StemCell Therapy

GERMANTOWN, Md., Nov. 18, 2019 /PRNewswire/ --Triple-Gene LLC, a clinical stage cardiovascular gene therapy company and majority owned subsidiary of Intrexon Corporation (NASDAQ: XON), yesterday presented preliminary data from the Phase I trial (clinical trial identifier: NCT03409627) of INXN-4001, a multigenic investigational therapeutic candidate under evaluation for the treatment of heart failure, in a poster at the American Heart Association (AHA) Annual Meeting.1 On November 7, 2019, Triple-Gene announcedthat enrollment in this Phase I study has been completed.

"We are pleased to have had the opportunity to share our early clinical data from this first-in-human study with the cardiology community at this prestigious meeting," stated Thomas D. Reed, PhD, Co-founder and Managing Director of Triple-Gene. "The data presented yesterday suggest that the combination of our transiently expressed, non-integrating naked plasmid DNA with the focused cardiac delivery enabled by Retrograde Coronary Sinus Infusion (RCSI) has the potential to open a new biologics treatment paradigm for treating cardiovascular diseases."

Triple-Gene's investigational therapy uses non-viral delivery of a constitutively controlled 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.

Dr. David Bull, who was the first investigator to dose a patient with INXN-4001, stated, "Having carefully reviewed the science behind Triple-Gene's product candidate, I was very excited to initiate the INXN4001 clinical trial at the University of Arizona. Heart failure is a devastating disease, and my patients on Left Ventricular Assist Devices (LVAD) have very few therapeutic treatment options. Triple-Gene's novel triple-effector plasmid, as delivered by the minimally invasive RCSI procedure, represents a potential game-changing approach for addressing disease pathology in this high-risk patient population."

Amit N. Patel, MD, MS, Co-founder and Clinical Director of Triple-Gene added, "We are pleased to have completed dosing of the twelve patients in this clinical trial between our two clinical sites at the University of Arizona, Tucson, AZ and The Christ Hospital, Cincinnati, OH, and look forward to sharing additional data for this investigational therapy once the trial is complete. Based upon the promising early results to date, we are now exploring clinical trial designs that contemplate repeat dosing as well as additional orphan-like heart failure subtypes."

1Jaruga-Killeen E, Bull DA, Lotun K, Henry T, Egnaczyk G, Reed TD and Patel AN. Safety of first-in-human triple gene therapy for heart failure patients. Presented at the American Heart Association Annual Meeting, November 17, 2019.

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 of Intrexon 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 at http://www.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 at http://www.dna.comor follow us on Twitter at @Intrexon, on Facebook, and LinkedIn.

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.

Safe Harbor Statement Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

For more information contact:

Investor Contact:

Steven Harasym

Vice President, Investor Relations

Intrexon Corporation

Tel: +1 (301) 556-9850

investors@dna.com

Corporate Contact:

Marie Rossi, PhD

Vice President, Communications

Intrexon Corporation

Tel: +1 (301) 556-9850

publicrelations@dna.com

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SOURCE Triple-Gene LLC

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Triple-Gene Presented Preliminary Phase 1 Trial Data of Investigational Multigenic Therapeutic Candidate INXN-4001 for Treatment of Heart Failure at...

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