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 […]

Operationalizing Cell and Gene Therapy Trials

Originally published by BioPharmaDive Small and mid-sized biotech companies face unique challenges when bringing novel therapies to market. While many large pharmaceutical companies have the knowledge and in-house talent to clear regulatory hurdles and conduct efficient clinical trials, smaller companies often do not. That’s why small and mid-sized biotechs need to THINK BIG when working to […]

Long-Term Follow-Up for CGT Trials: Best Practices to Operationalize

By Desmond Cabrera, Project Director, Cell and Gene Therapy With today’s cutting-edge technology, we are on the cusp of developing truly magnificent therapies using cell and gene science. However, as with any new therapy, we must always consider the patient first.   Guidelines to Ensure Patient Safety  As far back as 2001, as new cell and […]

A Regulatory Perspective on Cell and Gene Therapy in Oncology

By Jessica Lee, VP, Regulatory Strategy, Head, Cell/Gene Therapy The past decade has produced many promising innovative cell and gene therapies (CGTs) to treat human diseases. CGT products, which use genetic material to modify cells in vitro or in vivo to treat, prevent, or potentially cure diseases, have great potential to address unmet medical needs. […]

Developing Oligonucleotide Therapies

Oligonucleotides are short strands of DNA or RNA molecules that contain a small number of nucleotides. Each nucleotide consists of a nitrogenous heterocyclic base (or nucleobase), a five-carbon pentose sugar (deoxyribose in DNA or ribose in RNA), and a phosphate group. The development of nucleotide and oligonucleotide therapeutics has been growing in importance over the […]

Navigating Regulatory Strategies for Cellular & Gene Therapies

While any drug development program can face regulatory hurdles, developing cellular and gene therapies often presents unique challenges. Small and midsize biotech and pharma companies developing cellular and gene therapies should begin to understand critical nuances for successful regulatory meetings and submissions and how they impact strategic decisions during the product development lifecycle. At the […]

The Case for Early Regulatory Consideration for Clinical Development of Chimeric Antigen Receptor T cell (CART)

By Jessica Lee, VP, Regulatory Strategy, Head, Cell/Gene Therapy Chimeric Antigen Receptor T cell (CART) has revolutionized the treatment of hematological malignancies, including B-cell acute lymphoblastic leukemia, B-cell lymphomas, and multiple myeloma. Currently, there are five autologous CART therapies approved for various hematologic malignancies. However, there are limitations in producing autologous CART products, including manufacturing […]

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 […]