This week’s headlines include: Novartis strikes deal with Chinese firm to make Kymriah, Gene tweak kills whole population of malaria-carrying mosquitoes in lab, Congratulations. Your Study Went Nowhere, Gilead to Make Generic Hepatitis C Drugs and Cut Prices Up to 75%, With new blood test, Roche dives deeper into personal cancer care, and T-cell therapy for eradicating HIV reservoirs proves safe in small study.

Podcasts:

Check out our podcast channel. We have over 20 great podcasts covering drug discovery, stem cell culture, upstream and downstream biomanufacturing and more! Click below to download from iTunes or Google play:

Upcoming Conferences

Speed to IND for Biologics

October 17-18, 2018, Argonaut Hotel, San Francisco CA

CBI’s Speed to IND for Biologics provides a comprehensive, cross-functional format that brings together experts from every department to share their perspectives, achievements and lessons learned as a community working towards the common goal of accelerating speed while managing and mitigating risk.

CELL SERIES

The annual Cell Series features 4 outstanding programmes: our long-running 7^th Cell Culture and Bioprocessing, 5^thStem Cell & Regenerative Medicine, 4th Cell and Gene Therapy and Biobanking Congresses. The Cell Series provides a focused networking platform targeting Cell Culture & Cell Line Development and Bioprocessing; Cell & Gene Therapy highlighting CAR T Cell Development; Stem Cell & Regeneration Medicine Development as well as Biobanking.

In Case You Missed It, Recent Articles on Cell Culture Dish and Downstream Column:

Viral Vector Characterization: A Look at Analytical Tools

As cell and gene therapy field continues to push products through the clinical landscape, the need to develop appropriate analytical tools has become increasingly evident. Because of their intended use in patients, gene therapy products must meet rigorous safety guidelines highlighting the importance of well-characterized analytics…

Isolation of Mononuclear and Polymorphonuclear Leukocytes from Blood with Density Separation Media

Blood is a specialized body fluid composed of four main components: plasma, white blood cells (leukocytes), red blood cells (erythrocytes), and platelets (thrombocytes). Unlike red blood cells and platelets, all white blood cells are nucleated and can be classified by their nuclei structure as mononuclear or polymorphonuclear cells…

Biotech Week Boston 2018: Cell and Gene Therapy Bioprocessing and Commercialization Highlights

Biotech Week Boston, held at the Hynes Convention Center in Boston, MA, was a fantastic amalgamation of scientific conferences, training courses, and keynotes all focused on cell bioprocessing. The theme of this year’s Cell and Gene Therapy Bioprocessing and Commercialization conference was “transforming innovative research to drive the future of cell & gene therapy by relieving bottlenecks and pain points in clinical development, manufacture and commercialization of cell, gene and immunotherapies”…

Overview of the Regulatory Path to Commercializing a Gene Therapy

After decades of research, gene therapies now comprise much of the R&D and manufacturing pipelines in the United States and around the world. Huge strides in biological understanding and the development of key enabling technologies are at long last delivering on the promises of precision and personalized medicines. While there is still much more to learn and develop, regulatory constructs keeping pace with the speed of innovation remains a sizeable challenge…

A Defined Synthetic Growth Surface for Stable hiPSC Expansion

hiPSCs cultivation can be challenging with lot-to-lot coating variations, tedious preparation and increased contamination risk. In particular, biological coatings may negatively impact downstream applications due to the presence of undefined extracellular matrix (ECM) components and growth factors. Another common struggle is decreased reproducibility of data due to an inherently complex and non-defined growth surface. To address these common challenges, Eppendorf has introduced a ready-to-use consumable with synthetic fibronectin-derived motifs to support stem cell attachment by mimicking the native ECM protein fibronectin. The FN1 motifs surface allows expansion of stem cells in various xeno-free media and other restrictive culture conditions. Thus, you can establish a completely defined culture system for your stem cell culture without any animal or human components…

