"We are excited to have reached this important milestone in the clinical evaluation of INXN-4001 for treatment of end-stage heart failure," stated Amit Patel, MD, MS, Co-Founder and Medical Director of TripleGene. "Heart failure rarely results from a single genetic defect, and while single gene therapy approaches have been studied, these treatments may not fully address the causes of the disease. Our unique multigenic approach is designed to stimulate biological activity targeting multiple points in the disease progression pathway."
Triple-Gene's investigational therapy uses non-viral delivery of a constitutively expressed multigenic plasmid designed to express human S100A1, SDF-1, and VEGF165 gene products, which affect progenitor cell recruitment, angiogenesis, and calcium handling, respectively, and target the underlying molecular mechanisms of pathological myocardial remodeling. The plasmid therapy is delivered via RCSI which allows for cardiac-specific delivery to the ventricle.
"Heart failure is the leading cause of death worldwide and represents a significant and growing global health problem. Aside from heart transplant and LVAD, current treatment options for those patients with end-stage disease are limited," commented Timothy Henry, MD, FACC, MSCAI, Medical Director of the Carl and Edyth Lindner Center for Research and Education at The Christ Hospital and a member of the Triple-Gene Medical Advisory Board. "The INXN4001 investigational therapy represents a biologically-based method focused on repairing the multiple malfunctions of cardiomyocytes, and I look forward to seeing the results of this initial safety study and further exploring the promise of this innovative treatment approach."
Triple-Gene will present preliminary data from the Phase 1 study at theAmerican Heart Association Scientific Sessionsat the Pennsylvania Convention Center in Philadelphia. A poster titled "Safety of First in Human Triple-Gene Therapy Candidate for Heart Failure Patients" will be presented on Sunday, November 17thfrom 3:00 pm - 3:30 pm ETin Zone 4 of the Science and Technology Hall.
About the Phase 1 Trial of INXN-4001INXN-4001 is being evaluated in a Phase I open label study in adult patients with implanted Left Ventricular Assist Device (LVAD). The study is designed to investigate the safety and feasibility of supplemental cardiac expression of S100A1, SDF-1 and VEGF-165 from a single, multigenic plasmid delivered via Retrograde Coronary Sinus Infusion (RCSI) in stable patients implanted with a LVAD for mechanical support of end-stage heart failure. Twelve stable patients with an implanted LVAD were allocated into 2 cohorts (6 subjects each) to evaluate the safety and feasibility of infusing 80mg of INXN4001 in either a 40mL (Cohort 1) or 80mL (Cohort 2) volume. The primary endpoint of safety and feasibility is assessed at the 6-month endpoint. Daily activity data are also collected throughout the study using a wearable biosensor. Dosing on both Cohorts 1 and 2 has been completed, and patients continue follow-up per protocol.
About Triple-GeneTriple-Gene LLC is a clinical stage gene therapy company focused on advancing targeted, controllable, and multigenic gene therapies for the treatment of complex cardiovascular diseases. The Company's lead product is a non-viral investigational gene therapy candidate that drives expression of three candidate effector genes involved in heart failure. Triple-Gene is a majority owned subsidiary ofIntrexon Corporation(NASDAQ: XON) co-founded by Amit Patel, MD, MS, and Thomas D. Reed, PhD, Founder and Chief Science Officer of Intrexon. Learn more about Triple-Gene atwww.3GTx.com.
About Intrexon CorporationIntrexon Corporation (NASDAQ: XON) is Powering the Bioindustrial Revolution with Better DNAto create biologically-based products that improve the quality of life and the health of the planet through two operating units Intrexon Health and Intrexon Bioengineering. Intrexon Health is focused on addressing unmet medical needs through a diverse spectrum of therapeutic modalities, including gene and cell therapies, microbial bioproduction, and regenerative medicine. Intrexon Bioengineering seeks to address global challenges across food, agriculture, environmental, energy, and industrial fields by advancing biologically engineered solutions to improve sustainability and efficiency. Our integrated technology suite provides industrial-scale design and development of complex biological systems delivering unprecedented control, quality, function, and performance of living cells. We call our synthetic biology approach Better DNA, and we invite you to discover more atwww.dna.comor follow us on Twitter at@Intrexon, onFacebook, andLinkedIn.
TrademarksIntrexon, Powering the Bioindustrial Revolution with Better DNA,and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.
Here is the original post:
Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for...
