ToolGen, Inc.

June 15, 2022 12:00 UTC SUWON, South Korea--(BUSINESS WIRE)-- OliX Pharmaceuticals, Inc., a leading developer of RNAi therapeutics, today announced that the Company’s subsidiary mCureX Therapeutics, Inc. has recently signed a contract for joint research with ToolGen, Inc. to advance the development of gene therapy for rare eye diseases, leveraging mCureX’s mRNA technology. mCureX, specializing in mRNA vaccines and therapeutics, and ToolGen, having expertise in therapeutic gene-editing technologies, have been in active discussions since their signing of memorandums of understanding in August 2021. The two companies plan to conduct gene corrections in ocular tissues using mCureX's mRNA technology and ToolGen's CRISPR/Cas9 gene-editing technology for the treatment of rare hereditary eye diseases. “mRNA-based CRISPR/Cas9 is a powerful platform that could potentially lead to the development of treatments for incurable diseases,” said Sun Woo Hong, Ph.D., chief executive officer of mCureX. “Once we clinically validate the technology, we plan to expand the indications of treatments to various non-hereditary eye diseases as well.” About OliX Pharmaceuticals Inc. (KOSDAQ: 226950) OliX Pharmaceuticals is a clinical-stage pharmaceutical company developing therapeutics against a variety of disorders by down-regulating the expression of disease-causing genes based on its own proprietary RNAi technology. The Company’s core RNAi platform, asymmetric siRNA (asiRNA), is a unique gene silencing technology based on RNA interference (RNAi), which is considered the most efficient gene silencing technology. Utilizing this proprietary asiRNA technology, OliX has developed cell penetrating asiRNA (cp-asiRNA), a therapeutic RNAi platform to effectively target diseases locally, such as hypertrophic scarring, dry and wet age-related macular degeneration (AMD), subretinal fibrosis, and neuropathic pain. OliX has also developed another therapeutic RNAi platform, GalNAc-asiRNA, to target a variety of liver diseases. Learn more: About ToolGen Inc. (KOSDAQ: 199800) ToolGen is a biotech company with the CRISPR/Cas9 core technology in gene-editing. ToolGen's mission is to provide customers with innovative human gene/cell therapies and gene-edited animals or plants. ToolGen's research and business are based on ToolGen's CRISPR/Cas9-essential patents. ToolGen is the first patent filer successfully demonstrating a CRISPR/Cas9 application in eukaryotic cells in the world. ToolGen's CRISPR patents have been registered in ten countries, including the United States, Europe, China, Australia, and Japan. Learn more:

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong. CRISPR has the potential to be one of the most revolutionary (or dangerous) genetic manipulation technologies ever developed. It provides researchers with the ability to “edit” genetic information (including both structural genes encoding proteins as well as regulatory sequences that control when a gene is expressed, how much, and in what tissue) in ways heretofore only more crudely practiced; for example, by introducing a heterologous gene into a new cellular environment. It thus has implications for agriculture — increasing yield, for example, or reducing allergens like gluten — as well as human medicine. CRISPR was first reported by Jennifer Doudna and Emmanuelle Charpentier in 2012, as an outgrowth of their work on bacterial immunity at the University of California at Berkeley and the University of Vienna. They did not explicitly show that CRISPR could edit genes in eukaryotic cells — i.e., almost every type and species of cell except bacteria — in their earliest published work (although applying CRISPR to eukaryotic DNA was envisioned) and there is some evidence that the earliest efforts in achieving eukaryotic CRISPR were unsuccessful. The first scientific publication demonstrating that CRISPR could be effectively practiced in eukaryotic cells was by Zhang and colleagues at The Broad Institute, MIT and Harvard University; thereafter, several groups reported successful eukaryotic CRISPR results. Both the Broad and “CVC” (California, Vienna, Charpentier) groups accompanied their scientific work with patent applications. This is because both groups, and their universities, realized that in order to bring this technology to market there must be a reasonable likelihood of being able to recoup a sufficiently robust return on investment to make that investment worthwhile, and this reasonable likelihood depends on having patent protection. Both groups claimed inventorship over CRISPR applications to eukaryotic cells (which encompasses most of the most promising applications of the technology), and in the face of their competing claims, the Patent Trial and Appeal Board (PTAB) instituted an interference proceeding to make the determination of who invented eukaryotic CRISPR first. Two years ago, the outcome of the first of these interferences seemingly resolved the question, albeit imperfectly: the PTAB decided that the Broad Institute and their collaborators had the rights to eukaryotic CRISPR applications and that rights to CRISPR more generally were owned by the University of California at Berkeley, the University of Vienna and Emmanuelle Charpentier as an individual. This outcome had the benefit of certainty in identifying who owned the rights to eukaryotic CRISPR but suffered from the consequence that any third party wishing to bring CRISPR-modified eukaryotic organisms to market — or products made by such organisms — would likely need a license from both the Broad and CVC, which could result in delays in commercial development (because any third party would require licenses from both CVC and the Broad). In June 2019, the PTAB declared another interference between these same parties, involving again who has the rights to eukaryotic applications of CRISPR. After not prevailing in the first interference the CVC group had filed applications directed more narrowly at eukaryotic CRISPR embodiments, and the PTO declared an interference between these applications and most of the same Broad patents and applications involved in the first interference. Having completed the preliminary phase of the interference (without any substantive change in the posture of the parties regarding the presumption that the Broad as Senior Party is entitled to priority based on the parties’ respective filing dates) the current interference is in the priority phase, with both parties having filed evidence for their dates of conception and diligence in reducing to practice eukaryotic embodiments of CRISPR. CVC has shown evidence of conception as early as March 2012, and evidence of diligent efforts to show successful eukaryotic CRISPR experiments from that time until its earliest priority date recognized by the PTAB — Jan. 28, 2013. The Broad’s earliest date of conception is later than CVC’s, in June 2012, with asserted reduction to practice in July 2012. This timeline would ordinarily indicate that CVC should win the priority battle, but the Broad has asserted a nuanced counter-argument; the Broad contends that the unpredictability of using CRISPR in eukaryotic cells is sufficiently high that only by actually reducing the invention to practice in a eukaryotic cell could the invention be conceived. If the PTAB agrees with this argument the Broad should prevail. Further complicating this interference is that the PTAB has granted permission for CVC to depose two other scientists thought to have information relating to whether Broad scientists derived their invention from the CVC inventors. Initial resolution of the priority question should be made sometime this year. But because whatever the outcome the PTAB’s decision is sure to be appealed to the Federal Circuit Court of Appeals, final resolution is at least a year away. A further complication is that the PTAB declared two additional interferences, naming Korean company ToolGen as Senior Party — and presumptive first to invent — against CVC and Broad, separately. These interferences are in the early, so-called motions phase, and will not reach the priority phase (if either of them reach this phase) until this fall, with final resolution unlikely until sometime next year. In addition, another party, Sigma Aldrich, also has a claim to priority for eukaryotic CRISPR, although these claims are bottled up in a procedural morass in the PTO that has prevented Sigma from joining the fray despite their argument that joining them to the existing interference would give the Office (and the parties) a chance to resolve the ownership issue more expeditiously. These circumstances leave the ownership status of eukaryotic CRISPR technology in limbo for at least the foreseeable future. This state of affairs raises clear impediments to commercial development, at least until and if the parties decide some way to cross-license CRISPR to third parties. While the challenges of developing CRISPR technology over the next decade will undoubtedly be predominantly scientific and technological, economic realities mandate that ownership considerations will have a practical effect on what companies commercialize various aspects of the technology, where this commercialization occurs, and the licensing costs and complexities that arise in the process.