NEW YORK (GenomeWeb) – Guardant Health today announced the Guardant360 assay has been approved by New York State’s Clinical Laboratory Evaluation Program.
The assay is the first liquid biopsy permitted by the program, which Guardant said is one of the most demanding lab certification programs in the country, and the approval means Guardant360 can be offered in all 50 states.
The blood-based 73-gene test investigates actionable somatic alteration across all solid tumor sites to provide clinicians information to better manage their patients’ disease, according to Guardant. Last week, Medicare contractor Palmetto GBA released a draft local coverage determination for the test, proposing limited coverage for it in patients with advanced non-small cell lung cancer.
The test was launched in 2014 and has been ordered more than 40,000 times by more than 3,500 oncologists since, Guardant said.
May 12, 2017 | In August 2015 when Deep Genomics first launched, co-founder Brendan Frey made a prediction. “There’s a sea change coming,” he told Bio-IT World. “People are going to be focusing now on the machine learning component and trying to understand what the genome means, not just sequence a bunch of genomes.” It’s been nearly two years, and he’s been proven right as more and more efforts arise to apply deep learning, machine learning, and artificial intelligence to medicine.
But maybe Frey isn’t wholly prescient. Earlier this month Deep Genomics announced a shift in company focus. Genetic testing was the Deep Genomics business plan in August of 2015; today the company is working to develop genetic medicines.
The company’s technology and foundation haven’t changed. Frey’s vision is to use computer science to accurately model what’s going on in cells and how disease arises from mutations. “Closing the genotype-phenotype gap means understanding how mutations impact what’s going on in cells and how that impacts diseases, whether that’s cancer or Alzheimer’s Disease,” Frey told Bio-IT World earlier this week. Detecting mutations is the first step; figuring out what to do about the mutations is the second part.
But genetic testing as an industry is bound in regulatory constraints. “What we found is that the genetic testing community is very conservative and a lot of issues need to be sorted out. They’re political issues; they’re insurance issues; they’re FDA issues. Sorting that out is not something that we want to focus on in the short term. As the community moves forward and those issues get resolved, then we’ll re-engage with the genetic testing community. But right now we’re focused on genetic medicine,” Frey said.
Deep Genomics intends to understand disease starting with its genetics, and then rationally develop drugs to target the genetic underpinnings of disease. Frey believes the regulatory landscape for that type of genetic medicine is much more fertile, citing the 21st Century Cures Act.
“If you look at pharmaceuticals and therapeutics… everybody realizes that this $2.8b per drug [cost] to produce a drug doesn’t work and an 85% failure rate is just not acceptable. Many people are suffering because of this. So the regulatory constraints are dropping; it’s becoming easier and easier to develop drugs.”
Into The Wet Lab
You might expect that Deep Genomics would simply offer its deep learning platform to companies and groups as software-as-a-service or platform-as-a-service. Frey laughs when I ask him about it; he understands the assumption. But from the beginning, Deep Genomics has employed both computer scientists and cell biologists. The Deep Genomics platform has already identified genetic medicine candidates and the company is pursuing options for central nervous system, eye, and liver disorders, validating them now in tissue culture.
Deep Genomics has lab space at JLABS @ Toronto, a 40,000-square-foot life sciences incubator sponsored by Johnson & Johnson Innovation that just celebrated its first year. Frey is also making good use of the science-on-demand capabilities now available. “Nowadays, over the internet you can order compounds… companies will synthesize the compounds and do the chemistry for you. Companies like Transcriptic enable cloud labs, that allow you to do experiments by uploading basically a computer script.”
The company has previously been funded by angel investors and had revenue from clients and partners. But now Frey is securing additional funding for a Series A round to “massively scale up” the company’s experimental unit. He hopes to announce his own compounds in the next 18 months.
In addition, Deep Genomics is seeking pharma partners. “The path for us is to focus on the early-stage development right now, and collaborate with other pharmaceutical companies and help them get their products out as fast as they can, reduce risk for other pharmaceutical companies,” he said.
Deep Genomics’ vision, Frey stressed, hasn’t changed. “The core idea of Deep Genomics is that the pharmaceutical company of the future is going to look like a computer science company with an amazing team of biologists and chemists and experts in clinical trials rather than a traditional pharmaceutical company with biologists and chemists who are using computational tools. It’s a question of culture; it’ll be a culture of computer science.”
