INSIGHTS ON CELL & GENE CLINICAL TRIALS

• Advancing Novel Therapeutics With End-To-End Lipid Nanoparticle Development And Manufacturing

  An effective partner must possess the necessary unique analytical capabilities for LNP characterization and the ability to support clients from concept to commercialization.

• Considerations For Clinical Development of Cell & Gene Therapies

  Insights from Part 2 of Considerations for the Clinical Development of Cell & Gene Therapies, a two-part panel discussion featuring C-suite leaders from advanced therapy companies.

• Considerations For Cell & Gene Therapy Clinical Development, Part 1

  Part 1 of a 2-part series featuring C-suite leaders from advanced therapy companies at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting.

• FDA Releases Draft Guidance On Clinical Recommendations For “N of 1” Gene Therapies

  To eliminate ineffectiveness due to genetic differences, clinical trials of individual patients, described as an “N of 1” (single-subject) trial, can provide crucial insight. The regulatory path for conducting such gene therapy trials has been difficult to ascertain until now. This article shares the key takeaways of the FDA's draft guidance and provides an analysis for moving forward.

• A Five-Point Strategy For Building A CMC Dossier

  By adopting strategies to build a robust CMC package to help streamline the path to FIH trials, innovators can establish a quality foundation to support the development phases toward commercialization.

• Defining Meaningful Outcomes In Gene Therapy Trials For Duchenne Muscular Dystrophy

  Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is an X-linked recessive disease that affects approximately one in 5,000 live male births and has no curative therapy. This article discusses the limitations of classical therapeutic endpoints and how to consider novel strategies.

• How One Biotech Leveraged Advanced Preclinical Studies

  BriOri Biotech used cutting-edge technology to achieve the sensitivity and reduced carryover needed for analysis of reformulated COX-2 inhibitor.

• Patient Diversity In Clinical Studies: The Key To Advancing Precision Medicine

  Until we have better inclusion of participants with diverse demographics and behaviors – and can analyze that data – we won’t know the effectiveness of a product for these patients. This article shares the barriers to patient diversity in traditional studies, the efforts that the authors' company is putting forth, and considerations to keep in mind.

• Fixing The Patient Data Collection Process To Solve Bias In Precision Medicine

  A big problem in personalized medicine is that existing data overwhelmingly represents people of European descent, so a machine learning model built on such data sets can be fundamentally biased. However, it is often the data — not the algorithms — that are the root cause of the issues. These authors decided to look into fixing the processes that collect the data.

• Rare Disease Trial Strategies

  This white paper focuses on clinical trials, aligning the latest trends in rare disease R&D viewed through the lens of gold-standard clinical intelligence. From this emerges practical recommendations regarding clinical trial design, stakeholder engagement, and patient recruitment.