Kevin Eggan’s Post

Congratulations to QurCan Therapeutics Inc. for winning this year’s BioMarin Pharmaceutical Inc. golden ticket at LabCentral. We were impressed by their exciting new technology for improving brain delivery and are looking forward to interacting with them over the next year to encourage their success. Thanks to Pooja Agarwal, Ted Yun, Stuart Bunting and Gordon Vehar for leading our selection committee and Chieze Ibeneche-Nnewihe, Ph.D for spearheading the process here at BioMarin.

Tune in this morning to hear about our progress in R and D BioMarin Pharmaceutical Inc.

It’s great to have Dan with us leading our Genomics efforts. Looking forward to the many things he will bring to BioMarin Pharmaceutical Inc.’s R and D engine in the years ahead!

It has long been known that complete loss of gene function contributes to many of the most severe human maladies. However, it is only more recently that large scale genetic studies have revealed that many devestating conditions are caused by just partial loss of activity from certain essential genes. This discovery raises the key question of what the best therapeutic approach would be to “turn up” the function of these genes. Traditionally, it has been a challenging to identify small molecules that increase gene function, but recent discoveries around the mechanisms that regulate gene transcription suggest that small RNAs may represent a general approach for turning up the volume on gene function. At BioMarin Pharmaceutical Inc. we are excited to be entering into a collaboration with MiNA Therapeutics to explore whether this new small RNA approach can be used for identifying new therapies for several genetic conditions with very significant unmet need. https://lnkd.in/gY2XpgR9