ATSN-101

PreclinicalSmall molecule

AAtsena Therapeutics Inc.

ATSN-101 is one of 6 small molecule programs targeting Leber Congenital Amaurosis. It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Leber Congenital AmaurosisActive, not recruiting

Leber Congenital AmaurosisPreclinical

Active, not recruiting

Competition

Competitive Landscape

8 competitors in Leber Congenital Amaurosis

#DrugCompanyPhaseTrials

Sepofarsen

PProQR Therapeutics

Phase 3

1 2

AAV2-Hrpe65v2,voretigene Neparvovec-Rzyl

SSpark Therapeutics, Inc.

Phase 3

1 3

Rayos

RRoxane Laboratories

Preclinical

1 4

Voretigene Neparvovec-Rzyl

SSpark Therapeutics, Inc.

Phase 2

2 5

ATSN-101

AAtsena Therapeutics Inc.

Preclinical

1 6

Triamcinalone Acetonide

AAtsena Therapeutics Inc.

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

PreclinicalActive, not recruiting

Leber Congenital Amaurosis

NCT03920007

Clinical

Trials by Indication[1]

Leber Congenital Amaurosis1 trial

A Phase 1/2 Dose Escalation Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Active, not recruiting·AAtsena Therapeutics Inc.·Preclinical·NCT03920007·15 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for ATSN-101. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — ATSN-101 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for ATSN-101. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Atsena Therapeutics Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Preclinical[1]
Company: AAtsena Therapeutics Inc.

ATSN-101

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Trials by Indication[1]

Leber Congenital Amaurosis1 trial

References

Drug Info

Quick Facts

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