Category Archives: Human Genetic Engineering

When Amber Freed first told doctors her baby boy wasnt able to move his hands, they said that wasnt possible.

Freed had given birth to twins in March 2017. While her baby girl, Riley, squirmed and babbled and crawled through the first year of her life, her fraternal twin, Maxwell, was different. He didnt crawl or babble like Riley did. I would fill out their baby books each month, and Riley had met all of these milestones. Maxwell didnt reach one, she said. Most alarmingly, however, Freed noticed that he never moved his hands.

She knew the news was going to be bad when they sent her to the sad room at the hospital, a featureless conference space filled with grim-faced doctors, to hear the diagnosis.

You take your baby to the doctor and you say, He cant move his hands. And they look at you and they say, Of course he can, said Freed.

Then they look for themselves, and you can see from the look on their faces that they have never seen anything like this.

On June 14, 2018, at the Children's Hospital Colorado in Denver, Maxwell was diagnosed with a genetic disease called SLC6A1. The diagnosis explained why the infant hadnt moved his hands or learned how to speak for the first year of his life, while Riley was thriving. But it didnt explain much else: All the doctors who diagnosed Maxwell knew about the genetic disease came from a single five-page study published in 2014, the year of its discovery. It was too rare to even have a name, she was told, so the doctors just called it by the name of the affected gene: SLC6A1.

Now her 2-year-old son is at the center of a multimillion-dollar race against time, one thats come to include genetics researchers whom Freed personally recruited, paid for by $1 million that Freed and her husband, Mark, have raised themselves. At the center of their research will be specially crafted mutant mice that Freed paid scientists in China to genetically alter to have the same disease as Maxwell. The four mice are scheduled to arrive stateside next week, but Freed said shes prepared to smuggle them into the US disguised as pets if there are any problems.

In total, Amber and Mark will need to raise as much as $7 million to test a genetic treatment for their child. And unless they can find and fund a cure, SLC6A1 will condemn Maxwell to severe epileptic seizures, most likely starting before he turns 3. The seizures may trigger developmental disabilities for a lifetime, often accompanied by aggressive behavior, hand flapping, and difficulty speaking.

And the Freeds will have to do it largely alone there are only an estimated 100 other people diagnosed with SLC6A1 in the world. This is the rarest of the rare diseases, pediatric geneticist Austin Larson of the Children's Hospital Colorado told BuzzFeed News.

SLC6A1 is just one of thousands of untreatable rare diseases, and the perilous path it has set up for Freed, half science quarterback and half research fundraiser, is one that few parents can follow. My dream is to create a playbook of how I did this for those that come after me, said Freed. I never want there to be another family that has suffered like this.

You can think of SLC6A1 as a vacuum cleaner in the brain, genetic counselor Katherine Helbig of the Childrens Hospital of Philadelphia, told BuzzFeed News. Helbig will speak at the first conference on the gene at the American Epilepsy Society meeting in Baltimore on Dec. 5, an effort organized by Freed.

The protein made by the gene acts as a stop sign to message-carrying chemicals in the brain, halting them by vacuuming them up once they reach their destination brain cell, Helbig explained.

When one of the two copies of the SLC6A1 gene in every brain cell is damaged, like in Maxwells case, too little of its protein is available to perform its vacuuming duties, leading to miscommunication between cells, developmental disorders, autism-like symptoms, and, often, severe epileptic seizures.

Maxwell is about the age when epileptic seizures typically start in kids with the genetic disease, said Helbig, adding, There probably are many more children out there who have it, but they just havent had the right test to find it. At least 100 similar genetic defects cause similar kinds of epilepsy, afflicting about 1 in 2,000 kids, she said.

I was the one who presented this diagnosis to Amber, said Larson of the Children's Hospital Colorado. There was no medicine or diet or any other treatment for SLC6A1. It wasnt an easy conversation. Most of the time when we present a diagnosis for a genetic condition, there is not a specific treatment available.

At that moment, it was just vividly clear that the only option was for me to create our own miracle, said Freed. Nobody else was going to help.

Half the battle with a rare genetic disease is getting researchers interested, said Helbig.

At that moment, it was just vividly clear that the only option was for me to create our own miracle. Nobody else was going to help.

So that is what Freed set out to do. She quit her job as a financial analyst and started making phone calls to scientists, calling 300 labs in the first three months. For those who didnt respond, she sent them snacks via Uber Eats.

Her search, and a rapid-fire education on genetic diseases, led her to conclude the best hope for helping Maxwell was an experimental technique called gene therapy.

All the roads zeroed in on one scientist: Steven Gray of the University of Texas Southwestern Medical Center in Dallas. In 2018, a team headed by Gray reported the first human experiments of gene transfer by spinal injection, conducted in 5 to 10 children with mutations in a gene called GAN that causes swelling in brain cells.

The GAN gene transfer in that experiment, first tested in mice, attached a corrected version of the damaged gene to a harmless virus. Viruses reproduce by infecting cells and hijacking their DNA machinery to reproduce their own genes, making more viruses. The gene therapy virus in turn leaves behind a corrected gene in the DNA of cells they infect. Injected into the spinal cord, Grays virus can travel straight to the brain, leaving behind the corrected gene after the virus has run its course.

I gave him my 30-second equity analyst pitch. I told him why Maxwell was a good patient, that we would raise $4 million to $7 million, and quarterback every step of the research, she said. And it worked. He agreed to make it a priority if we could raise the money.

The SLC6A1 researchers with the Freeds at a science meeting. From left: Terry Jo Bichell, Frances Shaffo, Amber Freed, Katty Kang, and Mark Freed.

Less than a month after meeting Gray, Freed contacted a lab at Tongji University in Shanghai that was also researching SLC6A1. The lab agreed to develop a mouse with Maxwells specific mutation for less than $50,000, using a gene modification technology called CRISPR that has revolutionized genetic engineering in the lab. CRISPR mice are much more expensive in the US, and this lab had experience with the gene, said Freed.

By July of this year, an experiment with a gene therapy virus that corrects SLC6A1 was tested on normal lab mice, which showed no sign of a toxic response, an encouraging sign. And by September, a line of CRISPR mice with Maxwells exact genetic mutation had been created at Tongji University.

It is the literal mouse version of him, said Freed. Testing a therapy in this mouse is as close as science can get to testing in my son directly.