The platform has matured over the past two years. The company has diversified the types of molecular phenotypes it looks at, considering transcription initiation, polyadenylation, and mRNA stability, in addition to splicing errors, and has added protein-related molecular phenotypes.
For the past few months, Frey said, the company has been focusing on how to introduce genetic modifications or therapies to fix various mutations.
“Say there’s a mutation that causes a problem with splicing or a transcriptionally-related problem,” he proposes. “Now what kind of a genetic modification or genetic medication would be needed to fix that problem?”
Most at-home lab testing devices like Scanadu and Cor are still waiting for FDA approval, but simple lab testing can still be done in the confines of your own home and then shipped to a lab and that’s led to a handful of new startups offering services like STD or food allergy tests.
Everlywell, an Austin-based at-home lab testing startup (and a Disrupt Battlefield company), aims to make the testing it offers affordable and easily accessible to the masses. The company debuted two years ago and has grown quite a bit in a short amount of time. Founder Julia Cheek tells me Everlywell is now generating millions in sales and ships to 46 states in the U.S. The company recently announced it had pulled in another $2 million in seed funding, bringing the total to $5 million to help it grow its service offerings.
Everlywell so far offers eight different tests including food sensitivity, thyroid and metabolism testing or fertility testing to get a clear picture of how you are doing in those areas.
MyLabBox is another startup offering a detailed list of STD tests available for use in the home and at your convenience. The costs might seem high if not covered by insurance (and most at-home lab testing doesn’t seem to be) but you could still see about FSA/HSA reimbursements.
Though the debate wages on for how to cover America, these types of startups present a new range of abilities for the healthcare industry and could help lower the cost and ease of access for service workers and others who don’t work regular hours or don’t typically have insurance coverage.
The process is pretty simple for each startup — you order online and a kit arrives in the mail. Each test is different but some require a sample of blood or saliva. Just follow the instructions and then pop it back in the mail for analysis by a third-party lab.
The idea for these types of startups might be similar to something the embattled blood testing company Theranos once hoped to accomplish. Theranos held a lot of promise when it first launched, claiming it could test for hundreds of diseases on one drop of blood. However, you had to go into a Walgreen’s partner lab if you wanted to get results.
These newer lab startups offer the ability to test in privacy and instead use certified third-party facilities for accurate measurements. They may also be the preferred method for those who just want to see their results before determining whether they need to see a doctor as sitting face-to-face with someone who might tell them they have an STD can be a very real fear.
Both startups stand by the accuracy of their results and I’ve personally tried a food sensitivity test from Everlywell that I can say helped me determine certain foods that were causing stomach issues — including green peas, which is one I never would have thought of otherwise.
It’s not exactly full access to healthcare (that’s another debate I could get into but not today), but this new crop of health startups do offer a regulated option for those who can scrape up the money without paying insurance premiums to find out what might be going on inside their body — and with an easy and convenient solution to some of the fear they might have about going to the doctor’s office to get results.
When it comes to artificial intelligence (AI) and Chinese tech companies, thoughts often begin and end with Baidu. But Tencent, Asia’s second highest-valued tech company behind Alibaba, has reminded the world that it too is investing in the field.
Search giant Baidu was one of the first to make a major commitment to deep learning. It spent over $2.9 billion on R&D over a 2.5 year period, according to Bloomberg, and currently has more than 1,300 specialists working on a variety of technologies that include AI and augmented reality. Baidu, however, suffered a blow when its chief scientist Andrew Ng, who heads up its U.S.-based research team, announced his departure last week.
There’s one more exit to add to that list after Tencent announced today that it poached machine learning researcher Tong Zhang, who heads up Baidu’s Big Data Lab, to lead its own AI Lab. The Shenzhen-based lab is focused on computer vision, speech recognition, and natural language processing.
Tencent, best known for WeChat, China’s top messaging app, announced the lab last April. It said today that it has 50 AI specialists housed there. Aside from that development facility, Zhang — who received a PhD in Computer Science from Stanford and has worked at IBM and Yahoo — will lead a team of 200 product engineers that’s tasked with converting AI advances into tangible features and updates for Tencent’s apps and services.
