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The Power of Genomics: The Road Less Traveled

October 10th, 2016
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Landscape with fork rural roads in forest

You could say that we’re in an era of ambitious genomic projects and healthcare is getting smarter faster than ever. From Illumina’s plans to become the “Google of genetic testing” to pharmaceutical companies’ adoption of DNA sequencing as the foundation of their business strategy, an arms race is underway to sequence, commercialize, and democratize genetic information.

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Immuno-Oncology – Good Today, Great Tomorrow

May 2nd, 2016
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Researchers in the field of immuno-oncology have achieved remarkable success over the past five years. The ability to boost the immune system to attack cancer using a variety of biotechnology-derived approaches has led to the successful treatment of thousands of patients with advanced melanoma, lung cancer, and various types of leukemia. The success of these approaches has extended the lives of many patients, a significant number of whom have seemingly been cured. All of this has created a great deal of excitement and optimism among patients, researchers, and physicians after many years of frustration, false starts, and failed trials.

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Single Cell Biology: A Step Toward Precision Diagnostics

January 21st, 2016
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The past several years have seen a dramatic increase in the ability to isolate and characterize single cells – leading to advances in diagnostics, drug discovery, stem cell biology, cancer, and many other areas of biomedical research.

These advances have arisen thanks to growing capabilities in various single cell “omics” technologies – which have enabled RNA and DNA sequencing on a genome-wide scale (the interrogation of proteins, metabolites, and many other types of molecules that provide information about cellular growth, differentiation, and the underlying molecular basis of disease).

And why has single cell technology become so attractive within the biomedical research community? Because most studies are currently hampered by sample heterogeneity.

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Next-Gen Sequencing Could Unlock Ebola’s Secrets, Yield Outbreak-Halting Clues

September 23rd, 2014
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The Ebola outbreak in West Africa has dominated recent newscasts and shocked many people with the speed and ferocity of its spread. Any such outbreak raises questions about its origin, its rate of transmission, and measures that can be taken to control its propagation. Thus far more than 2,600 people have died and the numbers are rising. There currently is no cure or treatment, notwithstanding recent high profile reports of experimental drugs and vaccines that are under development. In the absence of treatment, the main goal of healthcare workers is to care for the sick and to limit the spread of the virus. And fortunately, researchers are now also applying the latest technologies to search for new ways to halt Ebola.

 

In an effort to better understand both the virus and the current outbreak, Pardis Sabeti and colleagues at the Broad Institute/Harvard University, and the Sierra Leone Ministry of Health and Sanitation recently reported in Science the genomic sequence of 99 Ebola isolates that were collected from 78 patients in Sierra Leone during the first 24 days of the outbreak. Reviewing this information will ultimately help in the development of both diagnostic tests and drugs to better manage the virus. The Ebola genome is just under 20,000 nucleotides in length, encoding just seven genes. The apparent simplicity of the virus belies the havoc that is wreaked upon infection based upon an assault on the immune system and disruption of the blood clotting process, leading often to fatal internal bleeding.

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Compound- to Target-Centric: A New View of Drug Discovery with H3 Biomedicine CEO Markus Warmuth

October 29th, 2012
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The cost and risk associated with biopharmaceutical R&D is enormous and the rewards are increasingly elusive. For many young firms, the “valley of death” is a very real obstacle. What can startups (and larger companies) do? In the first blog post based on my interview with H3 Biomedicine CEO Markus Warmuth, we discussed H3’s strategy to propel innovative therapies into the market using a tighter time frame based upon a new research and clinical paradigm that is almost the reverse of traditional biopharmaceutical development. In this segment, Warmuth shares with us some specifics about H3 Biomedicine’s unique approach to drug development.
 

Q: What is the current approach to drug discovery at H3?
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A Glimpse at Drug Discovery’s Future from H3 Biomedicine CEO Markus Warmuth

October 5th, 2012
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As many of you know, R&D progress in drug discovery has been a challenge for “big pharma” and biotech firms alike. The costs and risks are enormous, and the rewards are increasingly elusive. For many young firms, the “valley of death” is a very real obstacle as cash burn rates exceed the pace of successful delivery of new drugs to market. What can startups (and larger companies) do? Markus Warmuth, CEO of Cambridge, Massachusetts-based H3 Biomedicine, talked with us about his company’s strategies for giving drug discovery a much-needed boost.
 
