INSIGHTS ON CELL & GENE CLINICAL TRIALS

• 5 Key Takeaways: Insights On Alternative Designs To The Traditional 3+3 Design In Phase 1 Dose Escalation Studies

  During Premier Research’s recent webinar Alternative Designs to the Traditional 3+3 Design in Phase 1 Dose Escalation Studies, Abie Ekangaki, Vice President, Statistical Consulting, and Andreas Schreiner, Vice President, Medical Affairs, Neuroscience & Analgesia, discuss alternative dose-escalation paradigms introduced into the clinical trial landscape for Phase 1 trials. In this blog post, we share five of their key insights on alternative dose escalation strategies for Phase 1 studies.

• Developing Advanced Therapies: Considerations For Internal Versus External Manufacturing

  Due to their complexity and cost, developers of cell and gene therapies face unique manufacturing hurdles. Determining the right approach to manufacturing is crucial when bringing these advanced therapeutics from clinical to commercialization. This paper provides a distinct perspective on how to approach manufacturing and other roadblocks at every stage of development.

• Drug Repurposing Trends And Strategic Approaches For Shortening Timelines

  As the complexity of drug development increases, so does the industry’s focus on strategies and solutions that can help bring advanced products to market as quickly as possible.

• Preventing The Development Of Muscular Dystrophy Through Surrogate Gene Therapy

  Researchers have identified a key regulatory protein implicated in Galgt2 overexpression and began to elucidate its protective mechanism against muscular dystrophy.

• Advancing Patient Healthcare: Clarifying The 2019 Changes In India’s Drug And Clinical Trial Rules

  The Ministry of Health and Family Welfare, responsible for all health policy in India, took steps to clarify their regulatory requirements and address many of sponsors’ concerns with the publication of its New Drugs and Clinical Trials Rules in 2019. With these changes, it is worthwhile for sponsors, especially biotech and specialty pharmaceutical companies, to consider the many opportunities that India and other Asia-Pacific countries offer. We have summarized some of the most significant changes in this article.

• Rethinking Data Quality Best Practices In The Era Of Decentralized Clinical Trials

  Pandemic-related disruptions have accelerated much-needed change in clinical operations, but this change has been accompanied by questions about data collection and data quality. While the adoption of DCT approaches can benefit patients, sites, and sponsors alike, successful implementation of these approaches requires careful consideration of the regulatory guidance, processes, and technologies necessary to ensure data quality and manage risk.

• Optimizing Successful Development Of Viral Vector Gene Therapies, Gene Therapy Trials, And Companion Diagnostics

  Despite risks associated with gene therapy, the field is growing as a promising treatment for various diseases. In this paper we provide a general background of the gene therapy process and discuss hurdles to the development of viral vector gene therapies, with specific considerations for adeno-associated-virus (AAV)–based gene therapies, challenges to initiating a clinical trial with a gene therapy, and how Precision for Medicine can help develop gene therapy programs from bench to bedside.

• An Expanded, Multicenter Look At Gene Therapy For Spinal Muscular Atrophy

  In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been studied in children aged up to 8 months. In the new study, Dr. Mendell and colleagues from Nationwide Children’s and three other Ohio children’s hospitals report safety and early outcome data from 21 children (age 1-23 months) treated with onasemnogene abeparvovec-xioi in Ohio.

• The Value Of Gene Therapy In Spinal Muscular Atrophy

  A client was developing a potentially curative gene therapy, they needed to translate the clinical benefit into economic terms to engage payers and health technology organizations. By working with the client to develop a cost-effectiveness and budget impact model for a gene therapy in SMA that overcame unique challenges of modeling an early childhood neuromuscular genetic disorder they were able to change the prevailing mindset on how SMA progression is characterized.

• Process And Communication: Navigating Logistical Complexity In Matched Cellular Therapy Trials

  Autologous and other matched cellular therapies hold tremendous promise for oncology and other indications, yet the development of these transformational personalized investigational products can be operationally and logistically complex. Read how clear and consistent communication among the sponsor, sites, manufacturing facilities, and the CRO partner helps sponsors and sites mitigate any issues.