Advertisements
Feeds:
Posts
Comments

Archive for the ‘Global Partnering & Biotech Investment’ Category


Huge Data Network Bites into Cancer Genomics

Larry H. Bernstein, MD, FCAP, Curator

LPBI

 

Closer to a Cure for Gastrointestinal Cancer

Suzanne Tracy, Editor-in-Chief, Scientific Computing and HPC Source
http://www.scientificcomputing.com/news/2015/11/closer-cure-gastrointestinal-cancer

In order to streamline workflows and keep pace with data-intensive discovery demands, CCS integrated its HPC environment with data capture and analytics capabilities, allowing data to move transparently between research steps, and driving discoveries such as a link between certain viruses and gastrointestinal cancers.

 

SANTA CLARA, CA — At the University of Miami’s Center for Computational Science (CCS), more than 2,000 internal researchers and a dozen expert collaborators across academic and industry sectors worldwide are working together in workflow management, data management, data mining, decision support, visualization and cloud computing. CCS maintains one of the largest centralized academic cyberinfrastructures in the country, which fuels vital and critical discoveries in Alzheimer’s, Parkinson’s, gastrointestinal cancer, paralysis and climate modeling, as well as marine and atmospheric science research.

In order to streamline workflows and keep pace with data-intensive discovery demands, CCS integrated its high performance computing (HPC) environment with data capture and analytics capabilities, allowing data to move transparently between research steps. To speed scientific discoveries and boost collaboration with researchers around the world, the center deployed high-performance DataDirect Networks (DDN) GS12K scale-out file storage. CCS now relies on GS12K storage to handle bandwidth-driven workloads while serving very high IOPS demand resulting from intense user interaction, which simplifies data capture and analysis. As a result, the center is able to capture, store and distribute massive amounts of data generated from multiple scientific models running different simulations on 15 Illumina HiSeq sequencers simultaneously on DDN storage. Moreover, number-crunching time for genome mapping and SNP calling has been reduced from 72 to 17 hours.

“DDN enabled us to analyze thousands of samples for the Cancer Genome Atlas, which amounts to nearly a petabyte of data,” explained Dr. Nicholas Tsinoremas, director of the Center for Computational Sciences at the University of Miami. “Having a robust storage platform like DDN is essential to driving discoveries, such as our recent study that revealed a link between certain viruses and gastrointestinal cancers. Previously, we couldn’t have done that level of computation.”

In addition to providing significant storage processing power to meet both high I/O and interactive processing requirements, CCS needed a flexible file system that could support large parallel and short serial jobs. The center also needed to address “data in flight” challenges that result from major data surges during analysis, and which often cause a 10x spike in storage. The system’s performance for genomics assembly, alignment and mapping is enabling CCS to support all its application needs, including the use of BWA and Bowtie for initial mapping, as well as SamTools and GATK for variant analysis and SNP calling.

“Our arrangement is to share data or make it available to anyone asking, anywhere in the world,” added Tsinoremas. “Now, we have the storage versatility to attract researchers from both within and outside the HPC community … we’re well-positioned to generate, analyze and integrate all types of research data to drive major scientific discoveries and breakthroughs.”

About DDN

DataDirect Networks is a big data storage supplier to data-intensive, global organizations. For more than 15 years, the company has designed, developed, deployed and optimized systems, software and solutions that enable enterprises, service providers, universities and government agencies to generate more value and to accelerate time to insight from their data and information, on premise and in the cloud. Organizations leverage DDN technology and the technical expertise of its team to capture, store, process, analyze, collaborate and distribute data, information and content at largest scale in the most efficient, reliable and cost effective manner. DDN customers include financial services firms and banks, healthcare and life science organizations, manufacturing and energy companies, government and research facilities, and web and cloud service providers.

 

“Where DDN really stood out is in the ability to adapt to whatever we would need. We have both IOPS-centric storage and the deep, slower I/O pool at full bandwidth. No one else could do that.”

Joel P. Zysman

Director of High Performance Computing

Center for Computational Science at the University of Miami

The University of Miami maintains one of the largest centralized, academic, cyber infrastructures in the US, which is integral to addressing and solving major scientific challenges. At its Center for Computational Science (CCS), more than 2,000 researchers, faculty, staff and students across multiple disciplines collaborate on diverse and interdisciplinary projects requiring HPC resources.

With 50% of the center’s users come from University of Miami’s Miller School of Medicine with ongoing projects at the Hussman Institute for Human Genomics, the explosion of next-generation sequencing has had a major impact on compute and storage demands. At CCS, the heavy I/O required to create four billion reads from one genome in a couple of days only intensifies when the data from the reads needs to be managed and analyzed

Aside from providing sufficient storage power to meet both high I/O and interactive processing demands, CCS needed a powerful file system that was flexible enough to handle very large parallel jobs as well as smaller, shorter serial jobs. CCS also needed to address as much as 10X spikes in storage, so it was critical to scale and support petabytes of machine-generated data without adding a layer of complexity or creating inefficiencies.

Read their success story to learn how high-performance DDN® Storage I/O has helped the University of Miami:

  • Establish links between certain viruses and gastrointestinal cancers discovered with computation that were not possible before
  • Reduce genomics compute and analysis time from 72 to 17 hours
CHALLENGES

  • Diverse, interdisciplinary research projects required massive compute and storage power as well as integrated data lifecycle movement and management
  • Highly demanding I/O and heavy interactivity requirements from next-gen sequencing intensified data generation, analysis and management
  • Handle large parallel jobs and smaller, shorter serial jobs
  • Data surges during analysis created “data-in-flight” challenges

SOLUTION

An end-to-end, high performance DDN GRIDScaler® solution featuring a GS12K™ scale-out appliance with an embedded IBM® GPFS™ parallel file system

TECHNICAL BENEFITS

  • Centralized storage with an embedded file system makes it easy to add storage where needed—in the high-performance, high-transaction or slower storage pools—and then manage it all through a single pane of glass
  • DDN’s transparent data movement enables using one platform for data capture, download, analysis and retention
  • The ability to maintain an active archive of storage lets the center accommodate different types of analytics with varied I/O needs
Advertisements

Read Full Post »


Point of Care Diagnostics

Larry H. Bernstein, MD, FCAP, Curator

LPBI

 

Point-of-Care Diagnostics—An Expanding Field Driven by Technology Development 

Impact of Various Disease Classes on the Development of POC Diagnostics

Gary Oosta, Ph.D.Enal Razvi, Ph.D.

