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.
The recent FDA guidance titled, 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.
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.
A client engaged the Veristat team in a Phase II study of intracerebral stem cell injection in patients following an ischemic stroke. The goal was to look at limb recovery time and level in patients with stroke. The effort began with identifying the best time for a product to be delivered and then expanded into full-service involvement, including project management, clinical monitoring, medical writing, and regulatory affairs. Learn how we overcame unique challenges in this multi-center trial that led to the client publishing positive Phase IIa data.
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.
No two new drug applications (NDAs) or marketing authorization applications (MAAs) are the same. Learn what key strategies you can implement to propel even the most complex submissions to successful regulatory conclusions.