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Roche Acquiring RNA Medications Biotech Company

RNA illustration

RNA illustration (

5 August 2014. The pharmaceutical company Roche is acquiring Santaris Pharma, a biotechnology company developing therapies targeting ribonucleic acid or RNA that performs vital genetic functions. Roche is expected to pay as much as $450 million for Santaris, based in Hørsholm, Denmark.

Santaris designs therapies focusing on RNA, a fundamental molecular building block in the genetic system that controls chemical activities in cells. The company says its technology, called Locked Nucleic Acid, has advantages over earlier attempts to address RNA, including more potency, affinity for wider variety of targets, and smaller molecular size making it better able to interact with target cells.

Santaris’s lead candidate is miravirsen, an inhibitor of microRNA-122 controlling the expression of a gene that helps hepatitis C virus replicate and accumulate in the liver. Because miravirsen targets an enabler of the virus rather than the virus itself, it is believed to be less susceptible to resistance from mutations in the virus. The company says in lab tests miravirsen was shown to be active against all six types of hepatitis C virus.

Miravirsen is being tested in an intermediate-stage clinical trial against current drugs considered the standard of care for treating hepatitis C. The 20 patients enrolled in the trial are infected with genotype 1 of the hepatitis C virus, considered the most common, yet most difficult to treat. Current drugs, says the company, are effective in only about half of hepatitis C cases and associated with adverse side effects among some patients.

Under the acquisition, Roche is paying Santaris $250 million at signing, with another $200 million to be paid upon completion of specified milestones. Roche is expected to continue Santaris’s operations in Denmark under the name Roche Innovation Center Copenhagen.

Santaris and Roche were already collaborating on the discovery of RNA medications, in a deal announced in January. In that agreement, the companies would apply Santaris’s technology to several unspecified disease areas, in return for $10 million up front, and up to $138 million per product in subsequent milestones.

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Hat tip: FirstWord Pharma

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Consortium to Develop Common Neuroscience Data Format

Brain silhouettes (


4 August 2014. A coalition of research institutes and universities working in neuroscience are constructing a common format for sharing data about the brain to make emerging databases more useful to researchers. The Neurodata Without Borders project is an initiative of Allen Institute for Brain Science, California Institute of Technology, New York University School of Medicine, the Howard Hughes Medical Institute (HHMI) and the University of California in Berkeley, with funding from GE, Kavli Foundation, Allen Institute, HHMI, and International Neuroinformatics Coordinating Facility.

The field of neuroscience is growing quickly, with more researchers capturing greater volumes and more types of data. The field is also one where collaboration and data sharing are not only highly desirable, but also encouraged by undertakings such as the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) project in the U.S., and sometimes mandated by funding agencies like National Institutes of Health.

Neurodata Without Borders aims to break down the barriers and silos inhibiting the flow of neuroscience data to the broader scientific community. A team from the participating institutions will first concentrate devising a common data format for sharing cell-based neurophysiology data, including electrical and optical recordings, the kind sought most often by researchers building models of brain functions. The project has a one year timetable.

A key part of the project will tackle a common solution for data that describe the information collected about brain activity. These data about data are known as metadata and include key variables such as data collection methods and behavior of lab animals during data collection, as well as rudimentary details such as the gender and age of the animals.

Participants in the project aim to collect current data sets at Collaborative Research in Computational
Neuroscience or CRCNS, a repository at UC-Berkeley that already stores two lab animal data sets from participating research labs, with plans to add two more. The team then expects to invite proposals from data modelers and vendors for common formats to store the data.

The project also plans to write application program interfaces or APIs that make it possible for software developers to easily access the data in the common format. A Neurodata Without Borders Hackathon — an intensive, hands-on codefest to write software — is planned for 20-22 November at HHMI’s Janelia Farms in Ashburn, Virginia.

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Allied Minds, Bristol-Myers Squibb Form Life Science Venture

Drug screening samples

(Bristol-Myers Squibb)

4 August 2014. Allied Minds, a research commercialization company in Boston, and the pharmaceutical company Bristol-Myers Squibb in New York are starting a joint venture to discover new drug candidates and enterprises from promising life science research in U.S. university labs. Financial terms of the new venture, named Allied-Bristol Life Sciences LLC, were not disclosed.

