TITAN: NASA Solar system exploration

Titan іѕ bigger thаn Earth’s moon, аnd еvеn larger thаn thе planet Mercury. Titan іѕ thе оnlу moon іn оur solar ѕуѕtеm thаt hаѕ clouds аnd a dense atmosphere, аnd thе оnlу world apart frоm Earth wіth liquids оn іtѕ surface.

Saturn’s largest moon Titan іѕ аn extraordinary аnd exceptional world. Amоng оur solar system’s mоrе thаn 150 known moons, Titan іѕ thе оnlу оnе wіth a substantial atmosphere. And оf аll thе places іn thе solar ѕуѕtеm, Titan іѕ thе оnlу place bеѕіdеѕ Earth known tо hаvе liquids іn thе fоrm оf rivers, lakes аnd seas оn іtѕ surface.

Titan іѕ larger thаn thе planet Mercury аnd іѕ thе second largest moon іn оur solar ѕуѕtеm. Jupiter’s moon Ganymede іѕ just a little bit larger (by аbоut 2 percent). Titan’s atmosphere іѕ mаdе mоѕtlу оf nitrogen, like Earth’s, but wіth a surface pressure 50 percent higher thаn Earth’s. Titan hаѕ clouds, rain, rivers, lakes аnd seas оf liquid hydrocarbons like methane аnd ethane. Thе largest seas аrе hundreds оf feet deep аnd hundreds оf miles wide. Beneath Titan’s thісk crust оf water ice іѕ mоrе liquid—an ocean primarily оf water rаthеr thаn methane. Titan’s subsurface water соuld bе a place tо harbor life аѕ wе know іt, whіlе іtѕ surface lakes аnd seas оf liquid hydrocarbons соuld conceivably harbor life thаt uses different chemistry thаn we’re used to—that іѕ, life аѕ wе don’t уеt know іt. Titan соuld аlѕо bе a lifeless world.

Did Yоu Know?

Aѕ exotic аѕ Titan mіght sound, іn ѕоmе wауѕ it’s оnе оf thе mоѕt hospitable worlds іn thе solar ѕуѕtеm. Titan’s nitrogen atmosphere іѕ ѕо dense thаt a human wouldn’t need a pressure suit tо walk аrоund оn thе surface. Hе оr ѕhе wоuld, hоwеvеr, need аn oxygen mask аnd protection аgаіnѕt thе cold—temperatures аt Titan’s surface аrе аrоund minus 290 degrees Fahrenheit (minus 179 Celsius).

Titan’s dense atmosphere, аѕ wеll аѕ gravity roughly equivalent tо Earth’s Moon, mеаn thаt a raindrop falling thrоugh Titan’s sky wоuld fall mоrе slowly thаn оn Earth. Whіlе Earth rain falls аt аbоut 20 miles реr hour (9.2 meters реr second), scientists hаvе calculated thаt rain оn Titan falls аt аbоut 3.5 miles реr hour (1.6 meters реr second), оr аbоut ѕіx tіmеѕ mоrе slowly thаn Earth’s rain. Titan’s raindrops саn аlѕо bе pretty large. Thе maximum diameter оf Earth raindrops іѕ аbоut 0.25 inches (6.5 millimeters) whіlе raindrops оn Titan саn reach diameters оf 0.37 inches (9.5 millimeters), оr аbоut 50 percent larger thаn аn Earth raindrop.

Pop Culture

Titan wаѕ famously featured іn thе 2009 film “Star Trek”—the U.S.S. Enterprise соmеѕ оut оf warp іn Titan’s atmosphere іn order tо sneak uр оn thе Romulan ship attacking Earth. Titan аlѕо appeared іn thе film “Star Trek Nemesis,” аѕ wеll аѕ аn episode оf “Star Trek: Thе Nеxt Generation.” Sеvеrаl оthеr television shows аnd films feature Titan, ѕuсh аѕ thе film “Gattaca,” аnd thе shows “Futurama” аnd “Eureka,” аѕ wеll аѕ thе iconic anime series “Cowboy Bebop.” Dozens оf video games аlѕо feature Titan, аѕ dо ѕеvеrаl comics frоm Marvel аnd DC, аmоng оthеrѕ.

