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Gene Therapy Biotech Raises $60M in Venture Funds

Adeno-associated virus

Adeno-associated virus (

13 April 2015. Voyager Therapeutics, a biotechnology company developing gene therapies for central nervous system disorders, raised $60 million in its second venture financing round. Financing for the 1 year-old enterprise in Cambridge, Massachusetts was led by new investors Brookside Capital and Partner Fund Management, with participation by Wellington Management Company and Casdin Capital, also new investors, as well as other investors who were not disclosed.

Voyager is developing treatments for diseases of the central nervous system including Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, a rare inherited disease causing damage to the nervous system and movement problems, and Huntington’s disease, an inherited brain disorder that results in progressive loss of both mental faculties and physical control. The company is commercializing research by the company’s founders whose work covers technologies for harnessing viruses to deliver genetic therapeutics, and treatments using RNA interference to inhibit the expression of certain genes.

Voyager’s technology harnesses adeno-associated viruses to deliver healthy genetic material for expressing proteins missing from the mutated or damaged genes causing the disorder. Adeno-associated viruses are benign, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response. One of the company’s founders is Guangping Gao, professor of microbiology at University of Massachusetts Medical School, whose work  involves the discovery, development, and use of adeno-associated viruses for gene therapy of inherited diseases.

In February 2015, Voyager announced a licensing deal with Genzyme, a biotechnology subsidiary of the drug maker Sanofi, that could earn Voyager as much $845 million. The agreement covers several Voyager gene therapies for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other unspecified disorders of the central nervous system. Voyager is leading research and development of the therapies, working with Genzyme, a developer of treatments for multiple sclerosis and several rare diseases.

Genzyme has an option to license further rights to the therapies following early clinical trials. Voyager will continue to hold the U.S. rights to its lead treatments being developed for Parkinson’s disease and Friedreich’s ataxia, and will split profits in the U.S. with Genzyme from its Huntington’s disease therapy. Voyager’s ALS treatment is not included in the collaboration.

In addition to Guangping Gao, Voyager was founded by three other medical, genetics, and pharmacology faculty from University of Massachusetts, University of California in San Francisco, and Stanford University. First round financing of $45 million and early management for Voyager was provided by Third Rock Ventures, a life sciences venture capital company.

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Hat tip: Fortune/Term Sheet

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Janssen Licensing DNA Technology for Hep B Vaccine

Hepatitis B virus particles

Transmission electron micrograph of hepatitis B virus particles (Public Health Image Library, CDC)

13 April 2015. Janssen Pharmaceuticals, a division of Johnson & Johnson, is licensing an electronic DNA drug delivery technology from Ichor Medical Systems for vaccines to treat hepatitis B. Ichor, based in San Diego, expects to gain as much as $85 million in the deal.

Hepatitis B is a liver infection caused by a virus that can become a chronic condition, increasing the risk for life-threatening cirrhosis and liver cancer. The disease is spread through contact with blood and other bodily fluids, often from sexual contact or sharing hypodermic needles. World Health Organization calls hepatitis B a major global health problem, affecting some 240 million people with chronic conditions (lasting 6 months or more) and causing 780,000 deaths, mainly from cirrhosis.

Ichor Medical Systems is developing a drug delivery system using electrical impulses to enhance the effectiveness of DNA vaccines that introduce DNA plasmid molecules into cells. Those molecules give instructions to deposit antigens on cell surfaces for stimulating an immune response. The process, called electroporation, sends electrical impulses to create temporary pores in cell membranes, allowing for faster uptake of the DNA payload.

The company says its TriGrid drug delivery system overcomes efficiency problems with DNA vaccines using conventional injections, with a hand-held device that requires minimal user training. The system is being tested in clinical trials with vaccines for several types of cancer, HIV/AIDS, and other infectious diseases. Ichor says early-stage trials show TriGrid is safe and delivers vaccines that generate immune responses.

Under their agreement, Janssen is licensing the TriGrid technology, with the two companies collaborating on a hepatitis B vaccine. Janssen is responsible for some development costs and all commercialization costs in the deal, including expenses for manufacturing and distribution of TriGrid devices. Ichor will receive upfront, R&D, and milestone payments up to $85 million, as well as royalties on future product sales.

