- Exicure to collaborate with Friedreich’s Ataxia Research Alliance (FARA)
- Hank Paulson, MD, PhD and Susan Perlman, MD, added to Scientific Advisory Board
Exicure, Inc.
(NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic
drugs utilizing spherical nucleic acid (SNA™) technology, today
announced Friedreich’s ataxia (FA) as the therapeutic indication for the
company’s first neurology development program. Exicure also announced the expansion of its Scientific Advisory Board.
Patients living with FA experience a devastating and progressive loss of neurological function. There are no approved therapies for FA. Exicure’s FA program will be designed and developed with guidance from and in collaboration with FARA.
"FA is a rare, progressive and life-shortening disease, and there is a critical need for effective treatments," explained FARA's Chief Executive Officer, Jennifer Farmer. "Exicure’s SNA technology is unique in its ability to target the affected gene in FA. FARA has championed collaborative approaches to drug development, and we’re thrilled to work with Exicure as they develop their genetically targeted therapeutic candidate.”
Exicure expects IND-enabling work to begin in 2020. Exicure’s preclinical data in rodents and non-human primates showed distribution of SNAs to all brain regions following intrathecal administration, including those relevant in FA. Additional preclinical rodent data, in a head-to-head comparison with an FDA-approved oligonucleotide, nusinersen, showed improved potency in mice when put into an SNA format.
Exicure also is announcing the expansion of the company’s Scientific Advisory Board to include neurology experts Dr. Susan Perlman, MD, Professor of Neurology at University of California Los Angeles and Medical Director for the National Ataxia Foundation and Dr. Hank Paulson, MD, PhD, Lucile Groff Professor of Neurology for Alzheimer's Disease and Related Disorders in the Department of Neurology at the University of Michigan.
“We are pleased to bring together this fantastic group of patient advocates, medical leaders, and disease experts to join the Exicure team in our mission to launch a program in Friedreich’s ataxia,” Dr. Giljohann concluded.
About Friedreich’s Ataxia (FA)
FA is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use; diminished vision, hearing and speech; scoliosis (curvature of the spine); increased risk of diabetes; and a life-threatening heart condition. There are no FDA-approved treatments. An estimated 5,000 patients in the US and 15,000 patients worldwide are affected by FA.
About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia. www.CureFA.org
About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. Exicure believes that its proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure's lead program is in a Phase 1b/2 trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL and in Cambridge, MA.
For more information, visit Exicure’s website at www.exicuretx.com.
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