Titan hаѕ bееn a setting оr subject іn dozens оf short stories аnd novels bу giants оf science fiction including Arthur C. Clarke, Philip K. Dick, аnd Isaac Asimov, аѕ wеll аѕ legendary “Star Trek” actress Nichelle Nichols. Evеn literary greats ѕuсh аѕ Kurt Vonnegut hаvе included Titan іn thеіr fiction.

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Company Overview

The Company Overview

Contacts

Riku Rautsola, PhD
President and Chief Executive Officer
riku@virxsys.com

Gerard Fleury,
EVP and CFO
gerard@virxsys.com

Gary McGarrity, PhD
EVP Scientific and Clinical Affairs
gmcgarrity@virxsys.com
Address
VIRxSYS Corporation
200 Perry Parkway, Suite 1A
Gaithersburg, MD 20877
United States

Telephone
301-987-0480

Fax
301-987-0489

Year Founded
1998

Financial Summary
Fundraising and Strategic Outlook
The Company has raised more than $80 million in six previous private equity rounds of financing since 1999, through Signature Capital Securities, LLP. The most recent round of fundraising will fund the Company through the completion of Phase II clinical trials for VRX496, a novel gene delivery therapy for HIV.

Company Profile
The Company, founded in Gaithersburg, Maryland, in 1998, is a private biotechnology company whose mission is to develop gene-based therapies using its proprietary lentiviral vector-based delivery system. The first application of the Company’s novel gene delivery technology has been for the treatment of HIV, the virus that causes AIDS. After a successful first-in-class Phase I clinical trial that demonstrated the safety profile of the Company’s product VRX496, this new ex-vivo anti-HIV treatment using a lentivector is currently being evaluated in the first and only Phase II clinical trial approved by the FDA involving a lentiviral vector. The Company’s lentiviral vector platform is an ideal delivery system for the effective treatment of disease. The Company’s vector technology is also being investigated for applications in anti-cancer therapies and for the treatment of other various life-threatening diseases.

Technology
The Company has developed a proprietary HIV-based lentiviral vector platform for delivery of genetic therapeutic payloads. Its first product, VRX496, for treatment of HIV, may offer the patients a long-term alternative to antiretroviral drug regimens. Traditional drug regimens are toxic and produce drug resistance strains of HIV. The Company believes that its therapy is non-toxic and solves the resistance issue thus enabling patients to live drug free. This has the potential to represent a significant improvement in the quality of life for people with HIV compared to that with the current antiretroviral drug treatments. The Company’s manufacturing capabilities are unparalleled in terms of large-scale clinical grade vector production and cell processing (including purification of vector, and transduction and expansion of patient cells). The Company has developed a stable cell line, which will make clinical grade vector production more compatible with commercialization. The Company has several therapies in preclinical development that apply the Company’s technology to the treatment of other diseases, such as cancer.

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Company Milestones

VIRxSYS has taken proof-of-concept studies performed at The Johns Hopkins University, completed its Phase I clinical trials in humans, and has initiated Phase II clinical trials in less than seven years. Company milestones are as follows:

