Delving into the Latest Updates on GLYN122 with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Target

HTT

Action

inhibitors

Mechanism

Huntingtin inhibitors

Therapeutic Areas

Nervous System DiseasesCongenital DisordersOther Diseases

Active Indication

Huntington Disease

Inactive Indication-

Originator Organization

Galyan Bio

Active Organization

Chemotargets SLGalyan Bio

Medicxi Ventures UK LLP

Inactive Organization-

License Organization-

Drug Highest PhasePreclinical

First Approval Date-

Regulation-

AbstractCurrently, there are no therapies available to modify the disease progression of Huntington’s disease (HD). Recent clinical trial failures of antisense oligonucleotide candidates in HD have demonstrated the need for new therapeutic approaches. Here, we developed a novel in-silico fragment scanning approach across the surface of mutant huntingtin (mHTT) polyQ and predicted four hit compounds. Two rounds of compound analoging using a strategy of testing structurally similar compounds in an affinity assay rapidly identified GLYN122. In vitro, GLYN122 directly binds and reduces mHTT and induces autophagy in neurons. In vivo, our results confirm that GLYN122 can reduce mHTT in the cortex and striatum of the R/2 mouse model of Huntington’s disease and subsequently improve motor symptoms. Thus, the in-vivo pharmacology profile of GLYN122 is a potential new preclinical candidate for the treatment of HD.

100 Deals associated with GLYN122

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Huntington Disease	Preclinical	Spain	Chemotargets SL	16 Nov 2022
Huntington Disease	Preclinical	United Kingdom	Medicxi Ventures UK LLP	16 Nov 2022
Huntington Disease	Preclinical	United States	Galyan Bio	16 Nov 2022