Microsoft System Blamed for New Jersey Vaccine-Booking Glitches

New Jersey officials are blaming Microsoft systems that were supposed to help vaccination efforts for allegedly glitching over the past five weeks, hindering the ability for a smooth vaccination rollout in the state. 

The state’s Microsoft system for running the vaccination rollout has had issues daily from booking appointments to losing registrations, state government officials told Bloomberg News

State officials blamed issues they said seem to stem from Microsoft not having enough employees staffed to deal with the issues and having employees in time zones that make them unavailable to help during business hours.

Vaccination rollout has been a major operation for states struggling to keep track of vaccinations and standardize strict guidelines for who medical facilities are allowed to vaccinate. 

Microsoft told Bloomberg that they know of some issues with the system in New Jersey, but did not comment further. 

“We are working with the state of New Jersey to deliver vaccinations as quickly, safely and efficiently as possible, and that includes addressing some technical issues,” a Microsoft spokesperson told Bloomberg.

Hospital and county websites have picked up the slack in New Jersey where the Microsoft system has failed, Bloomberg reported. More than 1.2 million vaccinations have reportedly been scheduled in the state. 

New Jersey was hit hard by the coronavirus pandemic, with more than 700,000 confirmed cases and more than 22,000 deaths from COVID-19, according to The New York Times.

Joe Biden is still aiming for 100 million people to be vaccinated within his first 100 days in office, but the process has been difficult, involving delivery delays, scheduling issues and vaccine hesitancy.

By Lexi Lonas

Tags MicrosoftJoe BidenNew Jerseycoronavirus vaccineCoronavirus

Bloomberg Quicktake: Now

Five weeks of stumbles by Microsoft Corp. on New Jersey’s Covid-19 vaccine-booking software have left the state pushing for daily fixes on almost every part of the system and doubting it will ever operate as intended, according to members of Governor Phil Murphy’s administration. The glitches — and attempted fixes that forced one megasite to go off-line temporarily — have led New Jersey to rely more on the county- and hospital-operated websites that are working well and have helped schedule more than 1.2 million doses in the most densely-populated state in the country.

Officials say those systems are successfully booking thousands of people. They fear the state’s booking portal, run on Microsoft software and functioning for just a limited number of residents, won’t withstand broad demand as eligibility eventually is opened to millions of more people. Health care has become a major focus for Microsoft, which unveiled a package of industry-specific cloud software in May. The world’s largest software company, which has hired executives with medical backgrounds, also has been researching machine learning and artificial intelligence tools for areas including clinical trials and patient care. In late January, the Redmond, Washington-based company touted its Microsoft Vaccination Management platform — usable by those seeking shots and by health providers — to register, schedule, track supplies and otherwise streamline the biggest inoculation effort in U.S. history.

The platform has yet to work correctly for New Jersey in the state’s effort to inoculate its residents against the coronavirus, according to two administration officials who asked not to be identified discussing contractual issues. Governor Murphy and State Health Commissioner Judith Persichilli acknowledged there was an issue with Microsoft in a Feb. 10 briefing, but didn’t go into detail about the problems. Since the state’s CovidVaccine.nj.gov website went live Jan. 5, the software has booked thousands of appointments. But it’s also blocked users, lost registrations, double-booked residents and crashed for periods of five minutes to three days, the officials said. Though Microsoft has worked daily on the troubles, the officials said they had no confidence that they’ll get all the features called for in its contract with the company. In a statement, Microsoft acknowledged difficulties with booking shots but didn’t specify the problems. “We are working with the state of New Jersey to deliver vaccinations as quickly, safely and efficiently as possible, and that includes addressing some technical issues,” a Microsoft spokesperson said in an email.

The New Jersey officials declined to say whether the state is considering canceling the Microsoft contract, but said they are seeking solutions and workarounds of all kinds. The cost of the contract wasn’t readily available. New Jersey was among the earliest and hardest-hit U.S. states by Covid-19, recording almost 21,000 deaths with a lab-confirmed link to the disease caused by the coronavirus. Murphy, a first-term Democrat running for re-election this year, has committed to vaccinating 4.7 million people, or 70% of the state’s population, by late June. So far, New Jersey has administered nearly 1.2 million doses, representing a tenth of the population who have received at least one dose, according to the Bloomberg Vaccine Tracker. State officials said Microsoft appears to be using too few staffers, with some key personnel in overseas time zones that leave them unavailable during U.S. business hours. The officials said they’ve conferred with other states using versions of the same software, which is built on the Microsoft Dynamics customer-relationship management platform.

