Cell and Gene Therapies
Transforming Lives
According to ClinicalTrials.gov, there are over 1,500 registered ongoing clinical trials for cell and gene therapies (C>s). C> is poised to deliver on its promise of transforming lives. C> promises to deliver life-changing remedies to rare and orphan disease populations by finding a cure, as opposed to management or treatment.
While the Food and Drug Administration (FDA) approved the first therapy as recently as 2017, through 2022, the FDA approved a total of 27 C>s. This represents an increasing proportion of total approvals and approximately eight percent of the 340 approved biologics. The research landscape has been energized by recent significant therapeutic successes.
Our Insights
While gaining traction, biotech companies recognize the future of this unique research ecosystem means facing those challenges head-on in manufacturing, regulatory requirements, staffing, and investment from research to commercialization.
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Research to Commercialization
With C> sales expected to exceed $86 billion by 2028, accelerating the go-to-market journey is key for innovative life sciences companies. The unique research and development ecosystem for C> – distinct from their conventional biopharma cohorts – contributes to a high price tag for patients. While C> is risky to develop and is often difficult to target in small populations, there is a promise of replacing a lifetime of treatments with a single curative dose. In spite of this high price tag, C> continues to attract pharmaceutical companies as they race to selectively add late-stage, promising assets to their drug development pipelines.
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Optimizing Research Performance
A key imperative for sustaining fluid capital and achieving go-to-market success is the ability to meet critical milestones. Doing so means cell and gene companies must demonstrate to key stakeholders their clinical development plan is risk-based, eliminates research inefficiencies, streamlines clinical trial startup, and integrates advanced technology platforms.
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Streamlining Study Startup
Fast and effective site selection, streamlined contract negotiations, and efficient ethics and regulatory approvals all impact advancing studies onto Phases II and III. With preparation needed for C> drug development, identifying possible sites is critical. With a gene therapy ready (GTR) site, study startup is typically completed five to six weeks quicker than non-GTR sites, and eight to 12 weeks faster than sites using a local IBC. Additionally, streamlining study startup using a GTR network demonstrates expertise and readiness to conduct your trials involving genetically engineered therapies and vaccines.
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Advanced Technology Solutions
While C> organizations target small populations, ensuring compliant and confident study protocol execution is still a critical regulatory requirement, often impacting advancing milestones. For C>s, it is imperative for technology to empower and inform study participants while efficiently managing and monitoring their complex studies within a single, intuitive platform. Successful go-to-market launches hinge on streamlining key processes, keeping study teams, site staff, and participants compliant, connected, and moving forward.
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Compliance Complexities: Safety and Ethics Reviews
In addition to the institutional review board (IRB) review requirement for clinical research involving human participants, an institutional biosafety committee (IBC) also must review studies involving recombinant, synthetic, messenger nucleic acids (rNA, sNA, mRNA, siRNA), and other genetically engineered treatments as part of regulatory requirements for adequate data safety monitoring plans. Engage specialized review committees like data safety monitoring boards (DSMBs) and clinical event committees (CECs) if there is a reason for a particular safety concern if the study treatment has the potential for serious toxicity, and if the study involves a potentially vulnerable participant population.
Did you Know?
of the top 100 cell and gene companies have used Advarra Technology or Services
of the top 10 pharma companies have used Advarra Services
of the top 100 cell and gene companies have worked with Advarra Consulting
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Read our Related Resources
FDA Guidance Offers New Flexibility to Biotechs in Cell and Gene Therapy
FDA recently released new guidance, potentially impacting early-phase cell and gene therapy trials.
Regulatory Trends in Cell and Gene Therapies
Recent signals have indicated the FDA plans to provide greater support for cell and gene therapy research.
A Guide for Successful Cell and Gene Therapy Research
Understand the unique regulatory, clinical, and manufacturing requirements for cell and gene therapy research.