“Tencent is looking at four areas for AI application: content, social, online games and cloud services. At present, over a hundred Tencent products, including Weixin/WeChat, QQ and Tian Tian Kuai Bao, a Tencent news app, use AI technology,” the company added.
That’s a similar goal to Baidu, which got serious on deep learning when it hired Ng, a co-founder of online learning startup Coursera, in 2014.
A world-renowned AI expert, Ng previously made his name when it helped found Google’s deep learning team, Google Brain. Ng said in his departure note that Baidu’s AI efforts had been felt across its “existing businesses in search, advertising, maps, take-out delivery, voice search, security, consumer finance and many more” areas.
“The team is stacked up and down with talent; I am confident AI at Baidu will continue to flourish,” he added.
For now, Tencent is talking up its AI prowess in the field of Go, the strategic game that Google made its mark on when its AI (AlphaGo) triumphed over world champion Lee Seedol last year.
Tencent said its ‘Fine Art’ AI, which was developed by 13 Tencent engineers, defeated high-ranking Japanese Go player Ryo Ichiriki last week. All in all, the firm said the AI has taken on 100 “renowned human players,” winning 406 of over 500 rounds that it has competed in.
Uber has confirmed that its small fleet of autonomous vehicles in San Francisco will be back on the road today. The company grounded its entire self-driving test fleet in the U.S. on Saturday, following an accident involving one of its vehicles in Arizona earlier in the day.
“We are resuming our development operations in San Francisco this morning,” an Uber spokeswoman told us this morning.
The spokeswoman also confirmed Uber’s autonomous cars in Arizona and Pittsburgh remain grounded, but said the expectation is for them to shortly be back on the roads too.
Uber grounded all its self-driving test cars following the accident, initially suspending the operation in Arizona but also in the two other cities where it is currently testing the vehicles: San Francisco and Pittsburgh, pending the outcome of an investigation.
While Uber’s self-driving cars are able to drive autonomously, the test vehicles include a human driver sitting in the passenger seat so they are in a position to take over should it become necessary.
The two vehicles it’s testing in SF are apparently at a different stage of development vs the 12 test cars apiece it’s been piloting in the other locations — explaining why it’s feeling confident enough to return to the road in SF but not yet elsewhere.
Early reports of the Arizona incident appear to have cleared Uber’s technology of blame, with local police saying the accident occurred because a normal (i.e. human-driven) vehicle failed to yield to the Uber, which was in self-driving mode at the time.
The company’s self-driving test program has, however, previously faced safety-related criticism — including when one of the vehicles ran apparently ran a red light. In that instance Uber claimed the car had not been in self-driving mode at the time — though a New York Times report, citing two Uber sources, has suggested the opposite.
Beyond questions over the safety of its self-driving tech, Uber continues to face wider criticisms relating to its company culture — with accusations of sexism by a former employee continuing to pile pressure on the leadership (and apparently contributing to the decision of president Jeff Jones to leave the company earlier this month, amid the turmoil).
NEW YORK (GenomeWeb) – Editas Medicine has signed a deal potentially worth more than $90 million, giving Allergan the exclusive rights to license certain of its CRISPR genome editing-based treatments for eye diseases.
Under the terms of the deal, Allergan subsidiary Allergan Pharmaceuticals International Limited will have exclusive options to license up to five of Editas’ ocular drug candidates including LCA10, its preclinical treatment for Leber congenital amaurosis, an early candidate for the first disease to be treated medically with CRISPR. Allergan will also have access to CRISPR/Cpf1, an alternative DNA targeting nuclease developed by Editas Cofounder and Broad Institute Researcher Feng Zhang.
Editas will receive an upfront payment of $90 million for the development of the five drug programs, and stands to receive additional payments upon achieving undisclosed near-term milestones specifically related to LCA10. It may also receive payments tied to certain development and commercial milestones, plus royalties on a per-program basis.
Allergan will be responsible for all development and commercialization efforts for all optioned candidates, subject to an Editas’ option to co-develop and co-promote one or two of the drugs in the US.
Additional terms were not disclosed.
