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Vial and syringe with a vaccine

Clover Biopharmaceuticals, a clinical-stage firm developing a Covid-19 vaccine with potential manufacturing and distribution advantages over other vaccine technologies, has raised $230 million as it prepares to advance its lead candidate into pivotal testing.

Chengdu, China-based Clover said Tuesday that it expects to start a global Phase 2/3 clinical trial for its vaccine candidate, SCB-2019, in the first half of this year. The company added that it has already started planning for the production of potentially hundreds of millions of vaccine doses.

The Clover Covid-19 vaccine is protein-based. SARS-CoV-2, the virus that causes Covid-19 infection, is an enveloped RNA virus—the outer coating is dotted with spike proteins that bind to a receptor on the host cell. These spikes are trimeric, meaning they’re formed by three proteins.

Using its Trimer-Tag technology, Clover developed a trimeric spike protein that resembles the one found on the outer envelope of the novel coronavirus. The vaccine uses an adjuvant, an ingredient that boosts immune response, supplied by Dynavax Technologies.

As a protein-based vaccine similar to many of the vaccines developed for influenza, shingles, and hepatitis B, Clover said production can use manufacturing processes that are already well established. The company added that this production can be rapidly scaled up to large quantities.

Another advantage of the Clover vaccines are the temperature requirements. The company said its vaccines and adjuvant should be stable for long periods at refrigerator temperatures of 2 to 8 degrees Celsius. At room temperature, Clover has said its vaccines are stable for at least two months. Those temperature and storage requirements stand in contrast to messenger RNA vaccines, which must be distributed frozen and stored at temperatures well below what medical-grade freezers can achieve. Last week, Pfizer and BioNTech asked the FDA to approve a change in the storage temperatures permitted once vaccines reach a vaccination site.

The new financing follows publication in The Lancet earlier this month of peer-reviewed results from an early-stage test of two Clover Covid-19 vaccine candidates. The 150-patient study showed that the vaccines were well tolerated and safe. Both vaccines also induced neutralizing antibodies at levels comparable to or higher than those found in the blood of those who have recovered from Covid-19.

Clover said that its research includes vaccines that could address multiple variants of the novel coronavirus. In addition to supporting its Covid-19 vaccines, Clover said the new capital will support plans to advance multiple programs into human testing later this year. Other vaccines in the Clover pipeline include programs for rabies and influenza. The company also said it plans to expand its manufacturing and capabilities.

GL Ventures and Temasek both led the Series C financing. Oceeanpine Capital, OrbiMed, and Delos Capital also invested. Clover said it has raised more than $400 million in the past year.

Clover also has financial support from the Coalition for Epidemic Preparedness Innovations (CEPI), which has committed to finance development of the company’s Covid-19 vaccine candidate up through licensure with a total investment of $328 million. Some of that cash will fund the global Phase 2/3 study. If the Clover vaccine is shown to be safe and effective, it would be distributed through Covid-19 Vaccines Global Access, or COVAX, the World Health Organization’s initiative to ensure equitable vaccine access throughout the world.

Public domain photo by Flickr user Alachua County

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As 2021 dawned and vaccine distribution has picked up, many people had one overarching question on their minds: how and when will they get vaccinated.

At the same time, providers have had to grapple with another question:  how to best approach for this historic, monumental task.

For Stamford Health in Connecticut, the way forward was clear: Leverage existing partnerships with the city of Stamford, community organizations and the state to distribute the much-anticipated vaccines.

The health system has been sharing data and information with the city of Stamford throughout the pandemic, including positivity rates, hospitalizations and other key metrics, said Kathleen Silard, CEO of Stamford Health, in a phone interview. Stamford Health and the city worked collaboratively to set up testing sites, and in the last few months, they also worked together to set up vaccination sites — including one at an old hospital on Stamford Health’s campus.

“The collaboration [with the city] really is to pool our resources, because we know, together we are better,” Silard said.

The health system began vaccinating healthcare workers, first responders and other eligible essential workers on Dec. 17, when the state was in Phase 1a of its vaccine rollout. Back on Jan. 18, it began vaccinating people older than 75 and recently added those older than 65 to the list, as part of Phase 1b of the rollout.