To pay for all this, Maxwells family started fundraising last November and organized the first medical symposium on SLC6A1 in New Orleans that same month. They opened a GoFundMe account, which has raised $600,000, and held 35 fundraisers, which raised an additional $400,000 by October. In one charity competition, Larson from the Colorado Childrens Hospital, who diagnosed Maxwell, personally helped her raise $75,000.

It is the literal mouse version of him. Testing a therapy in this mouse is as close as science can get to testing in my son directly.

That money is helping to pay for the next step getting the CRISPR mice to Grays lab to test the SLC6A1-correcting virus on them. But its not as simple as putting the mice in a box and shipping them by mail. The mice will be transferred through a lab at Vanderbilt University headed by Katty Kang, an expert on the neurotransmitter disrupted by Maxwells mutation.

Amber is helping us to advance science, and everyone is making this a priority because of the young lives at stake not just Maxwell, but other children this could help, Kang told BuzzFeed News.

Once the four mice arrive, they will spend several weeks in quarantine, be tested to make sure they have Maxwells specific point mutation in the SLC6A1 gene, and breed with normal lab mice to produce generations of mixed-inheritance mice to serve as controls in future experiments. The mutant mice will be closely monitored before they head to UT Southwestern to make sure that they demonstrate the same problems and genetics as human patients with SLC6A1 and can therefore be used in any future clinical trials of gene therapy.

Right now at UT Southwestern, results from a safety test of the gene therapy virus conducted by Grays lab on young, normal lab mice is awaiting publication. If that works out, once the Chinese mice are sent over, they will also receive the gene-correcting virus. His team will see if their symptoms improve and to what extent their brain cells accept the corrected gene.

Maxwell's brain cells seen through a microscope (left), and a sample of his cells in a petri dish.

And then, Freed just needs another $5.5 million. Half a million dollars will go to test the virus in a second SLC6A1 animal model, likely a rat, as another safety step. Two million dollars will go toward creating more of the gene-correcting virus for a human safety study if that proves to be safe. And finally, if all that works out, $3 million will be needed to conduct the experiment on Maxwell and other children next year, following the path of the GAN clinical trial led by Gray.

Its a really horrible realization that the only thing standing in the way of a cure for your 2-year-old is money, said Freed.

Freed acknowledges that she has only been able to pursue a cure for Maxwell because her family has the resources to do so which she would never have had growing up in small towns in Texas, Montana, and Colorado in a poor family affected by alcoholism. I grew up visiting my parents in rehab and knew what to say to put a family member on a 72-hour psychiatric hold by age 12, she said. She dug herself out to build a career in finance, and hoped her kids would never have to experience the struggles she did growing up.

Even so, the fight hasnt been easy on them or on Maxwells sister, Riley.

Freed worries her daughter is growing up in doctors' waiting rooms, waiting on treatments for her brother to end. Maxwells disease has progressed, causing him to constantly clench his fingers, and sometimes pull his sisters hair. His 3-year-old sister will gently remind him, Soft hands, Maxie.

Families like the Freeds are at the forefront of efforts to turn diagnoses of rare genetic ailments, which often used to be the stopping point for medicine, into treatments. A similar case saw the family of a 3-year-old girl, Mila Makovec, raise $3 million for gene therapy to cure her Batten disease, a deadly genetic brain disease that affects 2 to 4 of every 100,000 children born in the US.

In a New England Journal of Medicine editorial on that case published in October, FDA officials questioned how high the agency should set the safety bar for such treatments, meant for severe diseases affecting so few people. In these cases, parents are often collaborators in developing treatments, and might not want to stop efforts that come with high risks. Even in rapidly progressing, fatal illnesses, precipitating severe complications or death is not acceptable, so what is the minimum assurance of safety that is needed? wrote senior FDA officials Janet Woodcock and Peter Marks.

This is way beyond what anyone expects of families.

Finally, Woodcock and Marks wrote, finding sustainable funding for such interventions may prove challenging, because the cost of production can be quite substantial, particularly for gene therapies.

In our era of financial inequality, the specter of wealthy parents buying custom genetic treatments for their childrens ailments while other parents desperately resort to GoFundMe accounts, or else do nothing looms as a possibility.

This is way beyond what anyone expects of families, said Larson. The pathway has been opened up by the brave new world of improved genetic diagnoses, and the coming of age of rapid genetic engineering tools like CRISPR.

But only 20 years ago, an experimental gene therapy that relied on a harmless virus killed an 18-year-old volunteer, Jesse Gelsinger, in a research misconduct case that brought gene therapy to a standstill. Now more than 2,500 gene therapy clinical trials have been conducted, and more than 370 are underway. The human genome was not sequenced until 2000; today, mapping an entire human gene map costs around $700. In this new era, customized treatments for rare genetic diseases like Maxwells are suddenly possible.

What I hope is that we are paving the way for other parents to help their children, said Freed.

Families of children with rare genetic diseases are also working together to make treatments like the one Freed is spearheading possible, said Larson.

They support each other and work together, he said. The best example might be the families of children with cystic fibrosis, who through the Cystic Fibrosis Foundation and the discovery of the gene responsible for the disease in 1989 have pushed for the discovery of new drug treatments. In October, the FDA approved a breakthrough pharmaceutical that could treat 90% of cases.

It is easier working with FDA on this kind of approach rather than starting from scratch, Gray told BuzzFeed News by email. After all, he said, its easier to follow a path that youve already walked down.

Similarly, Freed hopes the SLC6A1 Connect advocacy group she started can lead to similar treatments for other children with genetic epilepsies caused by the gene.

I dont think any parent should be expected to single-handedly cure his or her childs rare disease, said Helbig. Amber is a very tenacious and persistent person, and she will fight tooth and nail for her kids. But a lot of people dont have the resources and they shouldnt have to.

Helbig says that cautious optimism is appropriate on the chances of research yielding a genetic therapy for children like Maxwell. For SLC6A1, its really too early to say whether this is going to work.

But if it works, it might lead many more parents to get genetic tests for children that will reveal undiagnosed problems, she said. Many doctors discourage extensive genetic tests, thinking they wont find anything helpful. In the absence of known treatments, insurers are also reluctant to pay for such tests, discouraging all but the most fortunate and resourceful parents. Even for them, there are no guarantees.

The other tough reality is the possibility this treatment wont be completed in time to help Maxwell, said Freed. I love him with every ounce of my being, and I want him to know that I did everything humanly possible to change his outcome.

Read more:
This Mom Is Buying Mutant Mice From China To Find A Cure For Her Sons Rare Genetic Disease - BuzzFeed News

A conversation about why outsourcing the moral responsibilities of AI is not an option. Miguel Luengo, AI expert, entrepreneur and chief scientist at UN Global Pulse, speaks with Konstanze Frischen, Ashokas global leader on Tech & Humanity. (Full bios below.)