The task appears to be going smoother, they said, in places that asked for fewer applications — just scheduling, say, rather than more complex services. Subscribe to our YouTube channel: https://bit.ly/2TwO8Gm​ Bloomberg Quicktake brings you live global news and original shows spanning business, technology, politics and culture. Make sense of the stories changing your business and your world. To watch complete coverage on Bloomberg Quicktake 24/7, visit http://www.bloomberg.com/qt/live​, or watch on Apple TV, Roku, Samsung Smart TV, Fire TV and Android TV on the Bloomberg app. Have a story to tell? Fill out this survey for a chance to have it featured on Bloomberg Quicktake: https://cor.us/surveys/27AF30​ Connect with us on… YouTube: https://www.youtube.com/user/Bloomberg​ Breaking News on YouTube: https://www.youtube.com/c/BloombergQu…​ Twitter: https://twitter.com/quicktake​ Facebook: https://www.facebook.com/quicktake​ Instagram: https://www.instagram.com/quicktake

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More Contents:

Our view: It is not blasphemy to identify problems in NHS vaccine booking system http://www.oxfordmail.co.uk – February 15THERE has been much rejoicing this weekend at the much anticipated announcement that the rollout of the coronavirus vaccine has passed the 15……1

Monty Don addresses ‘puzzled’ fan after his vaccine news causes confusion | Celebrity News | Showbiz & TV http://www.express.co.uk – February 13[…] A fan stepped in to help Monty Don explain the vaccine booking process to a follower Monty Don took to Twitter to share the ‘wonderful’ news that he had boked his […]3

Microsoft System Blamed for New Jersey Vaccine-Booking Glitches http://www.youtube.com – February 13N/A

CommonWealth Magazine commonwealthmagazine.org – February 13[…] and availability issues in many regions across the state, and communications breakdowns in the vaccine booking system,” the speaker said […]1

Microsoft System Blamed for N.J. Vaccine-Booking Glitches http://www.itprotoday.com – February 12HYBRID CLOUD Five weeks of stumbles by Microsoft Corp. on New Jersey’s Covid-19 vaccine-booking software have left the state pushing for daily fixes on almost every part of the system.N/A

Vaccine booking error further frustrates medically vulnerable Floridians cbs12.com – February 12Elizabeth Kahn turns 48-years-old next week and has just one birthday wish this year. “My wish is to get the vaccine, or at least schedule it,” Kahn said. And for a moment, she thought her wish was coming true.6

News from the UK’s largest union – Issue #1 | Revue http://www.getrevue.co – February 12[…] announcement that care workers who haven’t had a dose yet can make an appointment The article Vaccine booking system now open to frontline care workers first appeared on the UNISON National site […]0

Microsoft system blamed for N.J. vaccine-booking glitches http://www.bloomberg.com – February 12Five weeks of stumbles by Microsoft on New Jersey’s COVID-19 vaccine-booking software have left the state pushing for daily fixes on almost every part of the system, according to state…546

Pritzker’s Stats Obsession Will Intensify Vaccine Hunger Games patch.com – February 12[…] — without consulting her, and if it would create even more problems for an already-overwhelmed vaccine booking system […]11

U.S. Orders More Doses; Japan to Rule on Pfizer: Virus Update http://www.bloombergquint.com – February 12[…] Vaccine-Booking Glitches (6 a.m. NY) Five weeks of stumbles by Microsoft Corp. on New Jersey’s Covid-19 vaccine-booking software have left the state pushing for daily fixes on almost every part of the system an […]0

February 10, 2021: COVID-19: US State Policy Report http://www.natlawreview.com – February 11[…] over 75 can get vaccinated beginning February 1 (see here) Gubernatorial announcement regarding vaccine booking program (see here) Michigan: Stay at home order expired on June 1 Shelter in Place order expired on […]0

February 9, 2021: COVID-19: US State Policy Report http://www.natlawreview.com – February 10[…] over 75 can get vaccinated beginning February 1 (see here) Gubernatorial announcement regarding vaccine booking program (see here) Michigan: Stay at home order expired on June 1 Shelter in Place order expired on […]0

Seniors report problems with state’s 211 vaccination hotline http://www.capecodtimes.com – February 10[…] To make the vaccine booking appointment as equitable as possible for eligible Cape Codders — which include those in phase on […]4

Vaccine web portal lacks accessibility gpo.ca – February 9[…] many Ontarians who do not have access to the technology or broadband needed to access the online vaccine booking portal […]4

Getting the COVID-19 vaccine: what to expect – http://www.which.co.uk – February 9[…] These can look highly convincing (as per the example of a fake vaccine booking website above), so it’s worth being vigilant […]43

COVID-19 vaccine FAQs http://www.barnsleyccg.nhs.uk – February 9[…] uk/covidvaccine Last updated: 08/02/21 Vaccine booking update: If you are 70 or over, or 18 or over and have been advised to shield by letter because yo […]N/A