So far, the health system has administered around 27,000 vaccines, and is averaging between 750 and 930 doses a day, Silard said. But Stamford Health has ambitious plans to increase this number three-fold.

The health system is planning to open a new, much larger, site around March 1, which will enable the provider to administer up to 3,000 doses a day, she said.

But getting shots in people’s arms is not without its challenges.

Vaccine availability has been one of the biggest hurdles the health system has faced, but working closely with Connecticut Gov. Ned Lamont and his team has helped the system get the doses and resources it needs, Silard said.

Aside from uncertainty with vaccine availability that has since receded into the background, Stamford Health is tackling a more intractable problem: vaccine hesitancy and health inequity. Both present a formidable barrier to its 3,000-a-day vaccination goal.

To help combat vaccine hesitancy, Stamford Health is conducting outreach programs, including setting up panels with people who have already received the vaccine to talk about their experience, Silard said. The system is also participating in Stamford Mayor David Martin’s weekly Zoom calls to further educate the public on the vaccine.

The Covid-19 pandemic shone a harsh light on existing health disparities in the country, with people in minority racial groups and low-income populations most likely to get the disease and die from it.

Stamford Health has put together a task force, which includes health system members, city officials and members of community health organization Vita, to ensure that vaccines are being administered in an equitable manner, said Silard.

In addition, the health system is partnering closely with the National Association for the Advancement of Colored People and Building One Community, an organization that works with the undocumented community, to focus vaccine administration efforts on underserved populations.

Building One Community has developed a great deal of trust with a population that is typically hesitant to use healthcare services, said Dr. Anka Badurina, executive director of the organization, in a phone interview.

Through the pandemic, the organization has been working to ensure immigrant and underserved communities are included in response efforts — from testing to, now, vaccination.

One of its main areas of focus has been helping the elderly in these communities get registered for vaccine appointments, Badurina said. Those currently eligible often don’t have access to the internet or an email address, which is typically required for registration. Building One Community, which has interpreters on hand, helps them with the process.

Further, the organization helps organize transport to vaccination sites.

“Stamford Health partners with organizations like Building One Community [because] you have to go to those that have a trusted voice in the community,” Badurina said. “They are the ones that know where the community is and know exactly what the community is lacking.”

With the help of its community partners Stamford Health has established a “No Barriers” day, where members from minority groups and under-resourced communities can come to a vaccination site without an appointment, get registered and get vaccinated, Silard said. No individuals are asked about their immigration status or other questions that might keep people from coming to get vaccinated.

Stamford Health wants to eliminate any traditional barriers to vaccination to ensure that the largest swath of eligible individuals can get vaccinated, Silard said.

“We see [vaccine administration] as our moral, ethical responsibility to help fight this deadly disease,” she said.

Photo: LarisaBozhikova, Getty Images

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The Covid-19 pandemic has affected every sector of our society and requires coordinating a broad coalition of assets to contain it. The response includes multiple federal and state government agencies, thousands of hospitals, and a broad swath of commercial manufacturing capabilities and supply chains. We saw this coordination and collaboration early on with personal protective equipment and ventilators, and we see it again as we ramp up vaccine distribution.

To coordinate an effective response, it is critical to integrate disparate data types from multiple domains and sources, something that has been a long-standing challenge in health care. Obstacles include government agency budget structures that don’t incentivize data sharing and legacy databases that create barriers to data integration. The commercial sector also brings the challenges of competition and proprietary systems. Even seemingly simple questions, such as how many ICU beds are available in a community, are maddeningly difficult to answer in near real time. While well-branded and user-friendly websites provide impressive updates on case counts, emergency operation centers have found it challenging to integrate that data with bed availability, hospitalization projections, work force data, supply chain data, mitigation interventions, social determinants of health, and other key data elements that allow for effective planning and response.

In addition to the public health challenges, new care delivery models have underscored the need to better integrate data to deliver care for chronic diseases. The pandemic has accelerated the adoption and use of telehealth. But again, tools, sensors, apps, and devices are often deployed on disparate data platforms that make it cumbersome for patients and providers to integrate data in a meaningful fashion.

The pandemic illustrates the need for better data integration to improve management of this crises as it continues to impede the everyday care of patients.