In November 2014, Ichor received a 5-year contract from Defense Advanced Research Projects Agency (DARPA) to develop a platform for producing vaccines that can work immediately with troops in the field, including those already exposed to pathogens. The contract, with a total value of $20.2 million, is supporting further development and clinical trials of the TriGrid system.

Disclosure: The author owns shares in Johnson & Johnson, parent company of Janssen Pharmaceuticals.

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Simple Avian Flu Test Designed for Poultry

Turkey (Agricultural Research Service, USDA)

(Agricultural Research Service, USDA)

10 April 2015. A bioengineering lab at University of Guelph in Ontario, Canada developed a quick, simple test for avian flu virus that infects poultry, including the type of virus now infecting turkeys in the U.S. and Canada. Guelph’s Bionano Lab led by engineering professor Suresh Neethirajan says a description of its device will appear in an upcoming issue of the journal Sensors.

Current testing techniques for avian flu viruses require taking blood samples from birds and sending them to remote labs for analysis. Just the analysis step takes 8 hours, says Neethirajan, and the entire process requires at least 2 days from sample to results.

When testing for avian flu outbreaks, particularly early on, getting results quickly is vital. “It’s critical to get out front of any outbreaks,” notes Neethirajanin in a university statement. “There are many strains, and we need to know the source of the flu. The identification of the strain determines what treatment options we should use.”

The Guelph test, returns results on the spot in about 2 to 3 minutes. The device designed by Neethirajanin and Longyan Chen, a postdoctoral researcher in the Bionano Lab, uses less blood from the birds than current techniques. In addition, the device tests the samples for characteristic surface proteins with a process using gold nanoparticles and quantum dots tuned to emit different colors. Quantum dots are pieces of semiconductor material that make it possible to measure and manipulate single electronic charges.

Not only can the Guelph test indicate the presence of avian flu virus, it can tell the strain of virus for determining the course of treatment. The researchers say their test can discriminate between H5N1 and H1N1 avian flu strains, and can be extended to indicate H5N2 strain causing the current outbreak. Of the 16 hemagglutinin subtypes of influenza — the “H” in virus codes — H5 is associated more with outbreaks in wild and domestic birds, while H1 can also affect humans.

The new avian flu test comes at a critical time for poultry producers in North America. The New York Times reports today that the deadly H5N2 virus, believed to originate in migrating wild birds, is causing turkey farmers Minnesota to euthanize some 525,000 birds, with quarantines occurring in the U.S. West and Midwest. That same virus hit British Columbia in December 2014 and January 2015, and this week was confirmed on a farm in southwestern Ontario by Guelph’s Animal Health Lab, which collaborated on the avian flu test.

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North Texas Campuses Researching Assistive Technologies

Ovidiu Daescu

Ovidiu Daescu (University of Texas, Dallas)

10 April 2015. Two University of Texas campuses, in Dallas and nearby Arlington, formed a research center to study software solutions for assistive technologies that benefit disabled and able-bodied people alike. The iPerform Center for Assistive Technologies to Enhance Human Performance, funded for five years by National Science Foundation, is also enlisting industry partners that would give them early access to the center’s work.

The joint project takes a broader view of assistive technologies, a term usually associated with solutions to help people with disabilities. The center plans to examine software for computer-assisted tools such as prosthetics for amputees, robotics to help people with failing eyesight, and smart wheelchairs. But the same techniques can be extended to solutions to help elderly people cope with day-to-day life and live securely in their own homes: e.g., better designed homes to prevent accidents, enhanced telecommunications, and application of games for rehabilitation.

Another extension of assistive technologies is for health care delivery, such as sensors to detect gestures or facial movements, along with speech recognition for personalized therapies. In addition, the same technologies can be applied to manufacturing to improve the efficiency of human-machine interactions and  increase worker safety.