Date Milestone
1998 VIRxSYS incorporated
1999 First round of fundraising completed
2000 Initial meeting with the FDA to discuss the VIRxSYS lentiviral vector, VRX496
2001
January 2001 Second round of financing completed
August 2001 Pre-clinical studies completed toward an Investigational New Drug (IND) application
September 2001 VIRxSYS Phase I protocol for VRX496 presented to the NIH Recombinant Advisory Committee (RAC) for public review
October 2001 VRX496 presented to the FDA Biological Response Modifier Advisory Committee (BRMAC) for formal protocol review
2002
April 2002 Third round of financing completed
August 2002 IND application submitted to the FDA for final review of VIRxSYS’s proposed Phase I clinical trial for VRX496
December 2002 VIRxSYS granted permission by the FDA to proceed with the Phase I clinical trial for VRX496 modified CD4 T cells as an anti-HIV therapy; this clinical trial is a first-in-class use of lentiviral vectors in humans for any indication
2003
January 2003 Final approval received from The Institutional Review Board (IRB) at the University of Pennsylvania for Phase I clinical trial to be held at that institution
July 2003 First patient in the Phase I clinical trial dosed with VRX496 modified CD4 T cells
2004
April 2004 VIRxSYS awarded $1.5 million by the NIH for construction of a cell processing center and completion of important safety studies for VRX496 insertion into CD4 T cells
July 2004 Fourth round of financing completed
July 2004 “Fast Track” status granted to VIRxSYS by the FDA for VRX496
September 2004 State-of-the-art cell processing center completed in Gaithersburg, MD
September 2004 Final patient of the Phase I trial dosed
2005
May 2005 All patients in the Phase I trial reached nine-month post infusion, the official end of the trial; participants continue to be monitored
September 2005 Phase II Trial to establish safety and tolerability of multiple infusions of VRX496 commenced
October 2005 Fifth round of financing completed
2006
July 2006 Multiple infusions of VRX496 completed for all patients receiving multiple doses in Phase II trial, demonstrating the safety and tolerability of multiple doses of VRX496
July 2006 Patient from Phase I trial shows safety of treatment and persistence of vector at three years post infusion
September 2006 Phase I/II Clinical trial commenced at the University of Pennsylvania to test the safety, tolerability, and efficacy of VRX496 in HIV-positive patients who are well-controlled by traditional drug regimens
September 2006 Phase I/II clinical trial approved for the  treatment of HIV positive patients who are not on drug therapy, to be conducted at Harvard University, The University of Pennsylvania, and The University of Amsterdam;  AIDS Clinical Trial Group funds received for clinical trial sites in the United States
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Product Pipeline

VIRxSYS has developed a proprietary HIV-based lentiviral vector gene delivery system, from which the disease-causing aspects of the virus have been removed, leaving behind an efficient delivery vehicle.

In order to provide a therapy against HIV, VIRxSYS has equipped the vector with a long antisense sequence against the HIV envelope protein to create VRX496.  The Company is currently evaluating VRX496, the first application of our lentiviral vector delivery platform, in Phase II clinical trials.  The progress made in developing VRX496 has led us to begin development of other therapies to administer to patients via our lentiviral vector system.

Development of additional methods of administration will allow us to treat a broad range of serious human diseases.  VIRxSYS is currently establishing three main approaches for administering therapies to patients via its lentiviral vector:

  1. CD4 T cell Immunotherapy – In this process, lymphocytes are extracted from a patient, and those cells are purified to select CD4 T cells. The lentiviral vector containing a designated gene or other therapy is transduced (inserted) into the CD4 T cells.  The T cells that are genetically modified by the lentiviral vector are reinfused back into the patient.
    Current therapy application: VRX496 for HIV/AIDS
  2. Hematopoietic Stem Cell – In this process, stem cells are extracted from a patient and the lentiviral vector containing a designated gene or other therapy is transduced (inserted) into the cells.  The stem cells that are genetically modified by the lentiviral vector are reinfused back into the patient.  These cells replicate and each of the progeny carry the modified genes.
    Potential therapy application: Cancer
  3. Direct Injectable – VIRxSYS is currently working on developing a method for direct injection of the lentiviral gene delivery vector.
    Potential therapy application: Vaccine

VIRxSYS has various collaborations to develop additional therapies for HIV, cancer, and genetic diseases with The Johns Hopkins University, University of Pennsylvania, and private companies in the United States and Europe.

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Investor Information

VIRxSYS has raised over $80 million through six rounds of financing thanks to the support of our investors.  Without their continued support and enthusiasm, the Company would not be able to develop technology that delivers on the promise of genetic medicine, allowing for the treatment of serious human diseases.

