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Key takeaways 

• Cell and gene therapies (CGTs) are revolutionizing treatment for a variety of complex conditions, offering hope for patients with previously untreatable diseases.  
• Prime Therapeutics (Prime) provides timely, expert insights to help payers, providers and decision-makers prepare for the rapid evolution and cost management of CGTs through its new Cell & Gene Pipeline Outlook publication.  
• The CGT pipeline is expanding with innovative therapies for both rare and common conditions, and several agents are expected to soon receive FDA decisions, highlighting the need for strategic planning and adaptability in health care.  

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Cell and gene therapies (CGTs) represent a revolutionary class of treatments that go beyond managing symptoms to target some of the root causes of diseases, including a person’s unique cellular structures and DNA. By modifying living cells and genetic material, these therapies offer the potential to treat conditions once considered untreatable, from rare genetic disorders to aggressive cancers.  

Advances in gene-editing tools like clustered regularly interspaced short palindromic repeats (CRISPR), breakthroughs in chimeric antigen receptor (CAR) T-cell therapy and accelerated U.S. Food and Drug Administration (FDA) review pathways have fueled an unprecedented wave of innovation. These advancements offer hope for treating complex conditions — but they also create concerns around affordability and health outcomes.  

Prime Therapeutics (Prime) stays atop the latest trends in the drug pipeline and delivers timely, credible insights into this fast-moving landscape with its new Cell & Gene Pipeline Outlook. This publication highlights therapies on the horizon and provides strategic context for payers, providers and health care decision-makers. The goal is simple: to help stakeholders be prepared as CGTs reshape health care delivery and manage its associated cost.  

To learn more about this publication and the insights it shares, the Prime newsroom team caught up with Maryam Tabatabai, PharmD, associate vice president of clinical information, who is part of the team of experts that curate this new publication.   

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What are cell and gene therapies? 

Cell and gene therapies are two separate and sometimes overlapping types of treatment that use cells and/or genetic material to treat or prevent disease. These personalized, regenerative treatments can take different forms, including different platforms, therapeutic targets and delivery mechanisms. They’re also incredibly costly. In fact, the most expensive drug currently in the market is the gene therapy atidarsagene autotemcel (Lenmeldy), which is approved to treat metachromatic leukodystrophy. The drug, from Orchard Therapeutics, came in at $4.25 million. 

The first gene therapy to be approved by the FDA was voretigene neparvovec-rzyl (Luxturna) from Spark Therapeutics in 2017 for the rare disease retinal dystrophy, a form of inherited blindness. Similarly, the first CAR-T cell therapy to be approved, also during the same year, was tisagenlecleucel (Kymriah) from Novartis. Kymriah is indicated for select patients with acute lymphoblastic leukemia (ALL) and was subsequently approved for certain types of non-Hodgkin lymphoma — blood cancers.  

In the last eight years, the FDA has approved dozens of CGTs. A recent approval from Krystal Biotech, for example, is used to treat the rare skin disorder dystrophic epidermolysis bullosa (DEB) and uses a novel mechanism. It is applied weekly as a topical gel until wound closure.  

Although some treatments require multiple doses, many hold the promise to cure intractable diseases with just a single dose. These are highly complex therapies and are not without risk. Because these treatments are so new and therefore have not been studied for long, their long-term effectiveness is still largely unknown. That said, CGTs have the potential to alter the trajectory of certain diseases and bring hope to patients and their families living with incurable or debilitating conditions.   

Why did Prime develop the Cell & Gene Pipeline Outlook publication? 

The groundbreaking discovery of the DNA double helix — the code of life — in the 1950s, the remarkable completion of the Human Genome Project in 2003 and the landmark paper on CRISPR-Cas9 gene editing technology in 2012 laid out the blueprint for advancements in human genetic innovation. We now live in a time where precision medicine and the genomic medicine landscape are evolving rapidly. From research and drug development to artificial intelligence, chemistry, manufacturing and controls (CMC), regulatory shifts and even the drugs themselves, CGTs are an important area of the pipeline with impact to human health. Prime’s Cell & Gene Pipeline Outlook is a timely and credible resource with curated expertise for these emerging therapies. Our team developed this publication to share proactive insights on what stakeholders can expect in the CGT pipeline.  