H3 Biomedicine Inc. is a privately held, uniquely structured oncology discovery enterprise. H3 is applying the expertise of leading scientists to the integration of insights from cancer genomics with innovative capabilities in synthetic chemistry and tumor biology to discover patient-based, genomics-driven, small molecule drugs, which represent the most promising current opportunity in cancer therapeutics. H3 Biomedicine will achieve its goals through a unique relationship with the pharmaceutical company Eisai Inc. Eisai has pledged up to $200 million in research funding to H3 Biomedicine, as well as additional support for the clinical development of H3 Biomedicine programs.
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Drug Development Gets Even More Personal, Precise and Tailored

March 27th, 2012
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As we’ve discussed, Eric Topol’s “How to Change Medicine” provides valuable insights on how tailoring treatments in the clinic could boost health care effectiveness and lower costs. But we at Popper and Company would like to see industry take an additional step by using the techniques Dr. Topol recommends for patient care to develop drugs more efficiently.
 
The technology Topol recommends to stratify patients could also streamline drug development. In fact, the FDA is suggesting that faster approvals of antibiotics could result from smaller clinical trials that test antibiotics targeted against drug-resistant bacteria.
 
We now know enough about molecular biology that we can apply genomics/proteomics/metabolomics to drug development (DNA screening, identifying and validating surrogate markers, or characterizing tumors by genetic makeup). However, tailored treatments can run into challenges, including  heterogeneity in tumor cells as recently described in the New England Journal of Medicine, that resist even targeted biological treatments.
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Roche’s Proposed Takeover of Illumina—Game On for Universal DNA Sequencing?

February 3rd, 2012
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There appears to be a full-scale race underway to bring affordable next-gen DNA sequencing into diagnostics and clinical medicine, as demonstrated over the past week or so by Roche’s hostile $5.7 billion-dollar bid for Illumina.  Roche’s move should come as no shock. The multi-national healthcare giant has been a leader in diagnostics by virtue of several earlier acquisitions. But this action signifies a formal acknowledgment that next-gen sequencing will be part of the diagnostic and clinical toolbox—perhaps sooner than we thought.
 
Roche’s aggressive move may be motivated by an optimism that arose from whole genome-sequencing on the individual level. We noted this rising tide of optimism early last year, and many advances have been reported since then. We’re certainly seeing many examples of the application of next-gen sequencing to diagnose disease and to help match the right drugs to the right patients. Examples of companies that are staking claims over the early application of targeted sequencing of specific panels of genes for diagnostics include our client Multiplicom, which develops CE-marked, multiplexed PCR kits to generate templates for next-gen sequencing, and Foundation Medicine, which is using targeted sequencing to help diagnose certain cancers and to guide the treatment of cancer patients.
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Through the Murky Slog: Part II—A New Light for Cancer Therapies?

November 9th, 2011
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Last week, we discussed new drug approvals that reflected a paradigm shift for cancer drug development and for treatment of people living with cancer. Finally, we are starting to see a matchup of specific diagnostics with targeted biotherapeutics to address (effectively, we hope) targeted treatments for smaller groups of cancer patients.
 
Just recently, the American Society for Clinical Oncology (ASCO) urged the use of new biological knowledge to develop treatments faster, design more targeted clinical trials, and use information technology to integrate once-separate translational and clinical research. ASCO even says that targeted therapies can improve clinical trial responses from 8 to 30 percent. Also, just recently, the FDA gave itself a pat on the back as it highlighted its recent innovative drug approvals, with targeted cancer therapies included among those on the list.
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Seeking Holy Grail: Quest for Stem Cells from Embryos Continues

October 20th, 2011
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In a recent paper that spawned news articles and blogs worldwide, researchers announced the generation of human embryos that could produce stem cells. The announcement marks another step in the use of stem cells to treat a number of disorders, including diabetes, cancer, and neurodegenerative disease. But while this research, conducted by Dieter Egli, Scott Noggle, and their colleagues at the New York Stem Cell Foundation, holds much promise, it also demonstrates the daunting obstacles that block the path to stem cell therapy.
 
The biggest technical obstacle to stem cell therapy has been generating and isolating enough early-stage stem cells. It is these cells that have the greatest developmental potential because they can most readily differentiate into a mature cell that, in turn, could be used for cell therapy and regenerative medicine.
 
The discovery that adult cells could be “reprogrammed” into inducible pluripotent stem cells (iPSC) offered promises of stem cell therapy while sidestepping ethical issues arising from embryonic stem cell therapies. However, iPSCs presented technical issues that so far preclude their therapeutic potential. Bone marrow, cord blood, and adipose tissue also produce stem cells, but on an extremely limited basis.
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