Path: /organizations/asceneuron
Type: Biotechnology Company (Clinical Stage)
Headquarters: Basel, Switzerland
Founded: 2017
Status: Private
Asceneuron is a Swiss clinical-stage biotechnology company focused on developing orally bioavailable small molecule therapeutics for tauopathies, with primary focus on Progressive Supranuclear Palsy (PSP). The company's proprietary approach targets the microtubule-associated protein tau (MAPT) through a novel mechanism involving enhancement of antisense oligonucleotide (ASO) activity, enabling oral delivery of tau-targeting therapies that have traditionally required intrathecal administration.
Asceneuron's lead program, ASN51, represents a first-in-class oral tau-lowering approach for PSP that could significantly improve patient convenience and treatment accessibility compared to existing antibody and ASO approaches that require intravenous or intrathecal delivery.
Asceneuron's platform is based on the discovery that certain small molecules can enhance the activity of antisense oligonucleotides targeting the MAPT gene. This approach offers several advantages:
ASN51 is an orally bioavailable small molecule that enhances MAPT ASO activity in the brain. By modulating splicing factors that regulate tau isoform expression, ASN51 can shift the equilibrium toward reduced 4R tau production — the predominant pathological isoform in PSP.
Development Status:
| Program | Mechanism | Indication | Stage |
|---|---|---|---|
| ASN51 | MAPT ASO enhancer | PSP | Phase 1 |
| ASN62 | MAPT ASO enhancer | Alzheimer's disease | Preclinical |
| ASN79 | Tau aggregation inhibitor | PSP/AD | Discovery |
PSP is classified as a 4R-tauopathy, meaning that the pathological form of tau in PSP brains is the 4-repeat (4R) isoform. Normal adult human brain has roughly equal amounts of 3R and 4R tau, but in PSP, 4R tau accumulates disproportionately. Asceneuron's approach:
Phase 1 Study Design:
Phase 2 Plans (PSP):
The ability to deliver a tau-lowering therapy orally addresses a major unmet need in PSP treatment:
Asceneuron operates as an independent biotech with strategic partnerships:
The company works with leading tauopathy researchers including:
Asceneuron competes in the tau-targeting therapeutic space, with differentiation through:
| Company | Approach | Administration | Stage |
|---|---|---|---|
| Asceneuron | MAPT ASO enhancer | Oral | Phase 1 |
| Biogen/Ionis | MAPT ASO | Intrathecal | Phase 1/2 |
| Roche | Anti-tau antibody | IV | Phase 2 |
| Pinteon | Conformant antibody | IV | Phase 1 |
| Oligomerix | Tau oligomer inhibitor | Oral | Preclinical |
Asceneuron's oral approach offers a significant differentiation if clinical efficacy is demonstrated.
Asceneuron has raised funding from: