With the rising incidence of cancer and neurological disorders, many of which have historically been considered untreatable, gene therapy and technological advances are working to rapidly change these therapeutic landscapes. As of 2022, the global market for gene therapy clinical research was valued at US$7.54 billion, with a compound annual growth rate (CAGR) of 19.1% from 2023 through 2030. Gene therapy is a promising field that has the potential to revolutionize the way we treat genetic diseases. This article will discuss the need for gene therapy and list several top gene therapy CROs leading this field of research.
The Need for Gene Therapy CRO Research Services
However, the development of gene therapies requires significant efforts to address the complexities of the diseases being treated, which can be expensive and time-consuming. That’s why many biotech and pharma companies turn to contract research organizations (CROs) for their experience and guidance in this therapeutic area. CROs specialize in providing a range of services that help companies develop gene therapies more quickly and efficiently, from initial research and development to clinical trials and regulatory approval. When it comes to gene therapy CROs, there are many factors to consider, including experience, facilities, and expertise. Read on as we list the top 7 gene therapy CROs, in no particular order, and discuss the qualities that are making them stand out in this growing industry!
1 | Veristat
CRO Headquarters: Southborough, North Carolina
Veristat is a highly experienced and specialized gene therapy CRO that offers a range of services to help companies accelerate their gene products to market. With over 160 successful cell and gene therapy (CAGT) projects, Veristat has a global team of scientific experts who are well-versed in both strategy and execution across the clinical development journey. They can provide tailored clinical and regulatory strategies and expert insights to help companies navigate the intricate and complex process of gene therapy research and development. Veristat has extensive global expertise across product types, including gene therapies, cell therapies, and tissue engineering, which they leverage to assist sponsors in bringing their products to the patients who need them.
2 | Medpace
CRO Headquarters: Cincinnati, Ohio
Medpace is one of the top gene therapy CROs that provides cross-functional therapeutic expertise and patient-centric approaches to overcome complex clinical and regulatory barriers in CAGT trials. Over the past five years, Medpace has conducted over 130 advanced therapy trials in 55 countries, including more than 7800 patients and 2100 sites. Medpace provides comprehensive strategies to overcome complex clinical and regulatory barriers in the field of gene therapy, including multi-disciplinary teams of trained clinical professionals, patient-centric designs, and integrated laboratory services. This depth of experience in various geographical regions and therapeutic areas enables Medpace to provide sponsors with comprehensive strategies that address the rapidly evolving clinical, operational, and regulatory challenges in gene therapy research.
3 | Charles River Laboratories
CRO Headquarters: Wilmington, Massachusetts
Charles River Laboratories is another frontrunner in cell and gene therapy CRO research as a preclinical CRO that offers a wide range of cell products and services. What sets this CRO’s reputation apart is its extensive experience in gene therapy research, having conducted over 1000 CAGT studies in 2021 and supported the approval of 10 of these therapies by the United States Food and Drug Administration (FDA). Their comprehensive end-to-end portfolio includes innovation and support with biologics testing solutions, advisory services for gene therapy regulations, genetic model creation, microbial quality control solutions, and cell sourcing. Working as an extension of their clients’ own team, Charles River Laboratories has a committed team of scientists and technicians who share their clients’ goal of delivering safe and effective products to waiting patients.
4 | Premier Research
CRO Headquarters: Morrisville, North Carolina
Premier Research is a global leader in CRO research services that specializes in cell and gene therapy trials. The field of CAGT poses extraordinary challenges, and Premier Research is equipped to help companies navigate the complex and challenging process of developing gene therapies. Their multidisciplinary team of experts have conducted over 90 CAGT studies in the past five years, demonstrating their significant experience in the field. Notably, this CRO has experience in viral vectors, which are commonly used in gene therapy, and in both oncology and non-oncology. In addition to these services, Premier Research also offers resources such as white papers and insights that can help companies navigate the challenges of gene therapy research and development.
5 | PPD
CRO Headquarters: Wilmington, North Carolina
PPD is a global gene therapy CRO that offers end-to-end services to meet the unique demands of CAGT clinical trials, leveraging over two decades of experience and more than 130 clinical trials in the past five years. In addition, they have supported more than 75 gene therapy trials across adenovirus/adeno-associated virus (AAV), lentivirus, and retrovirus delivery platforms. In addition to PPD’s specialized and customizable lab capabilities, this CRO skillfully navigates the sensitive timelines and storage demands of CAGT development with its global logistics management expertise. PPD also facilitates extensive team training in these therapies through its employee development programs, which include ongoing education and development for sites and investigators.
6 | Catalent Pharma Solutions
CRO Headquarters: Somerset, New Jersey
Catalent is a global biologics leader with more than 30 years of experience in biologics services. They are a trusted gene therapy contract development and manufacturing organization (CDMO) partner for custom viral vector development and scalable manufacturing of AAV vectors, next-generation vaccines, and oncolytic viruses. With partnerships with more than 20 of the foremost gene therapy companies, they have experience with over 60 gene therapy programs across various stages of development. Catalent has state-of-the-art facilities in Europe and North America, making them accessible to customers of all sizes, from top global multinational pharma to small and virtual companies. Catalent’s commitment to quality can be seen throughout its organization, from its inspection-ready sites to its efforts to continually refine and improve the type and scope of services it can provide its customers.
7 | IQVIA Biotech
CRO Headquarters: Durham, North Carolina
IQVIA is one of the largest CROs in the world, with a trusted track record of supporting gene therapy development for sponsors across several continents. They offer a range of solutions to help biotech companies overcome the operational challenges and regulatory scrutiny that come with developing successful gene therapy and cell therapy clinical trials. The company’s CAGT Study Management team is a multidisciplinary unit that is specifically designed to address the specific challenges of this type of drug development. They help sponsors navigate operational challenges specific to CAGT trials, including site selection and start-up, regulatory requirements, and the intense logistical demands of product distribution and biospecimen collection. Overall, IQVIA’s dedicated experts bring complementary strategic guidance plus delivery excellence, resulting in increased value for biotech customers and a better experience for patients.
Vial, Your CRO Partner
Vial stands as a prominent entity among the top 7 gene therapy contract research organizations (CROs) that are reshaping the landscape of medical innovation. Vial is a full-service, global CRO that recognizes the role of technology in the future of drug development. Vial not only conducts faster and more affordable gene therapy trials but also covers a broad spectrum of indications, servicing various therapeutic areas, including but not limited to oncology, dermatology, and neurology.
What sets us apart from the other seven CROs is Vial’s integrated technology platform that combines an in-house EDC, ePRO, and eSource in one connected system for streamlined clinical trials, saving sponsors time and money. Our site onboarding platform allows for rapid site start-up (30 days or less), eliminating the traditional bottlenecks associated with study start-up, and ensuring that research can commence swiftly and effectively. This agility not only accelerates the pace of trials but also significantly reduces operational costs, translating to substantial savings for sponsors.
Lastly, Vial’s fixed-fee pricing ensures affordability, predictability, and peace of mind assuring sponsors that they won’t face unexpected financial burdens as their trials progress. Vial’s success in conducting diverse trials across several therapeutic areas results from deep scientific expertise, adaptable infrastructure, and an expert team, positioning Vial as a leading CRO for gene therapy research and beyond.
Contact a Vial representative today to discover how we can help!
