ALK-001 is a drug candidate being developed by Alkeus Pharmaceuticals for the treatment of Stargardt Disease (STGD1). Vitamin A dimers are the primary cause of vision loss associated with Stargardt Disease due to retinal toxicity. ALK-001 is a modified form of vitamin A that prevents aggregation into dimers.

AAV1-FS344 is a gene therapy technology being developed by Milo Biotechnology for the treatment of Duchenne Muscular Dystrophy as well as other muscle diseases including Becker Muscular Dystrophy and Inclusion Body Myositis. Successful AAV1-FS344 delivery leads to the production of follistatin, a TGF-β inhibitor.

Updated: February 2, 2017

SAR 422459 is a drug candidate being developed by Sanofi and Oxford BioMedica for the treatment of Stargardt disease (STGD1). The primary cause of Stargardt disease is the buildup of toxic vitamin A aggregates due to a genetic defect. SAR 422459 aims to correct this by using a gene-based approach to insert a healthy copy of the gene into the retina.

Updated: November 17, 2016

​P1101 is a novel engineered PEGylated interferon alpha 2b currently being investigated by PharmaEssentia. P1101 boasts a higher purity composition compared to currently marketed PEGylated interferon alphas such as PEG-Intron by Merck and Pegasys by Roche, which may improve the side effect profile. Clinical trials are currently underway to evaluate P1101 in a number of treatments for hematological disorders and infectious disease. 

Updated: November 10, 2016

Ganaxolone is a GABA modulator currently being investigated by Marinus Pharmaceuticals. Clinical trials are currently underway to evaluate ganaxolone in the treatment of pediatric genetic orphan epilepsies, behaviors in Fragile X Syndrome, and status epilepticus.