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.

​In the first part of this two part series, we will be discussing techniques and associated challenges in identifying potential assets early in the development process. Please click here for the second part of this series – “Asset Valuation and Risk Assessment“.
 
Rationale
The cost of drug development, from initial research through NME approval, now exceeds $1.3 billion in out-of-pocket expenses [1], resulting in a substantial commitment as well as a serious financial drain on pharmaceutical companies. In order to combat these high barriers and reduce the strain on internal research and development teams, it is becoming more common to turn to outside sources [2]. Options for outsourcing can include identifying target groups for acquisition, as well as evaluating specific technologies for licensing opportunities. This article will provide a framework for the asset identification and selection process.

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.