Allied Minds acts as a holding company for science and technology-based start-ups in the U.S. The company forms new businesses based on research conducted in the U.S. at university and federally sponsored labs. Allied Minds then provides funding and management for the new enterprises through their initial stages. The company says it has relationships with 33 universities and 32 labs and research centers affiliated with the U.S. defense and energy departments.

Under the deal, Allied-Bristol Life Sciences will identify research discoveries at university labs with commercial potential, and form start-ups to develop those discoveries from early feasibility stages to preclinical therapy candidates. The joint venture will also provide capital for these initial stages. Once a discovery reaches drug candidate status, Bristol-Myers Squibb will have the option to acquire the enterprise, under agreed-upon terms, which were not disclosed.

In June 2013, Allied Minds reported raising $100 million for its next series of start-ups from discoveries in university and federal research labs. The company also has two subsidiaries similar to Allied-Bristol Life Sciences for the identification and development of new medical devices, and to commercialize discoveries in labs supported by U.S. government agencies. It currently supports 18 enterprises in the U.S.

Bristol-Myers Squibb says it will make available its drug discovery expertise to the university researchers. “We believe this new venture will enhance the translation of early-stage academic research,” says Carl Decicco who heads drug discovery at Bristol-Myer Squibb in a statement by the companies, “and will ultimately help advance important potential new medicines more efficiently.”

This venture is not Bristol-Myers Squibb’s first collaboration with academic research labs. In May 2012, the company established a network with 10 universities and research institutes in the U.S. and Europe to investigate opportunities in cancer immunotherapy.

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Challenge Seeks Biomarkers for Neuropathic Pain

Neuron illustration (NIH)

(National Institute on Aging, NIH)

1 August 2014. A new challenge on InnoCentive is seeking biomarkers that help predict the effectiveness of pain-killing therapies for patients with neuropathic pain. The challenge requires a detailed written proposal due on 27 September 2014, with a payout of $25,000. Free registration is required to see the challenge details.

InnoCentive in Waltham, Massachusetts conducts open-innovation, crowdsourcing competitions for corporate and organization sponsors, in this case the pharmaceutical company Astra-Zeneca. Innocentive calls this type of competition a theoretical-licensing challenge that describes a proposed implementation of an idea, but has not yet been proven as a concept.

Neuropathic pain is caused by dysfunction or disorder in peripheral nerves, those found in motor or sensory functions, that feed into the central nervous system. The label applies to various kinds of conditions and syndromes, with a range of causes — e.g., diabetes, chemotherapy for cancer, phantom limb syndrome — that makes finding clear therapy targets difficult. Those same properties of neuropathic pain make it difficult to translate results from preclinical tests with lab animals into clear behavioral targets for clinical trials.

Astra-Zeneca therefore is taking a different strategy for addressing neuropathic pain that identifies new types of biomarkers as targets for therapies. These biomarkers should make it possible to monitor the physiology and nerve functions of patients with neuropathic pain, and better predict responses of patients to pain-killing treatments.

Responses to this type of challenge require written proposals, which will be reviewed by Astra-Zeneca, and usually include detailed requirements, specifications, and descriptions. In this challenge, Astra-Zeneca is seeking a non-exclusive license to develop the winning solution. Proposals not selected retain full intellectual-property rights following the evaluation period.

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Engineered Protein Detoxes Pesticides, Chemical Weapons

Jim Kim Montclare

Jim Kim Montclare (New York University)

1 August 2014. Biochemists at New York University synthesized a protein that removes the toxic elements of chemicals found in pesticides and some chemical warfare agents. The team led by Jin Kim Montclare at NYU’s engineering school published its findings last week in the journal ChemBioChem (paid subscription required).

The process, which includes use of computational modeling software, produces a synthesized protein that removes the toxicity of organophosphates, compounds found in many commercial pesticides and sarin, a nerve agent considered a weapon of mass destruction under international law. Organophosphates limit enzymes regulating a chemical in the nervous system called acetylcholine that carries signals between nerves and muscles. Without that regulating capability, acetylcholine builds up in the nerves, resulting in paralysis of muscles, including respiratory muscles, leading to suffocation and death.