Point-of-Care Diagnostics—An Expanding Field Driven by Technology Development

Figure 1. POC diagnostics word cloud

  • There is growing interest in the point-of-care (POC) diagnostics field as the space is expanding and evolving from pure technology development to applications development in various settings. We sought to understand the dynamics of the point-of-care diagnostics field by examining the entire ensemble of publications in this space and analyzing trends from the bottom-up.

  • Click Image To Enlarge +
    Figure 2. POC diagnostics is a global field.

    We harvested and analyzed 14,046 publications from the academic literature where the search terms were “point-of-care” in the titles and/or abstracts of the publications.  Figure 1 presents a word cloud that shows a pictorial representation of the abundance of various terms within this space as a means to illustrate the dataset which we harvested.  Most of the data was collected in 2015 and therefore it represents the current scenario of the field.

    We subsequently sought to understand the nature of the POC diagnostics field by examining the geographic source of these publications—is this a concentrated marketplace or is it truly global in nature, vis-à-vis research and publications in this field.  Figure 2 presents the geographic breakout and this clearly shows that POC diagnostics is a global field.

  • Click Image To Enlarge +
    Figure 3. Growth of publications in the POC diagnostics space.

    The U.S. dominates publications in this space with 39% of total but it’s important to note the breakout is truly global in nature and attests to the fragmented nature of research in this field.

    We also sought to understand the pace of growth of this field—if the hypothesis is that the POC diagnostics field is expanding we should observe a steep climb in the growth of publications—Figure 3 presents this data, which shows a clear expanding space and this begs the question as to where the growth in the field is coming from. To address this question, we examined the compound annual growth rates (CAGRs) of the various segments of the broader POC diagnostics space and this data (not presented in this article) suggest that specific segments of the POC space are outperforming the growth of the broader POC diagnostics space and there are opportunities for applications development in these particular spaces.

  • Click Image To Enlarge +
    Figure 4. Cardiovascular disease classes are an important component of the POC space.

    Infectious diseases are a strong theme in the overall POC diagnostics field and estimates suggest that infectious diseases account for 70% of the overall POC diagnostics marketplace.  Our industry research however also suggests that the oncology space is experiencing growth in the POC diagnostics arena and this suggests to us that the technology development taking place in POC diagnostics is starting to branch out and impact other market segments.  Another space we investigated as part of our industry analyses was the impact of POC diagnostics into the cardiovascular diseases space.  Figure 4 presents the publications landscape of POC diagnostics impacting various cardiovascular disease classes.

  • Click Image To Enlarge +
    Figure 5. In POC, specific biomarkers are an almost unexplored space.

    Even though POC diagnostics seeks to interrogate biological marker[s], the specific biomarker[s] that are implicated in POC diagnostics are generally not well characterized and indeed studies of biomarkers do not crossover into POC diagnostics.  We interpret this data to mean that the majority of the efforts in the POC diagnostics field are focused on technology development, approaches for deployment into the point-of-need setting rather than the molecular characterization of the biomarkers that are the underlying molecular entities associating the readout with the biological phenotype studied. This is especially true in the cancer biomarkers space where there is an extensive body of literature where specific biomarkers or signatures thereof are associated either as prognostic or diagnostic biomarkers with specific pathological states—such levels of association are not observed in the POC diagnostics field.  This will evolve over time and such molecular associations of the lesions will be described.  Figure 5 presents the publications landscape of specific biomarkers that have been described thus far in the POC diagnostics space.

  • Click Image To Enlarge +
    Figure 6a. In the POC space, the penetrance of cancer is growing, albeit small currently.

    POC diagnostics are suited ideally for high-impact diseases for which quick time to diagnosis and treatments are crucial, cardiovascular diseases for example as well as infectious disease where time to diagnosis is a key determinant of the therapeutic regimen that is deployed.  The broader cancer space impact on POC diagnostics is small at the present time, but growing.  We believe that the impact of minimally invasive diagnostics such as biofluid/liquid biopsies will increase the penetrance and impact of POC diagnostics into the cancer diagnostics space.

    The impact of molecular diagnostics tools onto the POC diagnostics space is expanding, and we believe that this trend will drive the interrogation of specific molecular species [biomarkers] in the POC context.  In this vein, we have noted that the penetrance of PCR amplification in POC diagnostics is growing suggesting the potential move of molecular testing, MDx, precision medicine into the POC diagnostics space.  We will continue to monitor this trend closely as the impact of nucleic acid analyses into POC diagnostics is an important theme and will enable precision medicine at the molecular level in the point-of-need setting.

    Earlier in this article, we alluded to the observation that in the point-of-care diagnostics setting cancer is currently a small contributor but is a growing space—Figure 6a illustrates this trend.

  • Click Image To Enlarge +
    Figure 6b. Segmentation of cancer classes by penetrance by POC diagnostics.

    We have further characterized this market segment and found that specific cancers are represented to various degrees—breast cancer is the most penetrant.  Figure 6bbreaks-down the various cancer classes vis-à-vis their penetrance into the POC diagnostics space and the trends observed are very interesting as they reflect on clinical practices in cancer screening as well as early diagnostics testing.  We seek to analyze this trend over the coming year to observe shifts in cancer POC diagnostics with the mounting interest in the liquid biopsy space—liquid biopsy can be viewed as a POC diagnostic since it is minimally invasive and can be performed in out-patient settings—a hallmark of the POC diagnostics space.

  • In summary, we have framed the current landscape of POC diagnostics, seeking to bring together various disease classes and describing their impact on the overall POC diagnostics field.  We continue to monitor this space as it evolves and is impacted by other fields, such as liquid biopsies, which will affect the character and dynamics of this space. We believe that not only will new technologies (such as microfluidics) drive the POC diagnostics field forward in resource-rich and resource-limited settings but also the impact of new biomarkers and biomarker classes will drive the field forward as more precise tailored-medicine becomes the norm in clinical practice.  Therefore even though currently POC diagnostics is deployed for high-impact diseases, the trend is moving toward its utilization in many niches of clinical unmet need.

  • Click Image To Enlarge +

    At the 2nd Annual Biofluid Biopsies and High Value Diagnostics 2015 conference Select Biosciences brings together academic researchers, industry researchers, as well as biotechnology and pharmaceutical companies to explore the expanding and evolving field whereby Circulating Biomarkers of various classes are being evaluated for their potential to be developed into diagnostics for cancer as well as other disease classes.