Technology Advancement
VIRxSYS intends to develop technology internally, as well as acquire technology which fit an SSD strategy:

  • Strategic – must help us drive toward our mission
  • Synergistic – must take advantage of our core competency in lentiviral vectors
  • Disruptive – must provide a significant and unique therapeutic advantage


Business Model
VIRxSYS’s strategy is to develop gene therapy treatments for serious diseases and advance each of those therapies into the market along one of three avenues:

  1. Exclusive licensing to, or partnering with, a major bio-pharmaceutical company
  2. Non-exclusive licensing to a bio-pharmaceutical company, retaining co-promotion or co-marketing rights for the United States
  3. Go-it-alone strategy whereby VIRxSYS will take the lead in sales, marketing and distribution


The “go to market” track will depend on a number of factors including, but not limited to:

  • Size of the market
  • Capital requirements to take to market
  • Complexity of the manufacturing and distribution channels
  • Strength of potential partners


Each disease indication will be evaluated to determine the most appropriate track.

Initial Market Opportunity
Conventional drug therapies for HIV-positive people in the industrialized world are estimated to be more than $6 billion.  When ancillary medical costs are factored in, the cost of treating HIV/AIDS more than doubles to $13 billion each year.  Despite the enormous cost of these treatments, toxicities inherent in the current antiretroviral drugs available make them undesirable for long-term use.  Genetic medicine holds promise for well-tolerated, effective therapies to combat HIV.

In addition to HIV/AIDS, VIRxSYS is researching similar applications of the Company’s proprietary technology toward other life-threatening diseases such as cancer.  With the National Institutes of Health estimating the cost of cancer to be close to $190 billion each year in direct and indirect medical costs and lost productivity, there is a clear need for innovative solutions.

These examples represent a significant opportunity to deliver on the promise of genetic medicine for the millions of people suffering from HIV/AIDS and cancer, and with the technology being researched at VIRxSYS, the Company is poised to do just that.

If you are interested in finding out more information about VIRxSYS, contact: infinityofme@gmail.com.

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Viral Vector Technology for Gene Therapy

Getting to the Root Cause of Disease
Gene therapy is the treatment of hereditary or infectious diseases by repairing or re-engineering the genome. The therapy’s goal is to treat the genetic cause of disease rather than merely treating the symptoms. The advantage of gene therapy over traditional treatments is the elimination or reduction of the toxic side effects that are commonly seen with drugs. Examples of diseases caused by genetic flaws include cancer (genetic flaws in genes that control cell growth), Parkinson’s disease (genetic flaw in a critical neuronal gene) and infectious disease (foreign genetic material invades human cells; i.e. HIV, and HCV).

Solving the Delivery Challenge
The field of gene therapy holds great promise, however, most of these promises have been unfulfilled due to challenges in developing a transport system capable of delivering genetic and therapeutic payloads into targeted cells efficiently, reproducibly, and permanently.

Vectors are vehicles that deliver genetic material into cells. Viral vectors use the backbone of viruses for this purpose. Viruses are very effective at getting into cells, so the first challenge is to remove the disease-causing elements from the virus and leave behind a natural delivery vehicle. VIRxSYS’s HIV-based lentiviral vector platform has achieved this goal and has also overcome other existing challenges, such as high-efficiency gene transfer, stable transfer of genetic material into dividing and non-dividing cells, and a reduced risk of immunogenicity and insertional oncogenesis (cancer). VIRxSYS created VRX496 for the first application of our vector against the target disease, HIV/AIDS. To see how VIRxSYS is using gene therapy to treat HIV click here to view an animation.

VRX496 is created by removing the disease components of HIV and inserting an anti-HIV therapeutic payload, called antisense. The Company has demonstrated that such a vector can inhibit HIV replication by more than 99% (Humeau 2004).

HIV infects human cells very efficiently and, consequently, a vector derived from HIV has these remarkable properties for efficient gene delivery. VIRxSYS has demonstrated that its HIV lentiviral vector can deliver therapeutic payloads into human cells with greater than 90% efficiency, which is remarkable compared with other gene therapy vectors that have reported an efficiency of 20-50%. VIRxSYS’ vector efficiency can be achieved in several important human hematopoietic cell types such as lymphocytes, hematopoietic CD34+ cells, monocyte-derived dendritic cells, and a panel of tumor cell lines.

VIRxSYS is the only company currently testing a lentiviral vector permitted for use by the FDA in Phase II clinical trials. To date, patients in the Company’s clinical trials have experienced no adverse events due to treatment, thus supporting the safety of the Company’s vector for applications in humans.