Montclare and colleagues — including Richard Bonneau, a biology and computer science professor at NYU — engineered enzymes called phosphotriesterases, which in their natural state have properties that degrade organophosphates. But also in their natural state, phosphotriesterases are unstable; they easily lose their potency and breakdown under high temperatures.

The researchers thus sought a way of improving the stability of phosphotriesterases, while maintaining their ability to act on organophosphates. “We’ve known that phosphotriesterases had the power to detoxify these nerve agents,” says Montclare in a university statement, “but they were far too fragile to be used therapeutically.”

The NYU team turned to Rosetta, software for molecular modeling of protein structures that offers algorithms for analyzing molecular interactions and designing custom molecules. The researchers used Rosetta to identify mutations in fluoridated forms of phosphotriesterases, which they knew from earlier studies improved the protein’s folding, where amino acids in proteins take the shape that specifies their functions.

The mutations revealed through Rosetta improved the stability of phosphotriesterases even further, with one variation identified as pFF-F104A shown able to work at higher temperatures, and with enough stability to continue working for many days at room temperature. The university filed a provisional patent for the process that includes the computational design of the synthesized protein.

Montclare says the engineered protein can be the basis for therapies for farm workers overexposed to pesticides or victims of nerve gas attacks. It can also be formulated into chemicals for cleaning up or decommissioning chemical weapons stores that now require heat and caustic chemicals. “These proteins could accomplish that same task enzymatically,” notes Montclare, “without the need for reactors and formation of dangerous byproducts.”

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Early Trial Shows Parkinson’s Vaccine Creates Antibodies

Alpha-synuclein illustration

Alpha-synuclein illustration (Michael J. Fox Foundation)

31 July 2014. An early-stage clinical trial testing the safety of an experimental vaccine to treat Parkinson’s disease, shows the vaccine generates antibodies that fight the build-up of proteins in the brain associated with the disorder. The vaccine is made by the biotechnology company Affiris AG, which presented the results today at a press conference in New York with the Michael J. Fox Foundation that funds the vaccine’s early clinical trials.

Affiris, in Vienna, Austria, develops vaccine therapies with a technology based on peptides called epitopes, areas on molecules to which antibodies bind. The epitopes in Affiris vaccines connect to B-cells, white blood cells programmed to generate antibodies addressing a specific target.

That target in this case is alpha-synuclein proteins, the build-up of which in the brain are a pathology associated with Parkinson’s disease, a neurodegenerative disorder. The disease is believed to result from the loss of cells in the brain that produce dopamine, a chemical that helps control movement.

The build up of alpha-synuclein proteins are believed to damage neurons, or nerve cells in the brain. Affiris is developing a vaccine with the code-name PD01A that aims to control levels of alpha-synuclein in the brain, by protecting neurons from the build-up of this protein.

The clinical trial, held in Vienna, enrolled 32 patients between the ages of 40 and 65 with Parkinson’s disease, of which 12 patients each were given four injections each month of PD01A in doses of either 15 or 75 micrograms for a year. Another eight patients receiving medications to control Parkinson’s symptoms, considered the standard of care, served as a control group.

In the first results reported from the trial, Affiris says about half of the patients receiving the vaccine developed antibodies to alpha-synuclein, which the researchers found in serum samples, as well in the patients’ cerebrospinal fluid. The trial also measured changes in motor symptoms and cognition of the patients, for which the company reported more stabilization among the vaccinated patients than those not getting the vaccine. Affiris says the vaccine was safe and well-tolerated by the patients, the main measurement objective of the trial.

The company plans a follow-up study of patients receiving PD01A, checking for any adverse effects of the vaccine after another year, as well as the vaccine’s continued ability to generate antibodies and influence Parkinson’s symptoms. An intermediate-stage clinical trial is expected to begin recruitment in September.

