January 30, 2024

Cell and Gene Therapies are Hot: See What’s Steaming in the Pipeline 

Cell therapy has been in the works for over half a century, but it’s never been hotter than it is now. Throughout its history, scientists have demonstrated its many benefits, furthering researchers’ interest in expanding efforts in cell- and tissue-based therapies. Cell and gene therapies are so popular right now the U.S. Food and Drug Administration (FDA) is expected to approve as many as 20 cell therapies and 31 gene therapies in 2024.  

The market is very lucrative, estimated at $3.22 billion in 2022. That value is projected to rise significantly to $25.58 billion in 2028, with a compound annual growth rate (CAGR) of 41.25%.  

And that’s just in the United States. Europe boasted a $2.17 billion cell and gene therapy market in 2022, and it’s expected to rise to $15.5 billion in 2028, yielding a CAGR of 38.20% over that period. Also, the European Medicines Agency (EMA) approved more than 19 cell and gene therapy drugs. Its current pipeline consists of another 193 investigational therapies; more than 50% of those treatments have reached Phase 2 clinical trials. Over 1,500 clinical trials were underway as of March 2023 due to a heightened interest in funding.  

Most significantly, these regenerative therapies that focus primarily on oncology and rare diseases can potentially change lives—many lives! How we approach disease treatment could forever be changed, with approximately one in three people in the U.S. alone likely to benefit from these impactful treatments.   

Cell and Gene Therapies Make Medicine More Personal 

Personalized medicine isn’t a new concept. Essentially, the idea is that everyone is different, so medicine or treatment shouldn’t be one-size-fits-all. One article published by BioInsights online at Cell & Gene Therapy Insights describes personalized medicine as “providing the right patient with the right therapy at the right dose at the right time.” 

Likewise, cell therapies are critical in making personalized medicine a reality by analyzing a patient’s genetic makeup and identifying specific mutations or abnormalities that underlie their disease or condition. Armed with this information, scientists can then design customized cell-based therapies. This may involve extracting a patient’s cells, such as T-cells or stem cells, and modifying them ex vivo to correct or enhance their functions. Alternatively, it could entail using engineered cells designed to target the specific molecular drivers of the disease.  

When these personalized cells are reintroduced into the patient’s body, they can precisely target and combat the disease, minimizing damage to healthy tissues, reducing side effects, and maximizing therapeutic efficacy. In this way, cell therapies form the cornerstone of personalized medicine, offering an exact and individualized approach to treating a wide range of medical conditions, making it an increasingly common approach.  

According to scientists, clinical trials have shown success rates to be substantial. Positive outcomes include the remission of certain cancers and reversing blindness brought on by gene mutations.  

Gene Editing Can Save Lives… But Are We Ready for It? 

Despite common misconceptions, cell and gene therapies aren’t only targeting cancer. Once again, this recently became apparent when the FDA approved two groundbreaking treatments to combat sickle cell disease (SCD) in patients 12 years of age and older. One followed on the coattails of the U.K.’s approval just a few weeks prior. This milestone marked the approval of “the world’s first medicine built from the Nobel Prize-winning technology CRISPR,” or Clustered Regularly Interspaced Short Palindromic Repeats.  

In layman’s terms, CRISPRs are repetitive DNA sequences. Scientists are working to target and edit these stretches of genetic code at precise locations with programmable systems or treatments designed to mitigate various devastating, debilitating, and potentially life-threatening inheritable diseases.  

The other treatment approved only in the U.S. differs slightly in that it uses a benign virus that aids in the placement of an engineered gene. This artificial gene is programmed to “encode for functional hemoglobin into patients’ stem cells,” offering the same “healing” effects as its CRISPR companion.   

With these first-time cell-based gene therapy treatments for SCD gaining the greenlight in early December 2023, researchers and government regulators in the U.S. and U.K. are paving the way for increased innovation and advancement in gene therapy—with genetic material being an essential component of our cellular makeup and gene therapy being a similar approach to cell therapy in attacking disease.  

The success rates make an even more compelling argument favoring additional gene therapies. Throughout testing, 30 study participants were followed for 16+ months post-treatment to observe the results. All but one participant saw significant relief, remaining free of pain crises for at least one year. All but three participants have remained completely crisis-free, avoiding hospitalizations.  

However, despite recent victories, gene and cell therapies are still somewhat anomalies. There are many reasons, including cost, availability, eligibility, length and complexity of treatment, and unknowns about long-term side effects. The CEO of one of the companies granted an FDA approval points to the obvious, that healthcare systems are set up to cater to chronic conditions, meaning a reliance on lifetime medicinal treatments. Substituting one-time treatments could shift that dynamic, essentially upending traditional medicine and its resulting profits.  

In addition to the concrete effects, namely the sizeable monetary hit (in the millions) to both patients and healthcare systems, experimental medicine is not without several ethical considerations. According to the National Human Genome Research Institute, over 400 million people worldwide are affected by one of 7,000 diseases caused by single-gene mutations. Still, per data gathered by the Pew Research Center for the U.S. alone, many Americans are divided from the start regarding warding off these illnesses. 

It’s possible that gene editing could eventually be used to lower a person’s chances of ever developing severe disease by enhancing one’s health in infancy. However, roughly 40% of the population is undecided about using gene therapy for this purpose. And it’s nearly a 50/50 split when it comes to whether a parent would use this type of gene editing for their baby, with one percent more in favor of leaving nature (or, in this case, genetics) to do its thing, addressing it only after it becomes a problem.  

