Newsletter | January 14, 2021

01.14.21 -- How To Navigate Regulatory Frameworks For Gene Therapy Development

How To Navigate Global Regulatory Frameworks And Expedited Programs For Gene Therapy Development In The U.S., EU, And Japan

As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. This article reviews the regulatory frameworks for gene therapies in the U.S., EU, and Japan, with a focus on programs designed to streamline the product development and approval process.

Parkinson’s: Why Has Disease Modification Failed — And What Now?

Researchers have made many attempts at disease modification as they pursue breakthroughs in treating Parkinson’s disease, but so far without success. Why have these efforts failed, and what’s next in treating this degenerative disorder that affects an estimated 10 million people worldwide? This article examines these past attempts, why they failed, and what targets are currently being pursued.

The Role Of Long-Acting Injectable Antipsychotics In Schizophrenia

Use of long-acting injectable (LAI) antipsychotics is often reserved for patients with chronic or treatment-refractory schizophrenia or repeated noncompliance issues. However, recent studies have consistently found a role for these treatments, both soon after diagnosis and in the treatment of chronic disease. This article reviews the symptomatology and pathophysiology of schizophrenia and discusses the pros and cons of treatment with LAIs.

Conclusive And Positive Results Delivered Ahead Of Time — By Eight Months

Most patients with binge eating disorder (BED) seek help from psychiatrists, nutritionists, or obesity specialists, as there was no approved, effective pharmacologic treatment. With unanticipated interest in these pivotal Phase 3 studies, learn how a CRO was able to line up and train the right resources while collaborating effectively with the central laboratory, data manager, and other participants.