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The cell and gene therapy space remains fertile territory for growth, exploration, and discovery. How applying a data-driven model may be the best way to approach this complex ecosystem and assess the innovations of tomorrow.




Mapping Out the Future of Cell and Gene Therapy

Each year, the success of cell and gene therapy becomes clearer, but what do we really know about it?





The Future of Cell and Gene Therapy

AbbVie Pedro Valencia ADCs

PE: How is AbbVie using novel cancer biomarkers to develop potential first-in-class ADCs for the treatment of advanced solid tumors?

We're constantly looking for biomarkers that can help us to do two key things. The first is to tackle cancer cells. and spare normal cells. We’re looking for specific antigens that are over-expressing tumor cells, and you see limited expression in normal cells. We want to make sure that target that’s over-expressed is critical for the survival of the cancer cell. Once you block it, you can actually drive a cancer cell death. That’s what we're looking for. In that perspective, c-Met is one of those biomarkers that is over expressing multiple tumor types such as lung, colorectal, gastric, and other GA tumors. We as an industry have tried to tackle c-Met in the past but have been unsuccessful. It would actually be successful to tackle what we call met, which is a gene inside the cell.

There’s this thing called met amplification, where the cancer cell produces multiple copies of the gene, and around 30% of patients have it. We can actually tackle that with small molecules. But once the gene becomes a protein and gets over-expressed in the cell, that over-expression is something we haven’t been able to target. For example, we know that on lung cancer, c-Met over-expression is about 25% to 50% in colorectal cancer, and it could be anywhere from 30% to 70% depending how you measure it, so it’s quite prevalent.

With ADCs, we’re trying to tackle c-Met over-expression on the surface of cancer cells. That was novel, and we can do it through ADCs. 706 has a target called SEZ6, and it’s a target that is over expressed in neuroendocrine cells or neuroendocrine neoplasms. When you think about it, multiple tumor types kind of have a subset of cells that are neuroendocrine driven. Small cell lung cancer is one of the few that has everything driven by neuroendocrine cells. The beauty of SEZ6 is over-expressing those cells and limited expression in normal cells. So, we can tackle it with an ADC and drive the results that we're seeing today.

PE: ADCs have redefined the treatment of cancer in recent years. Can you please discuss the promise of this emerging class and what the future may hold?

At the end of the day, ADCs work like a trojan horse. You have a good antibody that cancer cells are happy to receive, but once you hook into the cancer cell receptor, you deliver the payload, which is pretty much chemotherapy. It’s actually a highly potent version of the regular chemotherapy cancer patients are given on a regular basis. So, from that perspective, our goal is to replace chemotherapy. Our grand goal with ADCs is to replace good old, decades old chemotherapy that is really toxic for some patients. That’s how we see the redefinition of cancer treatment in the future with ADCs. So, what do we see in the future? Well, if you think about it, chemotherapy is being given in combination with other agents. In the future, we see ADCs being given in combination with chemotherapy. There will be one with other agents such as immuno-oncology agents. So, if you combine the ADC and the PD1s, you can start seeing that synergy come together and kind of start really dreaming about cures for the patients. All in all, we're super excited about ADCs replacing chemotherapy. 

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses using novel cancer biomarkers to develop potential first-in-class antibody drug conjugates (ADCs) and the promise that they offer. 

Utilizing Biomarkers to Develop First-in-Class Antibody Drug Conjugates to Treat Advanced Solid Tumors

This study demonstrates high-throughput analysis of BTEX compounds from several matrices (air, water and soil). Detection limits in the single-digit part-per-billion concentration range (by volume) are readily achievable within seconds using SIFT-MS because sample analysis is achieved without chromatography, pre-concentration, or drying. We also present a calibration approach that enables speciation of ethylbenzene from the xylenes in real-time.


High-Throughput Analysis of Volatile Compounds in Air, Water, and Soil Using SIFT-MS

This application note summarizes key SIFT-MS results presented in a peer reviewed article entitled “Unreported VOC Emissions from Road Transport Including from Electric Vehicles.” Learn how the Wolfson Atmospheric Chemistry Laboratory at the University of York used SIFT-MS for VOC analysis in its platform to experimentally verify that motor vehicle screen wash is a significant unreported source of VOC emissions (especially for ethanol and methanol). 


Real-Time Roadside Monitoring of Unreported VOC Emissions from Road Transport by SIFT-MS

Real-Time Measurement of EPA Regulated HON Compounds and Environmental Pollutants Using SIFT-MS

Data from the CIBMTR trial demonstrated that Kite’s Yescarta (axicabtagene ciloleucel) produced high overall survival (OS), objective response rate (ORR), complete response (CR), and effectiveness consistent with pivotal trial outcomes in treating patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). According to the company, the analysis included real-world evidence (RWE) from the largest analysis of second-line treatment with Yescarta in 2022-2023 based on Center for International Blood and Marrow Transplant Research (CIBMTR) registry data.

“We are pleased that Yescarta’s overall survival benefit for patients with early relapsed/refractory large B-cell lymphoma is confirmed in the largest real-world analysis of a broader patient population,” said Dominique Tonelli, VP, global head, medical affairs, Kite, in a press release. “By studying outcomes in the real world, we consistently demonstrate that patients treated with Yescarta have the opportunity to live longer.”