The iPerform Center is led by computer science professors Ovidiu Daescu at UT-Dallas and Fillia Makedon at UT-Arlington. Daescu studies algorithms using computational geometry as well as biomedical computing applications. Makedon conducts research on human-centered computing, particularly in health care, manufacturing, and vocational safety. Several faculty from each campus plan to take part as co-investigators.

In a UT-Dallas statement Daescu notes “There currently is no research hub in the country for assistive technologies,” adding “The projects conducted within the center will help advance basic research in areas such as computer vision, machine learning, user interfaces, brain imaging, human robot interaction, human computer interaction, virtual reality and simulation.”

The joint project is funded under National Science Foundation’s Industry/University Cooperative Research Center program, where NSF makes a small initial seed grant, and the universities recruiting industry partners to fund the bulk of the center’s activity. UT-Dallas reports attracting three companies as industry partners — Bosch North America, Raytheon Co., and Texas Instruments — as well as National Institute of Standards and Technology, an agency of U.S. Department of Commerce.

An industry partnership, at an annual fee of $40,000, offers companies early first-hand access to the results of iPerform’s work and the opportunity to collaborate with faculty and graduate students at the two campuses. Industry partners also get to recommend research projects and vote on the center’s annual research agenda.

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Alzheimer’s Drug Granted Orphan Status for Fragile X

Nerve cells illustration

Nerve cells in brain illustration (

9 April 2015. A new treatment in development for Alzheimer’s disease was designated an orphan drug for Fragile X syndrome, an inherited neurological disorder, by the U.S. Food and Drug Administration. Neurotrope BioScience Inc. in Newark, New Jersey, received the designation for its drug bryostatin, now in clinical testing to treat Alzheimer’s disease.

Fragile X syndrome is a genetic condition that results in intellectual disability, behavioral, and learning difficulties such as general and social anxiety, with identifiable physical symptoms, such as long face and connective tissue problems. The condition occurs in both genders, but males are affected more than females, and with greater severity. Fragile X syndrome affects about 1 in 4,000 males and 1 in 8,000 females.

Neurotrope is the developer of bryostatin, a drug in development that activates protein kinase C-epsilon, an enzyme that regulates a range of human functions and associated with a number of disorders when missing or limited, including cancer. Protein kinase C-epsilon works through a complex pathway, but when activated under some conditions is believed to reverse nerve damage that occurs in Alzheimer’s patients. Bryostatin is currently in intermediate-stage clinical trials as a treatment for Alzheimer’s disease.

Neurotrope was founded in 2012 to commercialize therapies and diagnostic technologies developed from research conducted at Blanchette Rockefeller Neuroscience Institute at University of West Virginia. Protein kinase C-epsilon activators are among those discoveries licensed by Neurotrope for Alzheimer’s disease treatments, but also for other neurodegenerative disorders, including Fragile X syndrome.

Preclinical studies of bryostatin, says the company, show promise as a treatment for Fragile X syndrome. Daniel Alkon, Neurotrope’s chief scientist, says in a company statement, “We are very encouraged by the preclinical data we acquired in a Fragile X mouse study that suggests treatment with bryostatin can mature synapses and increase the number of synaptic connections resulting in improved learning and memory.” Alkon is also scientific director at Blanchette Rockefeller Neuroscience Institute.

Orphan drug designation is granted to treatments being developed for diseases affecting fewer than 200,000 people in the U.S. Therapies, both drugs and biologics, designated as orphan drugs qualify for incentives such as 7 years of market exclusivity, tax credits for clinical trials, and exemptions from marketing application fees.

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Trial Testing Mobile Apps for Mood Management

IntelliCare screen

IntelliCare screen. Click on image for full-size view. (Google Play)

9 April 2015. A lab at Northwestern University medical school developed a collection of mobile apps for helping people cope with feelings of depression and anxiety, and is testing the apps as personalized interventions in a clinical trial. The study is led by David Mohr, director of Northwestern’s Center for Behavioral Intervention Technologies, who is recruiting 200 individuals to test the apps in their day-to-day lives.

The mobile suite, known as IntelliCare, has 12 individual apps, each designed to address a specific type of worry, such as repeated negative thoughts, isolation, or lack of motivation. Users can download the apps individually, or the entire collection, for free from Google Play; only Android versions are currently available. Each app, says the developers, is based on validated techniques used in therapy.