Lessons learned from defense and intelligence communities
The health industry lags behind other commercial sectors in its adoption of data management and open-source innovations. The health community can learn a great deal about data management from defense and intelligence agencies, which must integrate vast amounts of data from disparate systems to create a common operating picture to support life and death decisions for warfighters.

The 9/11 attacks demonstrated that data gaps can be deadly. The 9/11 Commission Report revealed that information that could have prevented this tragedy was scattered across several different intelligence agencies’ databases. Following the Commission’s critique, the intelligence agencies adopted low schema data “lakes” that could accommodate multiple “streams and rivers” of disparate data and allow for easier integration. Think of these data systems as giant spreadsheets, with each cell containing an entry item. With automated meta-tagging, each cell of information can be correlated with any other item of data to reveal patterns that would otherwise have gone undetected. These data platforms also enabled the accumulation of massive data stores that optimize advanced analytics and artificial intelligence. Intelligence agencies also benefited from security protections at the individual cell level that enhanced data security, an important feature to consider as health information increasingly comes under cyberattack.

The intelligence community also embraced open source tools and open architectures for these data systems. Open source allows the rapid development of new tools at lower cost. Open architectures avoid costly and stagnating vendor lock, and it enables the adoption of new best-in-class tools and capabilities as they are often developed by small niche firms and start-ups

While novel 15 years ago, many of these innovations have been avidly adopted in the commercial sector. However, the same cannot be said for the health domain. That said, there are notable exceptions that are bright spots on the health care landscape.

Advana: Uniting disparate systems and users on a common platform
Advana, a Department of Defense (DoD) data platform, pulls together more than 200 business systems across the DoD and makes data discoverable, understandable, accessible, and usable for advanced analytics for more than 17,000 users across the Army, Navy, and Air Force who need to make decisions about mission readiness, contracts, supply chain logistics and more. The platform has helped the DoD coordinate its Covid-19 response by enabling the easy integration of a wide range of data, including case, bed, supply chain, readiness, and financial data, to inform critical health care decisions. The open architecture platform supports multiple projection models and analytic tools, which allows the DoD to validate findings in a way that would not have been feasible with a single approach.

Advana faced many of the data integration obstacles familiar to health care IT leaders: non-standard interfaces, duplicate data and systems, legacy technologies, and a history of different units pulling their own data for decision making. To integrate disparate data from spreadsheets, application programming interface (APIs), database dumps, and data warehouses from across the enterprise, Advana streams data feeds, automatically categorizing, tagging, and transforming them into a common data model to improve enterprise level analytics.

Preparing for the next health crisis
The value of big data in health care is clear but unless we can integrate and correlate disparate types of data, we can’t realize the benefits. The data challenges of the Covid-19 response illustrate this issue. The seams between government agencies, health systems, and departments within the same organization create chronic barriers to data sharing. Few organizations manage more data than the defense and intelligence agencies, and as with health care, their decisions often have life and death consequences. For critical decisions, they have developed effective strategies to create a common operating picture through robust data integration. As we continue to respond to this pandemic and prepare for the next crisis, the health care community should learn from these mission critical organizations.

Editor’s Note – The author is a Department of Defense consultant.

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When IBM first launched its Watson Health unit in 2015, it had to live up to a grandiose vision. The company’s AI creation, popularized by its win on Jeopardy!, was pitched to oncologists as a tool that could comb through medical literature and cancer patients’ health records, detecting patterns that they could not.

Reports later found that it fell short of those claims, and in some cases, offered ‘unsafe and incorrect’ suggestions. Now, IBM might be considering selling its Watson Health unit, as it focuses more on its cloud computing business, according to the Wall Street Journal. Citing anonymous sources, the Journal reported the health unit brought in $1 billion in revenue and isn’t currently profitable.

IBM declined to comment on the report, and it’s not clear who would buy the company. But it’s another example of a high-flying healthcare effort that might have tried to do too much all at once, and a case where marketing overtook the science.

Other tech behemoths have also stumbled in their much-vaunted plans to disrupt healthcare. Haven, a joint venture between Amazon, JPMorgan Chase and Berkshire Hathaway to combat rising healthcare costs, dissolved as the three companies pursued their own efforts.