“The Allergan team is excited to work with colleagues at Editas Medicine to develop and potentially deliver game-changing treatment for retinal diseases like LCA10,” David Nicholson, Allergan’s chief research and development officer, said in a statement. “This program is highly complementary to our ongoing eye care development programs where unmet medical need exists for patients.”
In August 2016, Editas signed a collaboration deal with Adverum Biotechnologies to develop delivery methods of genome-editing eye therapies.
Allergan has long been a leader in advancing innovative therapies to treat eye diseases,” said Editas CEO Katrine Bosley. “Working together with Allergan through their Open Science R&D model significantly enhances our ability to develop genome editing medicines to help patients with serious eye diseases.”
NEW YORK (GenomeWeb) – Palo Alto, California-based Freenome announced today that it has raised $65 million in Series A funding, which it will put toward conducting clinical trials to validate its non-invasive, early cancer detection technology.
The round was led by venture capital firm Andreessen Horowitz, which last year helped the company raise $5.6 million in seed funding. Google Ventures, Polaris Partners, Innovation Endeavors, Spectrum 28, Asset Management Ventures, Charles River Ventures, Third Kind Ventures, AME Cloud Ventures, and Allen and Company contributed to this Series A round, as did previous investors such as Data Collective and Founders Fund.
Andreessen Horowitz General Partner Vijay Pande will join Freenome’s board of directors, the company added.
“These funds will help bring Freenome’s technology to market faster by accelerating our ongoing research and clinical trials,” wrote Cofounder and CEO Gabriel Otte in a blog post.
Freenome’s early cancer detection testing utilizes genome-wide sequencing and a computational system, called Adaptive Genomics Engine, that can be trained to analyze patterns in the cell-free genetic material floating in a person’s blood.
The firm is working with 25 research partners on various studies of its technology, including the University of California, San Francisco, Moores Cancer Center at UC San Diego Health, and Massachusetts General Hospital. According to Otte, five pharmaceutical companies are also using Freenome’s system to explore personalized medicine strategies in cancer.
Once Freenome commercially launches its technology, it will enter a space occupied by Grail, which today announced it has raised more than $900 million through the first close of a Series B financing. The company is planning a second close that will bring the total funds raised in the Series B to more than $1 billion.
In January, Guardant Health — which also competes in this space — inked a multi-year deal with MD Anderson Cancer Center to make its Guardant360 test the preferred liquid biopsy test at the facility. Guardant and MD Anderson will also jointly fund interventional clinical utility studies on the non-invasive targeted sequencing approach.
Pharma veteran and MIT visiting scientist Mark Trusheim advances a provocative solution to overcome treatment costs.
In a recent article in Forbes, MIT Sloan School of Management visiting scientist Mark Trusheim and co-author Peter B. Bach, a physician at Memorial Sloan Kettering Cancer Center in New York, propose a startling response to the prevalence of hepatitis C and the high cost of the leading therapy: The U.S. government should not just pay for the pills — they should buy their maker.
The pharmaceutical company Gilead sells hepatitis C drugs that have 80 percent market share at an estimated average cost per patient as high as $42,000 per curative treatment course. In clinical studies it’s shown to halt, and in some cases reverse, liver damage caused by the disease. “In this unique case … there is an intersection of a clear winning therapy, large public health need, slow adoption, high product pricing,” and a solid business case, say the authors.
By taking the unorthodox approach of buying Gilead and divesting the parts not related to U.S. hepatitis C treatment, the United States “will make [the therapy] affordable to rapidly treat the 2.7 million Americans that the CDC estimates still have hepatitis C,” and save money in the long run, compared to the traditional approach of reimbursing for treatments, Trusheim and Bach say.
This unprecedented solution, backed by a break-up analysis and financial cost-benefit calculations, fulfills another public aim: “Improving the health and wellbeing of the U.S. is the very objective of public investment in research, medical care, and in this one case, the stock market,” the authors assert.
“In a desire to cure more patients faster, we discovered that private equity financial tools could provide a more effective route than what the current drug purchasing approaches are achieving,” said Trusheim, who is also a strategic director of the MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative. NEWDIGS convenes a broad sample of healthcare stakeholders — including global leaders in research, development, insurance, regulation, clinical care, and patient advocacy — to develop and pilot innovative biomedical innovation practices that benefit all.