COVID State Policy Report for CO, CT, IL, NY, OR, RI, SC, SD http://www.natlawreview.com – February 9[…] over 75 can get vaccinated beginning February 1 (see here) Gubernatorial announcement regarding vaccine booking program (see here) Michigan: Stay at home order expired on June 1 Shelter in Place order expired on […]N/A

London hospital trust signs up to new Covid-19 vaccine booking app london-post.co.uk – February 8[…] keep track of who has received a Covid-19 vaccine within their workforce thanks to a new Covid-19 vaccine booking service […]N/A

Massachusetts COVID Vaccine Hotline: What to Know – http://www.nbcboston.com – February 6[…] Her daughter, Susan Parker, had found the state’s vaccine booking website hard to navigate, and the Marblehead family felt a sense of urgency to secure a slot […]1

Book now – vaccinations available at three local hospitals: but you must be eligible http://www.leicestercityccg.nhs.uk – February 6[…] A vaccine booking can be made at one of the following hospital sites by clicking on the relevant link: Glenfiel […]5

February 4, 2021 COVID-19 US State Policy Report http://www.natlawreview.com – February 6[…] over 75 can get vaccinated beginning February 1 (see here) Gubernatorial announcement regarding vaccine booking program (see here) Michigan: Stay at home order expired on June 1 Shelter in Place order expired on […]0

Covid vaccination available for limited period for priority groups at three hospital sites | Measham Parish Council http://www.meashamparishcouncil.gov.uk – February 5[…] A vaccine booking can be made at one of the following hospital sites by clicking on the relevant link: Glenfiel […]0

Temple Vaccine Booking outlook.office365.com – February 5You can now book and manage appointments using our booking page.7

COVID-19: US State Policy Report – February 3, 2021 http://www.natlawreview.com – February 4[…] pharmacist refill flexibility (see here) Massachusetts: Gubernatorial announcement regarding vaccine booking program (see here) New Jersey: Executive Order 219 Raising Indoor Capacity Limits from 25 Percen […] over 75 can get vaccinated beginning February 1 (see here) Gubernatorial announcement regarding vaccine booking program (see here) Michigan: Stay at home order expired on June 1 Shelter in Place order expired on […]N/A

Legislative leaders stepping up oversight over coronavirus response http://www.lowellsun.com – February 4[…] and availability issues in many regions across the state, and communications breakdowns in the vaccine booking system,” Mariano said […]N/A

The Pharmacy in Ludgershall to roll out Covid-19 vaccine http://www.salisburyjournal.co.uk – February 4[…] ” More vaccine news: These three sites will now appear on the national coronavirus vaccine booking system, along with the large vaccination centres at Salisbury City Hall and Bath Racecourse […]6

Covid-19 vaccine update 4 Feb 2021 :: Kent and Medway Clinical Commissioning Group http://www.kentandmedwayccg.nhs.uk – February 4[…] All bookings must be through the national Covid-19 vaccine booking service […]12

UK ‘getting safer every day’ as almost 1 in 5 adults have had first vaccine, says Nadhim Zahawi http://www.telegraph.co.uk – February 4[…] 2:50am Teachers skipping Covid vaccine queue Teachers are abusing the vaccine booking system and skipping the queue ahead of the elderly, a council warned as it introduced tighter rules […] Teachers are abusing the vaccine booking system and skipping the queue ahead of the elderly, a council warned as it introduced tighter rules […]N/A

Waiting for the COVID vaccine? Don’t ignore ‘unknown’ or ‘private’ callers for now http://www.khou.com – February 4[…] So again, if you’re waiting on an email response for your coronavirus vaccine booking, you will want to keep a close eye on all of your email folders […]41

Eight Best Practices for COVID-19 Vaccine Scheduling Online: Insights From Our Health System Customers http://www.kyruus.com – February 4[…] Add a one day lead time for vaccine booking (i […]0

Marco police & fire chief suspended over COVID-19 vaccine booking scandal nbc-2.com – February 4[…] – The Marco Island police and fire chief have been suspended for 30 days over the COVID-19 vaccine booking scandal […]

Japanese Supercomputer Shows How Coronavirus Spreads In A Dining Setting

Earlier this month, the Centers for Disease Control and Prevention (CDC) revised its guidance to say that the Covid-19 virus can “linger in the air for minutes to hours” and occur between people spaced more than six feet apart.  

This followed a CDC study last month that found that adults with Covid-19 were twice as likely to have dined out at a restaurant within two weeks prior to being infected.

A new simulation from the Fugaku supercomputer in Japan demonstrates how the seating arrangement can make a difference to how easily the coronavirus is transmitted to dining companions at the same table. Recommended For You

Japanese researchers from Kobe University and the research giant Riken tasked Fugaku, the world’s fastest supercomputer, to model how the coronavirus spreads in a typical dining situation. The simulation shows the emission and flow of aerosol particles when four people are sitting a table and speaking without masks on.

