On April 13, 2016 Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported it has entered into Exclusive Channel Collaborations (ECC) with two startups backed by the Harvest Intrexon Enterprise Fund, sponsored by Harvest Capital Strategies, LLC (Press release, Intrexon, APR 19, 2016, View Source [SID:1234511218]). Through the proprietary technologies of Intrexon, these companies will pursue new approaches for unmet needs in human health:
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Relieve Genetics, Inc. will focus on a breakthrough, non-opioid gene therapy approach for neuropathic pain; and
Exotech Bio, Inc. will utilize a novel exosome-based platform for delivering therapeutic RNA to treat select cancer indications.
"We are excited to enter into these new collaborations within Intrexon’s Health Sector to advance medicine on multiple fronts," said Samuel Broder, M.D., Senior Vice President, Head of Intrexon’s Health Sector. "Neuropathic pain is a tremendous unmet need in healthcare today. The collaboration with Relieve Genetics offers a significant opportunity to materially impact the lives of many patients who are in desperate need of relief from pain refractory to other therapies including opiates."
Neuropathic pain is a common, chronic complication caused by a number of different medical conditions. Current standard of care focuses mainly on easing discomfort as treatments are limited by incomplete efficacy and dose-limiting side-effects. Relieve Genetics, Inc. will instead center its therapeutic efforts on the underlying pathophysiology of neuropathic pain through gene therapy. With a designed viral vector delivery system, the collaboration will target delivery of an immunomodulatory protein alone or in combination with multiple therapeutic effectors under the control of the RheoSwitch Therapeutic System, providing localized, persistent, and regulatable drug delivery for pain management.
Dr. Broder continued, "Intrexon’s work in engineering complex miRNAs holds particular promise for an exosome-based platform to address certain cancers, for which conventional approaches have failed. In addition to the significant progress underway with our partner ZIOPHARM Oncology in the utilization of gene and adoptive cellular therapies against numerous cancer types, we look forward to introducing a new and important modality with Exotech Bio to treat cancer patients who have limited options under current treatment approaches."
Exosomes are micro-vesicles that naturally contain RNA, proteins and small molecule metabolites and are transmitted between the body’s cells to facilitate intercellular communication, immune modulation and developmental cell differentiation. A growing body of research supports the re-engineering of exosomes to transport drugs, including various RNA classes, as cell-specific cargoes that can mediate therapeutic responses to a variety of cancer cell types for which conventional treatments have been unsuccessful at effectively addressing.
Intrexon’s unique expertise in the design of subcellular localization motifs, unique protein-protein interaction motifs, multimeric miRNAs, and other RNA-based modalities can be applied in a coordinated fashion for the purpose of increasing anti-cancer therapy efficacy while reducing side effects. The collaboration with Exotech Bio, Inc. is focused on developing engineered cell lines for production of tumor-targeted allogeneic exosomes carrying bioactive RNAs to act directly on intracellular cancer pathways to suppress or eliminate specific tumor cells.
Under the terms of the ECC agreements for both collaborations, Intrexon will receive a technology access fee in the form of equity equating to 25% of each startup, reimbursement for all research and development costs, as well as potential milestones and backend economics in the form of royalties.