This week’s biotechnology news headlines include: 1 Patient, 7 Tumors and 100 Billion Cells Equal 1 Striking Recovery, Gladstone’s BioFulcrum Produces Tenaya, Leveraging $50M to Cure Heart Disease, With Nod To Biden, Senate To Say Yes To “Cures” Spending, Reform Bill, Momenta’s Humira biosimilar succeeds in key psoriasis study, Parkinson’s Gene Therapy data sparks Voyager stock jump, Flickering lights may illuminate a path to Alzheimer’s treatment, and Yellow Fever Epidemic in Africa Shows Gaps in Vaccine Pipeline.
In Case You Missed It, Recent Articles on Cell Culture Dish and Downstream Column:
Neural progenitor cells (NPCs) derived from primary tissue and pluripotent stem cells have proven to be a powerful tool for gaining insight into the human nervous system as well as associated disease states. Impaired neurons and other differentiated cells that endogenously arise from NPCs are associated with many of the most challenging neurological diseases, such as Parkinson’s disease. These differentiated neural cells serve as useful tools for modeling such diseases and provide a platform for the screening of potential drug therapies. In addition, researchers are just beginning to look into their direct use toward Cell Therapy applications where it may be possible to replace damaged or diseased neurons with healthy ones.
A vital part of the biomanufacturing process is being able to move fluid under sterile conditions using sterile connections. However, this activity requires considerable time and resources. One challenge is that in most cases, current disposable sterile connectors only allow users to make a single sterile connection per device, which then requires the use of multiple devices per unit operation. In addition, the flow path must be dry and non-pressurized during connection and disconnection. Due to these limitations, sterile liquid transfer can be very inefficient.
We recently finished our Ask the Expert discussion, “Media Optimization Can Improve Glycosylation Patterns and Consistency to Impact Protein Efficacy”. During this Ask the Expert session, we discussed factors that influence glycosylation, the relationship between media and glycosylation, and the effect of glycosylation on the protein. Additional topics included glycosylation in biosimilars, glycoengineering, other post-translational modifications and protein aggregation.
Finding the right combination of cell culture media ingredients can be tricky and it isn’t always simple to find an off the shelf medium that meets all your requirements. Sometimes it takes several clicks around to locate one that will meet your needs. To address this challenge we’ve created an online cell culture media formulation tool.
As part of our Boston Biotech Week 2016 coverage, we will be writing about some of the posters presented at the conference. One poster that caught my eye for downstream was presented by Oncobiologics and JSR Life Sciences, “Optimization of a Protein A Chromatography Process for a Herceptin® Biosimilar (Trastuzumab).” In the poster, Oncobiologics and JSR Life Sciences describe the steps taken in identifying the most efficient chromatography process.
Only 1 out of each 50 biopharmaceutical new product candidates makes it through the research phase into clinical trial testing and subsequently to the market. This high attrition rate is predominantly in the early development phases and is attributed to (I) undesired pharmacokinetics profile (39%), (II) lack of efficacy (30%), (III) in vivo toxicity in preclinical model (11%), (IV) adverse effect in humans (10%), and (V) other reasons, of which most commonly commercial arguments based on cost of goods (10%). It is therefore imperative that technologies become available that allow significant de-risking of biopharmaceutical product trajectories in the early research and development phase. The importance of this has been recognized by the field with the introduction of the “Design of Experiments” (DoE) approach, identifying critical quality attributes and performance attributes like yield, glycosylation, potency, and consumable costs of a manufacturing process. Owing to the DoE approach, scientists now have a tool to strategize the development of a novel product candidate. That said, it is often found that due to the complexity of many novel molecules, the number of parameters that need to be tested still requires vast numbers of experiments which are time consuming and costly.
At this year’s Boston Biotech Week the 2016 BioProcess International Award Winners were announced. These awards recognize outstanding achievements in the area of biotherapeutic development and manufacturing processes. This year individuals and companies that made significant contributions to improving biotherapeutics were recognized. Novel technologies in upstream, downstream and analytical application areas were also awarded. I have listed the winners and finalists along with a brief description of the winning achievements for downstream technologies here. For a list of upstream and analytical technology winners, please see 2016 BioProcess International Award Winners – Upstream and Analytical.
Because Protein A is a valuable resource in any mAb purification strategy, companies often search for ways to improve the productivity of their affinity chromatography step. One strategy worth further investigation is variable column loading. By varying residence time (RT) over the loading phase, productivity from an affinity chromatography step can be significantly improved.
“The remarkable recovery of a woman with advanced colon cancer, after treatment with cells from her own immune system, may lead to new options for thousands of other patients with colon or pancreatic cancer, researchers are reporting.”
“Gladstone’s BioFulcrum Produces Tenaya, Leveraging $50M to Cure Heart Disease,” Genetic Engineering News
“Building on research at the Gladstone Institutes, Tenaya Therapeutics, a new biopharmaceutical company, will concentrate on regenerative medicine and drug discovery for cardiovascular diseases. The new company is supported by $50 million in Series A financing from The Column Group. JJ Kang, Ph.D., an associate at The Column Group, is the president of the new company.”
“The U.S. Senate cleared the way for the 21st Century Cures Act, an omnibus healthcare spending and reform bill, by voting 85-13 to end debate this evening. It passed the House with 392 votes last week. After a final Senate vote as early as tomorrow, it will head to President Obama’s desk.”
“U.S. biotech Momenta Pharmaceuticals Inc said on Tuesday its experimental biosimilar version of AbbVie Inc’s top-selling autoimmune drug, Humira, met the main goal in a late-stage trial involving patients with a form of psoriasis.”
“Shares in Voyager Therapeutics jumped 35% in after-hours trading on the back of interim data from an early trial of its Parkinson’s disease Gene Therapy. The surgically delivered Gene Therapy is aiming to make patients with advanced forms of the disease respond better to levodopa, an old drug that is fairly effective at controlling symptoms in the early stages of the condition.”
“New research demonstrates that, in mice whose brains are under attack by Alzheimer’s dementia, exposure to lights that flicker at a precise frequency can right the brain’s faulty signaling and energize its immune cells to fight off the disease.”
“The yellow fever outbreak in Africa this year came closer to being a disaster than is widely recognized, public health experts recently disclosed. The epidemic also revealed glaring weaknesses in the emergency vaccine supply pipeline.”