Part of the challenge is that it’s difficult for these large companies to move quickly, while in the same span, dozens of startups are bringing their own solutions to market.

“There’s a contextual dynamic that large companies will by definition not move as quickly as early-stage innovative companies,” said Michael Greeley, co-founder and general partner with Flare Capital Partners. “A product roadmap that a big tech company might set for the end of the year, by the time committees meet and budget, the year has gone by.”

Two years ago, IBM started winding down sales of Watson for Drug Discovery to pharmaceutical companies, because it wasn’t yielding big enough financial returns. Before that, the general manager of the division also stepped down for a different role at the company.

Facing declining revenues, in an investor call last month, the company’s new CEO, Arvind Krishna, said he was looking to redefine IBM’s future as a cloud platform and AI company.

“This is where we are focusing the bulk of our efforts, time and investments,” he said.

Over-hyped and under-delivered

With the way IBM had marketed Watson for Oncology, “There was clearly always a mismatch in the reality and the promise of what they were going to bring to market,” Greeley said.

More time would have been needed to get closer to that goal. Building AI tools for healthcare requires a huge amount of high-quality data that can be hard to get, and complicated to analyze.

“To date, there’s been far more heat than light,” wrote David Shaywitz, founder of health-tech advisory firm Astounding HealthTech. “There’s a lot of complexity to health data that requires domain expertise to understand, and just sticking a lot of values in a data lake or data swamp and then setting algorithms loose on it hasn’t proved especially productive to date.”

Despite that, Shaywitz still remains optimistic that AI will have a role in medicine in drug development in the future. He pointed to Flatiron Health as an example of one startup that has done well – it was acquired by Roche in 2018 for $1.9 billion.

He said that success relies on the ability for health and tech experts to collaborate as equal partners,  something that’s “vanishingly rare” at big tech, biopharma and healthcare companies.

 Whatever happens with Watson, Greeley still doesn’t see tech companies’ interest in healthcare waning anytime soon, as Amazon wades into the prescription drug market and Google tries to woo more health systems with cloud partnerships.

“I think we’re seeing renewed intrigue by consumer tech, the Googles and Facebooks of the world,” he said. “I think because healthcare is such an important part of the economy, they will continue to be active with acquisitions.”

Photo credit: Getty Images, wigglestick

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Pfizer and BioNTech are asking the FDA to approve a change that would permit storage of their Covid-19 vaccine within a temperature range found in medical-grade freezers.

To be clear, this proposed change doesn’t eliminate the need for ultra-cold temperatures as the vaccine moves throughout the supply chain—temperatures that require specialized equipment. Those storage requirements are a barrier to its distribution because not all facilities have equipment that can achieve the required temperatures. What Pfizer and BioNTech are proposing is that when the vaccine reaches its destination, such as a hospital or pharmacy, it could be stored for up to two weeks at (relatively) warmer freezer temperatures.

The FDA granted emergency use authorization to the messenger RNA vaccine, named Comirnaty, last December. According to the product’s label, the vaccine must be stored in ultra-cold temperatures between minus 112 degrees and minus 76 degrees Fahrenheit (minus 80 degrees and minus 60 degrees Celsius). At those temperatures, the vaccine can last up to six months. Pfizer ships the vaccines in specially designed containers that can be refilled with dry ice every five days. Those containers can serve as temporary storage for up to 30 days.

The vaccine can be stored at refrigerator temperatures for up to five days. Pfizer and BioNTech are asking the FDA to approve an additional option to store the vaccine at minus 13 degrees to 5 degrees Fahrenheit (minus 25 degrees to minus 15 degrees Celsius) for two weeks. That range would put it closer to the storage requirements for the mRNA vaccine from Moderna. The temperature range Pfizer and BioNTech are proposing would be in addition to the five days at refrigerator temperatures that is currently permitted under the authorization.

“If approved, this new storage option would offer pharmacies and vaccination centers greater flexibility in how they manage their vaccine supply,” Pfizer CEO Albert Bourla said in a prepared statement.

Pfizer and BioNTech are proposing the additional temperature option based on new data about their vaccine’s stability. The data span from the earliest clinical trials up to batches currently in production.