Miguel, you have spent a lot of time examining the AI & Ethics landscape. What did you find?

Konstanze Frischen: In the AI field, there is a lack of consideration for some of the core principles that went into constitutions around the world and inspired the Universal Declaration of Human Rights and the Sustainable Development Goals (SDGs). When you look at what principles most corporations, think tanks, or governments propose should underpin AI, youll see there is overwhelming emphasis ontrustworthy AI AI that is transparent, reliable, non-biased, accountable, safe, etc. This is indeed a necessary condition, but it basically means technology working as it should. And I am thinking: this is great, but its not enough.

Why not?

Take genetic engineering: We all like to have genetic engineering that is trustworthy in that it works as it should; but that doesnt imply it is okay to copy and paste pieces of genome to create chimeras. Same with AI.

What then?

I argue that we need to move to a humanity centric AI. If AI is a real game changer, we musttake into considerations the implications of AI for humanity as a whole - at present, and in the future. I call that solidarity. Yet, only 6 out of 84 ethical AI guidelines examined (by the Health Ethics & Policy Lab from the University of Zurich) mention solidarity as a principle.

Miguel Luengo, social entrepreneur and chief data scientist at UN Global Pulse.

How do you define solidarity in the AI context?

We need to a) share the prosperity and burden of AI, which implies the re-distribution of productivity gains, and collaboration to solve global problems caused by AI; and b) we must assess the implications that AI has on humanity long-term before developing or deployingAI systems. In other words, solidarity lives in the present and also is strategic long term thinking for the future.

How do we share prosperity?

For instance, by giving back the productivity gains that stem from AI, literally. We can look at it from two perspectives.One is that we share directly with the ones who inputteddataand actionsto create and train the AI models.Currently, the norm is that they dont benefit financially in the outcome. But what if the data didnt belong to the company supplying a service, butto the people who contributed? Lets assume patients provide data and doctors train an algorithm to detect a disease. Shouldnt they all be compensated each time this algorithm is being used for diagnosis somewhere else? Not in a one-off way, but each time?There could be a royalty model, like it happens in the music industry: each time you play a song, the artist gets remunerated.

And the other way to share prosperity is indirect, i.e. via taxes?

Yes, in my view, at the public level, taxes on AI or automation (i.e. robots) are an interesting option and could be a solution for a deep underlying problem: that these technologies will put many people out of jobs. As Yuval Harari says, we are in danger of creating a new, irrelevant class of people that cant play in the digital economy, and more importantly that is not needed to create wealth. This is especially dangerous with the platform economy system, where the winner takesall. But if all our data is extracted and used and the gain is concentrated in the hands of few corporations, well, then well need to tax the use of AI.

How would we get to an AI economy that works on the principle of solidarity?

The change will happen in overlapping and iterative stages. First, there is awareness: citizens are recognizing that the status quo is not okay - that our data is taken for free and sold. Second, new businesses and initiatives will emerge that will take solidarity principles into account: they will give back to the people who helped them create their AI. Social entrepreneurs and B-corps can pave a way forward here. This alignment with citizens motivations and interests can give them a competitive advantage.They will be the responsible choice. We expect big companies to then turn in this direction. And thirdly, that dynamic can push new regulation. We urgently need AI regulatory frameworks contextualized in each sector like marketing, healthcare, autonomous driving or energy.

This will also enable international coordination to respond when AI fails or spirals out of control.

Absolutely.That is solidarity with humanity. Take deep fakes, for instance. Anyone who is tech savvy can train a machine to automate hate speech. Fake videos are easily made and look 100% real. Imagine its election day in a country with a history of genocide, and thousands of deep fakes on ethnic violence circulate on the internet. That might constitute an emergency where the red lines violating Human Rights are crossed in the digital world and the international community needs to act. Even in seemingly less dramatic instances, the complexities to respond to AI failures can be huge: assume someone finds theres a bias in a widely used AI model that underpins X-ray analysis at scale or manages global telecommunications infrastructure.This finding will require an orchestrated, complex operation to roll that back everywhere.Right now, these digital cooperation mechanisms are not in place.

And the principle of solidarity will require us to develop mechanisms in both case prosperity and burdens.

Correct. The key is thinking far ahead and factoring in the impact of AI, even on future generations.I am concerned that leadership is too short-sighted when it comes to societal and economic implications. For instance, right now very, very few researchers and companies take into account the carbon footprint of their AI efforts.The CO2 implications of AI are huge. It should be the norm that you estimate the CO2 impact of creating and operating your AI model, and weigh it against the perceived benefits. Is it reasonable to waste incredible amounts of energy to teach a machine to spot pictures of cats and dogs on the internet?Not just solidarity, butsustainabilityshould also be a core principle for the development of AI. And the principles are just the frame. AI must be applied beyond internet businesses and marketing. There is a global untapped market for AI to accelerate the attainmentof the SDGs.

What do you think are some of the perhaps less-obvious obstacles that prevent a change towards thinking long-term about solidarity and sustainability in the tech industry?

Part of the problem is that people work in silos. Most of the time, discussions around ethical principles of AI and their practical implementations are made by lawyers and ethicists, or business people or technical experts separately. And they do not speak the same language, so a lot is lost in translation. Ive seen meetings with many high ranking policy makers talking about AI with expert technologists and business leaders; and the higher up you go the clearer it often is that theres a lack of real understanding that prevent people on either side to put all the pieces together. We need a new generation which deeply understands the technical details and societal and economic implications of AI.

It seems employeesof big tech companies are increasingly demanding that the industry takes more responsibility for their actions. Im thinking for instance of the letter thousands of employees signed that got Google to abandon a plan to provide AI for drone analysis to the Pentagon.

Yes, and that trend will grow. Top talent is starting to choose to work in companies whose values and impact make the world better. We cannot outsource the moral responsibilities of the technology we develop.Its time to be clear: Those who develop scalable technology, we need to think upfront about the potential risks and harms and take a precautionary approach if needed. And ultimately, we must be held accountable forthe consequences of using that technology at scale.

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Next Now: The 21st century has ushered in a new age where all aspects of our lives are impacted by technology. How will humanity anticipate, mitigate, and manage the consequences of AI, robots, quantum computing and more? How do we ensure tech works for the good of all? This Ashoka series sheds light on the wisdom and ideas of leaders in the field.