Current hepatitis C treatment costs are limiting their impact on patient care. “Science is making remarkable treatments possible,” said Gigi Hirsch, executive director of NEWDIGS and the MIT Center for Biomedical Innovation. “The creativity and analytic rigor that Peter and Mark demonstrate in this proposal stimulate the kinds of collaborative discussions we need to overcome the financial barriers between patients and the medicines they need.”
For the first time, biologists have succeeded in growing human stem cells in animal embryos, shifting from science fiction to the realm of the possible the idea of developing human organs in animals for later transplant.
The approach involves generating stem cells from a patient’s skin, growing the desired new organ in a large animal, and then harvesting it for transplant into the patient’s body. Since the organ would be made of a patient’s own cells, there would be little risk of immune rejection.
The human-organ-growing animals would be examples of chimeras, animals composed of two different genomes. They would be generated by implanting human stem cells into an early embryo, resulting in an animal composed of mixed animal and human cells.
One team of biologists, led by Jun Wu and Juan Carlos Izpisua Belmonte at the Salk Institute, has shown for the first time that human stem cells can contribute to forming the tissues of an animal, despite the 90 million years of evolution between the two species.
Another group, headed by Tomoyuki Yamaguchi and Hideyuki Sato of the University of Tokyo, and Hiromitsu Nakauchi of Stanford, has reversed diabetes in mice by inserting pancreas glands composed of mouse cells that were grown in a rat. The Salk team’s report was published in Cell and the Stanford-Tokyo team’s in Nature.
The two reports together establish the feasibility of trying to grow replacement human organs in animals, though such a goal is still far off.
“I think this is very promising work in principle,” said Rudolf Jaenisch, a stem cell expert at the Whitehead Institute in Cambridge, Masschusetts.
Many technical and ethical barriers have yet to be overcome, but the research is advancing alongside the acute need for organs; some 76,000 people in the United States alone are awaiting transplants.
Creating chimeras, especially those with human cells, may prove controversial, given the possibility that test animals could be humanised in undesirable ways. One would be if human cells should be incorporated into an animal’s brain, endowing it with human qualities. Almost no one wants a talking animal.
Another untoward outcome would be if human cells should come to compose the animal’s reproductive tissues. Few people want to see what might result from the union between an animal with human sperm and an animal with human eggs.
Izpisua Belmonte’s and Nakauchi’s teams have both pursued a strategy of directing the human donor cells to generate specific organs in the recipient species. This is desirable for both technical and ethical reasons.
Nakauchi has disabled the master gene in rats for making a pancreas so that when mouse stem cells are injected into the early embryo of such a rat, the growing embryo has no choice but to construct its pancreas of pure mouse cells, instead of the usual mixture of rat and mouse cells.
The result provides proof of principle that Type 1 diabetes can be treated by growing a pancreas from an individual’s cells in another animal, Nakauchi and colleagues conclude.
The next step is to repeat the experiment in bigger animals, which produce organs of a more suitable size for use in humans. Izpisua Belmonte’s team has now shown that human stem cells do survive in the animal’s embryos and help form their organs, although not very efficiently.
“The human cell doesn’t contribute much. To the brain we observed little or no contribution at all,” said his colleague Wu.”This is good news because we can guide the human cells to the organ we want.”
Both Izpisua Belmonte and Nakauchi said there was a long way to go before human organs could successfully be grown in animals. Chimeras will be more immediately useful in studying human embryogenesis, testing drugs and following the progress of disease.
Both scientists expressed confidence that ethical concerns about chimera research could be addressed. Chimeras are typically mosaics in which each organ is a mixture of the host and donor cells. But new techniques like the Crispr-Cas gene editing system should allow the human cells in an animal embryo both to be channelled into organs of interest and to be excluded from tissues of concern like the brain and reproductive tissues.
“This isn’t dangerous research. We’re not creating monsters,” Nakauchi said.
“There isn’t a need to get into a debate about moral humanisation if scientists target the organs where the human cells will go,” said Insoo Hyun, a medical ethicist at Case Western Reserve University.”Scientists are not making chimeras just for fun ” it’s to relieve the dire shortage of transplantable organs.”