IntelliCare is designed to gather data from individuals based on their use of the apps, which when added together from all users, will form a knowledge base for recommending specific interventions customized for each person. An algorithm also recommends new apps and opportunities to learn new skills based on user behavior. Mohr, in a university statement, calls the technique “precision medicine for treating depression and anxiety delivered directly to the user,” adding “It will help the millions of people who want support, but can’t get to a therapist’s office.”

People who download the IntelliCare apps are asked to take part in a field test of their utility as interventions for depression and anxiety, among people experiencing clinical symptoms of those disorders. According to Centers for Disease Control and Prevention, a large number of people in the U.S. experience those symptoms. Some 8 million people a year visit doctors’ offices or outpatient clinics for major depressive disorder, and nearly 400,000 people are hospitalized for the disorder with an average stay in the hospital of 6.5 days.

The clinical trial aims to enroll 200 individuals with depression and anxiety to get early indicators of IntelliCare’s feasibility and effectiveness of IntelliCare in improving those symptoms. The trial asks participants to use the apps daily for 8 weeks, including a brief weekly motivational session with a coach. Assessments will be made at the beginning of the  8 weeks, with feedback and assessments gathered at weeks 4 and 8.

The trial’s main indicator of feasibility is the ability of users to stick with the regimen over the 8 week period. The study is also measuring self-reported severity of depression and anxiety symptoms, as well as satisfaction with the apps.

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Purdue Spin-Off Designing Customized Synthetic Tissue

Sherry Harbin

Sherry Harbin (Purdue University)

8 April 2015. A one year-old company, based on research at a Purdue University biomedical engineering lab, is producing customized biomaterials designed to form into synthetic tissue for drug discovery and toxicity testing. GeniPhys, founded by Purdue biomedical engineering professor Sherry Harbin, aims to further develop the technology, licensed from the university, into engineered tissue for implants and regenerative medicine.

At Purdue, Harbin and colleagues study components and properties of extracellular matrix of human tissues, the materials secreted by cells that make it possible for cells to form into three-dimensional forms and structures. Among those materials is collagen, the main structural protein in animals, consisting of thin filaments called fibrils that assemble into human tissue, such as cartilage and bone.

Harbin’s research examines the processes in the body that generate and assemble collagen, as well as the signaling mechanisms between cells and collagen. Harbin’s research led to a greater understanding of processes that make it possible for collagen to form fibril filaments, the components of collagen that give it shape and structure.

This greater understanding, says Harbin, changed her view of collagen from a static structural element in tissue, to a dynamic participant in the tissue formation process. “[It] is now evident,” says Harbin in a university statement, “that collagen fibril microstructure, mechanical properties including stiffness, and proteolytic degradability [ability to break down proteins into peptides or amino acids] provide critical cues and instructions that control cell fate and tissue formation.”

Based on these findings, Harbin began to see the commercial potential of providing synthetic collagen-fibril tissue designed to meet specific properties and characteristics that offer greater consistency and and quality control. Among those needing this kind of synthetic tissue are pharmaceutical developers where, Harbin says, current lab technologies are limited in their ability to emulate human tissue conditions. She notes that “growth of cells in these over-simplified environments has been shown not to correlate well with human cell responses in the body.”

GeniPhys, located in Zionsville, Indiana produces materials for customized collagen-fibril tissue designed to meet specific formulations needed by drug development labs. “GeniPhys collagen polymers allow scientists to grow cells within a highly reproducible, physiologically relevant 3-D collagen fibril matrix that they can customize,” says Harbin. “In this way, scientists can determine how specific attributes of the collagen [extracellular matrix] affect cell behavior, including tumor metastasis and drug/toxin sensitivity.”

Harbin founded GeniPhys in May 2014, and licensed the technology based on her research from Purdue’s technology transfer office. She then received coaching on starting up and running an enterprise from Purdue Foundry, the university’s business incubator. Unlike some scientific entrepreneurs who leave day-to-day business operations to others, Harbin is a hands-on manager, with her husband Scott Harbin, a partner in the company and practicing veterinarian in McCordsville, Indiana.