Dr. Miguel Luengo-Oroz isa scientist and entrepreneur passionate about technology and innovation for social impact.As the first data scientist at the United Nations, Miguel has pioneered the use of artificial intelligence for sustainable development and humanitarian action. He is the Chief Data Scientist at UN Global Pulse, an innovation initiative of the United Nations Secretary-General. Over the last decade, Miguel has built teams worldwide bringing AI to operations and policy in domains including poverty, food security, refugees and migrants, conflict prevention, human rights, economic development, gender, hate speech, privacy and climate change. Miguel is the founder of the social enterprise Spotlab, which uses mobile phones, 3d printing and AI for diagnosis of global health diseases. He is the inventor of Malariaspot.org videogames for collaborative malaria image analysis, and is affiliated with the Universidad Politcnica de Madrid.He was elected anAshoka Fellow in 2013.

Konstanze Frischen is the global leader for Ashokas emerging focus onAI,technology, and ethics. A journalist,entrepreneur and social anthropologist, among other things, she was one of the key actors introducing social entrepreneurship to Western Europe, founded Ashoka in her native Germany and co-led Ashoka in Europe to build up the largest network of social innovators. She is a member of the organizations global leadership group and based in Washington, DC.

Read more:
AI, Solidarity And The Future Of Humanity - Forbes

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The central dogma of biology describes the flow of genetic information from DNA to RNA to proteins. While RNA was originally believed to be a carrier of genetic information, subsequent work has shown something completely different: RNA is now known to have function independent of proteins, with a rich layer of regulatory networks. In fact, a large amount of the RNA present in a cell does not actually make proteins. This increased appreciation and understanding has led to many fascinating mechanistic insights into RNA and its role as a central player in cellular regulation and human disease.

Helping to facilitate RNA function are a large number of proteins that can bind to and regulate RNA. These RNA-binding proteins, or RBPs, number in the thousands and are made up of many different independent modular segments similar to a childs set of building blocks. In much a similar fashion, these blocks or domains provide nature with a way of mixing and matching different domains to generate new functions. In recent years, researchers have sought to learn from biology and use these building blocks to engineer new proteins with unique functions that are helpful in research and human health. In a recent study published in WIREs RNA, Professor Andrew Berglund and his colleagues describe recent advances and challenges in engineering RBPs.

Engineering [RBPs] is a powerful tool for researchers to probe the mechanisms of RNA processing pathways says Dr. Andrew Berglund, the newly appointed Director of the RNA Institute at the University at Albany. It is also a promising approach for the development of novel therapeutic molecules.

Potential targets for this approach are abundant as many human diseases have a strong RNA or RBP component, including the most common cause of muscular dystrophy and amylotrophic lateral sclerosis. For therapeutic purposes, engineered RBPs can be designed to replace a defective cellular RBP or bind and destroy toxic RNA. RBPs can also be engineered with new functions and/or targets as well as being marked or tagged so that the researcher can follow their progression within the cell, like a GPS tracker for RNA. The modular nature of RBPs makes it possible to add or mix function to suit the goal of almost any researcher.

In their study published in WIREs RNA, the research team highlights two specific types of RBDs domains, PPR and PUF domains, which are the most straightforward choice for protein engineering. Researchers have studied these domains, understand how to design them to bind specific RNA sequences, and even have websites that can be used to design a domain to target your RNA of choice. Not all RBP engineering is this straightforward, with most researchers having to consider other factors such as the type of linker between domains, where in the tissue or the cell that protein must go, and how to attach other domains to give the engineered protein function.

Ultimately, as more functions of RNA are discovered and more diseases are linked to RNA misregulation, the greater the importance will be for designing, engineering, and testing novel RNA binding proteins. Somewhere in a jumble of RBP building blocks may lie the key to unlocking the next big discovery on RNA and potentially the next generation of therapeutics to improve human health.

Kindly contributed by the authors.

Follow this link:
Engineering RNA Binding Proteins to Improve Human Health - Advanced Science News

DUBLIN--(BUSINESS WIRE)--The "Global Vaccines Market Analysis 2019" report has been added to ResearchAndMarkets.com's offering.

The Global Vaccines market is expected to reach $71.04 billion by 2026 growing at a CAGR of 8.7% from 2018 to 2026.

Factors such as rise in prevalence of diseases, increasing government initiatives towards immunization and increasing number of prospects from the developing economies are driving the market growth. Though, high cost of development and complexities related to manufacturing are projected to inhibit the growth of the market. Moreover, high growth prospects in emerging markets may provide ample opportunities for the market growth.

By technology, recombinant vaccines segment acquired significant growth in the market owing to less side-effect of these vaccines in comparison to conventional ones. They are largely used in animals for prevention of diseases such as pneumonia, foot and mouth disease, septicaemia and pox disease that will further support the business growth. Developments in the field of molecular biology and genetic engineering will positively impact the growth of market.

The key vendors mentioned are Emergent Biosolutions, Glaxosmithkline, Pfizer, Sanofi Pasteur, Merck, Medimmune, LLC (A Subsidiary of Astrazeneca), CSL Limited, Serum Institute of India, Johnson & Johnson, Mitsubishi Tanabe Pharma Corporation, Astellas Pharma, Panacea Biotec, Bavarian Nordic, Biological E and Daiichi Sankyo Company.

Key Questions Answered in this Report

Key Topics Covered

1 Market Synopsis

2 Research Outline

3 Market Dynamics

3.1 Drivers

3.2 Restraints

4 Market Environment

5 Global Vaccines Market, By Patient Type

5.1 Introduction

5.2 Pediatric Patients

5.3 Adult Patients

6 Global Vaccines Market, By Type

6.1 Introduction

6.2 Monovalent Vaccines

6.3 Multivalent Vaccines

7 Global Vaccines Market, By Route of Administration

7.1 Introduction

7.2 Oral Administration

7.3 Intramuscular and Subcutaneous Administration

7.4 Injectable

7.5 Other Routes of Administration

8 Global Vaccines Market, By Indication

8.1 Introduction

8.2 Foot and Mouth Disease

8.3 Respiratory Syncytial Virus (RSV)

8.4 Cancer

8.5 Cholera

8.6 Human Papilloma Virus (HPV)

8.7 Influenza

8.8 Diphtheria, Pertussis, and Tetanus (DPT)

8.9 Meningococcal Disease

8.10 Hepatitis

8.11 Varicella

8.12 Herpes Zoster

8.13 Measles, Mumps, and Rubella (MMR)

8.14 Dengue

8.15 Rotavirus

8.16 Pneumococcal Disease

8.17 Polio

8.18 Disease

9 Global Vaccines Market, By Technology

9.1 Introduction

9.2 Attenuated Vaccines

9.3 Recombinant Vaccines

9.4 Inactivated & Subunit Vaccines

9.5 Toxoid Vaccines

9.6 Live Attenuated Vaccines

9.7 Conjugate Vaccines

10 Global Vaccines Market, By Distribution Channel

10.1 Introduction

10.2 Retail Pharmacies

10.3 Institutional Sale

10.4 Hospital Pharmacies

11 Global Vaccines Market, By End User

11.1 Introduction

11.2 Pediatric Vaccines

11.3 Traveler Vaccines

11.4 Adult Vaccines

10 Global Vaccines Market, By Geography

10.1 Introduction

10.2 North America

10.3 Europe

10.4 Asia Pacific

10.5 South America

10.6 Middle East & Africa

11 Strategic Benchmarking

12 Vendors Landscape

12.1 Emergent Biosolutions

12.2 Glaxosmithkline

12.3 Pfizer

12.4 Sanofi Pasteur

12.5 Merck

12.6 Medimmune, LLC (A Subsidiary of Astrazeneca)

12.7 CSL Limited

12.8 Serum Institute of India

12.9 Johnson & Johnson

12.10 Mitsubishi Tanabe Pharma Corporation

12.11 Astellas Pharma

12.12 Panacea Biotec

12.13 Bavarian Nordic

12.14 Biological E

12.15 Daiichi Sankyo Company

For more information about this report visit https://www.researchandmarkets.com/r/y1y6vk

See the article here:
Global $71Bn Vaccines Market Review 2016-2019 and Forecast to 2026 - ResearchAndMarkets.com - Business Wire

By Geoffrey Kabat

Originally published asKabat, Geoffrey. Whos Afraid of Roundup?Issues in Science and Technology36, no. 1 (Fall 2019): 6473. Reprinted with permission.

In May 2019, a California jury awarded $2 billion to a husband and wife who claimed that the weed-killer Roundup caused their non-Hodgkins lymphoma. The defendant in the suit was Bayer AG, which had recently acquired Monsanto, Roundups manufacturer.

Crucial in determining the judgment was Alameda County Superior Court judge Winifred Smiths denial of a request by Bayers lawyers to share with the jury the US Environmental Protection Agencys recent determination that the active ingredient in Roundup, glyphosate, is not carcinogenic and poses no risk to public health when used as directed. What is the relevance? the judge is reported to have asked.

Instead, the judge allowed the plaintiffs lawyers to base their case on the International Agency for Research on Cancers (IARC) 2015 determination that glyphosate is a probable carcinogen. Deprived of the opportunity to hear any countervailing evidence, the jury found for the plaintiffs.

This was the third Roundup trial, following other cases in which a total of $158 million was awarded to the plaintiffs. At present there are over 18,000 lawsuits in the United States pending against Bayer based on claims that exposure to Roundup was responsible for the plaintiffs cancers.

The stakes are not limited to Bayer and those involved in the lawsuits. They extend to farmers, the agricultural sector of every country, and consumers worldwide who depend on affordable food. And even beyond these impacts, what is at stake is societys ability to rely on the best scientific evidence on questions that are entangled with competing interests and deeply held worldviews.

Roundup, the worlds most widely used herbicide, has been in use for 45 years. By targeting a key enzyme present in all plants, it can kill a wide variety of weeds. Farmers value it because it enables them to manage weeds more easily and more effectively than other products, and because it reduces the need for tillage, thus improving soil conservation. Roundup also has low toxicity compared with products it has replaced, such as atrazine and alachlor (both of which are banned in Europe). A successful campaign to ban Roundup would result in a worsening of soil quality and deny farmers a crucial tool for controlling weeds, confronting them with the choice between a return to using more harmful herbicides or experiencing major reductions in agricultural productivity for many crops.

THE SOLE DISSENTING VOICE

In view of the prominence given IARC in the legal proceedings, it is noteworthy that the agency stands alone in its conclusion that glyphosate poses a carcinogenic risk. The US Environmental Protection Agencys recent assessment is only the latest in a succession of reports from national regulatory agencies, as well as international bodies, that support the safety of glyphosate. These include Health Canada, the European Food Safety Authority (EFSA), the European Chemicals Agency, Germanys Federal Institute for Risk Assessment, and the Food and Agriculture Organization of the United Nations, as well as health and regulatory agencies of France, Australia, New Zealand, Japan, and Brazil.

How has a chemical that has been exhaustively reviewed by regulatory agencies all over the world and repeatedly found to be safe become a vehicle for a torrent of lawsuits?

To answer this question, the place to start is IARC, which in March 2015 classified glyphosate as a probable carcinogen based primarily on what it termed sufficient evidence in rodent studies. However, revelations by the Reuters journalist Kate Kelland, and documents made public in the Monsanto lawsuits, paint a different picture from that presented by IARC regarding the agencys process in initiating and producing the report and its conclusions.

Unlike virtually all other agencies, IARC engages in hazard assessment rather than risk assessment. This means that IARC considers any scientific evidence of possible carcinogenicity, no matter how difficult to interpret or how irrelevant to actual human exposure. In doing so, the agency ignores a cornerstone of toxicology that states the dose makes the poison. The agencys approach fails to distinguish between exposures as they occur in the real world and far-fetched and improbable scenarios, and this in turn leads to an upward skewing of evaluations in terms of risk. (Unsurprisingly, then, of roughly 500 agents and chemicals evaluated by IARC, only one, caprolactam, a chemical used in the manufacture of synthetic textiles, was found unlikely to be carcinogenic). The problems with the IARC glyphosate classification, however, cannot be explained primarily by the distinction between hazard and risk evaluation.

How has a chemical that has been exhaustively reviewed by regulatory agencies all over the world and repeatedly found to be safe become a vehicle for a torrent of lawsuits?

First, IARC based its probable carcinogen assessment primarily on the results of studies in rodents, because the agency considered the human evidence limited. However, independent analysis (by a former statistician at the US National Cancer Institute, Robert Tarone) of the rodent studies relied on by IARC showed no consistent or robust evidence of increased tumor yields in exposed animals. The IARC Working Group that conducted the assessment selected a few positive results in one sex and used an inappropriate statistical test to declare some tumor increases significant. Comparable inverse associations, some statistically significant, were ignored.