Either way, scientists are getting closer to “cracking the code” for disease eradication using various cell and gene therapies.  

Overcoming Clinical Trial Barriers for Increased Approvals 

Only a handful of cell and gene therapies have been approved yearly for the last couple of years. While all progress is good, and the numbers certainly appear to rise each year, the percentage of approved novel treatments versus those in development is still relatively small.  

More than 1,500 cell and gene therapy trials are registered with ClinicalTrials.gov., with many still in the beginning phases. The average process is about nine years from Phase 1 testing to application for approval, with the success of cell and gene therapies from Phase 2 to FDA sign-off having a significantly low bar of only 14%—meaning a vast majority of these trials will be abandoned.   

However, access to these trials (although growing) is a major impediment. Patients suffering from specific ailments face significant hurdles to participate. Some barriers have to do with funding. For example, the National Cancer Institute’s (NCI) cooperative group clinical trial treatment program has an enrollment cap for its funded groups. This cutoff totals 17,000 patients annually, accounting for only 1% of the approximately 1.7 million new cancer diagnoses in the U.S. in 2015, which is largely under-representative of the majority cancer population.  

Other obstacles include structural barriers, physician and patient attitudes, demographic and socioeconomic disparities, and narrow eligibility windows. 

The benefits of overcoming these barriers are two-fold. Not only does higher enrollment yield faster treatment advances, but it can also mean lower death rates for those who can receive the trial treatments. Children younger than 15 years old are traditionally accepted into clinical trials at higher rates than adult cancer patients. Likewise, deaths among child cancer patients have decreased since the 1970s. Mortality rates for adults have only been decreasing since the 1990s, showing a seeming correlation between trial enrollment numbers and reduced deadly outcomes.  

By adjusting the contributing factors limiting access to trial participation, mandatory clinical trials could be conducted more quickly, allowing life-saving cell and gene therapies to reach the market sooner. With nearly two-thirds of American cancer patients willing to participate in clinical trials but only less than 5% enrolled, those involved in the decision-making process are missing an opportunity to quickly roll out imperative medicines that could change the face of pharmaceuticals and healthcare. 

More FDA Approvals on the Horizon 

Since the pandemic, the FDA has been promoting increased approvals for cell and gene therapies, treating health issues like bladder cancer, Hemophilia B, glaucoma, sickle cell disease, and more. As of December 8, 2023, the FDA has approved 34 cell and gene therapies. However, that number doesn’t even begin to address the magnitude of advanced therapies awaiting a chance at commercialization.  

According to the American Journal of Managed Care (AJMC), “the current gene therapy pipeline contains 2075 candidates.” Yet only 8-10 gene therapies through the end of 2023 have advanced to the market after receiving approval from the FDA, and only about four are expected to make it to market in 2024. While affordability and accessibility are still two primary concerns, once these therapies are made available, the cell and gene therapy pipeline will continue to grow.  

The urgent push behind these therapies has a lot to do with their singular-use curative intent, meaning these treatments get to the root of the problem by providing a verifiable fix rather than simply reducing or alleviating symptoms.  

CRISPR gene editing is expected to pick up steam in the new year. Recent research shows that CRISPR-Cas molecules, originating from ancient bacterial antiviral defenses, can be used to modify cells to make cell-based therapies. These cells, known as T-cells, would be extracted from each patient’s body and reprogrammed to target cancer cells when reintroduced into the respective patient’s bloodstream.  

This therapeutic method, termed Chimeric Antigen Receptor or CAR T-cell therapy, was first introduced in the U.S. in 2017. Since then, it has been highly effective in helping patients with B cell leukemias, lymphomas, and other blood cancers achieve long-term remission.  

When used with CRIPSR-Cas systems, the CRISPR-Cas molecules might be helpful in replacing faulty genes or adding genes to enhance cellular function and provide CAR Tcells with new abilities, such as recognizing or withstanding tumors.  

As of September 2023, 12 cellular therapies were also in the pipeline for treating acute myeloid leukemia (AML). Two cell therapies involving gamma-delta T cells also address a systemic disorder that results in the rejection of cell transplants. This disorder is called graft versus host disease (GvHD), and currently, there is nothing available to reduce its occurrence in AML patients. These cell therapies may also prove helpful in effectively treating other hematological malignancies if trials show success in AML patients.  

Fortunately, the U.S. isn’t the only region to acknowledge the benefits of CRISPR gene editing technology and other cell and gene therapy-based technologies. As discussed above, the European Medicines Agency is also pushing for heightened approvals of these life-changing treatments.   

Oxford Can Help Support Cell and Gene Therapy Advancements  

Oxford has the talent and expertise to support companies in advancing their cell and gene therapy efforts. We can help get biotech companies started or promote their continued growth in the cell and gene therapy realm. Whether you are looking to expand your operations or complete specific projects to keep you up to date with current technology trends, Oxford can help.   

We strive to stay informed on developments that propel the industry forward so that we can promote innovation and encourage a competitive advantage. When you decide to partner with Oxford, we guarantee you’ll get The Right Talent. Right Now. We will be with you every step of the way, solidifying more than just a business transaction. Instead, we pride ourselves on building strong relationships that are mutually beneficial, leading companies into a more promising and secure future.  

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Whether you want to advance your business or your career, Oxford is here to help. With nearly 40 years’ experience, we know that a great partnership is key to success. Start a conversation today.