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Pfizer Acquires Baxter Vaccines Business

Vaccination (U.S. Army Corps of Engineers)

(U.S. Army Corps of Engineers)

31 July 2014. Baxter International in Deerfield, Illinois sold its vaccines operations to Pfizer Inc. in New York for $635 million. The deal covers two current Baxter vaccines and part of a plant in Orth, Austria where the vaccines are produced.

One of the vaccines acquired by Pfizer, FSME-IMMUN, protects against tick-borne encephalitis, a viral infection affecting the central nervous system. The disease is spread by ticks, which are often hosted on rodents. Humans, however, can act as accidental hosts. The symptoms range from headaches, malaise and vomiting to confusion, sensory disturbances, and paralysis.

FSME-IMMUN is given as an injection with an adjuvant or booster. The vaccine is available in Europe, but recommended for travelers to Europe who may be visiting regions where ticks are more numerous, such as grasslands and wooded areas, including outdoor enthusiasts, hikers, and armed forces personnel.

The second Baxter vaccine is NeisVac-C that protects against meningitis from the group C meningococcal bacteria. NeisVac-C is made from inactivated extracts of group C bacteria. It is given as an injection and available outside the U.S.

Meningitis is an inflammation of the membranes surrounding the brain and spinal cord, which can be caused by bacteria or viruses, but bacterial meningitis is considered more serious. Early symptoms of headache, fever, and a stiff neck can lead to more serious complications including hearing loss, brain damage, kidney failure, or death.

Pfizer’s current vaccines program includes developing a vaccine for group B  meningococcal bacteria, as well as Staphylococcus aureus and Clostridium difficile bacteria associated with health care associated infections.

The companies expect the deal to close by the end of the year. Before then, Baxter expects vaccine sales of about $300 million, including $50 million in one-time milestone payments from government collaborations for developing influenza vaccines.

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Hat tip: FirstWord Pharma

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Biochemical Signaling Chip Design Techniques Devised

William Bentley

William Bentley (University of Maryland)

30 July 2014. Engineers at University of Maryland in College Park developed techniques for designing a chip device that controls biological functions of cells with electronic and biochemical signals. The team led by bioengineering professor William Bentley published its findings earlier this week in the journal Nature Nanotechnology (paid subscription required).

Bentley and colleagues are seeking ways to provide greater electronic controls on microfluidic chip devices that emulate physiological functions, even entire human organs. These devices are being developed to simplify and miniaturize medical lab tests, as well as test drugs for potential toxicity with more reliability than lab animals.

In their research, the team conducted a series of experiments to design and test a biological process with enzymes programmed on a gold microchip. The process programmed on the chip controls the amount of enzymes assembled and their activity, with cell signals from the chip tested on bacteria.

The process involves a protein assembled from multiple genes called (His)6-LuxS-Pfs-(Tyr)5 or HLPT, with which Bentley’s lab worked previously. The researchers send several levels of electric charge through chips with the HLPT protein, where the charge affects the amount of enzyme activity taking place. The resulting biochemical output of the chips is then measured through a signaling molecule called autoinducer-2, known to cue bacterial behavior, and tracked with reporter cells that fluoresce in a blue color.

One effect of autoinducer-2 signals is to stimulate quorum sensing where bacteria and other species coordinate their activities, often as a result of population density. The researchers found the bacteria, when sent the autoinducer-2 signals, exhibit more coordinated activity rather than acting as individual cell organisms. In addition, the team found the amount of electrical charge sent to the chip correlates directly with the amount biochemical signal it generates, and the level of bacterial cell activity.

This proof-of-concept study, say the authors, indicates it is possible to control bacterial communication with electrical signals, which opens up their use with microfluidic labs-on-chips measuring enzymes, cells, and other biological components emulating human functions. The university’s Biochip Collaborative, in which Bentley is a member, aims to design this kind of hybrid bio-electronic components embedded in microelectronic systems to interact with microfluidic devices.