Second, IARC was aware of the availability of relevant results regarding non-Hodgkins lymphoma (NHL) from the large National Cancer Institute-funded Agricultural Health Study (AHS), a prospective study of 54,000 pesticide applicators in Iowa and North Carolina. Although only very early results for glyphosate and NHL from the study had been published when the IARC Working Group met to evaluate glyphosate, a senior investigator on the AHS served as chair of the group. This scientist would have been aware that updated results from the AHS showed no significant increases for NHL from glyphosate exposure.

IARC argues that these results were not included in its 2015 assessment of glyphosate due to its rule that excludes unpublished findings. However, if the objective was to produce a valid assessment of glyphosate, this explanation is inadequate. The characteristics and methods of the AHS were widely known, and the details of the statistical methods used in the analysis of NHL had been described in a 2014 paper. Given that the existence of high-quality results from a large, carefully designed prospective studyprecisely the type of human evidence that regulators most valuewas known to at least one member of the Working Group, IARCs decision to proceed with the report but ignore the existence of the AHS results requires a more forthcoming explanation. Indeed, when the results for glyphosate and cancer incidence in the AHS were finally published in theJournal of the National Cancer Institute, in 2018, the paper reported no significant increases for more than 20 solid or lymphopoietic malignancies, including NHL and several NHL subtypes.

Third, in the past two years other improprieties in IARCs glyphosate assessment have come to light. Kate Kelland, the health reporter for Reuters, examined drafts of the chapter of the monograph devoted to animal studies and found that early drafts more accurately summarized the evidence, whereas later drafts progressively emphasized findings that appeared to indicate a positive association.

Finally, the role played by Christopher Portier in the glyphosate assessment has become apparent in transcripts from litigation involving Monsanto. Portier, an American scientist who had worked for the federal government, chaired an IARC committee that prioritized glyphosate as an agent to be evaluated, and subsequently served as an invited specialist on the Working Group that evaluated glyphosate. Although IARC is hyper-alert to conflicts of interest involving industry, the agency seems not to be concerned about anti-industry bias. Two weeks after publication of the IARC report, Portier signed a lucrative contract to act as a litigation consultant with a law firmLundy, Lundy, Soileau, and Southengaged in bringing lawsuits against Monsanto for Roundup exposure.

In sum, IARCs classification of glyphosate diverged from the conclusions of other agencies worldwide, and the divergence resulted from a flawed assessment of the scientific evidence by the IARC Working Group.

A POWERFUL COUNTER-NARRATIVE

How can a respected scientific agency and its supporters take such a different view of the safety of Roundup/glyphosate from the mainstream?

Although glyphosate spraying has been practiced since 1974, its use has increased almost 15-fold globally since the 1996 introduction of Roundup-ready genetically engineered, glyphosate-tolerant crops. As a result, use of Roundup and cultivation of genetically modified foods have become indissolubly linked not just in agricultural practice but in public debates about genetically modified organisms (GMOs). Indeed, a powerful alliance of groups that oppose agricultural biotechnology has entered the fray concerning the carcinogenicity of glyphosate. These groups are anti-GMO, anti-pesticide, and anti-Big Ag, favoring instead natural farming and organic foods. One prominent organization is US Right to Know (USRTK), funded by the Organic Consumers Association, which advocates for organic agriculture while opposing genetic engineeringas well as, it might be noted, vaccines. USRTK and similar groups, including GM Watch, the Environmental Working Group, Greenpeace, and many others, ignore the enormous body of evidence that demonstrates the benefits of genetic engineering of crops, for example through improved tolerance to drought, increased resistance to pests, and enhanced nutrient content (as in the case of Golden Rice). Now the low-toxicity pesticides that enhance the value of GM crops are in the crosshairs as well. To these groups, IARC represents the sole agency that has not been corrupted by making compromises with industry.

Anti-GM agriculture groups have been waging an all-out campaign on their websites and in social media attacking journalists, scientists, and agricultural experts who defend modern farming and criticize IARC, alleging that they sow misinformation, ignore evidence of risks, and are compromised by conflicts of interest. Their targets have included academic experts Nina Fedoroff of Penn State, Kevin Folta of the University of Florida, Drew Kershen of the University of Oklahoma, Alison Van Eenennaam of the University of California, Davis, and many others (including myself). To counter the activist anti-GMO, anti-pesticide organizations, groups such as the Genetic Literacy Project, the American Council on Science and Health, and the Cornell Alliance for Science see their mission as trying to explain the science and its implications on these contested topics to the public.

What distinguishes the two sides is that the latter groups pay more attention to the quality of the scientific evidence and are interested in gene editing, development of more resilient crop varieties, strategies for reducing pesticide use, and other advances that have the potential to feed more people with fewer chemical inputs using less land. In contrast, the former groups tend simply to assert that there are serious risks associated with genetic engineering of plants and animals and with pesticides, and to tar all who disagree as being associated with agrichemical companies and their front organizations. They dont have to point to any substantive evidence of the implied risks or cover-ups. They dont have to distinguish between solid studies and those that are questionable. All thats needed is to assert that the figures they single out are part of a sinister and corrupt network featuring,as USRTK says, secret financial arrangements and close collaborations between corporations, their PR firms, and supposedly independent academics who promote corporate interests.

In addition, both American and European activists have been lobbying bureaucrats and politicians in the European Union to have glyphosate banned. Christopher Portier and Carey Gillam, a spokesperson for USRTK, have testified before the European Parliament in support of a ban. The European Union provides fertile soil for activists opposed to modern agricultural practices because it has enshrined the precautionary principle as part of its regulatory framework. As explained in a 2017 European Commission document, the precautionary principle allows that regulatory intervention may still be legitimate, even if the supporting evidence is incomplete or speculative and the economic costs of regulation are high. IARC, by declaring glyphosate a probable carcinogen, provides groups such as USRTK the authoritative scientific cover they need to pursue their campaign against Bayer and in support of a glyphosate ban. In California, the IARC findings allow the state to list glyphosate as a carcinogen under its 1986 Safe Drinking Water and Toxic Enforcement Act, better known as Proposition 65, and thus provide an apparent scientific basis for litigation.