The first takeaway from the Fugaku simulation is that the seating arrangement matters. When an infected individual speaks to dining companions seated across the table, four times as many exhaled aerosol droplets reach the person seated directly across the table compared to the person seated diagonally from the speaker.

The person seated next to an infected person is the most at risk. When an infected person turns his head sideways to speak to the dining companion next to him, that individual is exposed to more than five times the amount of exhaled droplets than the individual directly across the table from the speaker.

This research also implies that diners can further reduce risk by keeping face masks on when conversing before food arrives and after they have finished eating.

“When people with Covid-19 cough, sneeze, sing, talk, or breathe they produce respiratory droplets,” explains the CDC guidance. “These droplets can range in size from larger droplets (some of which are visible) to smaller droplets. Small droplets can also form particles when they dry very quickly in the airstream.”

A second takeaway from the same Japanese research is that humidity levels can have a significant impact on how easily droplets are transmitted. The scientists found that fewer droplets are dispersed when humidity is higher, which suggests that the use of humidifiers in indoor settings may help limit infections if window ventilation is not possible.

Public health experts like Dr. Anthony Fauci, the nation’s top infectious disease expert, have expressed concern about dining in dry, heated indoor environments during the the winter months. “People will be spending more time indoors, and that’s not good for combating a respiratory-borne virus,” Fauci told MSNBC.

Toward that effort, the leaders of New York City and Chicago and other cities are creating initiatives to make outdoor dining a reality throughout the coming winter.

Fugaku is the product of a $1 billion, decade-long mission by several thousand developers from the government-run Riken Center for Computational Science and computer maker Fujitsu. Since the pandemic began, Fugaku has been creating simulations that demonstrate the ease with which the coronavirus spreads in various settings, including on trains, in work spaces and in classrooms.

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Suzanne Rowan Kelleher

Suzanne Rowan Kelleher

I’m always looking for new ways to travel better, smarter, deeper and cheaper, and spend a lot of time watching trends at the intersection of travel and technology. As a longtime freelance travel writer, I’ve contributed hundreds of articles to Conde Nast Traveler, CNN Travel, Travel Leisure, Afar, Reader’s Digest, TripSavvy, Parade, NBCNews.com, Good Housekeeping, Parents, Parenting, Esquire, Newsweek, The Boston Globe and scores of other outlets. Over the years, I’ve run an authoritative family vacation-planning site; interviewed Michelin-starred chefs, ship captains, taxi drivers and dog mushers; reviewed hundreds of places to stay, from stately castles and windswept lighthouses to rustic cabins and kitschy motels; ridden the iconic Orient Express; basked in the glory of Machu Picchu; and much more. Follow me on Instagram (@suzannekelleher) and Flipboard (@SRKelleher).

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Nippon TV News 24 Japan 10.8K subscribers Researchers use the supercomputer Fugaku to simulate how droplets of the novel coronavirus spread. ********************** Subscribe to our YouTube channel: https://www.youtube.com/c/NipponTVNew… Like us on Facebook: https://www.facebook.com/NipponTVNews… ********************** #NipponTVNews24Japan#NTV#日テレ#Japannews#Japanvideo#Japan#Coronavirus#COVID19#Fugaku#Supercomputer#SupercomputerFugaku#coronavirusinfection#Riken#coronavirusresearch#coronavirusdroplet

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After Push From Experts, World Health Organization Says It’s Possible COVID-19 Spreads by Air

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he World Health Organization (WHO) on Thursday heeded calls from more than 200 scientists, who urged the global health authority to acknowledge that COVID-19 may spread by air.

Previously, the WHO said contact with large respiratory droplets, like those expelled in a sick person’s cough or sneeze, appeared to be the primary way COVID-19 spreads. But in a highly publicized letter published earlier this week, a large group of scientists argued the WHO’s guidance neglected to adequately address another important route of transmission: inhaling tiny respiratory particles generated by a sick person, which can remain suspended in the air indoors for hours.

In a scientific brief published Thursday, the WHO allowed that “short-range aerosol transmission, particularly in specific indoor locations, such as crowded and inadequately ventilated spaces over a prolonged period of time with infected persons cannot be ruled out.” Still, it maintained that further studies on airborne transmission are needed, and said the evidence is strongest for respiratory droplet transmission.

The WHO hinted at its evolving opinion during a press briefing on Tuesday, when Maria Van Kerkhove, the agency’s technical lead for COVID-19, said its scientists had been “talking about the possibility of airborne transmission and aerosol transmission.” Many of the scientists who signed the letter on airborne transmission celebrated her comments.

“Nice work, team Indoor Air,” signatory Joseph Allen, an indoor air expert and assistant professor at the Harvard T.H. Chan School of Public Health, tweeted on Tuesday.

In addition to making peace with the scientific community, the WHO’s recognition could change public-health officials’ disease-prevention advice, especially as more indoor spaces reopen to the public. That could mean new standards for indoor ventilation, for example, or mandates for wearing face masks inside stores, restaurants and other public facilities.

The WHO’s new scientific brief also addressed another COVID-19 controversy: the role asymptomatic people play in spreading the virus.

Van Kerkhove sparked a flurry of confusion last month, when she said it was “very rare” that people without symptoms of coronavirus infect others—a statement seemingly contrary to months of public-health messaging. Van Kerkhove later qualified her comments, emphasizing that asymptomatic spread is possible but happens with unknown frequency.

The WHO’s new brief echoes her comments. “While someone who never develops symptoms can also pass the virus to others,” it says, “it is still not clear to what extent this occurs and more research is needed in this area.”

By:  Jamie Ducharme

Source: https://time.com/

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This Startup Might Finally Cure Sickle Cell Disease After A Century Of Racist Neglect

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The painful blood disorder, which mostly affects Black people, is just one of thousands of rare diseases that could be cured by Beam Therapeutics’ revolutionary gene editing technology.

Beam Therapeutics CEO John Evans loves rocket launches—and not just the ones that succeed. He makes his team watch all the SpaceX failures, too, when the unmanned rockets explode. “For the first many years, all the rockets crash and there’s a lot of failure along the way, and then suddenly, they start working,” he says.

Gene editing, Evans believes, is on a similar trajectory, poised for a series of successful launches in the years ahead. “The stuff we can do now in genome editing would have seemed like magic five or ten years ago,” he says, as technological advancements suddenly have biotechs competing to cure rare genetic diseases long out of reach.

The first generation of gene editing was Crispr, a technology developed in 2012 that can target and cut sections of DNA like a pair of scissors. Now Cambridge, Massachusetts-based Beam is pioneering Crispr 2.0. Its advancement, known as base editing, works more like a pencil, which can target a single misspelling in the DNA code allowing for much greater precision, says Evans, who is 43. Beam’s technology, which has yet to be tested in humans, could theoretically cure thousands of the genetic diseases caused by single letter misspellings, known as point mutations. Topping the company’s targets is sickle cell disease—a point mutation that predominantly affects Black people and has been neglected through more than a century of racist attitudes.

Beam, which was founded in 2017, went public in February and now has a market cap of $1.5 billion despite having no revenues and losing $95 million over the past year. What the company does have is 12 research programs for 10 different rare diseases, including beta thalassemia, another inherited blood disorder, and two types of blood cancer. None of the treatments have been cleared for clinical trials, but the company hopes to file a slew of applications in 2021.

Sickle cell disease is the most promising opportunity. It’s the most common inherited blood disorder in the United States, affecting about 100,000 people. The disease produces abnormal hemoglobin, the molecule that helps red blood cells carry oxygen throughout the body. While normal red blood cells are shaped like doughnuts, sickle cells look like pointy crescent moons (thus the name), which clog up blood vessels and cut off oxygen supply to the bones and organs, causing “excruciating pain,” explains Dr. Robert Liem, director of the comprehensive sickle cell program at the Lurie Children’s Hospital of Chicago.


With one out of every 365 Black babies born with sickle cell, the disease has an ugly racial history. By the mid-1900s, the presence of sickle-shaped cells in the blood viewed under a microscope were a “marker for race,” says Shawn Bediako, professor of psychology at the University of Maryland Baltimore County, who focuses on stigma in healthcare. “When people who were not Black had sickle cell disease then the societal assumption was that then that person wasn’t completely white,” even though the disease is found in people of many other ethnicities. In the 1960s, the Black Panther Party began to champion the right to health and implemented a national sickle cell screening program in the wake of government inaction.

But racist attitudes and a lack of federal funding still persist today. Sickle cell patients, who can end up in hospital emergency departments with serious pain, waited, on average, 25% longer than general patients and 50% longer than patients with bone fractures to be seen, according to a study in the American Journal of Emergency Medicine. Sickle cell received an average of $812 in federal research funding per person over the last decade, while cystic fibrosis, a lung disease that predominantly affects white people, received more than $2,800 per person, even though sickle affects three times as many people, according to a paper in JAMA Network Open. “If we really have to have this debate about whether or not Black life matters, I think we need look no further than sickle cell and how it’s been treated as a medical condition to indicate that it really doesn’t,” says Bediako.

Sickle cell was the first “molecular disease” discovered, revealing how a change in one single amino acid could disrupt blood and oxygen supply to the entire body. The first drug to treat sickle cell, hydroxyurea, wasn’t approved until 1998, even though the disease has been known in the medical literature since 1910, with three more drugs coming to market since 2017. The only cure is a bone marrow transplant, which is limited to a small percentage of patients who have a matching sibling donor. But Beam is hoping to change that with its potentially curative base editing technology. “We’re going to go in and land our editor right on the mutation that causes sickle cell and change it to something that’s normal,” Evans says.