 

 

Read Full Post »


The Pharmaceutical Consulting Consortium International (PCCI) June Meeting: Envisage-Wistar Partnership and Immunacel LLC

An early stage healthcare venture creation and management firm

Presenter: Vic Subbu, COO of Immunacel & Managing Partner of Envisage and Heather Steinman, VP of Business Development & Executive Director Tech Transfer Wistar Institute

Monday, June 8, 2015

Embassy Suites, Chesterbrook, Pennsylvania (directions)

Announcement from the PCCI website:

Much has been said lately about how to improve the tech transfer situation. Wistar is meeting this challenge. Immunacel is the first of a series of developmental challenges and the Envisage-Wistar partnership solution becomes the meat of the evening’s discussion.

The Wistar Institute is the nation’s first independent institution devoted to medical research and training. The Wistar Institute has evolved from its beginnings as an anatomical teaching museum to its present-day status as an international leader in basic biomedical research.

Envisage LLC is an early stage healthcare venture creation and management firm. By focusing on key healthcare segments, Envisage aims to identify and advance promising healthcare innovations into value-add ventures.

IMMUNACCEL LLC is a Wistar Institute spin-out focused on accelerating the development of immune-mediated treatments for cancer and other unmet medical needs:

MMUNACCEL’s 3-D cancer-immune cell organotypic culture system is a physiologically relevant culture system utilizing primary human cancer cells and cytotoxic T cells (CTL) generated from patient T-cells, amongst fibroblasts and collagen assembled in a 3-D organotypic model.

Other related articles on PCCI and Philadelphia Biotech were published in this Open Access Online Scientific Journal, include the following:

PCCI’s 7th Annual Roundtable “Crowdfunding for Life Sciences: A Bridge Over Troubled Waters?” May 12 2014 Embassy Suites Hotel, Chesterbrook PA 6:00-9:30 PM

Protecting Your Biotech IP and Market Strategy: Notes from Life Sciences Collaborative 2015 Meeting

The Vibrant Philly Biotech Scene: Focus on KannaLife Sciences and the Discipline and Potential of Pharmacognosy

The Vibrant Philly Biotech Scene: Focus on Computer-Aided Drug Design and Gfree Bio, LLC

The Vibrant Philly Biotech Scene: Focus on Vaccines and Philimmune, LLC

The Bioscience Crowdfunding Environment: The Bigger Better VC?

R&D Alliances between Big Pharma and Academic Research Centers: Pharma’s Realization that Internal R&D Groups alone aren’t enough

BIO Partnering: Intersection of Academic and Industry: BIO INTERNATIONAL CONVENTION June 23-26, 2014 | San Diego, CA

Diagnostics and Biomarkers: Novel Genomics Industry Trends vs Present Market Conditions and Historical Scientific Leaders Memoirs

Read Full Post »


Icelandic Population Genomic Study Results by deCODE Genetics come to Fruition: Curation of Current genomic studies

Reporter/Curator: Stephen J. Williams, Ph.D.

 

UPDATED on 9/6/2017

On 9/6/2017, Aviva Lev-Ari, PhD, RN had attend a talk by Paul Nioi, PhD, Amgen, at HMS, Harvard BioTechnology Club (GSAS).

Nioi discussed his 2016 paper in NEJM, 2016, 374:2131-2141

Variant ASGR1 Associated with a Reduced Risk of Coronary Artery Disease

Paul Nioi, Ph.D., Asgeir Sigurdsson, B.Sc., Gudmar Thorleifsson, Ph.D., Hannes Helgason, Ph.D., Arna B. Agustsdottir, B.Sc., Gudmundur L. Norddahl, Ph.D., Anna Helgadottir, M.D., Audur Magnusdottir, Ph.D., Aslaug Jonasdottir, M.Sc., Solveig Gretarsdottir, Ph.D., Ingileif Jonsdottir, Ph.D., Valgerdur Steinthorsdottir, Ph.D., Thorunn Rafnar, Ph.D., Dorine W. Swinkels, M.D., Ph.D., Tessel E. Galesloot, Ph.D., Niels Grarup, Ph.D., Torben Jørgensen, D.M.Sc., Henrik Vestergaard, D.M.Sc., Torben Hansen, Ph.D., Torsten Lauritzen, D.M.Sc., Allan Linneberg, Ph.D., Nele Friedrich, Ph.D., Nikolaj T. Krarup, Ph.D., Mogens Fenger, Ph.D., Ulrik Abildgaard, D.M.Sc., Peter R. Hansen, D.M.Sc., Anders M. Galløe, Ph.D., Peter S. Braund, Ph.D., Christopher P. Nelson, Ph.D., Alistair S. Hall, F.R.C.P., Michael J.A. Williams, M.D., Andre M. van Rij, M.D., Gregory T. Jones, Ph.D., Riyaz S. Patel, M.D., Allan I. Levey, M.D., Ph.D., Salim Hayek, M.D., Svati H. Shah, M.D., Muredach Reilly, M.B., B.Ch., Gudmundur I. Eyjolfsson, M.D., Olof Sigurdardottir, M.D., Ph.D., Isleifur Olafsson, M.D., Ph.D., Lambertus A. Kiemeney, Ph.D., Arshed A. Quyyumi, F.R.C.P., Daniel J. Rader, M.D., William E. Kraus, M.D., Nilesh J. Samani, F.R.C.P., Oluf Pedersen, D.M.Sc., Gudmundur Thorgeirsson, M.D., Ph.D., Gisli Masson, Ph.D., Hilma Holm, M.D., Daniel Gudbjartsson, Ph.D., Patrick Sulem, M.D., Unnur Thorsteinsdottir, Ph.D., and Kari Stefansson, M.D., Ph.D.

N Engl J Med 2016; 374:2131-2141June 2, 2016DOI: 10.1056/NEJMoa1508419

Abstract
Article
References
Citing Articles (22)
Metrics

BACKGROUND

Several sequence variants are known to have effects on serum levels of non–high-density lipoprotein (HDL) cholesterol that alter the risk of coronary artery disease.

METHODS

We sequenced the genomes of 2636 Icelanders and found variants that we then imputed into the genomes of approximately 398,000 Icelanders. We tested for association between these imputed variants and non-HDL cholesterol levels in 119,146 samples. We then performed replication testing in two populations of European descent. We assessed the effects of an implicated loss-of-function variant on the risk of coronary artery disease in 42,524 case patients and 249,414 controls from five European ancestry populations. An augmented set of genomes was screened for additional loss-of-function variants in a target gene. We evaluated the effect of an implicated variant on protein stability.