More broadly, IARCs flawed assessment both relies on and lends apparent scientific credibility to a variety of powerful beliefs and biases that infect the public discussion of environmental exposures to chemicals such as glyphosate. By bracketing out much of what is known about the causes of cancer and by focusing peoples attention solely on what are trace environmental residues, activist organizations reinforce these beliefs and biases, which seem prevalent enough to merit being labeled memes. From my own work, and building on decades of research by decision scientists such as Paul Slovic, Cass Sunstein, Daniel Gardner, and Peter Sandman, I identify at least four such memes:

In the case of glyphosate, 40 years of science demonstrating the safety of the chemical is quite consistent and is supported not only by industry-affiliated scientists but by independent scientists, including agricultural experts, toxicologists, and regulatory officials who are familiar with pesticide use, as evidenced by the fact that so many regulatory bodies worldwide are in agreement. Why, then, are the attacks on glyphosate in courtrooms and governments succeeding? Part of the explanation of course is that the widely shared memes I cite allow advocacy groups and others skeptical of GM crops and agrochemicals to discount the body of science documenting glyphosates safety and focus entirely on the IARC assessment.

SCIENCE DIVIDED

The more interesting and difficult question is why a substantial number of scientists appear to support the IARC assessment. Indeed,a November 2015 letterto the European Commissioner for Health and Food Safety signed by 96 scientists attacked the European Food Safety Authoritys determination that glyphosate was not carcinogenic, and supported IARCs contrary determination.

But Bernhard Url, the head of EFSA, in an address to his organization, provided a different perspective: People that have not contributed to the work, that have not seen the evidence most likely, that have not had the time to go into the detail, that are not in the process, have signed a letter of support [for a ban on glyphosate]. Sorry to say that, for me, with this you leave the domain of science, you enter into the domain of lobbying and campaigning. And this is not the way EFSA goes.

Its possible to understand why scientists without direct and deep expertise on a specific subject might weigh in through such a letter. Because, of course, scientists are human too. Scientists who have worked with IARC appear to feel a strong loyalty to the institution and rally to its defense, often without appearing to know the details of the substantive criticisms that have been made by outside scientists. But if loyalty to IARC and alignment with its mission can explain the support of IARCs broad base, it is still necessary to explain how the IARC leadership that organized and oversaw the glyphosate review can defend their position. Here, it is difficult to escape the conclusion that there are bigger issues at stake than the narrow interpretation of the evidence regarding glyphosate.

IARCs flawed assessment both relies on and lends apparent scientific credibility to a variety of powerful beliefs and biases that infect the public discussion.

My own belief is that an extreme precautionary approach to evaluating risks is at the root of both the recent conduct of the IARC program to identify human carcinogens and that of IARC-associated epidemiologists who are, it seems, often willing to give weight to evidence from weak observational studies and from other types of studies that appear to point to a risk. It must also be said that being in a position to make authoritative pronouncements about risks that are of public concern is not a negligible source of influence and career advancement. Because of their political or professional stake in the issue, scientists may find particularly credible and draw attention to certain studies that purport to show an association, while ignoring other higher-quality studies. For example, an expert providing testimony for the plaintiffs in one of the Monsanto cases cited crude case-control studies of glyphosate as evidence that exposure is associated with increased risk of NHL, while ignoring the higher-quality findings of the Agricultural Health Study. A recent paper in the journalMutation Researchcombined five small case-control studies with the much larger AHS in a meta-analysis, and, by selecting only the highest of five risk estimates from the AHS, the authors asserted that exposure to glyphosate increased the risk of NHL by 41%. If they had included the other estimates, there likely would have been no risk. One could give many more examples of this kind of selective approach to the evidence.

Of course, other scientists may have biases that push in the other direction, sometimes indeed because their interests or sympathies lie with industry, or with farmers. But thats why scientists representing a variety of institutional perspectives need to be included in any process to assess small environmental risks in large populations using complex statistical tools. And failure to have such representation sets IARC apart from the many other environmental risk assessment bodies that have concluded that glyphosate does not pose a cancer risk.

FACEBOOK SCIENCE IN ACTION

For years, IARC has positioned itself as the voice of independent scientific authority on the carcinogenicity of physical, chemical, and biological agents. When specific assessments of IARC have been questioned or criticized by qualified scientists, the agencys default response has been to assert its preeminent position and its authority, rather than to address the specific substantive criticisms or engage in a discussion of the evidence on its merits. In addition, IARC and its defenders typically argue that any criticism must be motivated by conflicts of interest and subservience to industry. For example, an article published in 2015 in the journalEnvironmental Health Perspectivestitled IARC Monographs: 40 Years of Evaluating Carcinogenic Hazards to Humans, signed by 124 authors, sought to win the public debate by insinuating that critics of IARC have venal motives. Yet the article consistently failed to address legitimate specific points raised by critics.

This pattern of refusing to engage in a discussion of the evidence for its classifications goes back more than 10 years. In the most recent publications of IARC supporters addressing the glyphosate issue, the authors restate yet again IARCs conscientious approach to its mission, focus on alleged questionable behavior by Monsanto, and imply that IARCs critics have conflicts of interest. However, they continue to avoid discussing the evidence and ignore the fact that all other regulatory agencies have found glyphosate to be safe and noncarcinogenic. Nor, with the exception of acknowledging Portiers becoming a litigation consultant immediately after publication of the glyphosate assessment, do they acknowledge any of the other irregularities pertaining to the glyphosate report.

IARCs supporters in the scientific community consistently paint a picture of selfless scientists motivated by protecting public health pitted against powerful corporations aided by compliant scientists and politicians. Quite intentionally, this Manichean picture leaves no room for a discussion of the scientific evidence on its merits. You are either for IARC and science and public health, or you are okay with corporations assaulting public health because they dont care if people get cancer so long as they get profits. There is no middle ground. What needs emphasizing, however, is that the effect of IARCs strategy is to transform a debate about science and evidence into a crusade for moral and political purity against which there can be no defense. In this highly polarized climate, those who see things differently may be reluctant to speak out.

The memes that shape peoplesincluding some scientistsviews on complex issues of risk coalesce, and reinforce and amplify each other, contributing to what the Nobel Prizewinning behavioral psychologist Daniel Kahneman terms an availability cascade a self-sustaining chain of events, which may start from media reports of a relatively minor event and lead up to public panic and large-scale government action. This cycle, he adds, is sometimes sped along deliberately by availability entrepreneurs, individuals or organizations who work to ensure a continuous flow of worrying news. The danger is increasingly exaggerated as the media compete for attention-grabbing headlines. Scientists and others who try to dampen the increasing fear and revulsion attract little attention, most of it hostile: anyone who claims that the danger is overstated is suspected of association with a heinous cover-up.

