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CHICAGO & CAMBRIDGE, Mass .– (BUSINESS WIRE) – July 8, 2021–

Exicure, Inc. (NASDAQ: XCUR), a pioneer of gene regulatory drugs using spherical nucleic acid technology (SNA â„¢), will host a virtual R&D day on Thursday, July 15, 2021 from 10:00 a.m. to 11:00 a.m. ET. The event will showcase Exicure’s neuroscience pipeline, including its main program for Friedreich’s ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.

Exicure will present new and unpublished preclinical data and discuss the progress of XCUR-FXN, which is on track for IND filing at the end of Q4 2021. Additionally, Exicure will discuss its perspective on differentiation competitive XCUR-FXN in FA and the program’s path to validation.

Additionally, Exicure will provide an update on its expanding pipeline in several rare high unmet need neurodegenerative diseases and highlight progress with two preclinical programs targeting SCN9A (Na v 1.7) for neuropathic pain and CLN3 for Batten’s disease.

“We believe our SNA platform is ideally suited to treat neurological disorders based on the demonstrated ability of ANS in non-clinical studies to achieve higher cellular uptake in all key cell types and broad biodistribution in CNS, including deep brain regions, ”said Dr. David Giljohann, CEO of Exicure. “We are delighted to translate the capabilities of our platform into potential benefits for patients, as demonstrated by our bispecific ANS, XCUR-FXN for Friedreich’s ataxia, scheduled to enter the clinic in the first half of 2022. “

A live webcast will be available in the Events & Presentations section of Exicure’s website on July 15, 2021 at 10 a.m. ET. An archived version will be available on the company’s website after the event. Additional information can be found here:

About Friedreich’s ataxia

Friedreich’s ataxia is the most common inherited ataxia, a degenerative neuromuscular disease that causes progressive loss of coordination and causes severe childhood disability and early death, in most cases before the age of 40. It is a monogenic disease caused by mutations in the FXN gene resulting in reduced levels of frataxin protein. FA affects approximately 13,500 people in the United States, Europe, Canada and Australia combined. There is currently no approved therapy for patients with Friedreich’s ataxia.

About SCN9A

SCN9A is the gene encoding Na v 1.7, a transmembrane sodium channel, which plays an essential role in pain signal signaling. Na v 1.7 is highly expressed in dorsal root ganglion (DRG) neurons, which transmit peripheral pain signals to the brain. The critical role of Na v 1.7 for pain signaling has been biologically validated by gain-of-function mutations in humans leading to severe pain conditions such as hereditary erythromelalgia and small-fiber neuropathy and mutations loss of function in humans lead to congenital insensitivity to pain. Therapies targeting Na v 1.7 could provide a new treatment option for painful neuropathic conditions in which currently available therapies are largely ineffective.

About CLN3 Latte disease

CLN3 Batten disease is a monogenic, autosomal recessive, lysosomal overload disease caused by mutations in the CLN3 gene causing latte protein deficiency. Those affected suffer from childhood blindness, pediatric dementia syndrome, seizures and premature death between the ages of 20 and 30. CLN3 Batten is estimated to affect over 1,800 people in the United States and Europe. There is currently no approved treatment for these patients.

About Exicure, Inc.

Exicure, Inc. is a clinical-stage biotechnology company that develops therapies for neurology, immuno-oncology, inflammatory diseases, and other genetic disorders based on our proprietary spherical nucleic acid or ANS technology. Exicure believes that its proprietary SNA architecture has distinct chemical and biological properties that may offer advantages over other nucleic acid-based therapies and may have therapeutic potential to target diseases that are typically not treated with d ‘other nucleic acid therapies. Exicure is in the preclinical development of XCUR-FXN, an ANS-based therapeutic candidate, for the treatment of Friedreich’s ataxia (FA). Exicure’s drug candidate, cavrotolimod (AST-008), is currently in a Phase 2 clinical trial in patients with advanced solid tumors. Exicure is in Chicago, IL and has an office in Cambridge, MA.

For more information, visit the Exicure website at

Forward-looking statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release other than statements of historical fact can be considered as forward-looking, including, but not limited to y limit, statements regarding the advancement of the Company’s clinical programs and its expansion into neuroscience; its clinical development of XCUR-FXN, including development and regulatory submission deadlines and anticipated data reads; and the timing and success of its preclinical programs, including a program targeting SCN9A for neuropathic pain and a program targeting CLN3 Batten disease. The forward-looking statements contained in this press release speak only as of the date of this press release, and the company does not undertake to update these forward-looking statements. Forward-looking statements are based on management’s current beliefs and assumptions which are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement due to various factors, including, but not limited to: the risks that the ongoing COVID-19 pandemic could disrupt the business of the business and / or the global healthcare system more severely than it is to date or more severe than expected, which may impact or delay the Company’s ongoing Phase 1b / 2 clinical trial; unforeseen costs, charges or expenses that reduce the capital resources of the business; the Company’s preclinical or clinical programs do not advance or result in approved products in a timely or cost-effective manner, or at all; the results of the first clinical trials are not always predictive of future results; the cost, timing and results of clinical trials; that many drug candidates do not become approved drugs in a timely or cost-effective manner or have no capacity at all to enroll patients in clinical trials; possible safety and efficacy concerns; regulatory changes; and the company’s ability to protect its intellectual property rights. For a discussion of other risks and uncertainties, and other important factors, each of which could cause the Company’s actual results to differ from those contained in forward-looking statements, see the section entitled “Risk Factors” in the annual report on company form. 10-K for the fiscal year ended December 31, 2020, as updated by the company’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of publication, and the company does not undertake to update this information except as required by law.

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Karen sharma



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SOURCE: Exicure, Inc.

Copyright Business Wire 2021.

PUB: 08/07/2021 7:00 a.m. / DISC: 08/07/2021 7:01 a.m.

Copyright Business Wire 2021.


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