- Where is medical device approval headed? The horse's mouth speaks... [Last Updated On: August 17th, 2024] [Originally Added On: May 3rd, 2010]
- Starting a medical device company? You are better off without some of these "VC"s! [Last Updated On: August 17th, 2024] [Originally Added On: May 4th, 2010]
- Silicon Valley Business of Engineering - A great event and resource for local medical device folks and others... [Last Updated On: August 17th, 2024] [Originally Added On: May 5th, 2010]
- MedTech: A great bioscience resource for folks in New York [Last Updated On: August 17th, 2024] [Originally Added On: May 7th, 2010]
- Blog brief: Inventables - a nice resource for your medical device projects [Last Updated On: August 17th, 2024] [Originally Added On: May 17th, 2010]
- Want to help out a biotech hackerspace AND learn about medical device regulations? Come to my class... [Last Updated On: August 17th, 2024] [Originally Added On: June 8th, 2010]
- A prototyping tip & a quick update [Last Updated On: August 17th, 2024] [Originally Added On: August 2nd, 2010]
- The Healthcare bill of rights is here... [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- Come join me for a workshop on "The Rules of Brainstorming" [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- DIYBio gets a little more local...Bangalore just got one - is there one in your area? [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Are you an entrepreneur? A job seeker? Where are your major hurdles coming from? [Last Updated On: August 17th, 2024] [Originally Added On: November 28th, 2010]
- The World Diabetes Day is here [Last Updated On: August 17th, 2024] [Originally Added On: November 28th, 2010]
- Healthgamescamp, San Francisco is tomorrow, barcamps and more... [Last Updated On: August 17th, 2024] [Originally Added On: December 13th, 2010]
- Healthgamescamp - liveblog: "It's crazy and wild and fun" [Last Updated On: August 17th, 2024] [Originally Added On: December 13th, 2010]
- Healthgamescamp - liveblog; part 2 - break time! [Last Updated On: August 17th, 2024] [Originally Added On: December 13th, 2010]
- Quick Post: Are others (guests) allowed to post on this blog? [Last Updated On: August 17th, 2024] [Originally Added On: December 19th, 2010]
- Entrepreneurship Opportunities: The Health 2.0 challenge for 2011 is announced [Last Updated On: August 17th, 2024] [Originally Added On: December 19th, 2010]
- A heartwarming story, a foundation and a few medical device challenges for the future [Last Updated On: August 17th, 2024] [Originally Added On: December 27th, 2010]
- What will your 2011 medical device design resolutions be? [Last Updated On: August 17th, 2024] [Originally Added On: January 2nd, 2011]
- Elan was recently asked: Your workers or your private jet..guess what they chose? [Last Updated On: August 17th, 2024] [Originally Added On: February 6th, 2011]
- Blog Briefs: A great resource: The National Academies Press makes all pdf books free! [Last Updated On: August 17th, 2024] [Originally Added On: June 12th, 2011]
- On Scott Brown's unfactual rant... [Last Updated On: August 17th, 2024] [Originally Added On: June 26th, 2011]
- There's trouble ahead for Pharma... [Last Updated On: August 17th, 2024] [Originally Added On: July 3rd, 2011]
- The Acne Apps Scandal - how a select few make everyone suffer... [Last Updated On: August 17th, 2024] [Originally Added On: September 18th, 2011]
- Faculty Interview | MIT Introduction to Bioengineering, Spri [Last Updated On: August 17th, 2024] [Originally Added On: October 10th, 2011]
- Destroying Humanity with Social [Last Updated On: August 17th, 2024] [Originally Added On: October 10th, 2011]
- Bioengineering- Clemson Graduate School [Last Updated On: August 17th, 2024] [Originally Added On: October 10th, 2011]
- Faculty Interview | MIT Introduction to Bioengineering, Spri - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 13th, 2011]
- Grant to benefit Bioengineering research - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 14th, 2011]
- BioEngineering Associates, Inc. - Mad River Bluffs Stabilization Project - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 14th, 2011]
- BIO ENGINEERING - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 15th, 2011]
- Cross-Species Hybridization and Bioengineering: We're Not in Kansas Anymore! - Alex Jones Tv 2/2 - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Paul Root Wolpe: It's time to question bio-engineering - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 19th, 2011]
- BioEngineering Insights 2009 - BioMaterials Part 2 - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 22nd, 2011]
- Blog Action Day - Blogging for Food... [Last Updated On: August 17th, 2024] [Originally Added On: October 23rd, 2011]
- [KAIST-Bioengineering]Biomimetics - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- [KAIST Bioengineering] Bioengineering through movies - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 26th, 2011]
- PhD Research in Belgium (Bioengineering at the University of Leuven) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Penn Bioengineering: Modeling Contractile Forces - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Global 3000 | How One Bio-engineering Seed Producer is Dominating Markets - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- White House Awards UC San Diego Bioengineering Professor Shu Chien National Medal of Science - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- [KAIST BioEngineering] Olleh! BioEng! - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 9th, 2011]