Under such circumstances, positive studies or assessments, such as IARCs assessment of glyphosate, reinforce the prevailing beliefs and fears, while negative studies or assessments, such as those of the other government organizations that do not reveal a cancer risk, fail to find a receptive audience. The availability cascade has in turn led to the juggernaut of litigation cases against Monsanto/Bayer, each one enacted as a morality play in which a plaintiff with a rare, poorly-understood cancer is pitted against a powerful corporation.

In this case, the availability entrepreneurs include IARC itself, along with some scientists, advocates, plaintiffs lawyers, and nongovernmental organizations with an agenda (and, it should be said, with their own set of financial interests, such as funding from the organic foods industry and manufacturers of green environmental products). Collectively, they spin the evidence for their purposes. The result is what EFSAs Bernhard Url has termed the Facebook age of science. As he put it: You have a scientific assessment, you put it on Facebook, and you count how many people like it. For [EFSA], this is no way forward. We produce a scientific opinion, we stand for it, but we cannot take into account whether it will be liked or not.

The glyphosate controversy may be the most glaring example of Facebook science, but it should come as no surprise that the same factors that are at work here are at work in many other areas, whether electromagnetic fields, cell phone radiation, so-called endocrine disrupting chemicals, numerous aspects of diet, cosmetic talc, GMOs, vaccines, nuclear power, or climate change.

Todays highly interconnected world faces serious problems that are in large part the result of the unprecedented progress that has been made over the past 150 years in science, technology, public health, and nutrition. These problems include, among others, the emergence of new pandemic virus strains and increasing antibiotic resistance; degradation of the environment, leading to loss of habitat and loss of species diversity; the challenge of producing adequate food for a growing population; and the pressing need to transition to a realistic energy policy as part of a response to a changing climate. These challenges will not be met by appeasing activists who seem to believe that the world would be better off today without many of the scientific and technological advances of the past, who exaggerate the risk associated with those advances by misrepresenting the scientific evidence, and who have nothing to offer but the simplistic and moralistic narratives of Facebook science.

I realize that complex issues of risk and the environment create a near-impenetrable thicket of uncertainties, values, interests, and competing experts views of the evidence. But sometimes the clear weight of evidence coupled with a dose of common sense is enough to show whats right, even if that means going against the tide of popular outrage. Glyphosate is a boon to agriculture and humanity. Lets refocus the energy and resources spent on trying to demonize this useful and valuable chemical on problems that really matter.

Geoffrey Kabatserves on the ACSH Board of Science Advisors and is a cancer epidemiologist and the author ofGetting Risk Right: Understanding the Science of Elusive Health Risks.

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Who's Afraid of Roundup? - American Council on Science and Health

XconomyBoston

A cancer tumor is a veritable patchwork of cells with a variety of genetic fingerprints.

Immunitas Therapeutics is using single-cell genomicsan approach that studies the genetic activity of individual cellsto peer deeply into patient tumors and more precisely determine what is fueling the growth.

With that knowledge, the company plans to develop new targets for treating forms of the disease based on what it learns about the interactions between immune cells and cancer cells around tumors.

Now the Boston company has raised $39 million to advance compounds discovered with its computational platform into human testing by the end of 2022.

The startup was founded by venture capital firm Longwood Fund, itself started about a decade ago by a trio of biotechies who worked together at Sirtris Pharmaceuticals through its 2008 acquisition for $720 million by British drug giant GlaxoSmithKline (NYSE: GSK).

Lea Hachigian, a Longwood principal, is president of Immunitas. She told Xconomy that Longwood found out about the platform, which had been developed and in use in the labs of its scientific cofounders for about three years, this winter.

The progress it had madeImmunitas already has multiple potential monoclonal antibody treatments in its pipelineprompted the venture firm to turn the tech into a company.

Treatments for cancer based on the genetic signature of a tumor, known as checkpoint inhibitors, have been able to help many cancer patients who previously had few options for treatment. But those treatments are only relevant for about 15 percent to 20 percent of cancer patients, Hachigian says.

Combination approaches, in which drug developers mix and match some of those therapies, havent proven to be a panacea either.

Those approaches are exciting, but they have been limited so far in what theyve yielded in the clinic in terms of efficacy, she says.

There a bunch of patients who havent been able to benefit from some of these treatments, she saysand those are the people for whom Immunitas is aiming to develop new treatments.

It plans to analyze cells from specific patient subgroups, such as people with a well-defined form of a disease or those who have developed resistance to a certain kind of treatment. The companys technology has also led it to identify biomarkers that it intends to use to guide its selection of patients for clinical trials. The idea is that a drug developed from those samples would be targeted at that group.

It is also looking to set itself apart from other drug discovery efforts by analyzing human samples, avoiding the misleading signals that can be sent by animal tests.

Single cell genomics pioneer Aviv Regev, a computational biologist and core member of the Broad Institute of MIT and Harvard, was an early collaborator on the project.

Hachigian likened the platform to noise-canceling headphones for tumor biology in how it allows researchers to hone in on drivers of tumor progression.

The companys lead program is designed around a target Immunitas discovered by studying a tumor that is resistant to an existing treatment. Since then it has determined the target is overexpressed in other tumor types, too, both liquid and solid.

Hachigian says the companys deep immunology expertise also set it apart from others using single-cell genomics to find cancer drugs. One of its scientific founders, Kai Wucherpfennig, heads the Dana-Farber Cancer Institutes department of cancer immunology and virology. (Its others are Mario Suv, a physician-scientist in the department of pathology at Massachusetts General Hospital; and MITs Dane Wittrup, the Carbon P. Dubbs Professor in Chemical Engineering and Biological Engineering.)

Immunitas isnt the only startup thats looking cell by cell in hopes of making new biological discoveries that lead to treatments. Regev, in fact, is a co-founder of Cambridge, MA-based Celsius Therapeutics, another new company using single cell genomics to advance its drug discovery efforts.

Celsius launched last year with $65 million in Series A funding led by Third Rock Ventures.

In addition to Longwood, two big pharma companies are among Immunitass biggest backers. Its Series A was led by Leaps by Bayer and Novartis Venture Fund, those companies respective venture arms. Other institutional investors in the round include Evotec, M Ventures, and Alexandria Venture Investments.

The company has five full-time employees and is based in BioLabs, an incubator in Kendall Square. By the end of next year, it plans to have added another 10 or so. And the following year, when it projects it will move into human testing, Immunitas plans to tack on perhaps another 10 more employees to fuel its clinical development efforts.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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With Cell-By-Cell Take on Drug Discovery, Immunitas Debuts With $39M - Xconomy