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U.S. Patent Awarded for Prodrug Cancer Imaging, Treatment

USPTO building (


30 July 2014. A new patent was awarded for a technology that detects and treats solid tumor cancers with a prodrug, a precursor compound activated inside the body — made by GenSpera Inc., a biotechnology company in San Antonio. U.S. patent number 8,772,226 was awarded on 8 July to inventors Samuel Denmeade and John Isaacs of Johns Hopkins University and Søren Brøgger Christensen at University of Copenhagen in Denmark, and assigned to Johns Hopkins University. GenSpera, founded by Denmeade and Issacs, licenses the technology from Johns Hopkins.

The patent covers the use of thapsigargin, a substance derived from the thapsia plant, a weed grown in Mediterranean regions and toxic to sheep and cattle. GenSpera synthesizes a form of thapsigargin that remains dormant until activated by enzymes associated with the targeted cancers, both to attack the tumor as well as the supporting blood supply.

The patent also covers the activation mechanisms when thapsigargin comes into contact with the enzymes prostate specific membrane antigen (PSMA), prostate specific antigen (PSA), and human glandular kallikrein 2 (hK2). One or more of these enzymes are associated with liver, breast, brain, renal, and colon, as well as prostate cancer. The activation process employs peptides that in the presence of these enzymes open up to release the thapsigargin payload. Thapsigargin, says the company, kills tumor and supporting cells independently of the rate of cell division, and thus can be used to treat both slow and fast-growing cancers.

This activation process can be applied as well to detecting cancer, particularly in early stages or when tumors are growing slowly. Current imaging methods such as trans-rectal ultrasound or magnetic resonance spectroscopy are established  techniques for detecting prostate cancer, for example, which provide images of regions where the cancer may occur.

The patent instead describes a process for detecting and highlighting cancer cells rather than regions. The techniques in the patent label the prodrugs with radioactive isotopes, such as tritium, which in contact with the enzymes emit rays that illuminate to allow for imaging and detection.

GenSpera’s lead product, code-named G-202, is in intermediate-stage clinical trials as a treatment for liver cancer and glioblastoma, a form of brain cancer. A trial testing G-202 with prostate cancer completed early-stage trials.

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Genome Institute Grant Funds 23andMe Database Upgrades

DNA illustration


29 July 2014. National Human Genome Research Institute, part of National Institutes of Health, is funding enhancements to the research database capabilities of personal genetics company 23andMe in Mountain View, California. The two-year, $1.37 million project aims to help the company better mine its genetic and survey data collections for research connecting genomic variations to physical traits.

23andMe analyzes saliva samples and returns DNA analyses to reveal their customers’ ancestry. The analysis focuses on key genetic variations, called single nucleotide polymorphisms or SNPs that determine inherited traits, but can also trace ethnic heritage and even the presence of Neanderthal ancestors.

The company asks customers as well to complete online profiles and questionnaires about personal health, which makes it possible to associate SNP variations with physical traits including occurrence of disease. These associations are made in the aggregate, not for individuals; the company received a stiff warning from the Food and Drug Administration in November 2013 for making individual health assessments without the proper clearances from FDA.

The NHGRI grant is expected to help 23andMe upgrade its databases to make them more useful to genomic researchers. The enhancements include refinements to its current online questionnaires as well as adding 15 new questionnaires to its collection. The new surveys will be designed to improve longitudinal data analysis as well as add online tests to assess cognitive abilities.

The funding is expected as well to help 23andMe upgrade its capabilities to better accommodate whole genome sequencing data to help find new associations, particularly with rare genomic variations. This upgrade also aims to refine the company’s research accelerator program that offers geneticists access to its databases of SNPs and physical trait information, with personal identities removed.

The program is now in a pilot stage where a limited number of researchers are studying associations between genomic and physical trait variables. 23andMe says the upgrades should result in databases with survey data on diseases and traits from 400,000 of its customers, as well as 40 million SNPs.

An example of this research potential is a study published earlier this week in the journal Nature Genetics that analyzed genome-wide associations of more than 13,000 individuals with Parkinson’s disease compared to more than 95,000 individuals without the disorder. The analysis identified 6 genetic regions previously not associated with Parkinson’s disease, as well as confirming 24 known associations. 23andMe provided data from than 4,000 of its participants with Parkinson’s disease, and 62,000 without the condition for the study.

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