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Six years after the first approved cell and gene therapy, the field is living a renaissance. 5 additional FDA-approved therapies were added in 2022 and could be doubled in 2023, setting a new pace to cell & gene therapies. The BioRN Annual Conference on September 29th in Heidelberg will highlight next-generation approaches in the field.

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Six years after the first approved cell and gene therapy, the field is living a renaissance. 5 additional FDA-approved therapies were added in 2022 and could be doubled in 2023, setting a new pace to cell & gene therapies. The BioRN Annual Conference on September 29th in Heidelberg will highlight next-generation approaches in the field.

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Exosomes are extracellular vesicles ranging from 30 to 150 nm in diameter that are released from various types of cells, including tumor cells. They can induce apoptosis, modulate the immune system, and function as biomarkers for diagnosis. In addition, as an important component of cell-to-cell communication, exosomes can regulate the tumor microenvironment and are involved in the development, progression, and metastasis processes of numerous cancers1,2.

Figure 1: Humanization of therapeutic antibodies has reduced their immunogenicity.

Antibodies play a vital role in the immune system by providing protection against foreign invaders, such as bacteria and viruses. Produced by B cells, antibodies are protein molecules that can be harnessed to treat cancers, autoimmune disorders, and various infectious diseases. However, the human immune system may recognize modified therapeutic antibodies as foreign substances, reducing their efficacy. To overcome this challenge, the following two processes have been developed: antibody humanization and affinity maturation.

Picture: Lonza

Physiologically based pharmacokinetic (PBPK) modelling is a tool that can have a huge impact in speeding up drug development. It simulates a range of physiological factors that have an effect on how an orally dosed drug behaves, flagging up any areas where performance might be expected to be sub-optimal. This allows them to be addressed at an early stage, rather than causing bigger problems later on that might hold up development while a solution is sought.

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Transmembrane proteins play a variety of roles in physiological processes such as protein ligation, recognition, transport, anchoring, and transduction.

The Catalent Biologics facility in Madison, WI, USA offers expanded mammalian cell line engineering and biomanufacturing capabilities. Picture: ?Catalent Biologics

DSP?Tosoh Bioscience recently launched Octave© BIO, the first in a series of MCC instruments
targeting all stages of biomolecule manufacturing. Here, Tosoh discusses the potential of MCC to help alleviate downstream bottlenecks and a process intensification collaboration they recently performed with Catalent Biologics. Emily Schirmer, Interim General Manager at Catalent Biologics, provides ­selected insights on Catalent’s findings from the collaboration.

The Catalent Biologics facility in Madison, WI, USA offers expanded mammalian cell line engineering and biomanufacturing capabilities. Picture: ?Catalent Biologics

DSP Tosoh Bioscience recently launched Octave© BIO, the first in a series of MCC instruments
targeting all stages of biomolecule manufacturing. Here, Tosoh discusses the potential of MCC to help alleviate downstream bottlenecks and a process intensification collaboration they recently performed with Catalent Biologics. Emily Schirmer, Interim General Manager at Catalent Biologics, provides ­selected insights on Catalent’s findings from the collaboration.

Kimberly Shepard, Ph.D.

There is increasing interest in delivering a range of drugs via inhalation because of the benefits it can offer patients. Lonza’s Kim Shepard looks at the advantages of pulmonary delivery, and the prospect for inhaled biologics.

© Vetter Pharma International GmbH: Supporting efficient drug development with dedicated clinical development expertise.

Both the US Skokie and European Rankweil sites address customer needs in early-phase drug development