| Name | Title | Contact Details |
|---|
We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Translate Bio is a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Our proprietary mRNA therapeutic platform (MRTTM) is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. We believe that our MRTTM platform is applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system, lymphatic system and circulatory system. Our two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.
Janpix is discovering and developing monovalent small molecule degraders of Signal Transducer and Activator of Transcription (“STAT”) proteins. Most members of this family of transcription factors have been known for some time and the relevance of STAT3 & STAT5, in particular, in certain cancers is well validated. Although disrupting the function of STAT proteins is very challenging, Janpix has developed new chemistry to address this complex problem.
Bioskryb is a venture-backed startup bringing new solutions for cellular heterogeneity, in both the research and clinical space studies, using a proprietary genome amplification system.
Scientia Advisors is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.