We are back with biotech weekly, exploring the biotech and MedTech highlights from the past week. We look at Novavax delaying the regulatory submissions of its Covid-19 vaccine, Orchard Therapeutics’ gene therapy efficacy in kids with no immune system, and CRISPR-Cas9 expanded to a new type of immune cells. We will also look at how MedTech companies are expected to transition to decentralized and patient-centered care. Apart from these, we will talk about AI in biotech and MedTech, both in terms of new discoveries and the regulatory framework, as the EU’s new proposed AI regulations have led to widespread criticism among MedTech scientists. Meanwhile, Mayo Clinic’s AI algorithm is 32% more effective in detecting early signs of heart disease than a physician, and Gladstone Institutes’ scientists have developed an AI approach to track cells.
Novavax continues to delay filing for regulatory approval of its Covid-19 vaccine, with the newest reports stating that the company will not seek approval until July, at the earliest. The delays are attributed to manufacturing issues. The initial plan for seeking regulatory approval in Q2 of 2021.
“We know that we are delayed from where we thought we would be at this point. Now we’re giving guidance that nearly all of the major challenges have been overcome,” said Novavax CEO Stanley Erc.
Erc ensured that the company is confident they will be able to hit full capacity manufacturing which stands at 150 million doses per month until the end of the year, slightly later than the previously expected Q3 of 2021.
“Although demand in the US has clearly been met by the mRNA vaccines, demand outside the U.S. remains very high and the company remains in communications with governments that want vaccine now and for 2022,” wrote Jefferies analyst Kelechi Chikere in a note to investors on Monday.
In an early-stage study, the gene therapy by Orchard Therapeutics has shown to be 100% effective among patients with an inherited disease that damages the immune system.
The data from a study that was conducted in the last three years, was just published in the New England Journal of Medicine. 50 ADA-SCID patients took part in the study and at two and three years, 100% of the patients were alive and 95% survived without having to go through another treatment. While all of the patients experienced certain adverse effects, Orchard claims they are majorly mild or moderate. Out of the sample, four have experienced serious complications. Having said that, that complication can occur even when the body recovers from antiretroviral drugs.
“With sustained engraftment of up to three years, these data show the potential of HSC gene therapy to correct the underlying genetic cause of ADA-SCID, delivering positive outcomes in a single treatment,” said Bobby Gaspar, M.D., Ph.D., CEO of Orchard Therapeutics.
Orchard was initially planning to submit for an FDA clearance early this year, however, Covid-19 has delayed these plans.
Mayo Clinic’s AI algorithm proved effective in discovering the early stages of heart disease during a standard 12-lead electrocardiogram (EKG) reading.
In a study exploring the effectiveness of the algorithm, 22 600 patients were given an EKG as a part of the primary care checkups and were randomly assigned to have their results examined either by their physician as usual or the AI algorithm. The results showed that the use of the algorithm led to 32% more diagnoses.
“The AI-enabled EKG facilitated the diagnosis of patients with low ejection fraction in a real-world setting by identifying people who previously would have slipped through the cracks,” said Peter Noseworthy, a Mayo Clinic cardiac electrophysiologist and senior author on the study.
“The information was readily available in the electronic health record, and care teams could see the results and decide how to use that information,” Noseworthy continued. “The takeaway is that we are likely to see more AI use in the practice of medicine as time goes on. It’s up to us to figure how to use this in a way that improves care and health outcomes but does not overburden frontline clinicians.”
Today, Frost & Sullivan issued a report showing that medtech firms are under a large transformation, as hospitals are moving from high-dependency care to decentralized patient-centric care.
It is expected that by 2024, Medtech solutions based on managing diseases through remote patient monitoring and connected care will generate a revenue of $171.65 billion. The expected compound annual growth rate (CAGR) stands at 14.3%
“MedTech companies’ business models no longer aim to sell products with superior features addressing clinicians’ needs. These models focus on saving the cost of care for payers, improving outcomes for patients, and enabling operational or workflow efficiencies for providers,” said Srinath Venkatasubramanian, Healthcare & Life Sciences Industry Analyst at Frost & Sullivan. “Additionally, the evolution of regulatory framework and processes for breakthrough devices and digital capabilities such as artificial intelligence-based solutions have boosted the innovation capability and improved market access for medical device companies.”
