thermo Fisher Scientific

Healthier

Quotation marks
"Our aspiration is to increase the accessibility of life-saving cell and gene therapies to patients, and we're doing that by accelerating the adoption of our best-in-class technology, products and services through close collaboration with our customers."
Betty Woo
Vice President, Cell, Gene and Advanced Therapies
Thermo Fisher Scientific

“As innovators in this space, we have an obligation to extrapolate backwards from commercial cost and work proactively with our cell and gene therapy development partners to be more critical around process,” says Panteli Theocharous, global vice president, cell and gene therapy strategy lead, clinical research at Thermo Fisher Scientific. “We also want to ensure that diverse patient populations are included in global trials so that all of them may gain access to these life-changing therapies post-regulatory approval.

“Beyond therapy development, we have the expertise to help our customers build strategies that address the needs of regulators and payers,” he says. “We are always looking at the practicalities of bringing these highly complex therapies to more patients.”

To date, regulators have approved more than 30 new cell and gene therapies, and as many as 13 additional new ones could be approved for use in the United States, Europe or both by the end of 2023.

Less than a decade since the U.S. Food and Drug Administration (FDA) approved the first CAR-T cell therapy, the science of cell and gene therapy is now moving into broader applications beyond cancer. Three gene therapies have been approved for hemophilia A and B, the most recent one in June this year by the FDA, and new treatments for sickle cell disease are anticipated to come online within a year.

The majority of cell and gene therapy’s success so far has focused on autologous applications, but the next major frontier for developers and their collaborators, including Thermo Fisher, is the advancement of allogeneic therapies.

Panteli Theocharous, global vice president, cell and gene therapy strategy lead, clinical research, Thermo Fisher Scientific

While autologous therapy treats one patient with one product, allogeneic therapies leverage cells from one donor to develop thousands of cell products. This approach of developing “off-the-shelf” solutions has the potential to democratize cell therapies by further reducing costs and increasing accessibility to more patients, while also making them easier for clinicians to use.

 

“It’s all about strong partnerships, attention to detail and demonstrating sensitivity and empathy as we place ourselves in the shoes of patients and support them through a complex, long-term journey,” Theocharous says. “Personally, I always wanted to make a real difference to each and every patient journey. I want to help them achieve something very special – long-term remissions, cures and overall survival.” 

 

1.    Rare Disease, National Institutes of Health; Feb. 2023

2.  Regenerative Medicine: The Pipeline Momentum Begins, Alliance for Regenerative Medicine; Sept. 2022

3.   Workforce Report: Gap Analysis for the Cell and Gene Therapy Sector, Alliance for Regenerative Medicine; March 2023

4.  Cell Therapy Market Size, Share, & Trends Analysis Report by Therapy Type (Autologous, Allogenic), By Therapeutic Area (Oncology, CVD, Dermatology), By Region, And Segment Forecasts, 2023 – 2030; Grand View Research, 2022

Thermo Fisher’s Impact on CGT Industry

  • 15+ network facilities worldwide
  • 130+ clinical trials management/support
  • 250+ clinical trials using cGMP-grade products
  • 300+ peri- and post-approval projects
  • $3B manufacturing capabilities investment (since 2019)
  • 16 (out of top 20) biopharma customers
  • 35+ countries/regions with supply distribution
  • 160+ viral vector products produced globally
  • 700+ cGMP viral vector lots manufactured
  • 2 regulated viral vector products (U.S. and EU) – more pending

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