RO7248824

Phase 1Small molecule

Roche

RO7248824 is one of 12 small molecule programs targeting Angelman syndrome. It is currently in Phase 1.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Angelman SyndromeActive, not recruiting

Angelman SyndromePhase 1

Active, not recruiting

Competition

Competitive Landscape

16 competitors in Angelman Syndrome

#DrugCompanyPhaseTrials

Lodosyn

Gaboxadol

1 3

Minocin

TTriax

Phase 2

2 4

Bendopa

MMerz Pharmaceuticals GmbH

Phase 2

1 5

GTX-102

Ultragenyx

Phase 3

4 6

ION582

Ionis Pharmaceuticals

Phase 3

2 7

OV101

HHealx AI

Phase 3

2 8

Levodopa/carbidopa

Orion Corporation, Orion Pharma

Phase 1

1 9

NNZ-2591

NNeuren Pharmaceuticals Limited

Phase 2

1 10

Cystadane

RRECORDATI GROUP

Preclinical

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

Clinical

Trials by Indication[1]

Angelman syndrome1 trial

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

Completed· Roche logo

Roche·Phase 1·NCT04428281·74 enrolled

Central Nervous System1 trial

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants

Completed· Roche logo

Roche·Phase 1·NCT04863794·21 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for RO7248824. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — RO7248824 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for RO7248824. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Roche company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 1[1]
Company: Roche

RO7248824

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Angelman syndrome1 trial

Central Nervous System1 trial

References

Drug Info

Quick Facts

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