RESULTS

We found a rare noncoding 12-base-pair (bp) deletion (del12) in intron 4 of ASGR1, which encodes a subunit of the asialoglycoprotein receptor, a lectin that plays a role in the homeostasis of circulating glycoproteins. The del12 mutation activates a cryptic splice site, leading to a frameshift mutation and a premature stop codon that renders a truncated protein prone to degradation. Heterozygous carriers of the mutation (1 in 120 persons in our study population) had a lower level of non-HDL cholesterol than noncarriers, a difference of 15.3 mg per deciliter (0.40 mmol per liter) (P=1.0×10−16), and a lower risk of coronary artery disease (by 34%; 95% confidence interval, 21 to 45; P=4.0×10−6). In a larger set of sequenced samples from Icelanders, we found another loss-of-function ASGR1 variant (p.W158X, carried by 1 in 1850 persons) that was also associated with lower levels of non-HDL cholesterol (P=1.8×10−3).

CONCLUSIONS

ASGR1 haploinsufficiency was associated with reduced levels of non-HDL cholesterol and a reduced risk of coronary artery disease. (Funded by the National Institutes of Health and others.)

 

Amgen’s deCODE Genetics Publishes Largest Human Genome Population Study to Date

Mark Terry, BioSpace.com Breaking News Staff reported on results of one of the largest genome sequencing efforts to date, sequencing of the genomes of 2,636 people from Iceland by deCODE genetics, Inc., a division of Thousand Oaks, Calif.-based Amgen (AMGN).

Amgen had bought deCODE genetics Inc. in 2012, saving the company from bankruptcy.

There were a total of four studies, published on March 25, 2015 on the online version of Nature Genetics; titled “Large-scale whole-genome sequencing of the Icelandic population[1],” “Identification of a large set of rare complete human knockouts[2],” “The Y-chromosome point mutation rate in humans[3]” and “Loss-of-function variants in ABCA7 confer risk of Alzheimer’s disease[4].”

The project identified some new genetic variants which increase risk of Alzheimer’s disease and confirmed some variants known to increase risk of diabetes and atrial fibrillation. A more in-depth post will curate these findings but there was an interesting discrete geographic distribution of certain rare variants located around Iceland. The dataset offers a treasure trove of meaningful genetic information not only about the Icelandic population but offers numerous new targets for breast, ovarian cancer as well as Alzheimer’s disease.

View Mark Terry’s article here on Biospace.com.

“This work is a demonstration of the unique power sequencing gives us for learning more about the history of our species,” said Kari Stefansson, founder and chief executive officer of deCode and one of the lead authors in a statement, “and for contributing to new means of diagnosing, treating and preventing disease.”

The scale and ambition of the study is impressive, but perhaps more important, the research identified a new genetic variant that increases the risk of Alzheimer’s disease and already had identified an APP variant that is associated with decreased risk of Alzheimer’s Disease. It also confirmed variants that increase the risk of diabetes and a variant that results in atrial fibrillation.
The database of human genetic variation (dbSNP) contained over 50 million unique sequence variants yet this database only represents a small proportion of single nucleotide variants which is thought to exist. These “private” or rare variants undoubtedly contribute to important phenotypes, such as disease susceptibility. Non-SNV variants, like indels and structural variants, are also under-represented in public databases. The only way to fully elucidate the genetic basis of a trait is to consider all of these types of variants, and the only way to find them is by large-scale sequencing.

Curation of Population Genomic Sequencing Programs/Corporate Partnerships

Click on “Curation of genomic studies” below for full Table

Curation of genomic studies
Study Partners Population Enrolled Disease areas Analysis
Icelandic Genome

Project

deCODE/Amgen Icelandic 2,636 Variants related to: Alzheimer’s, cardiovascular, diabetes WES + EMR; blood samples
Genome Sequencing Study Geisinger Health System/Regeneron Northeast PA, USA 100,000 Variants related to hypercholestemia, autism, obesity, other diseases WES +EMR +MyCode;

– Blood samples

The 100,000 Genomes Project National Health Service/NHS Genome Centers/ 10 companies forming Gene Consortium including Abbvie, Alexion, AstraZeneca, Biogen, Dimension, GSK, Helomics, Roche,   Takeda, UCB Rare disorders population UK Starting to recruit 100,000 Initially rare diseases, cancer, infectious diseases WES of blood, saliva and tissue samples

Ref paper

Saudi Human Genome Program 7 centers across Saudi Arabia in conjunction with King Abdulaziz City Science & Tech., King Faisal Hospital & Research Centre/Life Technologies General population Saudi Arabia 20,000 genomes over three years First focus on rare severe early onset diseases: diabetes, deafness, cardiovascular, skeletal deformation Whole genome sequence blood samples + EMR
Genome of the Netherlands (GoNL) Consortium consortium of the UMCG,LUMCErasmus MCVU university and UMCU. Samples where contributed by LifeLinesThe Leiden Longevity StudyThe Netherlands Twin Registry (NTR), The Rotterdam studies, and The Genetic Research in Isolated Populations program. All the sequencing work is done by BGI Hong Kong. Families in Netherlands 769 Variants, SNV, indels, deletions from apparently healthy individuals, family trios Whole genome NGS of whole blood no EMR

Ref paper in Nat. Genetics

Ref paper describing project

Faroese FarGen project Privately funded Faroe Islands Faroese population 50,000 Small population allows for family analysis Combine NGS with EMR and genealogy reports
Personal Genome Project Canada $4000.00 fee from participants; collaboration with University of Toronto and SickKids Organization; technical assistance with Harvard Canadian Health System Goal: 100,000 ? just started no defined analysis goals yet Whole exome and medical records
Singapore Sequencing Malay Project (SSMP) Singapore Genome Variation Project

Singapore Pharmacogenomics Project

Malaysian 100 healthy Malays from Singapore Pop. Health Study Variant analysis Deep whole genome sequencing
GenomeDenmark four Danish universities (KU, AU, DTU and AAU), two hospitals (Herlev and Vendsyssel) and two private firms (Bavarian Nordic and BGI-Europe). 150 complete genomes; first 30 published in Nature Comm. ? See link
Neuromics Consortium University of Tübingen and 18 academic and industrial partners (see link for description) European and Australian 1,100 patients with neuro-

degenerative and neuro-

muscular disease

Moved from SNP to whole exome analysis Whole Exome, RNASeq

References

  1. Gudbjartsson DF, Helgason H, Gudjonsson SA, Zink F, Oddson A, Gylfason A, Besenbacher S, Magnusson G, Halldorsson BV, Hjartarson E et al: Large-scale whole-genome sequencing of the Icelandic population. Nature genetics 2015, advance online publication.
  2. Sulem P, Helgason H, Oddson A, Stefansson H, Gudjonsson SA, Zink F, Hjartarson E, Sigurdsson GT, Jonasdottir A, Jonasdottir A et al: Identification of a large set of rare complete human knockouts. Nature genetics 2015, advance online publication.
  3. Helgason A, Einarsson AW, Gumundsdottir VB, Sigursson A, Gunnarsdottir ED, Jagadeesan A, Ebenesersdottir SS, Kong A, Stefansson K: The Y-chromosome point mutation rate in humans. Nature genetics 2015, advance online publication.
  4. Steinberg S, Stefansson H, Jonsson T, Johannsdottir H, Ingason A, Helgason H, Sulem P, Magnusson OT, Gudjonsson SA, Unnsteinsdottir U et al: Loss-of-function variants in ABCA7 confer risk of Alzheimer’s disease. Nature genetics 2015, advance online publication.