- Calling BS on the link between tax increases and medical device innovation... [Last Updated On: August 17th, 2024] [Originally Added On: January 1st, 2012]
- Stanford's Stephen Quake, Bioengineer - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 1st, 2012]
- Plandai Biotechnology, Inc. Announces Appointment of World-Renowned Physicist to Chair Plandai Scientific Board [Last Updated On: August 17th, 2024] [Originally Added On: January 30th, 2012]
- Need muscle for a tough spot? Turn to fat stem cells [Last Updated On: August 17th, 2024] [Originally Added On: January 31st, 2012]
- India gets her own stem cell hosptial [Last Updated On: August 17th, 2024] [Originally Added On: February 5th, 2012]
- A marketable major [Last Updated On: August 17th, 2024] [Originally Added On: February 7th, 2012]
- US Navy here to talk biofuels [Last Updated On: August 17th, 2024] [Originally Added On: February 7th, 2012]
- Carlsberg-invested Chongqing Brewery to hire independent auditors [Last Updated On: August 17th, 2024] [Originally Added On: February 7th, 2012]
- Industry linkage beefs up livestock nutrition [Last Updated On: August 17th, 2024] [Originally Added On: February 13th, 2012]
- The Hertz Foundation Selects 50 Finalists for 2012-2013 Hertz Fellowship Supporting Gifted Young Leaders in Applied ... [Last Updated On: August 17th, 2024] [Originally Added On: February 13th, 2012]
- US Navy eyes Aussie biofuel research [Last Updated On: August 17th, 2024] [Originally Added On: February 13th, 2012]
- Researchers make breakthrough in stem cell research [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2012]
- UQ researchers make breakthrough in stem cell research [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2012]
- Brand Marvel Worldwide Consumer Products Corporation Update [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2012]
- U.S. and Canadian Scientists Form a Global Alliance for Nano-Bio-Electronics in Order to Rapidly Find Solutions for ... [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2012]
- Speaker to discuss Honor Code at UPUA meeting [Last Updated On: August 17th, 2024] [Originally Added On: February 15th, 2012]
- Understand the Lessons Learned from the FDA QbD Pilot Programme - Case Study Merck Serono [Last Updated On: August 17th, 2024] [Originally Added On: February 15th, 2012]
- Lewis: Fully caffeinated professor dreams up business [Last Updated On: August 17th, 2024] [Originally Added On: February 15th, 2012]
- Titan Spine Acquires Additional Patent Protection for its Spinal Implant Surface Technologies [Last Updated On: August 17th, 2024] [Originally Added On: February 15th, 2012]
- Histogenics to Present at 7th Annual New York Stem Cell Summit [Last Updated On: August 17th, 2024] [Originally Added On: February 16th, 2012]
- Microchip successfully delivers bone-loss drug: study [Last Updated On: August 17th, 2024] [Originally Added On: February 16th, 2012]
- Microchip delivers drug; can it replace shots? [Last Updated On: August 17th, 2024] [Originally Added On: February 16th, 2012]
- Bioengineering company bringing ideas to Huntington [Last Updated On: August 17th, 2024] [Originally Added On: February 18th, 2012]
- UCLA Discovery that Migrating Cells "Turn Right' has Implications for Engineering Tissues, Organs [Last Updated On: August 17th, 2024] [Originally Added On: February 18th, 2012]
- After scrapping bigger plans, HP says webOS still has an open-source future [Last Updated On: August 17th, 2024] [Originally Added On: February 19th, 2012]
- Discovery that migrating cells 'turn right' has implications for engineering tissues, organs [Last Updated On: August 17th, 2024] [Originally Added On: February 19th, 2012]
- Biotechnology and Bioengineering: Cell stamping from PEG-oleyl surfaces - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 21st, 2012]
- American Oriental Bioengineering Inc. Announces Plans for Reverse Stock Split of its Common Stock [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- Injectable gel could repair tissue damaged by heart attack [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- World’s First Lab-Engineered Burger Just Months Away [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- For UC Berkeley’s New Helios Energy Research Facility: Firespray’s Ductwork Solves Lab Construction Challenge [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- IBM and IBN Treating MRSA With Nanotechnology and Nanomedicine in February's Edition of Healthcare Global [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- Injectable gel could repair damaged cardiac tissue [Last Updated On: August 17th, 2024] [Originally Added On: February 22nd, 2012]
- DNA2.0 Partners with Cosmo Bio to Deliver Bioengineering Solutions to Japanese Researchers [Last Updated On: August 17th, 2024] [Originally Added On: February 23rd, 2012]
- Three UI professors win Sloan fellowships [Last Updated On: August 17th, 2024] [Originally Added On: February 23rd, 2012]
- IBM and IBN Treating MRSA with Nanotechnology and Nanomedicine in February’s Edition of Healthcare Global [Last Updated On: August 17th, 2024] [Originally Added On: February 23rd, 2012]
- Now, injectable gel that could repair tissue damaged by heart attack [Last Updated On: August 17th, 2024] [Originally Added On: February 23rd, 2012]
- India's drug discovery innovation prognosis is "poor". Is anyone surprised? [Last Updated On: August 17th, 2024] [Originally Added On: February 26th, 2012]