Messenger RNA vaccines are a new technology that employ a snippet of genetic material from the novel coronavirus. This mRNA serve as a blueprint that a cell’s protein-making machinery use make the characteristic spike protein found on the surface of the novel coronavirus. That protein is what triggers an immune response and confers immunity.

Though mRNA vaccines are administered at room temperature, they must be kept at ultra-cold temperatures in the supply chain because mRNA is fragile. The extremely cold temperatures keep the components of the vaccine from breaking down. As mRNA companies study the storage data they have for vaccines, they are getting a better understanding of how long these vaccines can last at certain temperatures. Last November, Moderna released data that it said supported storage of its vaccine at refrigerator temperatures for up to 30 days.

Temperature requirements can be a barrier to the distribution of the vaccine to rural areas, which may not have facilities with appropriate freezers. In that regard, the Johnson & Johnson Covid-19 vaccine is seen as offering an advantage. In addition to requiring only a single shot (both authorized mRNA vaccines require two), the J&J vaccine can be stored at standard refrigerator temperatures. An FDA advisory committee is scheduled to hold a hearing about that vaccine on Feb. 26.

Photo by BioNTech

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The journey toward achieving interoperability in healthcare needs to now move beyond data exchange, and instead, focus on data management.

This is the opinion of a panel of experts who gathered at the all-virtual Health Datapalooza and National Health Policy Conference Thursday to discuss one of healthcare’s most hotly debated concepts: interoperability.

The healthcare industry has come a long way with regard to interoperability, especially with the new rules proposed by the Department of Health and Human Services, set to take effect April 5. These rules aim to provide patients with unprecedented access to their data.

But the healthcare problems of today require solutions that support data management, and not just data exchange, said Claudia Williams, CEO of California-based Manifest MedEX, at the Health Datapalooza conference.

The industry has focused on enabling the basic exchange of health records between providers and made great progress, but the connective tissue that enables data management — including matching and cleaning data — is lacking, she said. And it’s the smaller providers that are being left behind.

“In California, a slim share of the health delivery systems, mostly the big health systems, have hundreds of people doing this work, and safety net providers and small Medicaid plans and others really are stuck with just being able to pile up CCDAs [consolidated clinical document architecture] or maybe not even process CCDAs at all,” she said.

There needs to be policies and strategies enacted at the state and federal levels to create an infrastructure that has more to do with the management of data than with health record exchange, Williams said.

While policy actions are necessary, there also needs to be more alignment between the needs of the providers on the ground and the health IT technology and capabilities available today, said Dr. Farzad Mostashari, CEO of Bethesda, Maryland-based Aledade, during the panel discussion.

Through Aledade, which operates accountable care organizations in partnership with more than 800 primary care practices, Mostashari has experienced that disconnect firsthand.

“What EHRs do today have nothing to do with what I need,” he said. “Well, not nothing, but they really don’t fill the thermometer of what I need to do for population health.”

It is costing Aledade millions to map, match and translate EHR data, he added.

Looking ahead, care quality measurement needs to be automated within the EHR and providers should get free access to information that is already mapped in accordance with data standards, Mostashari said.

The technical tools needed to push interoperability forward already exist, but the regulatory landscape needs to catch up, said Donald Trigg, president of North Kansas City, Missouri-based Cerner, during the session.

The government is now both the biggest healthcare regulator in the country and the biggest payer. This means it is in a unique position to use health IT certification and provider reimbursement to help create the interoperability architecture that is necessary for the coming decade, Trigg said.

“I’m still an optimist,” he said. “And I think that Covid and this administration will be an accelerant for the next wave of meaningful data exchange.”

Trigg’s advice for the new administration’s HHS is to tackle the inter-agency complexity that exists at the federal level.

Coordination between agencies like the HHS, Federal Trade Commission and Food and Drug Administration is necessary. This means, it will be important to create clarity around the inter-agency landscape and data management so that the healthcare industry can innovate and do more, Trigg said.

Photo: LeoWolfert, Getty Images

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health insurance, Obamacare

A rise in insurer participation in the Affordable Care Act individual marketplace indicates that payers are anticipating a fruitful year ahead. The installation of a new president and ongoing Covid-19 pandemic appear to be driving this trend.