In abstract #526, 446 patients from 89 centers in the United States from the CIBMTR registry who received second-line Yescarta treatment showed outcomes consistent with the ZUMA-7 trial, with a demonstrated ORR of 79% with a CR rate of 64%. Additionally, the one-year rate of duration of response was 66%, progression-free survival (PFS) rate was 53%, event-free survival was 53%, and OS was 71%.

“It is reassuring that the largest real-world dataset for axi-cel as a second-line treatment for relapsed/refractory large B-cell lymphoma, across a broader patient population than the ZUMA-7 pivotal study or Phase II ALYCANTE study for transplant-ineligible patients, has demonstrated consistent outcomes at 12-months median follow-up as in ZUMA-7,” said lead investigator Caroline Lee, fellow, hematology medical oncology, Stanford Medicine, in the press release. “These data should provide further confidence to physicians that earlier use of [Yescarta] can provide the best chance for overall survival and possibly a cure for these patients.”

In abstract #527, RWD from 615 patients with R/R LBCL from 109 centers from the CIBMTR registry demonstrated a decreasing trend in incidence, severity, and duration of cytokine release syndrome (CRS) and immune-effector cell-associated neurotoxicity syndrome following treatment with Yescarta in the third-line setting. Additionally, these patients had significantly lower incidences of Grade ≥ 3 CRS compared to those treated during 2017–2019. Further, patients treated in the later time periods of 2022-2023 and 2020-2021 experienced significantly shorter duration of CRS compared to 2017-2019.

Lastly, in abstract #4505, findings from the Phase II ALYCANTE study in patients with R/R LBCL after one prior line of therapy who were deemed ineligible for high-dose chemotherapy and autologous stem cell transplantation found that after a short initial deterioration at one month post-infusion, patients reported longer-term stability or improved quality of life across multiple parameters, after up to a year of follow-up. Results found that 61 patients reported a lower symptomatic level at three months compared to baseline. After three months, 45% of patients presented a stable global health condition and 73% stable physical functioning. After a year, most patients reported a more stable condition, with clinically and statistically significant improvement.

“ALYCANTE is the first study to assess [Yescarta] as second-line therapy in transplant-ineligible relapsed/refractory large B-cell lymphoma patients, with previous study findings confirming its efficacy in this patient population,” said Roch Houot, head, hematology department, University Hospital of Rennes, France, coordinator, ALYCANTE study, in the press release. “This current study shows that, in this frail and elderly population, [Yescarta] not only increased the quantity of life but also improved the quality of life which was comparable to that of the transplant-eligible patients, and allowed recovery of a fatigue score close to the general French population.”

1. Kite Presents New Data Underscoring Curative Potential of Yescarta® in Relapsed/Refractory Large B-cell Lymphoma at ASH. 

December 8, 2024. Accessed December 9, 2024. https://www.gilead.com/news/news-details/2024/kite-presents-new-data-underscoring-curative-potential-of-yescarta-in-relapsedrefractory-large-b-cell-lymphoma-at-ash 

Real-world evidence from the largest analysis of second-line treatment with Yescarta in 2022-2023 demonstrated a high overall survival rate in patients with relapsed/refractory large b-cell lymphoma. 

Yescarta Demonstrates Promise in Treating Relapsed/Refractory Large B-Cell Lymphoma 

Peer Exchange: Technology's Role in Achieving Greater DE&I in Clinical Trials

In this roundtable discussion, industry experts gathered to address the state of achieving greater diversity in clinical research. The group highlighted a variety of topics including current challenges they are seeing, the use of technology, and more.

Out-of-Pocket Costs for MS Medications Rise While They Drop for Other Neurologic Conditions

The out-of-pocket cost for MS disease-modifying therapies jump by 217% going from $750 per year in 2012 to $2,378 per year in 2021.

OpenAI Adds $200 Monthly ChatGPT Pro Subscription With New Model

OpenAI is introducing a higher-priced paid tier for its chatbot ChatGPT that includes unlimited access to its most powerful artificial intelligence models, including an improved version of its software that can perform human-like reasoning tasks.

The new ChatGPT Pro option, announced in a livestreamed event on Thursday, will cost $200 a month and offer access to an expanded version of o1, its reasoning model. OpenAI previously introduced a preview of the model in September, but the company said the new version is faster and better at solving competitive math and code problems.

Cencora Launches Accelerate Pharmacy Solutions to Enhance Support for Hospital and Health System Customers

Cencora announced the launch of Accelerate Pharmacy Solutions, a unified portfolio of solutions designed to help hospital and health systems customers optimize operations and improve financial performance.

Accelerate Pharmacy Solutions brings together Cencora’s suite of pharmacy and supply chain solutions, enabling Cencora to deliver an enhanced customer experience and integrated support to address customers’ evolving needs. The solutions range from specialty drug purchasing and cost management strategies to logistics, transport, supply chain and inventory management solutions.

Don't miss part 4 of my interview with AllazoHealth's William Grambley!

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