Other post related to DECODE, population genomics, and NGS on this site include:

Illumina Says 228,000 Human Genomes Will Be Sequenced in 2014

CRACKING THE CODE OF HUMAN LIFE: The Birth of BioInformatics & Computational Genomics

CRACKING THE CODE OF HUMAN LIFE: The Birth of BioInformatics and Computational Genomics – Part IIB

Human genome: UK to become world number 1 in DNA testing

Synthetic Biology: On Advanced Genome Interpretation for Gene Variants and Pathways: What is the Genetic Base of Atherosclerosis and Loss of Arterial Elasticity with Aging

Genomic Promise for Neurodegenerative Diseases, Dementias, Autism Spectrum, Schizophrenia, and Serious Depression

Sequencing the exomes of 1,100 patients with neurodegenerative and neuromuscular diseases: A consortium of 18 European and Australian institutions

University of California Santa Cruz’s Genomics Institute will create a Map of Human Genetic Variations

Three Ancestral Populations Contributed to Modern-day Europeans: Ancient Genome Analysis

Impact of evolutionary selection on functional regions: The imprint of evolutionary selection on ENCODE regulatory elements is manifested between species and within human populations

Read Full Post »


Presentations Content for All Business Track Panels and the Scientific Panel on Immunotherapy @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

 REAL TIME Curator: Aviva Lev-Ari, PhD, RN

9:00 am – 9:30 am 3/26/2015  Welcome Remarks & MassBio Board Elections @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/22/900-am-930-am-welcome-remarks-massbio-board-elections-massbio-annual-meeting-2015-cambridge-ma-326-327-2015/

9:30 am – 10:15 am 3/26/2015, Keynote: Kathy Giusti, Founder & Executive Chairman of the Multiple Myeloma Research Foundation, @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/930-am-1015-am-3262015-live-keynote-kathy-guisti-founder-executive-chairman-of-the-multiple-myeloma-research-foundation-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel/

10:30 am – 11:30 am, 3/26/2015 – LIVE Better Business Track: It’s Not Your Grandfather’s Manufacturing @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/1030-am-1130-am-3262015-better-business-track-its-not-your-grandfathers-manufacturing-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

11:45 am – 1:30 pm, 3/26/2015,  LIVE – The MassBio Annual Awards Luncheon @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/1145-am-130-pm-3262015-the-massbio-annual-awards-luncheon-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

1:30 pm – 2:20 pm 3/26/2015, Precision Medicine: Who’s Paying? @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/130-pm-220-pm-3262015-live-precision-medicine-whos-paying-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

2:25 pm – 3:15 pm 3/26/2015 Better Business Track: Externalizing Pharma R&D @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/225-pm-315-pm-3262015-better-business-track-externalizing-pharma-rd-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

3:30 pm – 4:20 pm LIVE Trends in Science Track: Immunotherapy – Oncology and Beyond @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/26/330-pm-420-pm-3262015-trends-in-science-track-immunotherapy-oncology-and-beyond-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

8:40 am – 9:30 am   3/27/2015  Better Business Track: Innovative Ways to Fund Your Early-Stage Company @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/27/840-am-930-am-3272015-live-better-business-track-innovative-ways-to-fund-your-early-stage-company-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

9:40 am – 10:30 am 3/27/2015 Better Business Track: The Evolving Reimbursement Landscape @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/27/940-am-1030-am-3272015-live-better-business-track-the-evolving-reimbursement-landscape-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

11:00 am – 12:00 pm, 3/27/2015 Defining Value @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/27/1100-am-1200-pm-3272015-live-defining-value-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

12:45 pm – 1:30 pm  3/27/2015  LIVE Keynote: Andrew Lo, Director of the MIT Laboratory for Financial Engineering  @ MassBio Annual Meeting 2015, Cambridge, MA, Sonesta Hotel, 3/26 – 3/27, 2015

https://pharmaceuticalintelligence.com/2015/03/27/1245-pm-130-pm-3272015-live-keynote-andrew-lo-director-of-the-mit-laboratory-for-financial-engineering-massbio-annual-meeting-2015-cambridge-ma-sonesta-hotel-326-327-2015/

Read Full Post »

Protecting Your Biotech IP and Market Strategy: Notes from Life Sciences Collaborative 2015 Meeting


 

Protecting Your Biotech IP and Market Strategy: Notes from Life Sciences Collaborative 2015 Meeting

Achievement Beyond Regulatory Approval – Design for Commercial Success

philly2nightStephen J. Williams, Ph.D.: Reporter

The Mid-Atlantic group Life Sciences Collaborative, a select group of industry veterans and executives from the pharmaceutical, biotechnology, and medical device sectors whose mission is to increase the success of emerging life sciences businesses in the Mid-Atlantic region through networking, education, training and mentorship, met Tuesday March 3, 2015 at the University of the Sciences in Philadelphia (USP) to discuss post-approval regulatory issues and concerns such as designing strong patent protection, developing strategies for insurance reimbursement, and securing financing for any stage of a business.

The meeting was divided into three panel discussions and keynote speech:

  1. Panel 1: Design for Market Protection– Intellectual Property Strategy Planning
  2. Panel 2: Design for Market Success– Commercial Strategy Planning
  3. Panel 3: Design for Investment– Financing Each Stage
  4. Keynote Speaker: Robert Radie, President & CEO Egalet Corporation

Below are Notes from each PANEL Discussion:

For more information about the Life Sciences Collaborative SEE

Website: http://www.lifesciencescollaborative.org/

Or On Facebook

Or On Twitter @LSCollaborative

Panel 1: Design for Market Protection; Intellectual Property Strategy Planning

Take-home Message: Developing a very strong Intellectual Property (IP) portfolio and strategy for a startup is CRITICALLY IMPORTANT for its long-term success. Potential investors, partners, and acquirers will focus on the strength of a startup’s IP so important to take advantage of the legal services available. Do your DUE DIGILENCE.