Over the past year, insurer participation in the ACA individual marketplace has grown and benchmark premiums have declined, according to a new analysis by the Robert Wood Johnson Foundation. The total number of insurance offerings sold on the marketplace is now 9,144, which is about 75% of the 2015 record high.

This suggests that insurers are anticipating increased enrollment as a result of federal policy changes and the impact of the Covid-19 pandemic, said Katherine Hempstead, senior policy adviser at RWJF and author of the analysis, in an email.

“The Biden administration has been emphatic about its commitment to the ACA marketplace,” she said. In particular, the administration has committed to preserving and expanding health coverage to help Americans during the pandemic.

In his American Rescue Plan, President Joe Biden called on Congress to subsidize continuation health coverage through the end of September and to expand and increase the value of the Premium Tax Credit. The latter move will help lower or eliminate health insurance premiums and ensure enrollees will not pay more than 8.5% of their income for coverage.

Further, Biden recently reopened the HealthCare.gov insurance markets for three months to enable Americans to sign up for coverage amid the ongoing pandemic.

Though these policy changes are temporary, there is a commitment on the part of the administration to try to make them permanent, Hempstead said.

Drilling down into participation among major insurers, Hempstead found that Anthem, UnitedHealth and Cigna currently comprise about two-thirds of the national commercial offerings on the individual marketplace. Centene, which dominates the Medicaid managed care organizations category, made its largest single-year increase, nearly doubling its marketplace offerings from 2020 to 2021.

In addition, participation by newcomers like Oscar and Bright Health has grown steadily. Bright Health is now in 10 states, and Oscar is in 19.

Another key analysis finding is that states that have yet to expand Medicaid saw increases in insurer participation. Increased participation in the ACA individual marketplace was particularly focused in Florida, Georgia, North Carolina and Texas, where the number of offerings increased by almost 50% in the last year.

“This is where the greatest number of uninsured people live, so it is the biggest opportunity for membership growth,” Hempstead said.

Looking ahead, it is clear that insurers are hopeful that a significant expansion in healthcare coverage is due, and they see the ACA marketplace as an increasingly important part of the coverage landscape, according to the analysis.

Photo: BrianAJackson, Getty Images

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A major concern during the Covid-19 pandemic has been that Americans, especially those with underlying conditions, will delay necessary care. New survey results show this concern is not unfounded.

As of last September, about 40% of Americans with one or more chronic health conditions reported delaying or avoiding care, according to a new report from the Urban Institute and Robert Wood Johnson Foundation.

Report authors analyzed data from the second wave of the Urban Institute’s Coronavirus Tracking Survey, a nationally representative survey conducted Sept. 11-28, 2020. The survey polled 4,007 adults, ages 18 to 64 years.

About 36% of Americans said they delayed or did not receive healthcare due to a fear of exposure to the coronavirus or because a provider limited services during the pandemic, the report states. Black adults (39.7%) were more likely than white (34.3%) or Hispanic/Latinx (35.5%) adults to report delaying or forgoing care because of concerns about virus exposure.

About four in 10 adults with one or more chronic health conditions (40.7%) said they delayed or avoided care because of the pandemic, as compared with 26.4% of adults with no chronic conditions.

In addition, more than half of adults with both a physical and mental health condition (56.3%) reported delaying or avoiding healthcare due to the pandemic. About 43% of this group also reported delayed or forgoing multiple types of care.

The impacts of delaying or avoiding care were acutely felt by those with chronic conditions, the report shows. An estimated 23.2% of these adults reported that going without or delaying care worsened a health condition, 21% said it limited their ability to perform daily activities and 15.2% said it limited their ability to work.

Further, the report shows the kinds of care that Americans were avoiding. Dental care was the most common type of care adults delayed or did not receive because of the pandemic (25.3%), followed by seeing a general doctor or specialist (20.6%) and receiving preventive health screenings or medical tests (15.5%).

“Tackling unmet healthcare needs requires effectively assuaging fears about exposure to the coronavirus,” report authors concluded. Providers need to reassure patients that they are following public health guidelines and that these precautions can effectively prevent virus transmission.

