Author Archives: BPI Staff

Understand viral titer and quality

The cell and gene therapy field has burgeoned in the last decade with drug candidate treatments for many different diseases. Determining the correct patient dosage has become more important than ever to extend the reach of the multi-million-dollar manufacturing runs required to generate these viral therapies. Genome copies (GC), capsids, GC/capsid ratios, percent full, functional titering… What does it all mean? As of today, there is no single perfect method for capturing the qualities of viral therapies. Thus, manufacturers often…

Bristol-Myers forks out $4.1bn to acquire Turning Point Therapeutics

Bristol-Myers Squibb will acquire oncology firm Turning Point Therapeutics for 4.1 billion and gains its lead asset, repotrectinib aiming to treat lung cancer and solid tumors. The merger agreement will see Bristol-Myers Squibb (BMS) pay $76.00 per share to acquire Turning Point Therapeutics (TPT) and the deal is expected to close during the third quarter of this year. As part of the deal, BMS will take on TPTs lead asset, repotrectinib, a next generation tyrosine kinase inhibitor (TKI) targeting ROS1…

Aseptic closed small-volume processing: Today’s options

Small-volume biopharmaceutical processes continue to grow as more biopharmaceutical, cell therapy and gene therapy companies develop products. A 2021 Association for Regenerative Medicine (ARM) report states that there are 1,085 cell, gene and tissue-based therapy developers worldwide. These companies are engaged in many small-volume (<10L) processes including early-stage drug development and autologous therapies. The increase in small-volume processes coincides with the ongoing need to create fast, reliable aseptic closed systems. Historically, few convenient options have existed to facilitate sterile processing.…

Pharmaron’s cell and gene therapy webinar series

Pharmaron has taken their platform beyond small molecules and are pleased to now offer a full array of services for biologics and cell and gene therapies (CGTs). Our new CGT webinar series focusses on identifying key cell and gene therapy questions that arise during drug discovery and development. Register here for the series. Our second webinar, entitled Downstream AAV Production: A Targeted Approach to Optimization takes place on Thursday, March 24 at 9.00am ET. Join us as we highlight our…

CMC Testing Support for Gene and Cell Therapy

With the promise to enhance treatment, greatly reduce side effects, and potentially cure many types of diseases and disorders, Advanced Therapy Medicinal Products (ATMP), such as gene and cell therapy products, are in high demand, and biopharma companies are in a race to the clinic. However, these technologies are very complex in nature and are vastly different than traditional biopharmaceutical products, especially when it comes to the use of these products for personalized medicine. The complexities span the development pipeline,…

Is the AAV payload impacting the stability of your gene therapy?

The ability to use adeno-associated viruses (AAVs) for targeted therapeutic payload delivery has dramatically accelerated the development of life-saving gene therapies.  Development can further be accelerated by monitoring critical quality attribute parameters like subvisible particles (SVP) concentration early in the development process. Capsid degradation and nucleic acid leakage can both occur under different formulation and storage conditions and contribute to the accumulation of aggregates. Characterizing candidates earlier in the development process is best as it identifies and eliminates inherently unstable…

In brief: Vetter opens clinical filling site in Austria

Vetter has received manufacturing authorization for a fill and finish site Austria, which the CDMO acquired in 2020. Contract development and manufacturing organization (CDMO) Vetter bought the clinical production site in Rankweil, on the Austrian border with Switzerland and Liechenstein, from Impletio – part of Rentschler, with which Vetter inked an alliance in July 2020. The firm has been working to modify and equip all laboratory, technical and production areas at the 10,000 square-meter facility and has announced this week…

WACKER’s plug-and-play technology serves growing pDNA demand for cell and gene therapy

The global demand for nucleic acid-based gene therapies, novel vaccines and innovative therapeutic agents, including messenger RNA (mRNA) and viral vectors, is extremely high and projected to increase in the future. Plasmid DNA (pDNA) is the basis for all of these advanced therapies. Plasmid DNA can be used either directly for vaccines and gene therapies, or as starting material, e.g., to manufacture mRNA. The CDMO market for pDNA is expected to grow by 19 percent by 2025 (Global Viral Vector…

Univercells adds DNA synthesis through SynHelix buy

Univercells has entered the synthetic biology space through the acquisition of French DNA synthesis firm SynHelix. The deal, financials of which have not been disclosed, sees Belgian bioprocess firm Univercells add technology aimed at simplifying biotherapeutic development through a scalable and automated DNA synthesis platform. SynHelix’s technology is based on the GMP production of long DNA fragments in large quantities and has been touted as an alternative to DNA amplification on bacteria in the synthetic biology, gene therapy, and diagnostics…