Panelists:

John F. Ritter, J.D.., MBA; Director Office Tech. Licensing Princeton University

Cozette McAvoy; Senior Attorney Novartis Oncology Pharma Patents

Ryan O’Donnell; Partner Volpe & Koenig

Panel Moderator: Dipanjan “DJ” Nag, PhD, MBA, CLP, RTTP; President CEO IP Shaktl, LLC

Notes:

Dr. Nag:

  • Sometimes IP can be a double edged sword; e.g. Herbert Boyer with Paul Berg and Stanley Cohen credited with developing recombinant technology but they did not keep the IP strict and opened the door for a biotech revolution (see nice review from Chemical Heritage Foundation).
  • Naked patent licenses are most profitable when try to sell IP

John Ritter: Mr. Ritter gave Princeton University’s perspective on developing and promoting a university-based IP portfolio.

  • 30-40% of Princeton’s IP portfolio is related to life sciences
  • Universities will prefer to seek provisional patent status as a quicker process and allows for publication
  • Princeton will work closely with investigators to walk them through process – Very Important to have support system in place INCLUDING helping investigators and early startups establish a STRONG startup MANAGEMENT TEAM, and making important introductions to and DEVELOPING RELATIONSHIOPS with investors, angels
  • Good to cast a wide net when looking at early development partners like pharma
  • Good example of university which takes active role in developing startups is University of Pennsylvania’s Penn UPstart program.
  • Last 2 years many universities filing patents for startups as a micro-entity

Comment from attendee: Universities are not using enough of their endowments for purpose of startups. Princeton only using $500,00 for accelerator program.

Cozette McAvoy: Mrs. McAvoy talked about monetizing your IP from an industry perspective

  • Industry now is looking at “indirect monetization” of their and others IP portfolio. Indirect monetization refers to unlocking the “indirect value” of intellectual property; for example research tools, processes, which may or may not be related to a tangible product.
  • Good to make a contractual bundle of IP – “days of the $million check is gone”
  • Big companies like big pharma looks to PR (press relation) buzz surrounding new technology, products SO IMPORTANT FOR STARTUP TO FOCUS ON YOUR PR

Ryan O’Donnell: talked about how life science IP has changed especially due to America Invests Act

  • Need to develop a GLOBAL IP strategy so whether drug or device can market in multiple countries
  • Diagnostics and genes not patentable now – Major shift in patent strategy
  • Companies like Unified Patents can protect you against the patent trolls – if patent threatened by patent troll (patent assertion entity) will file a petition with the USPTO (US Patent Office) requesting institution of inter partes review (IPR); this may cost $40,000 BUT WELL WORTH the money – BE PROACTIVE about your patents and IP

Panel 2: Design for Market Success; Commercial Strategy Planning

Take-home Message: Commercial strategy development is defined market facing data, reimbursement strategies and commercial planning that inform labeling requirements, clinical study designs, healthcare economic outcomes and pricing targets. Clarity from payers is extremely important to develop any market strategy. Develop this strategy early and seek advice from payers.

Panelists:

David Blaszczak; Founder, Precipio Health Strategies

Terri Bernacchi, PharmD, MBA; Founder & President Cambria Health Advisory Professionals

Paul Firuta; President US Commercial Operations, NPS Pharma

 

Panel Moderator: Matt Cabrey; Executive Director, Select Greater Philadelphia

 

Notes:

David Blaszczak:

  • Commercial payers are bundling payment: most important to get clarity from these payers
  • Payers are using clinical trials to alter marketing (labeling) so IMPORTANT to BUILD LABEL in early clinical trial phases (phase I or II)
  • When in early phases of small company best now to team or partner with a Medicare or PBM (pharmacy benefit manager) and payers to help develop and spot tier1 and tier 2 companies in their area

Terri Bernacchi:

  • Building relationship with the payer is very important but firms like hers will also look to patients and advocacy groups to see how they respond to a given therapy and decrease the price risk by bundling
  • Value-based contracting with manufacturers can save patient and payer $$
  • As most PBMs formularies are 80% generics goal is how to make money off of generics
  • Patent extension would have greatest impact on price, value

Paul Firuta:

  • NPS Pharma developing a pharmacy benefit program for orphan diseases
  • How you pay depends on mix of Medicare, private payers now
  • Most important change which could affect price is change in compliance regulations

Panel 3: Design for Investment; Financing Each Stage

Take-home Message: VC is a personal relationship so spend time making those relationships. Do your preparation on your value and your market. Look to non-VC avenues: they are out there.

Panelists:

Ting Pau Oei; Managing Director, Easton Capital (NYC)

Manya Deehr; CEO & Founder, Pediva Therapeutics

Sanjoy Dutta, PhD; Assistant VP, Translational Devel. & Intl. Res., Juvenile Diabetes Research Foundation

 

Panel Moderator: Shahram Hejazi, PhD; Venture Partner, BioAdvance

  • In 2000 his experience finding 1st capital was what are your assets; now has changed to value

Notes:

Ting Pau Oei:

  • Your very 1st capital is all about VALUE– so plan where you add value
  • Venture Capital is a PERSONAL RELATIONSHIP
  • 1) you need the management team, 2) be able to communicate effectively                  (Powerpoint, elevator pitch, business plan) and #1 and #2 will get you important 2nd Venture Capital meeting; VC’s don’t decide anything in 1st meeting
  • VC’s don’t normally do a good job of premarket valuation or premarket due diligence but know post market valuation well
  • Best advice: show some phase 2 milestones and VC will knock on your door

Manya Deehr:

  • Investment is more niche oriented so find your niche investors
  • Define your product first and then match the investors
  • Biggest failure she has experienced: companies that go out too early looking for capital

Dr. Dutta: funding from a non-profit patient advocacy group perspective

  • Your First Capital: find alliances which can help you get out of “valley of death
  • Develop a targeted product and patient treatment profile
  • Non-profit groups ask three questions:

1) what is the value to patients (non-profits want to partner)

2) what is your timeline (we can wait longer than VC; for example Cystic Fibrosis Foundation waited long time but got great returns for their patients with Kalydeco™)

3) when can we see return

  • Long-term market projections are the knowledge gaps that startups have (the landscape) and startups don’t have all the competitive intelligence
  • Have a plan B every step of the way

Other posts on this site related to Philadelphia Biotech, Startup Funding, Payer Issues, and Intellectual Property Issues include:

PCCI’s 7th Annual Roundtable “Crowdfunding for Life Sciences: A Bridge Over Troubled Waters?” May 12 2014 Embassy Suites Hotel, Chesterbrook PA 6:00-9:30 PM
The Vibrant Philly Biotech Scene: Focus on KannaLife Sciences and the Discipline and Potential of Pharmacognosy
The Vibrant Philly Biotech Scene: Focus on Computer-Aided Drug Design and Gfree Bio, LLC
The Vibrant Philly Biotech Scene: Focus on Vaccines and Philimmune, LLC
The Bioscience Crowdfunding Environment: The Bigger Better VC?
Foundations as a Funding Source
Venture Capital Funding in the Life Sciences: Phase4 Ventures – A Case Study
10 heart-focused apps & devices are crowdfunding for American Heart Association’s open innovation challenge
Funding, Deals & Partnerships
Medicare Panel Punts on Best Tx for Carotid Plaque
9:15AM–2:00PM, January 27, 2015 – Regulatory & Reimbursement Frameworks for Molecular Testing, LIVE @Silicon Valley 2015 Personalized Medicine World Conference, Mountain View, CA
FDA Commissioner, Dr. Margaret A. Hamburg on HealthCare for 310Million Americans and the Role of Personalized Medicine
Biosimilars: Intellectual Property Creation and Protection by Pioneer and by Biosimilar Manufacturers
Litigation on the Way: Broad Institute Gets Patent on Revolutionary Gene-Editing Method
The Patents for CRISPR, the DNA editing technology as the Biggest Biotech Discovery of the Century

 

 

Read Full Post »


The Vibrant Philly Biotech Scene: Focus on KannaLife Sciences and the Discipline and Potential of Pharmacognosy

Curator and Interviewer: Stephen J. Williams, Ph.D.

 

philly2nightThis post is the third in a series of posts highlighting interviews with Philadelphia area biotech startup CEO’s and show how a vibrant biotech startup scene is evolving in the city as well as the Delaware Valley area. Philadelphia has been home to some of the nation’s oldest biotechs including Cephalon, Centocor, hundreds of spinouts from a multitude of universities as well as home of the first cloned animal (a frog), the first transgenic mouse, and Nobel laureates in the field of molecular biology and genetics. Although some recent disheartening news about the fall in rankings of Philadelphia as a biotech hub and recent remarks by CEO’s of former area companies has dominated the news, biotech incubators like the University City Science Center and Bucks County Biotechnology Center as well as a reinvigorated investment community (like PCCI and MABA) are bringing Philadelphia back. And although much work is needed to bring the Philadelphia area back to its former glory days (including political will at the state level) there are many bright spots such as the innovative young companies as outlined in these posts.

In today’s post, I had the opportunity to talk with both Dr. William Kinney, Chief Scientific Officer and Thoma Kikis, Founder/CMO of KannaLife Sciences based in the Pennsylvania Biotech Center of Bucks County.   KannaLifeSciences, although highlighted in national media reports and Headline news (HLN TV)for their work on cannabis-derived compounds, is a phyto-medical company focused on the discipline surrounding pharmacognosy, the branch of pharmacology dealing with natural drugs and their constituents.

Below is the interview with Dr. Kinney and Mr. Kikis of KannaLife Sciences and Leaders in Pharmaceutical Business Intelligence (LPBI)

 

PA Biotech Questions answered by Dr. William Kinney, Chief Scientific Officer of KannaLife Sciences

 

 

LPBI: Your parent company   is based in New York. Why did you choose the Bucks County Pennsylvania Biotechnology Center?

 

Dr. Kinney: The Bucks County Pennsylvania Biotechnology Center has several aspects that were attractive to us.  They have a rich talent pool of pharmaceutically trained medicinal chemists, an NIH trained CNS pharmacologist,  a scientific focus on liver disease, and a premier natural product collection.

 

LBPI: The Blumberg Institute and Natural Products Discovery Institute has acquired a massive phytochemical library. How does this resource benefit the present and future plans for KannaLife?

 

Dr. Kinney: KannaLife is actively mining this collection for new sources of neuroprotective agents and is in the process of characterizing the active components of a specific biologically active plant extract.  Jason Clement of the NPDI has taken a lead on these scientific studies and is on our Advisory Board. 

 

LPBI: Was the state of Pennsylvania and local industry groups support KannaLife’s move into the Doylestown incubator?

 

Dr. Kinney: The move was not State influenced by state or industry groups. 

 

LPBI: Has the partnership with Ben Franklin Partners and the Center provided you with investment opportunities?

 

Dr. Kinney: Ben Franklin Partners has not yet been consulted as a source of capital.

 

LPBI: The discipline of pharmacognosy, although over a century old, has relied on individual investigators and mainly academic laboratories to make initial discoveries on medicinal uses of natural products. Although there have been many great successes (taxol, many antibiotics, glycosides, etc.) many big pharmaceutical companies have abandoned this strategy considering it a slow, innefective process. Given the access you have to the chemical library there at Buck County Technology Center, the potential you had identified with cannabanoids in diseases related to oxidative stress, how can KannaLife enhance the efficiency of finding therapeutic and potential preventive uses for natural products?

 

Dr. Kinney: KannaLife has the opportunity to improve upon natural molecules that have shown medically uses, but have limitations related to safety and bioavailability. By applying industry standard medicinal chemistry optimization and assay methods, progress is being made in improving upon nature.  In addition KannaLife has access to one of the most commercially successful natural products scientists and collections in the industry.

 

LPBI: How does the clinical & regulatory experience in the Philadelphia area help a company like Kannalife?

 

Dr. Kinney: Within the region, KannaLife has access to professionals in all areas of drug development either by hiring displaced professionals or partnering with regional contract research organizations.

 

LPBI  You are focusing on an interesting mechanism of action (oxidative stress) and find your direction appealing (find compounds to reverse this, determine relevant disease states {like HCE} then screen these compounds in those disease models {in hippocampal slices}).  As oxidative stress is related to many diseases are you trying to develop your natural products as preventative strategies, even though those type of clinical trials usually require massive numbers of trial participants or are you looking to partner with a larger company to do this?

 

Dr. Kinney: Our strategy is to initially pursue Hepatic Encephalophy (HE) as the lead orphan disease indication and then partner with other organizations to broaden into other areas that would benefit from a neuroprotective agent.  It is expected the HE will be responsive to an acute treatment regimen.   We are pursuing both natural products and new chemical entities for this development path.

 

 

General Questions answered by Thoma Kikis, Founder/CMO of KannaLife Sciences

 

LPBI: How did KannaLife get the patent from the National Institutes of Health?

