ABOUT VERISTAT, INC.

Veristat, a scientific-minded global clinical research organization (CRO), enables sponsors to solve the unique challenges associated with bringing cell and gene therapies to market. With experience in supporting over 100 cell and gene therapy trials, including the first gene therapy approved in Europe, Veristat is adept at strategy and execution across this complex clinical development pathway.

Veristat understands how high the stakes are with cell and gene therapy programs and that each therapy is unique, often coming with a complicated regulatory approval process and therapeutic challenges. We offer tailored solutions to combat these challenges, including:

  • The ability to develop your therapeutic with accelerated approval pathways and/or special designations
  • A review of operational aspects of the protocol and feedback on any potential obstacles that could impact the protocol execution at the clinical research sites
  • Novel strategies including implementation of a centralized site model and remote monitoring with direct-to-patient capabilities
  • Expertise in logistics and handling of live cellular material
  • Long-term follow-up strategies to meet global regulatory requirements

The complexities of developing cell and gene therapies requires a committed and experienced partner. Veristat has assembled an extraordinary team of scientific experts worldwide who have mastered therapeutic development intricacies in this specialized area. Whatever the study’s unique considerations – patients, products, process, follow-up, regulatory – Veristat can successfully get you through it. For more information, visit www.veristat.com.

PODCASTS

  • Listen as we replay some of Ewan Campbell’s insights from a recent webinar where Ewan provides a historical recap of the approved cell and gene therapy treatments over the past decade along with insights on where we are headed.

  • Listen as Robin Bliss describes the appropriate execution of PROs using clearly defined endpoints, measurable changes within context to the indication and disease progression, and several other key considerations for deployment in cell and gene therapy studies. 

  • Administering advanced therapy products during a clinical trial can bring about many unique circumstances. Listen as Rachel Smith discusses Convection Enhanced Delivery (CED) which delivers medicines directly into the brain. Learn how to prepare for its challenges and what it can mean for patients, clinical teams, and sponsors.

  • Listen as Ewan Campbell, Advanced Therapy and Biotech Director for Veristat outlines some of the unique challenges faced when developing advanced therapies and shares some of his experience gained in neurodegenerative disease studies over the past 20 years.

  • Listen as Kevin, Senior Regulatory Strategist for Veristat, identifies some of the key components of a cell & gene therapy marketing application in the US and EU, and outlines several of the most common pitfalls and gaps that he has encountered with sponsor applications. 

  • The pros, cons and future of allogeneic versus autologous therapies are explored as Rachel Smith, Portfolio Director for Veristat, shares her observations on the impact of these techniques on advanced cell-based therapies and what the next decade may hold.

  • Rachel Smith, Portfolio Director for Veristat, discusses the three types of post-marketing requirements that can be requested by the EMA and draws upon the recently approved therapies – Tecartus, Zolgensma and Libmeldy, Skysona and Abecma– for real-world context.

  • Robin Bliss, PhD, and VP of Strategic Consulting for Veristat, takes us through the how-to’s of selecting appropriate endpoints for an early phase study, the importance of considering endpoints in the context of clinical meaning, and the value of ensuring that endpoints are measurable within a reasonable amount of time.

  • Mariana Oviedo, Project Manager for Veristat, provides an overview of the key items that must be taken into consideration when designing and running a long-term follow-up study for a gene therapy product.

  • Hear from Rachel Smith, Portfolio Director for Veristat, as she uses our recent work with a biotech company to illustrate the importance of a comprehensive shipping and traceability strategy, tight management of the patient journey, and adoption of a well-conceived protocol and process documentation to support the commercialization strategy.

  • Rachel Smith, Portfolio Director for Veristat, takes us through a number of pitfalls to avoid when planning a gene therapy study. With the stakes high and having conducted trials for the first gene therapy approved in Europe, put Rachel’s lessons learned to work when determining your regulatory pathway, logistics strategies and donor screening requirements.

  • Listen as Kevin Hennegan, Senior Regulatory Strategist for Veristat, shares some of the common hurdles drug developers face when bringing cell therapies to market including international regulatory variability, patient safety assurance, planning for long-term follow-up and more.

CONTACT INFORMATION

Veristat, Inc.

134 Turnpike Road, Suite 200

Southborough, MA 01772

UNITED STATES

Phone: 508-306-6281

Contact: Lauren Willis

FEATURED ARTICLES

  • The recent FDA guidance entitled "Human Gene Therapy for Neurodegenerative Diseases" (ND) provides recommendations for Sponsors who are developing human gene therapy (GT) products for neurodegenerative diseases, which affect adult and pediatric patients. It focuses on considerations for product development, preclinical testing, clinical trial design and marketing approval pathways. This article outlines several areas to consider when preparing for regulatory agency interactions.

CASE STUDIES

  • A small biotech company engaged Veristat early in clinical development for help with a novel biologic being tested for treatment of an ultra-rare and aggressive hematologic malignancy with no available effective therapies. Discover what happens in this case study.

  • A mid-size pharmaceutical company and its development partner, a small biotechnology company engaged us to complete the medical writing for their product’s Marketing Authorization Application (MAA), New Drug Application (NDA), and New Drug Submission (NDS). Learn how our medical writing team worked closely with the sponsors to write these marketing applications in an accelerated timeline.

  • A mid-sized biopharmaceutical company dedicated to developing innovative therapies for patients with debilitating rare diseases acquired the rights to a new investigational drug from a large pharmaceutical company with the intention of preparing the data for a Marketing Authorization Application (MAA). Learn how our team had to think creatively to enter, clean and standardize the clinical trial data from previous and ongoing trials in order to get the MAA ready for submission in an extremely short timeline.

  • Veristat was brought in to create and manage a full development program for a gene therapy for a very rare inherited disorder. The work involved charting the course in completely uncharted waters – our team had to create industry best practices that didn’t exist before, anywhere. With no classic route to market, Veristat’s experts wrote the map on patient recruitment, regulatory and health agency engagement, natural history studies and the Central Site Model.

  • A long-term client called upon the Veristat team for help conducting two investigational Phase I/II studies. These global studies focused on proving the safety and efficacy of gene therapy for two rare genetic disorders. Working side-by-side with the client, our cell and gene therapy experts addressed many complicated challenges – including site start-up issues, stringent regulatory requirements, and the logistics of handling highly sensitive genetic material.

  • A clinical-stage biotechnology start-up asked Veristat to run a new European trial of their complex gene therapy. Veristat experts complimented the client’s team taking on their first clinical-stage program. Our global cell and gene therapy team brought knowledge in genetic material handling, relationships with key global regulatory agencies, and expertise in accelerated pathways to approval.

WEBINARS

  • In this webinar experts explore how to develop your marketing application strategy, analyze the benefits and risks associated with your data migration strategy and coordinate with the medical writing team so that all the information needed to write and complete the modules of the submission is provided with adequate time for review and quality control.

  • Submitting an Investigational New Drug (IND) application to the US FDA is a significant step in the development of a new drug or biologic product. But, are you ready? In this webinar, experts explore the minimum required content for an IND submission and the strategic considerations for developing more than the minimum necessary data before filing an IND.

  • Nothing is standard about the study design, study conduct or the regulatory process in cell and gene therapy trials. Every cell and gene therapy is different due to the specific characteristics of each product, with a complicated regulatory approval process and unique therapeutic challenges. In this webinar, participants will learn an end-to-end approach to bringing a cell and gene therapy to market.

WHITE PAPERS