Cell and Gene Therapy Advancements: Early-Stage CMC Strategies for Exosome Innovation

By Marites T. Woon (Associate Director, Nonclinical), Mialy Ramaroson (Director, CMC), Jessica Lee (Principal, Global Head Cell & Gene Therapy) Welcome back to our exploration of the transformative world of exosomes in the second installment of our blog series. In our initial dive, we uncovered the intriguing potential of exosomes as dynamic messengers in intercellular […]

Exosomes: Unveiling Their Potential in Cell and Gene Therapy

By Marites T. Woon (Associate Director, Nonclinical), Mialy Ramaroson (Director, CMC), Jessica Lee (Principal, Global Head Cell & Gene Therapy), William Lee (Vice President, Regulatory Affairs), and Richard Stewart (Vice President, Nonclinical)   Welcome to our three-part blog series on exosomes, the fascinating messengers of the cellular world. In this captivating exploration, we delve into the […]

The Potential of Biosimilars – for Drug Makers and Patients

The production of biosimilar drugs remains at a rapid pace and represents not only an advantage for patients but also a remarkable profit opportunity for drug makers. And for patients, the approval of biosimilars may represent the only alternative of treatment due to the extremely high cost of many original biologic drugs. That said, in […]

CAR T-Cell Therapy Challenges to Development & Approval

Chimeric antigen receptor T-cell (CAR T) therapy is a revolutionary immunotherapy that uses cells from the patient’s immune system to fight certain kinds of cancer. The first two FDA approved CAR T therapies, Yescarta™ and Kymriah™, were both approved in 2017. Former FDA Commissioner, Scott Gottlieb, noted the significance of these approvals in his October […]

Strategizing for Your Submission: Planning Ahead for Your NDA or BLA

Pharmaceutical marketing applications, whether a new drug application (NDA) or biologic licensing application (BLA), are massive undertakings. They can contain tens to hundreds of thousands of pages of documents. Therefore it behooves a sponsor to plan such an application well in advance. Often planning for the application is postponed until the pivotal Phase 3 clinical […]

Cellular & Gene Therapy: Implications on Drug Development

What is Cellular & Gene Therapy? Genes are defined by the NIH as the “basic physical and functional unit of heredity.” We receive these valuable sequences of DNA from our parents, however genes go far beyond inheritance. Genes are code for the creation of all proteins in living organisms and serve as the metaphorical instructional manual for […]

eCTD Regulatory Submissions: Hints & Common Mistakes to Avoid

The electronic common technical document (eCTD) is the standard format for submitting applications, amendments, supplements, and reports to FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). Understanding eCTD requirements and successfully applying them to your submissions is critical. However, these requirements are not always intuitive, and headaches […]