Knowledge Center

In this webinar, we'll discuss materials and methods to enable the expression of biomolecules on EVs. Data will be shared for using protein scaffolds, such as PTGFRN and BASP-1, that are clinically proven to effectively load therapeutic proteins on the su...

Abstract: Recombinant adeno-associated virus (rAAV) has emerged as the vector of choice for in vivo gene delivery, with numerous clinical trials underway for the treatment of various human diseases. Utilizing rAAV in gene therapy requires a highly precise...

Women In Science Spotlight featuring Inbar Friedrich Ben-Nun, Director of R&D, Lonza Cell & Gene Therapies

Exosomes represent one type of extracellular vesicle (EV) generated by all eukaryotic cells. Exosomes are a new modality with tremendous potential to enable delivery of drug substances that are too toxic for free systemic administration, to what were prev...

Dr. Lisa Prendergast leads innovation for Lonza's Licensing Business Unit, supporting the growth of expression system platforms which are available through Lonza in Your Lab® programs. Her role is to provide expertise in Molecular Technologies, including ...

Performance data of the 9-day process for functionally-closed and automated manufacture of CD19-CAR-T cells using the Lonza Cocoon® Platform Contributors: Kathleen Zohorsky, Aman Mehrotra, Beth Macleod, Khalil Dayekh, Kelly A Purpura, Rahul Purohit, Janet...

Abstract: During this webinar, our experts will explore recent developments that highlight the ability to adapt an automated manufacturing platform to different processes, such as positively or negatively selecting T cells directly from apheresis. Automat...

A complex process, prone to misconceptions and pitfalls, to find the “right human tissue” that will yield the desired cell quality and quantity, and pass BLA approval. Download our infographic to learn more

Abstract: During this webinar, our expert will share insights on how precise analytics and process design can ensure your extracellular vesicle (EV) therapies succeed in clinical development. Key learning points: - Why characterization is the cornerstone ...

Performance data of the Cocoon® Platform using Buoyancy-Activated Cell Sorting™ for T cell isolation from leukopaks: isolation of T cells directly from cryopreserved leukopaks: - No additional upstream equipment required - Reduced operator interactions - ...

Demand for lentiviral vectors (LVVs) as delivery vehicles for gene therapies is rising significantly, creating a need for robust, highly productive and scalable manufacturing processes. Lonza’s ready-to-use, suspension-based Xcite® Lenti Platform Technolo...

Abstract: During this webinar, our experts will share insights on how our cutting-edge AAV transient transfection and producer cell line platforms can significantly accelerate your journey towards Investigational New Drug (IND) application success. Curren...

Abstract: The superior safety profile and transduction efficiency make AAV an excellent viral vector for human gene therapy. However, current AAV manufacturing suffers from low productivity and scalability, high raw material cost and supply chain complexity, and high batch-to-batch variation. To overcome these limitations and industrialize AAV manufacturing, we have generated a high-performing, helper virus-free, Xcite® AAV stable producer cell line (PCL). The cell line is built upon our proprietary high-producer HEK293 cells used for AAV transient transfection platform, grown in chemically defined, serum-free, ADCF medium in suspension. The DNA expression vectors are designed to tightly control viral gene expression via engineered inducible promoters, and stably integrated into the cell genome in one step by our VS piggyBacTM transposon system, which markedly increase the efficiency of cell line construction. Upon small chemical doxycycline induction at regular cell density, our selected PCL clones outperform transient transfection method with even higher AAV titer (>1E12 vg/mL) and comparable AAV genome packaging (>...

Abstract: The genome packaging efficiency (full to total capsid ratio, or F/T ratio) for Adeno-Associated Virus (AAV) is a critical quality attribute for the manufacturing and commercialization of AAV-based gene therapies. A higher F/T ratio has the poten...

Abstract: Extracellular Vesicles (EVs), including exosomes, are emerging as promising natural nano-scale platforms for delivering nucleic acids, proteins, and small molecules. However, precision EV engineering has been challenging to date. Such technologi...

Abstract: Recombinant adeno-associated virus (rAAV) has emerged as the vector of choice for in vivo gene delivery, with numerous clinical trials underway for the treatment of various human diseases. Utilizing rAAV in gene therapy requires a highly precise...

Abstract: Due to their ability to deliver larger cargo, transduce both dividing and non-dividing cells and maintain long-term and high expression levels, lentiviral vectors (LVV) are increasingly becoming a tool of choice in cell and gene therapy applicat...

Performance data of the Cocoon® Platform using magnetic separation: cell viabilities, purity, etc. Contributors: Carlene Petes, Michele Vicentini-Hogan, Chad Ibbotson, Kathleen Zohorsky, Ashkan Tabibian, Khalil Dayekh, Aman Mehrotra, and Kelly A. Purpura

Adeno-associated virus (AAV) gene therapy has shown immense promise in treating various genetic disorders. Reducing AAV production costs will enable its use on a larger scale, and make gene therapy more accessible to a broader population. Enhancements in ...

Many of the decisions made during the early days of building and compiling the manufacturing information for your IND submission will directly impact the future cGMP manufacturing operations and simply become foundational for the commercial viability of t...

Extracellular Vesicles (EVs), including exosomes, are emerging as promising natural nano-scale platforms for delivering nucleic acids, proteins, and small molecules due to their immune silencing properties. However, precision EV engineering has been chall...

The packaging efficiency (full to total capsid ratio, or F/T ratio) for Adeno-Associated Virus (AAV) is a critical quality attribute for the manufacturing and commercialization of AAV-based gene therapies. A higher F/T ratio has the potential for reducing...

Despite the remarkable progress and vast growth in the number of CAR-T cell programs transitioning into the clinic and revolutionizing the oncology landscape, the complex manufacturing process associated with these therapies presents challenges impacting ...

After entering Phase 3, cell and gene therapies experience nearly five times more discontinuations or significant delays due to CMC issues than monoclonal antibodies. The highly manual processes, lack of robust analytical methods, and lack of quality stan...

From clinical stages to FDA-approved gene therapies: At the 2023 CAR-TCR Summit in Boston, bluebird bio and Lonza took the opening plenary stage to share the unique story of our 10-year journey commercializing two gene therapies. Ten years ago, in January...

Abstract: Autologous cell-based therapeutics have gained increasing attention in recent years because of their efficacy at treating diseases with limited therapeutic options. Chimeric antigen receptor (CAR) T-cell therapy has demonstrated clinical success...

Performance data of T cell expansion: high viability and purity Contributors: Carlene Petes, Kathleen Zohorsky, Kelly A. Purpura, Matteo Da Ros, Seyedehosna Hadavi, Aman Mehrotra, Michele Vicentini-Hogan, Steven Bouman, Janet J. Sei, Tamara Laskowski.

Abstract: Recombinant adeno-associated virus (rAAV) has been utilized successfully for in vivo gene delivery for treatment of a variety of human diseases. To sustain the growth of recombinant AAV gene therapy products, there is a critical need for the dev...