Base editing was pioneered in 2016 in the lab of David Liu, a core faculty member at the Broad Institute and a professor at Harvard University, along with postdoctoral fellows Alexis Komor, now an assistant professor at the University of California San Diego, and Nicole Gaudelli, the head of gene editing technologies at Beam. Liu cofounded Beam in 2017 along with Feng Zhang, a professor at the McGovern Institute at the Massachusetts Institute of Technology and core faculty member at the Broad Institute, and Dr. J. Keith Joung, endowed chair in pathology at Massachusetts General Hospital and professor at Harvard Medical School. This is the second of three gene editing companies the scientific power trio has founded since 2013, along with Cambridge-based Editas Medicine, which is developing Crispr-based therapeutics, and Durham, North Carolina-based Pairwise Plants, which is using both Crispr and base editing to develop more nutritious crops. In 2019, Liu founded Prime Medicine, a third generation “search and replace” genome editing technology, which he likens to a word processor.


“I certainly, in my wildest dreams, never imagined this kind of precise capability to edit the genome,” says Dr. Francis Collins of the National Institutes of Health.


Liu calls the human genome the “most important gift your parents ever gave you.” It’s made up of 6 billion combinations of four letters known as bases: A, T, G, and C. A person with sickle cell disease has one base pair misspelling at a crucial location in their adult hemoglobin gene: a ‘T-A’ where there should be an ‘A-T’. That typo, which appears twice among the 6 billion letters, is the difference between normal hemoglobin and the abnormal hemoglobin that causes the rigid crescent-shaped cells.

Beam is the first company to try to directly fix the base pair misspelling, though they can’t yet switch a T to an A. Instead, Beam switches the T to a C and the A to a G. While it appears odd to swap one typo for another, the new typo mimics a natural phenomenon, known as the Makassar variant, which results in functional red blood cells instead of the sickle shape.

Beam is also trying another approach to curing the disease: Introducing a second mutation in a different location to override the production of sickle hemoglobin. It mimics a naturally occurring phenomenon in which a person has two sets of sickle hemoglobin genes, but doesn’t show signs of the disease. The reason? A different mutation in the fetal hemoglobin gene, which usually turns off in favor of adult hemoglobin as people age, but remains turned on and producing normal hemoglobin. “They won the genetic lottery after losing it twice,” says Liu. Both programs showed promising results in mice.

Liu, 47, prefers to focus on his academic and research pursuits, rather than commercialization of the technologies, as do his cofounders Zhang and Joung. The trio like to hire well-credentialed executives to run their companies, like Evans, who has a track record of successfully bringing precision medicines from the lab to the market.


In 2009, Evans, who earned an MBA from Wharton and a Masters in biotechnology from the University of Pennsylvania, was an early employee at Agios Pharmaceuticals, then a tiny biotech with a pre-clinical target related to cancer metabolism. The following year he helped broker a unique alliance between Cambridge-based Agios and big biopharma Celgene, ultimately leading to two FDA approvals for treatments for acute myeloid leukemia in the span of ten years, which sounds like a long time, but is much faster than the usual drug development timeline.

“Celgene, gave us $130 million dollars upfront to explore this area of biology and Agios was able to preserve commercial right, and many other important features, so it was a very creative deal,” says David Schenkein, the former CEO of Agios and a general partner at Mountain View, California-based venture firm GV (formerly known as Google Ventures), which invested in Beam.

After eight years at Agios, Evans left to become a venture partner at Arch Venture Partners, an early investor in Beam. In late 2016, he met Liu in his office in Cambridge to discuss the company, which was in stealth mode at the time. “I couldn’t sleep that night, I was so excited about it,” Evans recalls.

Since base editing is a platform, rather than a single drug, once the technology works in one disease, it will likely work in others. “That ease of retargeting is going to mean that as we get it up and running, we can very quickly go through and treat a whole bunch of diseases and create a kind of sustainable flow of new medicines,” says Evans. He started in an interim CEO role at Beam and officially took the top job in January 2018.

Beam can’t attack every disease so Evans has pursued strategic partnerships and licensing agreements with other gene editing companies, including Editas, Prime and another Cambridge-based biotech called Verve, which is using base editing to develop heart disease therapies. Joung is a Verve cofounder, and Evans serves on the boards of Prime and Verve. “It’s kind of a divide and conquer approach, where we’re reducing redundancy and now I think more diseases, more patients are potentially going to benefit from the technology than otherwise,” Evans says.