Venkatasubramanian added: “The influx of disruptive startups and pure technology firms into the care provision landscape has increased the competitive intensity in the sector. As a result, large MedTech companies are exploring options to build adjacent assets in the digital space through mergers and acquisitions (M&As) to sustain revenue growth. The rising competition has led to the emergence of platform solutions beyond hardware with value-added services to differentiate from low-cost peers.”
As the EU is toughening up its artificial intelligence regulations, one of the sectors impacted will be Medtech. As MedTech and in vitro diagnostics devices are on the high-risk AI systems list, they can be placed on the EU market only “if they comply with certain mandatory requirements”.
The companies that will not comply with the proposed EU requirements will be faced with hefty fines that can go up to $36 million, or if it is a company, 6% of its total global annual turnover. To put it in perspective, a maximum fine for a company like Johnson & Johnson would reach almost $5 billion.
Many researchers have spoken against the proposed regulations, blaming EU for being too vague and creating regulations that are too complex and will ultimately lead to more harm than good.
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The team led by the Gladstone Institutes scientist, Todd McDevitt, has developed an AI tool that allows for an extensive view of how cells behave and collaborate to form complex organs. The program can detect patterns and analyze the locations of hundreds of cells growing together.
“This technique gives us a much more comprehensive view of how cells behave, how they work cooperatively, and how they come together in physical space to form complex organs,” says Gladstone Senior Investigator Todd C. McDevitt, PhD, senior author of a new paper published in the journal Stem Cell Reports.
Clusters of stem cells have the ability to form any tissue in the human body when exposed to the right mixture of signaling molecules. But researchers have a poor understanding of how those cells form patterns in space to eventually give rise to complex three-dimensional organs.
“Going in, we didn’t really expect there to be that much cell motion, so we had to come up with new approaches to understand the apparent chaos of the cells,” says Gladstone Graduate Student David Joy, first author of the new paper.
However, the team has discovered that the cell nearest the edges of each group moved the most and cells tended to start and stop to a higher degree than it was anticipated. Then the researchers went on to illustrate how exposing cells to different drugs can change the patterns of their movement. To add, while certain cells behaved like “leaders”, others acted more like “followers”
“Some cells move with a lot of persistence in one direction, while others move around and around but never get far from where they started,” says Ashley Libby, PhD, a former graduate student in McDevitt’s lab who helped lead the work. The diversity surprised the team; they had expected most cells to follow similar patterns of movement, she says.
McDevitt points out that the findings reported so far are just a fraction of the range of observations that are possible because of the AI approach applied to cell tracking.
“If I wanted to make a new human heart right now, I know what types of cells are needed, and I know how to grow them independently in dishes,” says McDevitt, who is also a professor of bioengineering and therapeutic sciences at UC San Francisco. “But we really don’t know how to get those cells to come together to form something as complex as a heart. To accomplish that, we need more insights into how cells work cooperatively to arrange themselves.”
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The researchers at Gladstone Institutes and UC San Francisco (UCSF) have added human monocytes to the list of CRISPR-Cas9 gene editing. Monocytes are immune cells that have a variety of roles in defending the human body from pathogens. While the team points out that editing monocytes was challenging, it is something they felt was crucial for the development of CRISPR-Cas9
“These experiments set the stage for many more studies on the interactions between major infectious diseases and human immune cells,” says senior author Alex Marson, MD, Ph.D., director of the Gladstone-UCSF Institute of Genomic Immunology and associate professor of medicine at UCSF.
“This technology opens doors for identifying the human genes most important to the function of monocytes and for coming up with new therapeutic strategies against a range of pathogens,” adds co-senior author Nevan Krogan, Ph.D., senior investigator at Gladstone and director of the Quantitative Biosciences Institute at UCSF.
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