A Guide for Selecting the Correct Filter Integrity Test Instrument

Selecting the right filter integrity test instrument is an important task. An informed, balanced choice will support the seamless integration of the instrument into critical processes and provide trouble-free operation for years to come. The right choice may be made more difficult by different departments, or user groups, who may assign a different value to the same feature. A rational approach is beneficial to ensure the selected instrument can fulfill all the selection criteria for all interested parties, with any compromises being limited to non-critical characteristics…

Downstream Manufacturing of Gene Therapy Vectors

The goal of downstream processing is to separate the viral vector from the various impurities produced during upstream processing and to get the virus into the appropriate state for formulation and administration to patients. Viral vector batches destined for clinical use must comply to increasing regulatory standards for impurities and contaminants as these can affect product safety and potency. In addition to providing purity, purification processes need to meet production scale, which can be quite large for clinical trials and commercial applications…

How Robots Are Changing The Landscape Of The Medical Field

Robotics are becoming increasingly present in many different industries. In the medical world, robots and AI are helping medical professionals conduct procedures, understand the risk of surgical complications, and provide more precise patient care….

Continuous biomanufacturing – Key drivers for adoption, economic modelling and regulatory considerations

The Medicine Maker recently published a multi-authored eBook on Continuous Biomanufacturing, titled, “The Continuous Way”. The publication includes seven articles covering the topic of continuous biomanufacturing from a variety of angles. I really enjoyed the eBook and it provided a great update on the current state of continuous biomanufacturing as well as information about key drivers for adoption, new economic modeling, and integration of continuous operations. Following is a sampling of articles with key highlights…

Cool Tool – Inline Diafiltration Closes the Gap for End-to-End Continuous Bioprocessing

Diafiltration, while fulfilling several functions in protein purification, has also presented a major obstacle in achieving continuous biomanufacturing. Technologies launched over the last decade have moved the industry closer to completing an integrated end-to-end continuous bioprocessing platform; however adequately addressing continuous final ultrafiltration/diafiltration of drug substance has been challenging. Now, inline diafiltration offers a solution to both traditional bottleneck hurdles and continuous biomanufacturing platform gaps…

Headlines:

“CAR T-Cell Therapy for Pediatric Leukemia Is Cost-effective,” Medscape

“Chimeric antigen receptor (CAR) T-cell therapy has been hailed as a major advance and a veritable game changer for pediatric leukemia, but it comes with an exceedingly hefty price tag. Tisagenlecleucel (Kymriah, Novartis) is priced at $475,000 for one treatment; but when it works, it offers a cure for pediatric and young adult patients (aged 3 – 25 years) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) who have run out of options…”

“FDA approves use of HPV vaccine for adults 27 to 45,” CNN

“The US Food and Drug Administration on Friday approved the use of a human papillomavirus vaccine called Gardasil 9 for men and women between the ages of 27 and 45. The FDA previously approved the HPV vaccine for people age 9 to 26…”

“U.S. FDA panel backs Celltrion copycat drug of Roche blood cancer drug,” Reuters

“An advisory panel to the U.S. Food and Drug Administration on Wednesday voted unanimously in favor of Celltrion Inc’s copycat drug of Roche Holding AG’s blood cancer drug Rituxan…”

“The CRISPR-Cas9 system is often compared to scissors, because the technology cuts out segments of DNA. Researchers at Stanford University have designed a new CRISPR gene-editing tool that that they say is more like molecular “tweezers,” and they believe the invention might someday be used in treating diseases like cancer…”

“Growing number of U.S. children not vaccinated against any disease,” CBS News

“A small but growing proportion of the youngest children in the U.S. have not been vaccinated against any disease, worrying health officials. An estimated 100,000 young children have not had a vaccination against any of the 14 diseases for which shots are recommended, according to a Centers for Disease Control and Prevention report released Thursday…”

“Gilead strikes deal with NHS England on Yescarta access,” PharmaTimes

“Gilead has struck a deal with NHS England that allows some adults with lymphoma access to its “game-changing” CAR-T therapy Yescarta. The agreement marks the first time adults will have access to CAR-T on the NHS, after NHS England reached a deal last month to make CAR-T available for children and young people with a rare form of leukaemia…”