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Brain Organoids Might Lead to Neurological Disease Therapies

In vitro models were created using stem cells from a patient with a rare gene mutation that affects myelination. The model showed that key aspects of this cell bundle were consistent with the disease. The model is a step forward in the study of human myelination and drug development, but treatments tested on this model are still some way from being offered to patients.

Raising Awareness of 4.0 in Biomanufacturing—Part II

Adopting 4.0 in one suite at one site may cost tens of thousands of dollars, but it’s important to keep in mind that plant-wide deployment or implementation at ten facilities will cost millions.

Avrobio Keeps Its Platform Closed and Compact

The company’s off-the-shelf instruments were turned into a system that accommodates methods controlled by the company’s proprietary algorithm to make lentiviral-based gene therapy.

Solving Contract Manufacturing Shortages by Going In-House

A cell therapy company has built a 45,000-square-foot manufacturing facility as a response to perceived shortages of contract development and manufacturing (CDMO) capacity.

Biopharma’s Resilience to Future Pandemics Relies on Manufacturing Innovation

Automation is viewed as key to improving the quality of drug products, increasing the speed to market as well as lowering manufacturing cost.

The State of Gene Therapy

In the May edition of GEN Live, sponsored by Cytiva, we discuss the transformation of gene therapy from an academic endeavor to a billion-dollar biotech industry. We’ll discuss the latest research advances and hurdles, highlights from ASGCT, trends in delivery, and hear the panel’s vision for the future.

Fashion, Space, and Medicine Stitched by Algae

Living materials, which are fabricated by encapsulating living biological cells within a nonliving matrix, have gained increasing attention in recent years. Now, for the first time, researchers at the University of Rochester and Delft University of Technology report they used 3D printers and a novel bioprinting technique to print algae into living, photosynthetic materials that are tough and resilient.

Scientists Develop Cryomicroneedle Tech for Intradermal Delivery of Therapeutic Cells

A research team led by City University of Hong Kong (CityU) scientists developed a new cryomicroneedle technology for the intradermal delivery of living cells in a minimally invasive manner. Their studies demonstrated that using the technology to delivery therapeutic cells into melanoma-bearing mice elicited robust anti-tumor immune responses, and resulted in improved therapeutic outcomes compared with traditional approaches to cell delivery.

Targeting Bowel Cancer with Nanotechnology

Approximately two million people are diagnosed with bowel cancer each year and half of those are not expected to survive, according to the WHO. Now, animal experiments by scientists from the Manipal Academy of Higher Education, the Indian Institute of Science, and the University of South Australia show that nanoparticles containing the Capecitabine attach themselves directly to diseased cells, bypassing healthy cells and therefore reducing toxic side effects as well as the size and number of tumors.

Neutralizing Antibodies Making Their Mark in Next Wave of Biologics

In this eBook, sponsored by Sartorius, we highlight the research efforts and related applications utilized to study binding affinity, potency and target specificities of neutralizing antibodies against SARS-CoV-2 and their potential for future use against diseases caused by viruses from the same coronavirus subgroup.

Human Organ Chips Shift Amodiaquine from Old Antimalarial to Promising COVID-19 Treatment

New work has identified the antimalarial drug amodiaquine as a potent inhibitor of SARS-CoV-2 infection in human lung cells and in living preclinical models. The breakthrough was made possible by a human Organ Chip-based drug testing ecosystem that was established through a multi-institution collaboration. The research helped secure the inclusion of amodiaquine in a COVID-19 clinical trial that is currently underway in Africa where the drug is inexpensive and widely available.

From Pandemic to Pipeline: What’s Next for Moderna?

Two weeks ago, Morningstar initiated coverage of Moderna and assigned the company its “very high uncertainty” and “no-moat” ratings, concluding in a report that Moderna is still building its “moat” or sustainable, competitive advantage.

CLOSE TO THE EDGE: Exclusive Live Interviews with Biotech Executives from GEN EDGE

In this Close to the Edge new live series, we’ll be interviewing chief executives and scientific and business leaders from the biotech spectrum. In this inaugural episode on May 12 GEN Edge welcomes Christian Henry, the CEO of PacBio, for an exclusive interview. We’ll have lots to discuss, including PacBio’s technological edge in long-read sequencing, the ramifications of the big merger that didn’t happen, and the company’s growth plans in both the biotech and clinical space.

Scholar Rock: A New Twist on Targeting Growth Factors

Within the wide range of targets it aspires to address, Scholar Rock is currently pinning its hopes on these two growth factor-targeting therapies and betting on their novel approach—targeting latent forms to improve selectivity.

Potential Strategy for Acute Myeloid Leukemia Therapy Identified

Studies by scientists at the Wellcome Sanger Institute provide new insights into how a loss-of-function mutation in the CUX1 gene leads to the development and survival of acute myeloid leukemia, and suggest that identifying a pathway that is essential for these cancer cells to continue growing could lead to new, targeted therapies for some patients.

Researchers Evaluate the First “Tumor-Uninformed” Test That Detects Cancer DNA Post-Surgery

Postsurgical circulating tumor DNA (ctDNA) analysis is a promising tool for the identification of patients at highest risk of cancer recurrence. Most tests require knowing the mutations that were present in a patient's tumor. Now researchers at Massachusetts General Hospital report they have evaluated the first test that detects cancer DNA circulating in the blood of patients following treatment without knowing the particular mutations that were present in the patient's tumor.

Viral Evolution Insights Will Help Us Fight COVID-19

Even as governments and corporations push to declare an end to the COVID-19 pandemic, questions about the virus that causes COVID-19 remain unanswered. Will...

The Microbiome Gains Momentum in Cancer Immunotherapy

Although a decade has passed since the first immunotherapy for cancer was approved, the reason for such mixed responses is still not well understood. Now that a variety of efforts are underway to account for the disappointing response rates, some scientists and several new companies are hoping that harnessing the microbiome will push the rates upward. Once an object of skepticism, microbiome-augmented cancer immunotherapy is being advanced by companies such as Synlogic, Vedanta Biosciences, and Persephone Biosciences.

Better Outfitted and Screened Viral Vectors Are Safer and More Effective

Adeno-associated virus (AAV) vectors are the leading vectors for gene delivery. AAV vectors pose challenges including costly manufacture, high percentages of empty capsids, and safety issues associated with high-dose regimens. To address this last challenge, developers are working to improve the impact of each AAV particle so that fewer particles will be required. Rather than unleash hordes of low-impact vectors, we should deploy cadres of high-impact vectors, reducing collateral damage while overcoming the toughest therapeutic challenges.

CRISPR’s Rapid Rise Shakes Up Genome-Wide Screening

CRISPR has been grabbing headlines because of its potential as a form of gene therapy. But CRISPR also deserves attention because of its contributions to genome-wide screening. Although these contributions usually escape public notice, they are nothing less than revolutionary. A truly disruptive technology, CRISPR screening roughly displaces its predecessors then refines itself, as shown by its functional genomics applications and its ability to complement single-cell transcriptomics
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