“More data showing healthcare settings are not common sources of transmission and better communication with the public to promote the importance of seeking needed and routine care are also needed,” the authors wrote.

Photo: YinYang, Getty Images

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Paula Soteropoulos

Gene therapy may offer potential cures, but its promise comes with a price. Some experimental approaches require a multi-step process to prepare stem cells for the procedure—a burden to a patient and to the healthcare system, according to Paula Soteropoulos, executive chair of startup Ensoma.

Soteropoulos’s company is proposing an alternative. The Boston-based biotech is developing technology that won’t require hospitalization in a specialized medical center. Furthermore, the Ensoma technology does its therapeutic work in vivo—inside the patient. These features could make genomic medicine more accessible, turning a lengthy hospital process into a single visit to a doctor’s office.

“Our hope with our technology is to be able to do it outpatient,” Soteropoulos said. “It’s an injection that can be done anywhere, it doesn’t require specialized centers.”

Ensoma emerged last week with details about its technology and $70 million in Series A financing. The company also revealed something unusual for a preclinical startup coming out of stealth: a research partnership with a large pharmaceutical company. Takeda Pharmaceutical is collaborating with Ensoma on up to five disease targets.

Soteropoulos, the former CEO of rare-disease drug developer Akcea Therapeutics, said Takeda and others that want to be in gene therapy are looking for in vivo innovations. Ex vivo approaches, in which a patient’s cells are removed and manipulated in a lab before being reintroduced, pose complexities and challenges for companies trying to commercialize them and to healthcare facilities that will provide them, she said.

Gene therapies reach target cells as cargo carried on engineered viruses. But these viruses come with limitations. Adeno-associated virus (AAV), a commonly used vector, can trigger an immune response. AAV also has limited capacity, which makes it hard to deliver a therapy consisting of a larger gene.

An alternative vector, lentivirus, has more capacity but is still limited in its ability to carry a big payload. This approach requires collecting a patient’s stem cells and engineering them outside the body. Before the stem cells are reintroduced, patients must undergo conditioning, comprised of chemotherapy. This step helps the stem cells carrying a therapeutic gene to be taken up by bone marrow, where they will proliferate. But conditioning can lead to side effects such as greater susceptibility to infection and bleeding. Avrobio and bluebird bio are among the biotechs developing lentiviral stem cell gene therapies that require patient  conditioning.

Soteropoulos describes Ensoma’s engineered adenoviruses as “gutless.” On the inside, they’re stripped of viral DNA or RNA that could trigger an immune response. On the outside, the viruses are engineered to specifically target hematopateic stem cells in the bone marrow. They can also target the cells that arise from these stem cells, such as T cells, B cells, and myeloid cells.

There’s another benefit to Ensoma’s gutless viruses. Removing their DNA or RNA creates more room for the genetic payload—more than three times as much as what the viruses used to deliver the current generation of gene therapies can carry. With that extra space, Ensoma’s vectors can carry larger genes as well as gene-editing tools, such as CRISPR or zinc finger nucleases.

“It allows us to do things that other gene therapies cannot,” Soteropoulos said.

Ensoma’s science is based on 20 years of research from the company’s scientific co-founders, Hans-Peter Kiem of the Fred Hutchinson Cancer Research Center, and André Lieber of the University of Washington School of Medicine. In the past five years, that research started forming the foundations of a company. In 2017, the scientists published research showing how their cells were taken up by the bone marrow in a monkey study. Last year, they published study results showing how their approach corrected two genetic disorders, beta thalassemia and sickle cell disease, in mice.

Ensoma was founded about 18 months ago, backed by seed financing from 5AM Ventures, Soteropoulos said. The startup licensed technology from Fred Hutch and UW, then added to the research, building on the intellectual property surrounding it. She said the research reached the point where additional financing was needed to support the next step of selecting which diseases to pursue.

Along the way, the startup drew interest from larger companies that had followed the science of its founders, Soteropoulos said. One of those companies was Takeda. In addition to investing in Ensoma’s Series A financing, the Tokyo-based pharmaceutical giant is also a research partner. The collaboration grants Takeda an exclusive global license to Ensoma’s technology for up to five rare diseases. That alliance could lead to up to $100 million in upfront and preclinical research payments to Ensoma. If all five programs reach the market, Ensoma could receive as much as $1.25 billion in milestone payments plus royalties from sales.