Of course, it’s impossible to predict whether Beam’s technological edge with base editing will ultimately prevail over earlier Crispr technologies that have the first-mover advantage, says David Nierengarten, a biotechnology analyst at Wedbush Securities. But what sets Beam apart so far based on their work in mice is “more efficient” gene editing, meaning that “higher numbers of modified cells” will get into patients, says Nierengarten.


Small Targets

Beam is researching a number of other rare diseases it hopes to treat, or even cure, with its precision gene editing technology. Here are the seven it has disclosed.

Sickle Cell Disease

Estimated U.S. Patients: 100,000

Inherited blood disorder causes severe pain.

Beta Thalassemia

Estimated U.S. Patients: 1,000-2,000

Inherited blood disorder causes severe anemia.

T-Cell Acute Lymphoblastic Leukemia

Estimated U.S. Patients: 500-1,000 per year

Fast-growing blood cancer.

Acute Myeloid Leukemia

Estimated U.S. Patients: 20,000 per year

Fast-growing blood cancer.

Alpha-1 Antitrypsin Deficiency

Estimated U.S. Patients: 60,000

Inherited disorder causes lung and liver disease.

Glycogen Storage Disoder 1a

Estimated U.S. Patients: 1,400

Inherited disorder where body can’t store sugar.

Stargardt Disease

Estimated U.S. Patients: 5,500

Inherited eye disorder causes progressive vision loss.


Beam has one key advantage: with its technology, the DNA double helix doesn’t need to be cut, like with first generation Crispr technologies. This means greater precision with less risk of random insertions and deletions of code.

Beam’s true test will come next year, when the company plans to apply for authorization from the U.S. Food and Drug Administration to begin clinical trials in humans. Even in these early stages, Dr. Francis Collins, director of the National Institutes of Health, the $41.7 billion (2020 budget) federal research agency, says he’s “really excited” about the potential of base editing to take on the 7,000 rare diseases caused by DNA misspellings.

“By delivering this kind of base editor to the right tissue at the right time, you can imagine many of these [rare diseases] becoming treatable, maybe even curable,” says Collins, who is working with Liu on an NIH-funded research project using base editing (currently in mice) to correct the point mutation for progeria, a disease that causes children to rapidly age and die by the time they are teenagers. “I certainly, in my wildest dreams, never imagined this kind of precise capability to edit the genome, where you could go and find one letter out of 3 billion that needed to be fixed, and provide the apparatus to do that with relatively little risk of causing trouble elsewhere,” Collins says reflecting on his career and the rapid progress in gene editing over the past few years.

But one of the big technical challenges ahead for many diseases will be refining the delivery methods of successfully getting base editors into the patient’s body. While the sickle cell therapy can be done outside the body and inserted, for other diseases, like progeria, the base editor will have to be directly inserted into the patient, and there are several methods being developed. “You have to come up with a delivery system that is going to take that base editing apparatus and efficiently and safely get it to the cells where it needs to do its magic,” says Collins. “And that’s a big challenge.”

The other hurdle on the horizon will be affordability. Existing gene therapies tend to be outrageously expensive. Novartis, for instance, made headlines in 2019 for charging $2.1 million for Zolgensma, a breakthrough cure for spinal muscular atrophy. The majority of adult sickle cell patients don’t even have access to hydroxyurea, which is the cheapest generic treatment on the market. “We just have a long way to go before we can realistically say that a substantial number of patients might benefit from these therapies,” says Dr. Liem, who chaired the American Society of Hematology clinical practice guidelines on sickle cell disease. While gene therapies are exciting, “the vast majority of patients out there still need basic care,” says Liem.

But Evans hopes Beam can fundamentally change the rules of engagement. “We’re going to have profound impacts for patients’ lives in the very near future,” he says, since patients who participate in phase one could potentially leave the trial cured of sickle cell. “That’s the amazing thing about these one-time therapies.”

This post was updated at 11:30 am ET July 10 to incorporate two additional co-inventors of base editing.

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I am a staff writer at Forbes covering health care. I was previously a health care reporter for POLITICO covering the European Union from Brussels and the New Jersey Statehouse from Trenton. I am a Knight-Bagehot Fellow in business and economics reporting at Columbia University. Email me at kjennings@forbes.com or find me on Twitter @katiedjennings.

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Nearly Half of Coronavirus Spread May Be Traced to People Without Any Symptoms

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One of the more insidious features of the new coronavirus behind COVID-19 is its ability to settle into unsuspecting hosts who never show signs of being sick but are able to spread the virus to others.

In a study published June 3 in the Annals of Internal Medicine, researchers at the Scripps Research Translational Institute reviewed data from 16 different groups of COVID-19 patients from around the world to get a better idea of how many cases of coronavirus can likely be traced to people who spread the virus without ever knowing they were infected. Their conclusion: at minimum, 30%, and more likely 40% to 45%.

