Aperture Therapeutics
Biotechnology company focused on discovery and preclinical development of oligonucleotide therapeutics for neurodegenerative diseases. Uses large-scale human genomic and clinical datasets combined with machine learning to identify protective genetic variants, prioritize targets, and design antisense oligonucleotide programs aimed at modulating neuroinflammation and drivers of neural vulnerability.
Industries
Nr. of Employees
small (1-50)
Products
ASO development candidate targeting CD33 (preclinical development candidate)
Antisense oligonucleotide program designed to modulate CD33 activity to restore microglial homeostasis; development candidate described as recapitulating protective loss-of-function effects observed in human genetics.
ASO candidate targeting MMP9 (preclinical program)
Antisense oligonucleotide program aimed at reducing MMP9 activity to protect motor neurons, reduce matrix remodeling and neuroinflammatory damage in models related to ALS and motor neuron vulnerability.
Undisclosed preclinical programs
Additional preclinical programs listed as undisclosed development programs.
APRTX-001
A first-in-class molecule for targeted suppression of microglial-driven neuroinflammation, modulating CD33 activity to reduce neuroinflammation and pathological biomarkers linked to neurodegenerative diseases including ALS, FTD, PD, and AD.
APRTX-003
A first-in-class molecule for targeted rescue of motor neuron vulnerability and microglial-driven neurotoxicity, selectively modulating MMP9 activity to protect motor units and delay neurodegeneration in ALS and related disorders.
APRTX-002
Undisclosed product in development by Aperture Therapeutics.
ASO development candidate targeting CD33 (preclinical development candidate)
Antisense oligonucleotide program designed to modulate CD33 activity to restore microglial homeostasis; development candidate described as recapitulating protective loss-of-function effects observed in human genetics.
ASO candidate targeting MMP9 (preclinical program)
Antisense oligonucleotide program aimed at reducing MMP9 activity to protect motor neurons, reduce matrix remodeling and neuroinflammatory damage in models related to ALS and motor neuron vulnerability.
Undisclosed preclinical programs
Additional preclinical programs listed as undisclosed development programs.
APRTX-001
A first-in-class molecule for targeted suppression of microglial-driven neuroinflammation, modulating CD33 activity to reduce neuroinflammation and pathological biomarkers linked to neurodegenerative diseases including ALS, FTD, PD, and AD.
APRTX-003
A first-in-class molecule for targeted rescue of motor neuron vulnerability and microglial-driven neurotoxicity, selectively modulating MMP9 activity to protect motor units and delay neurodegeneration in ALS and related disorders.
APRTX-002
Undisclosed product in development by Aperture Therapeutics.
Services
Business development and licensing collaborations with pharma and biotech to progress therapeutic candidates into clinical trials and expand delivery routes and indications.
Business development and licensing collaborations with pharma and biotech to progress therapeutic candidates into clinical trials and expand delivery routes and indications.
Expertise Areas
- Genetic target discovery
- Antisense oligonucleotide therapeutics
- Preclinical in vivo pharmacology and humanized models
- Biomarker discovery and assay development
Key Technologies
- Antisense oligonucleotides (ASOs)
- RNA interference (siRNA)
- Machine learning / AI for genomics
- Single-cell and multi-omic profiling
News & Updates
Interview with the company's founder discussing genetics-guided approaches to neurodegenerative disease and ASO therapeutics.
Awarded a prize to use AI/ML to mine large ALS patient datasets to identify protective disease modifiers driving neuronal resilience.
Public announcement of a first-in-class MMP9 antisense oligonucleotide program for ALS described in media releases.
Public announcement of a development candidate designed to mimic protective CD33 loss-of-function variants to restore microglial homeostasis.
Listed in media coverage as awarded or recognized by Mission BioCapital programming.
Interview with the company's founder discussing genetics-guided approaches to neurodegenerative disease and ASO therapeutics.
Awarded a prize to use AI/ML to mine large ALS patient datasets to identify protective disease modifiers driving neuronal resilience.
Public announcement of a first-in-class MMP9 antisense oligonucleotide program for ALS described in media releases.
Public announcement of a development candidate designed to mimic protective CD33 loss-of-function variants to restore microglial homeostasis.
Listed in media coverage as awarded or recognized by Mission BioCapital programming.