The Ensoma technology offers the potential to go beyond rare diseases. Soteropoulos said that the in vivo approach does away with all the complexity of working with a therapy outside of the body, making these therapies simpler to manufacture and easier to administer. She added that the fact that these therapies won’t require conditioning or stem cell donors helps extend the reach of these genetic medicines to common diseases.

Ensoma is pursuing rare diseases first. The technology is new, so regulators will need time to understand it, Soteropoulos said. Takeda and Ensoma aren’t disclosing the disease targets they have in mind and Soteropoulos said it’s too soon to say when the technology will reach human testing. But she added that because Ensoma’s approach holds promise to address many diseases, there are plenty to choose from. The startup may look for other collaborators in the future but in the near term, the company will focus on developing its own therapies in addition to working with Takeda.

“There are some rare diseases where there is already validation from being able to make these modifications and cure,” Soteropoulos said. “We would be working off of that for our first indications and then see how we can explore other areas.”

5AM led Ensoma’s Series A financing. Besides Takeda, the new round of funding included the participation of F-Prime Capital, Viking Global Investors, Cormorant Asset Management, RIT Capital Partners, Symbiosis II, and Alexandria Venture Investments.

Photo by Ensoma

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MedCity News will host its annual INVEST conference online, April 19-23. INVEST is a premier international healthcare investing event hosted in partnership with MHIN, a Midwest healthcare investor organization. The conference brings together active investors and promising startups in biopharma, diagnostics, medical devices, health IT, and health services.

INVEST will address six broad themes through a mix of live and pre-recorded sessions:

  • Public Health and Health Equity
  • Healthcare Delivery
  • Healthcare Investing & Financing
  • Empowering Patients
  • Behavioral Health
  • Employer Health

Among the confirmed speakers so far are:

  • Ellen Herlacher, Principal, LRV Health
  • Dennis Depenbusch, Director, New Ventures, BCBS Kansas
  • Austin Duke, Senior Venture Associate, UnityPoint Health Ventures
  • Christina Farr, Principal, OMERS Ventures
  • Ami Bhatt, MD, Director, Outpatient/TeleCardiology & Adult Congenital Heart Disease Program, Massachusetts General Hospital
  • Harsh Vathsangam, PhD, co-founder and CEO, Moving Analytics
  • Kevin Dedner, Founder and CEO, Hurdle
  • M Daniele Fallin, PhD, Chair, Department of Mental Health, Johns Hopkins Bloomberg School of Public Health
  • Morgan Cheatham, Investor, Bessemer Venture Partners
  • Tina Hernandez-Boussard, PhD, Associate Professor of Biomedical Informatics and Surgery, Stanford University School of Medicine
  • Sahil Choudhry, Managing Director, Cigna Ventures & Strategy

Click here to register now


Pitch Perfect Startup Competition

As in previous years, promising startups will get the opportunity to present to investors directly as part of the INVEST Pitch Perfect competition. In order to apply to get selected, startups must fulfill one of the following criteria:

  • Have raised at least $1 million, or
  • Have raised a Series A round, or
  • Have one qualified institutional investor or a strategic investor that they can name

There is no application fee. The deadline for submissions is Feb 28, so please click here to apply now.

Ask the Investor

One new feature of the conference will be intimate sessions to give startups an overview of investment strategies for leading healthcare investors and provide them the opportunity to engage in a Q&A with them. Each session will feature one investor who will outline his or her investment philosophy and area of interest, and 10 startups will be able to participate. Startups who choose to register for this session will be asked to provide company and contact information that will be shared with the presenting investor to facilitate interaction after the session.

Space is limited to 10 startups per meeting. Please register here.

If you are a VC or corporate investor and want to be considered for a judging role, or you’d like to speak on one of our panels at the event, please click here.

To learn about sponsorship opportunities, contact Stephanie Baum, Director of Special Projects, at sbaum@medcitynews.com or email the sales team at advertising@medcitynews.com.

For general information about the conference, contact Laura Kittredge, Director of Events at lkittredge@medcitynews.com.

Illustration: DrAfter123, Getty Images

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