 

My name is Thoma Kikis I’m the co-founder of KannaLife Sciences. In 2010, my partner Dean Petkanas and I founded KannaLife and we set course applying for the exclusive license of the ‘507 patent held by the US Government Health and Human Services and National Institutes of Health (NIH). We spent close to 2 years working on acquiring an exclusive license from NIH to commercially develop Patent 6,630,507 “Cannabinoids as Antioxidants and Neuroprotectants.” In 2012, we were granted exclusivity from NIH to develop a treatment for a disease called Hepatic Encephalopathy (HE), a brain liver disease that stems from cirrhosis.

 

Cannabinoids are the chemicals that compose the Cannabis plant. There are over 85 known isolated Cannabinoids in Cannabis. The cannabis plant is a repository for chemicals, there are over 400 chemicals in the entire plant. We are currently working on non-psychoactive cannabinoids, cannabidiol being at the forefront.

 

As we started our work on HE and saw promising results in the area of neuroprotection we sought out another license from the NIH on the same patent to treat CTE (Chronic Traumatic Encephalopathy), in August of 2014 we were granted the additional license. CTE is a concussion related traumatic brain disease with long term effects mostly suffered by contact sports players including football, hockey, soccer, lacrosse, boxing and active military soldiers.

 

To date we are the only license holders of the US Government held patent on cannabinoids.

 

 

LPBI: How long has this project been going on?

 

We have been working on the overall project since 2010. We first started work on early research for CTE in early-2013.

 

 

LPBI: Tell me about the project. What are the goals?

 

Our focus has always been on treating diseases that effect the Brain. Currently we are looking for solutions in therapeutic agents designed to reduce oxidative stress, and act as immuno-modulators and neuroprotectants.

 

KannaLife has an overall commitment to discover and understand new phytochemicals. This diversification of scientific and commercial interests strongly indicates a balanced and thoughtful approach to our goals of providing standardized, safer and more effective medicines in a socially responsible way.

 

Currently our research has focused on the non-psychoactive cannabidiol (CBD). Exploring the appropriate uses and limitations and improving its safety and Metered Dosing. CBD has a limited therapeutic window and poor bioavailability upon oral dosing, making delivery of a consistent therapeutic dose challenging. We are also developing new CBD-like molecules to overcome these limitations and evaluating new phytochemicals from non-regulated plants.

 

KannaLife’s research is led by experienced pharmaceutically trained professionals; Our Scientific team out of the Pennsylvania Biotechnology Center is led by Dr. William Kinney and Dr. Douglas Brenneman both with decades of experience in pharmaceutical R&D.

 

 

LPBI: How do cannabinoids help neurological damage? -What sort of neurological damage do they help?

 

Cannabinoids and specifically cannabidiol work to relieve oxidative stress, and act as immuno-modulators and neuroprotectants.

 

So far our pre-clinical results show that cannabidiol is a good candidate as a neuroprotectant as the patent attests to. Our current studies have been to protect neuronal cells from toxicity. For HE we have been looking specifically at ammonia and ethanol toxicity.

 

 

– How did it go from treating general neurological damage to treating CTE? Is there any proof yet that cannabinoids can help prevent CTE? What proof?

 

We started examining toxicity first with ammonia and ethanol in HE and then posed the question; If CBD is a neuroprotectant against toxicity then we need to examine what it can do for other toxins. We looked at CTE and the toxin that causes it, tau. We just acquired the license in August from the NIH for CTE and are beginning our pre-clinical work in the area of CTE now with Dr. Ron Tuma and Dr. Sara Jane Ward at Temple University in Philadelphia.

 

 

LPBI: How long until a treatment could be ready? What’s the timeline?

 

We will have research findings in the coming year. We plan on filing an IND (Investigational New Drug application) with the FDA for CBD and our molecules in 2015 for HE and file for CTE once our studies are done.

 

 

LPBI: What other groups are you working with regarding CTE?

 

We are getting good support from former NFL players who want solutions to the problem of concussions and CTE. This is a very frightening topic for many players, especially with the controversy and lawsuits surrounding it. I have personally spoken to several former NFL players, some who have CTE and many are frightened at what the future holds.

 

We enrolled a former player, Marvin Washington. Marvin was an 11 year NFL vet with NY Jets, SF 49ers and won a SuperBowl on the 1998 Denver Broncos. He has been leading the charge on KannaLife’s behalf to raise awareness to the potential solution for CTE.

 

We tried approaching the NFL in 2013 but they didn’t want to meet. I can understand that they don’t want to take a position. But ultimately, they’re going to have to make a decision and look into different research to treat concussions. They have already given the NIH $30 Million for research into football related injuries and we hold a license with the NIH, so we wanted to have a discussion. But currently cannabinoids are part of their substance abuse policy connected to marijuana. Our message to the NFL is that they need to lead the science, not follow it.

 

Can you imagine the NFL’s stance on marijuana treating concussions and CTE? These are topics they don’t want to touch but will have to at some point.

 

LPBI: Thank you both Dr. Kinney and Mr. Kikis.

 

Please look for future posts in this series on the Philly Biotech Scene on this site

Also, if you would like your Philadelphia biotech startup to be highlighted in this series please contact me or

http://pharmaceuticalintelligence.com at:

sjwilliamspa@comcast.net or @StephenJWillia2  or @pharma_BI.

Our site is read by ~ thousand international readers DAILY and thousands of Twitter followers including venture capital.

 

Other posts on this site in this VIBRANT PHILLY BIOTECH SCENE SERIES OR referring to PHILADELPHIA BIOTECH include:

The Vibrant Philly Biotech Scene: Focus on Computer-Aided Drug Design and Gfree Bio, LLC

RAbD Biotech Presents at 1st Pitch Life Sciences-Philadelphia

The Vibrant Philly Biotech Scene: Focus on Vaccines and Philimmune, LLC

What VCs Think about Your Pitch? Panel Summary of 1st Pitch Life Science Philly

1st Pitch Life Science- Philadelphia- What VCs Really Think of your Pitch

LytPhage Presents at 1st Pitch Life Sciences-Philadelphia

Hastke Inc. Presents at 1st Pitch Life Sciences-Philadelphia

PCCI’s 7th Annual Roundtable “Crowdfunding for Life Sciences: A Bridge Over Troubled Waters?” May 12 2014 Embassy Suites Hotel, Chesterbrook PA 6:00-9:30 PM

Pfizer Cambridge Collaborative Innovation Events: ‘The Role of Innovation Districts in Metropolitan Areas to Drive the Global an | Basecamp Business

Mapping the Universe of Pharmaceutical Business Intelligence: The Model developed by LPBI and the Model of Best Practices LLC

 

 

Read Full Post »

« Newer Posts - Older Posts »