GeniPhys plans to develop its technology into production of medical-grade synthetic tissue for implants and tissue engineering, with uses such as wound dressings, organ replacements, and hybrid medical devices. As part of this effort, Harbin is taking a leading role in development of an international standard for collagen polymer formulations to enable more consistent design, development, production, and quality control of collagen-based medical products.

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Uninsured Patients Paying Far More for Cancer Drugs

Stacie Dusetzina

Stacie Dusetzina (Univ. of North Carolina, Chapel Hill)

8 April 2015. An analysis of cancer drug costs and reimbursement practices shows people without health insurance are paying much more for chemotherapy drugs than people covered under private insurance or Medicare. The team led by pharmacy professor Stacie Dusetzina at University of North Carolina in Chapel Hill published its findings in the April issue of the journal Health Affairs (paid subscription required).

The cost of pharmaceuticals, particularly cancer drugs, is becoming a more urgent issue for the health care industry as new payment models and formulas take hold in the U.S. that move away from traditional fee-for-service to performance-based measures. A study published in 2013 shows the price of cancer drugs doubled in the past decade from about $5,000 to more than $10,000 a month.

In some instances, health care providers are questioning the high cost of cancer drugs. In one case, Memorial Sloan-Kettering Cancer Center in New York declined to treat patients with a new, more expensive drug for colorectal cancer, when a less expensive alternative considered just as effective was available at less than half the price, with the decision explained in a 2012 New York Times op-ed.

Dusetzina and colleagues examined the issue of cancer drug costs from a different angle, looking at the prices paid by patients depending on their health insurance. While claims data have long been available to show amounts paid by insurance companies and public sources (e.g., Medicare) for drugs, other sources showing drug prices charged by health care providers before negotiated discounts became available only in the past few years. The Medicare Provider Utilization and Payment Data Public Use File was one of those newer sources examined by the researchers that reveal drug costs before and after insurance companies and public agencies negotiate their discounts.

The researchers, including colleagues from UNC’s and Harvard’s medical schools, investigated prices charged by health care providers for drugs in outpatient chemotherapy, and found the prices varied widely in 2012 depending on the drug and source of funds to pay for it. Costs for a single chemotherapy infusion ranged from $59 for 500 milligrams of fluorouracil, a generic drug, to $9,225 for 10 milligrams of bevacizumab, marketed as Avastin. Both drugs are prescribed to treat various types of solid tumor cancer. And average reimbursements varied that year for chemotherapy from 40 percent for Medicare to 56 percent for private insurance plans.

Uninsured patients, however, found themselves often paying full price for their chemotherapy drugs, since they were not able to take advantage of discounts negotiated by insurance companies or public agencies. Uninsured patients paid prices as much as 5 times higher than amounts reimbursed by private health plans and 43 times as much as Medicare reimbursements for the same drugs. The researchers found similar discrepancies in amounts and reimbursements in prices charged and paid by uninsured, private plan insured, and Medicare patients for office visits to their doctors.

“One key concern here is that patients without insurance could be asked to pay more,” says  Dusetzina in a university statement, “not just more than an insured person but more than Medicare or private health insurance plans.” She adds that “There needs to be more transparency and less variability in health care pricing.”

Health care costs for people without insurance are paid by the individuals or their families, or absorbed by public agencies and hospitals. The Affordable Care Act aims to reduce the number of uninsured by providing subsidies to households, under certain income levels, for buying private insurance through online exchanges or expanding Medicaid. Expansion of Medicaid coverage is up to the states, with 22 states yet to expand their Medicaid rolls leaving some 5.1 million people without health insurance, according to White House estimates.

In addition, subsidies for people who enrolled for private insurance in states where the Federal government runs the online exchange are being challenged in the King vs. Burwell case now before the U.S. Supreme Court. The Robert Wood Johnson Foundation estimates a decision in favor of the plaintiffs will raise the number of uninsured Americans by 8.2 million, an increase of 44 percent.