How does Prime manage CGTs?  

Prime offers a comprehensive suite of solutions to help manage the complexity and cost of CGTs, prioritizing optionality to meet clients where they are with their unique needs. These include a product portfolio featuring risk protection through reinsurance, patient navigation via a national Center of Excellence network and outcomes-based contracts with manufacturers to tie payment to clinical success. Prime also provides predictive analytics, proactive pipeline monitoring and utilization management strategies to help guide appropriate therapy use, along with flexible value-based contracting and affordability programs. These solutions are designed to help mitigate financial risk, address access issues and assist in delivering better health outcomes for members across both pharmacy and medical benefits. 

What’s the FDA’s approach to CGTs? 

The FDA evaluates CGTs under a rigorous but flexible framework designed to ensure safety, efficacy and quality; however, all therapies have risks associated with use. These products are regulated as biologics by the Center for Biologics Evaluation and Research (CBER) and require an Investigational New Drug (IND) application for clinical trials and a Biologics License Application (BLA) for approval. The agency emphasizes substantial evidence of effectiveness, robust manufacturing controls, and long-term patient follow-up (up to 15 years based on product type). To accelerate access for serious conditions, the FDA may use expedited pathways like Regenerative Medicine Advanced Therapy (RMAT) designation, while requiring post-approval evidence collection to monitor durability and safety.  

As part of a roundtable on CGT, the agency communicated its interest in evolving and facilitating regulatory processes and increasing efficiency while maintaining rigorous standards for safety and efficacy. The FDA has published draft guidance documents on CGT with focus on expedited programs, innovative study designs, and post-approval monitoring, especially because CGTs often target rare diseases with small patient populations. Moreover, the FDA has signaled its openness for a potential "plausible mechanism” pathway. The agency has stated that study requirements for rare diseases will be different than for those with large populations.  

The FDA has also awarded its Commissioner’s National Priority Review Voucher (CNPV) to two gene therapies. Voucher recipients will receive a decision within 1–2 months, instead of 10–12 months, following filing of a complete application for an agent.  

• Exagamglogene autotemcel (Casgevy) age expansion for sickle cell from Vertex Pharmaceuticals 
• DB-OTO for a type of rare deafness from Roche 

What is in store for the CGT pipeline? What are therapeutic areas of focus?  

A hallmark of Prime’s Cell & Gene Pipeline Outlook publication is not only the inclusion of novel, experimental medicines but also new uses or formulations for existing CGTs including several pending FDA approval. While the bulk of the agents profiled are for rare or orphan conditions, we also feature therapies for more common conditions as well. Each listing includes development status — whether an FDA application has been submitted or if safety and efficacy are being studied in Phase 2 or 3 clinical trials.  

In the inaugural edition of the Cell & Gene Pipeline Outlook, we share some key insights including:  

• Nine of the reported agents have FDA decision dates. Of these, four are anticipated in 2025 including one new moiety.  
• While most CGTs in the pipeline are for treating rare diseases, there are those profiled for common conditions including type 1 diabetes, wet age-related macular degeneration and certain cancers such as lung and bladder cancers. 
• An off-the-shelf CAR T-cell therapy is included. These allogenic therapies can be manufactured in advance and potentially increase access.  
• Approximately 10 agents in Prime’s report may undergo FDA’s Accelerated Approval, which allows expedited approval based on surrogate markers instead of clinical outcomes. Confirmatory trials are needed to show benefit and for the drugs on remain on the market.  

Oncology, metabolic and rare disease are the top categories. Some examples include therapies for multiple myeloma, a blood cancer; Hunter syndrome, an inherited lysosomal storage disease; and stiff person syndrome, a rare autoimmune neurological condition that received additional awareness after Canadian singer Celine Dion revealed she was diagnosed with the condition.  

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To read the Cell & Gene Pipeline Outlook, visit PrimeTherapeutics.com/Read.  

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