Concern about human cells’ incorporation into a reduce animal’s mind is not with out basis. Dr. Steven Goldman of the College of Rochester Clinical Center discovered in 2013 that mice injected with a particular style of human mind mobile experienced enhanced understanding talents. But other kinds of humanized mice, this sort of as mice engineered to have a human immune system, are schedule laboratory animals that appear to be to event very little angst.
Dr. Izpisua Belmonte’s insertion of human stem cells into pig embryos was not influenced by the N.I.H. moratorium on this sort of chimeras mainly because he utilized personal cash. His experiment was accredited by the authorities in Spain and in California, and following their guidance, the improvement of the pig chimeras was stopped following four weeks in the womb.
Dr. Nakauchi moved his lab to Stanford from Tokyo in 2014 mainly because Japanese rules do not permit chimera exploration, only to be strike with the N.I.H. moratorium a year afterwards, which prevented him from generating chimeras with human cells. His mouse pancreas experiment has taken eight or nine many years to finish. “I have been in a very aggravating problem,” he said.
Despite tough talk by President-elect Donald Trump about a possible trade war, Chinese investors are hungry for U.S. biotech and they’ve got money to spend.
In the latest purchase following a record 2016 for Chinese health-care takeovers, Nanjing-based Sanpower Group Co., said Tuesday it would buy a cancer treatment business from Valeant Pharmaceuticals International Inc. for $820 million.
Sanpower’s interest is far from unique: Chinese venture capitalists and individual investors are looking as well. Last Sunday morning, in a hotel ballroom a short hop from the San Francisco airport, about 250 jet-lagged potential dealmakers and American biotech executives could be heard chatting in Mandarin and English. They were attending a get-together hosted by boutique investment firm CTIC Capital, hoping to forge alliances before the start of the industry’s biggest annual gathering, the J.P. Morgan Healthcare Conference, at the Westin St. Francis in San Francisco, which runs this week.
China has a “huge pile of money’’ and a lot of new venture capital firms, but a limited number of homegrown drug startups to invest in, said Kevin Chen, a partner at the China Fund of Menlo Park, California-based Sequoia Capital. The fund, which spends about a quarter of its $3 billion under management on health care, last year started founding U.S.-based startups with Chinese ties, helping bridge the gap for investors.
U.S. investors are beginning to notice. The J.P. Morgan conference has an entire track dedicated to China-based health-care companies at the conference. And last year, Chinese firms announced or completed about $8 billion in cross-border acquisitions, according to data compiled by Bloomberg.
“The gate is open and it won’t be closed again,” said Lan Huang, chief executive officer of BeyondSpring Pharmaceuticals Inc., a maker of cancer drugs with headquarters in New York and offices in Dalian, China. Her company, which plans to go public, has been meeting with investors from both countries.
The conference comes at a pivotal moment for the two nations. Trump’s rhetoric toward China has some investors worrying about a chilling effect on business. He has promised tariffs on Chinese goods, and China has said it’s prepared to step up its scrutiny of U.S. companies.
“If there’s no clear sign that it’s safe to invest in the U.S. and that innovation will import to China, that’s going to hurt investors,’’ said Jennifer Hu, a partner at China-based Qiming Ventures Partners. “They could go to Australia or Canada for that innovation instead.’’
Qiming, which spends about 40 percent of its $2.7 billion in funds on health care, is currently raising money for its first fund to be dedicated to U.S. health investments. Up to now, Qiming had focused its investments on Chinese companies.
Yet unless there’s “dramatic change” in U.S. policy, Sequoia’s Chen sees Chinese investors continuing to push money toward U.S. biotechs.
Out of Reach
Reasons include a shortage of Chinese companies whose financials pencil out. Many that have gone public are “unicorns” worth more than $1 billion — valuations that may be out of reach, said Yanhong Lin, founder and managing partner of CTIC, which is based in Palo Alto, California, and has offices in Shanghai and Nanjing.
While Chinese regulators are becoming more amenable to approving homegrown drugs, the U.S. is still perceived as the place to go for growth.
“The U.S. still represents the highest technology,” said Huang, the BeyondSpring CEO. As Chinese investors “increasingly are trying to think globally, this meeting is their opportunity.”