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Mobile Diabetes Device Maker Lands $20M in Venture Funds

InTouch smart glucose meter

InTouch smart glucose meter (Livongo Health)

7 April 2015. Livongo Health, a developer of mobile devices to help people with chronic disorders manage their conditions, raised $20 million in its second venture financing round. Funding for the Mountain View, California start-up, founded 7 months ago, was led by venture capital company Kleiner Perkins Caufield & Byers, with participation from DFJ Ventures and previous investor General Catalyst Partners.

Livongo Health is developing its Livongo for Diabetes system designed for people with either type 1 or type 2 diabetes. The system includes a smart blood glucose meter that connects to cellular networks, and transmits data from the meter to family members, clinicians monitoring the person’s condition, and third-party diabetes counselors certified by Livongo. The meter also collects other data related to the person’s health, such as physical activity.

People connected to the meter can provide feedback to the individual via voice telephone, e-mail, or text message. Data from the smart meter are sent as well to a database in the cloud, where a rules-based inference engine analyzes the data and offers personalized guidance to the individual with diabetes and his or her physician.

The company says its Livongo for Diabetes system benefits health plans and employers providing health insurance to their employees, because it helps people with diabetes proactively manage their conditions, reduces absenteeism and presenteeism (going to work while ill), and helps lower health care expenses. Large self-insured companies including Office Depot and Iron Mountain, agreed to offer the Livongo system to their employers, as well as health care providers Mission Health System and HealthCare Partners for their patients with diabetes.

Livongo Health says it expects to use the proceeds from the new financing to speed adoption of its diabetes management system, as well as develop an ecosystem for chronic diseases beyond diabetes. CEO Charles Tullman, a Silicon Valley entrepreneur and venture financier, started Livongo in September 2014, and soon received $10 million in first-round funds from General Catalyst Partners.

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Collaboration Collecting Data to ID Precise Cancer Meds

Blood vials


7 April 2015. A partnership between Caris Life Sciences and COTA, short for Cancer Outcomes Tracking and Analysis, is combining data on the chemical makeup of cancer patients with clinical outcomes information to create better profiles of cancer tumors and identify more personalized therapies. Financial details between Caris Life Sciences in Irving, Texas and COTA, based in New York, were not disclosed.

Caris provides a service called molecular intelligence that analyzes the molecular composition of tumors from cancer patients, and compares the results with data from clinical studies to provide their doctors with treatment recommendations best fitting the patients’ tumor profiles. The company says it has more than 70,000 such tumor profiles in its databases.

COTA is a medical analytics company that specializes in cancer diagnostics during the course of a patient’s care. The COTA platform draws data from cancer patients’ electronic health records and provides a precise analysis of each patient’s condition into more fine-grained sub-types. That analysis continues during the time of a patient’s treatment, providing doctors with individualized reports as treatments and outcomes change for the patient.

In their collaboration, Caris and COTA plan to merge their different approaches to individualized cancer care, with the goal of providing an even more precise diagnosis and actionable results for doctors and their patients. The partnership will combine Caris’s molecular data of tumors, including genomic and proteomic factors, with COTA’s precise classification scheme monitored over the course of a patient’s care. All data in this combined knowledge base, say the companies, will have identifying details of individuals removed.

The collaboration will be part of what Caris calls its centers of excellence for precision medicine network. “This collaboration combines our robust database of molecular information,” says Caris chairman and CEO David Halbert in a joint statement, “with COTA’s unique classification and real-time, longitudinal patient tracking capabilities to further expand access and utilization of our tumor profiling service, and to gain insights into cancer treatment outcomes, costs of care, and advance the discovery and delivery of more personalized targeted therapies.”

Eric Schultz, CEO of COTA, notes this approach will help the health care industry better fit in with new reimbursement models encouraging alternatives to traditional fee-for-service payments. “The combination of genomic and proteomic molecular data with COTA’s precise classification and clinical outcomes information and economic data,” says Schultz, “will provide meaningful insights for both physicians and payers, especially as the industry moves toward value-based treatment and reimbursement practices.”

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