Saturday, August 17, 2019

Measuring peripheral nerve involvement in Friedreich’s ataxia

Creigh, P. D., Mountain, J. , Sowden, J. E., Eichinger, K. , Ravina, B. , Larkindale, J. and Herrmann, D. N. (2019). Ann Clin Transl Neurol. doi:10.1002/acn3.50865

MC density, ENFD, and QST thresholds provide structural and physiologic markers of sensory involvement in FRDA. Longitudinal evaluation is needed to determine whether these measures can identify changes associated with disease progression or treatment.



Exicure, Inc.Announced important preclinical data supporting development of SNA technology in the central nervous system (CNS)

Exicure, Inc. (Nasdaq: XCUR), 8 Aug 2019, pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, today reported financial results for the second quarter ended June 30, 2019 and provided an update on corporate progress.

The first half of 2019 continued to bring advancements across our pipeline,”said Dr. David Giljohann, Exicure’s chief executive officer. “We are extremely pleased with the strong progress in both our neurology and oncology franchises, while we simultaneously strengthened our board leadership and balance sheet. Our non-human primate data confirmed our enthusiasm for neurological applications of our SNA platform. Our recent $63.3 million public offering, which closed on August 2, 2019 provides the financial resources to advance these opportunities,” he added.
Presented results from a biodistribution study of SNAs in the CNS of non-human primates.
SNAs were observed in all 46 regions of the brain examined.
Preclinical research underway in indications including Huntington’s disease, spinocerebellar ataxia type 3 (SCA3), SCA2, SCA1, Friedreich's ataxia and Batten disease.
Expect to nominate first candidate for neurological conditions late in 2019.