Such so-called asymptomatic spread is unique for a respiratory virus; most cause symptoms and disease once they infect their hosts. SARS-CoV-2, the virus responsible for COVID-19, is, however, particularly wily because it can also infect hosts silently and use them as unwitting pawns in its infectious campaign. “The range we found is extraordinarily high,” says Dr. Eric Topol, director and founder of the Institute and one of the authors of the paper. “That means the range of what can happen with SARS-CoV-2 is from no symptoms to [death]. That’s not at all similar to any virus or pathogen we’ve experienced that has killing potential in the past. What we have here is an extraordinary spectrum, including this quiet, stealth mode of infecting somebody.”

Topol and his co-author, Daniel Oran, searched for published and unpublished studies that included asymptomatic people and focused on 16 different groups of people screened or tested for COVID-19 around the world. Among others, these included a cohort of more than 13,000 people in Iceland who volunteered to be tested for COVID-19 (the largest group included), as well as residents of Vo, Italy; passengers on the Diamond Princess cruise ship where an outbreak occurred; visitors to homeless shelters in Boston and Los Angeles; prison inmates; college students; and nursing home residents in King County, WA.

Five of these studies included follow up testing of the participants; they showed that only a small fraction of people who were asymptomatic when they tested positive the first time then went on to develop symptoms. That allowed the researchers to distinguish between people who are pre-symptomatic—those who test positive but eventually go on to develop symptoms—and those who are truly asymptomatic, and test positive for COVID-19 but never develop obvious symptoms. Among the more than 2300 people sampled in the Vo population, none of the 41% who had no symptoms when they tested positive ever developed symptoms over a 14 day period.

That wouldn’t be worrisome if it weren’t for other research beginning to show that levels of virus in people who are asymptomatic are similar to those among people who develop symptoms. That suggests that while they may not be outwardly showing any signs of illness, asymptomatic people are still carrying a potentially dangerous burden of infectious virus that they can spread to others.

Another concern, says Topol, is that the virus may be damaging the bodies of asymptomatic in other, silent ways. Among the 331 passengers on the Diamond Princess cruise ship who tested positive but did not have symptoms, 76 people had CT scans of their lungs and nearly half showed signs of lung tissue damage typical of coronavirus infection. “People who are getting infection without symptoms are actually doing a lot of damage to their bodies and they don’t know it,” says Topol. Another small study in South Korea that studied 10 asymptomatic people from a group of 139 COVID-19 patients supports these findings.

Putting all of the data together, he says, supports “basically the reason why we have to all wear masks—because nobody knows who is an asymptomatic carrier. The person doesn’t know it and the person’s contacts don’t know it. That has enormous implications and it’s an area we need to study more, on how to test people without symptoms on a very large scale to understand these people better and follow them to determine precisely their ability to transmit [the virus].”

Given that public health officials aren’t testing the entire population, there are still huge gaps in understanding what asymptomatic disease, he says. Are people infected but not showing symptoms because their immune systems are better at controlling the virus, or because the virus they harbor is somehow less potent? Or are these people asymptomatic because they have immunity to other, more prevalent coronaviruses that are responsible for the common cold and therefore already might already have a level of protection against SARS-CoV-2 as well?

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Another question that the data raise involves how long asymptomatic people are infectious. In Topol’s analysis, the cases from U.S. aircraft carrier U.S.S. Theodore Roosevelt suggests that they may be able to spread the virus for longer than the presumed 14 days, which would have wide-ranging implications for public health policies focused on reopening cities and states safely—and further support the need for wearing masks in public settings.

While widespread testing of populations would be a way to capture more of these asymptomatic cases, and help public health officials to educate these people about the need for social distancing and other measures to prevent the spread of the virus, Topol says there’s a need for other options as well. He and his team are studying changes in heart rate that could be captured on smartwatch apps and fitness bands and might signal possible infection. Such changes may not be useful on an individual level, since heart rate changes can be attributable to a number of different factors including stress and heart disease. However, if, for example, resting heart rate levels for a specific community rise and remain high for a period of time, that could indicate a possible COVID-19 cluster and flag individuals and their doctors to increase testing and follow up care in the community.

“If even a portion of the 100 million Americans who have a smartwatch or fitness band are involved, then we could go in and do studies for information we are missing now—antigen testing, antibody testing and we can look for transmissibility,” says Topol. “The priorities during a pandemic are absolutely to look after the sick. But we also shouldn’t miss how important this area of asymptomatic spread is to understand. For every one person who is sick, there are a whole lot of people who have the virus and don’t know it.”

By Alice Park

Source: http://www.time.com

The World Health Organization is walking back a comment suggesting that the spread of COVID-19 from an asymptomatic person is rare. Dr. Ashish K. Jha, director of the Harvard Global Health Institute, joins CBSN to discuss when patients are the most contagious, and a new Harvard Medical School study which suggests the coronavirus may have been in China as early as August.

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