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Advisory Boards

Medical Advisory Board
VIRxSYS has assembled a Medical Advisory Board comprised of experts in HIV medicine.  The Medical Advisory Board offers guidance in the design and execution of the Company’s HIV clinical trials, and guidance in positioning VRX496.  The members of the Medical Advisory Board include:

Member Affiliation 
Brigitte Autran, MD, PhD
Groupe Hospitalier Pitie-Salpetriere
Paris, France
Steven Deeks, MD  University of California at San Francisco
San Francisco, California
Carl June, MD University of Pennsylvania
Philadelphia, Pennsylvania
 Christine Katlama, MD Groupe Hospitalier Pitie-Salpetriere
Paris, France
 Daniel Kuritzkes, MD  Harvard University
Cambridge, Massachusetts
 Joep Lange, MD  Academic Medical Centre
Amsterdam, The Netherlands
 Julio Montaner, MD  British Columbia Centre for Excellence in HIV/AIDS
Vancouver, British Columbia
 Robert Murphy, MD  Northwestern University
Evanston, Illinois
 Richard Pollard, MD  University of California at Davis
Sacramento, California
 John Sullivan, MD  University of Massachusetts Medical Center
Worcester, Massachusetts
 Andrew Zolopa, MD  Stanford University
Stanford, California
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Intellectual Property

VIRxSYS has developed an extensive portfolio of intellectual property which is protected by a combination of patents, trade secrets, and trademark law. Below is a summary of the Company’s parent patents in the United States.  VIRxSYS also has many foreign counterparts in countries around the world.

Patent/Ser No. Issue / Publication Date Title
US 5,885,806 March 23, 1999 Methods to Prepare Conditionally Replicating Viral Vectors
US 6,114,141 September 5, 2000 Method to Express Genes from Viral Vectors
09/667,893 Filed September 22, 2000 Conditionally Replicating Vectors and Methods for Their Production and Use
US 6,168,953 January 2, 2001 Genetic Antiviral Agents and Methods for Their Use
US 6,207,426 March 27, 2001 Conditionally Replicating Viral Vectors and their Use
US 6,410,257 June 25, 2002 Method to Express Genes from Viral Vectors
US 6,498,033 December 24, 2002 Lentiviral Vectors
US 6,627,442 September 30, 2003 Methods for a Stable Transduction of Cells with HIV-Derived Viral Vectors
US 20040203017 October 14, 2004 High-Throughput Methods for Identifying Gene Function using Lentiviral Vectors
US 6,835,568 December 28, 2004 Regulated Nucleic Acid Expression System
US 20050123514 June 9, 2005 Increased Transduction Using ABC Transporter Substrates and/or Inhibitors
US 20060003452 June 30, 2005 Vector Packaging Cell Line
US 20050196381 September 8, 2005 Two-vector Complementary Systems for Generating Immune Responses
11/424,673 Filed October 20, 2005 Antibody Complexes
US 20050257277 November 17, 2005 Regulation of Transcription with a Cis-acting Ribozyme
10/587,437 Filed May 22, 2006 Transduction of Primary Cells
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Manufacturing Facilities

VIRxSYS is the only company currently testing a lentiviral vector permitted for use by the FDA in Phase II clinical trials. Consequently, VIRxSYS is uniquely positioned to manufacture clinical-grade lentiviral vectors for therapies treating a wide range of life-threatening diseases.

VIRxSYS is initially focused on using lentiviral vectors in cellular therapies for HIV/AIDS.

VIRxSYS both manufactures clinical grade lentiviral vectors and performs cell processing for clinical trials at its Gaithersburg, Maryland facility. Our manufacturing facility includes two class 10,000 clean rooms for vector reproduction and patient cell processing. In addition, the company has a dedicated plasmid production room, a suite of quality control labs, and a variety of support rooms.

In addition to our manufacturing facilities, VIRxSYS has extensive research and devlopment facilites and capabilities dedicated to supporting the manufacturing and cinical trial initiatives, as well as to developing a pipeline of therapies for additional disease indications.

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