Drug Pipeline

19 drugs associated with Angelman Syndrome

DrugPhaseSponsor
Circadin 10 mg
Phase 1Neurim Pharmaceuticals Ltd.
Circadin 2 mg
Phase 1Neurim Pharmaceuticals Ltd.
OV101
Phase 1Healx AI
RO7248824
Phase 1Hoffmann-La Roche
Alogabat
Phase 2Hoffmann-La Roche
EPI-743
Phase 2Edison Pharmaceuticals Inc
GTX-102

Estrogen receptor alpha agonist

Phase 2Ultragenyx Pharmaceutical Inc
MVX-220
Phase 2MavriX Bio, LLC
NNZ-2591

Insulin-like growth factor 1 (IGF-1) is a hormone secreted by glia and neurons of the CNS that plays an important role in normal brain development and response to pathologies. It is broken down into two separate molecules in the brain, both of which have synthetic analogues in development. [Glypromate], or GPE, comprises the last three peptides of IGF-1 and acts on glial cells, in contrast to its parent compound which tends to act on neurons - a synthetic analogue, [trofinetide], is currently un

Phase 2Neuren Pharmaceuticals Limited
Circadin 2/5/10 mg
Phase 3Neurim Pharmaceuticals Ltd.
Gaboxadol
Phase 3Healx AI
ION582
Phase 3Ionis Pharmaceuticals, Inc.
Levodopa

Levodopa by various routes crosses the blood brain barrier, is decarboxylated to form dopamine. This supplemental dopamine performs the role that endogenous dopamine cannot due to a decrease of natural concentrations and stimulates dopaminergic receptors.

Phase 3Wen-Hann Tan
Betaine

Homocystinuria is a hereditary disorder characterized by high levels of the amino acid homocysteine. This condition can be caused by deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylenetetrahydrofolate reductase (MTHFR), and cobalamin cofactor metabolism (cbl). CBS converts homocysteine to cystathionine, and a deficiency in this enzyme can lead to an accumulation of homocysteine. MTHFR is responsible for producing 5-methyltetrahydrofolate, a methyl donor that participates

unknownUniversity of California, San Diego
Creatine

In the muscles, a fraction of the total creatine binds to phosphate - forming creatine phosphate. The reaction is catalysed by creatine kinase, and the result is phosphocreatine (PCr). Phosphocreatine binds with adenosine diphosphate to convert it back to ATP (adenosine triphosphate), an important cellular energy source for short term ATP needs prior to oxidative phosphorylation.

unknownUniversity of California, San Diego
Metafolin
unknownUniversity of California, San Diego
minocycline

mechanism of action The tetracyclines are primarily bacteriostatic and are thought to exert their antimicrobial effect by the inhibition of protein synthesis. The tetracyclines, including minocycline, have a similar antimicrobial spectrum of activity against a wide range of gram-positive and gram-negative organisms. Cross-resistance of these organisms to tetracycline is common.

unknownUniversity of South Florida
Rugonersen
unknownOak Hill Bio Ltd
Vitamin B12
unknownUniversity of California, San Diego