Tailored RNA Therapy Solutions

This young biotech company engaged Organix to design an SAR (Structure Activity Relationship) study focused
on the discovery of novel lipid compounds to stabilize their propriety mRNA compounds and allow for different half-lives within a particular cell. The optimization of the cationic lipid nano particle (LPN) half-life depends on the stability
of the LPN and the ease of which it falls apart. This half-life can be dialed in using different moieties on the building block lipid that is used to create the LPN. Organix had a fruitful collaboration with this biotech company over a period of 7 years and scaled up many of their lead lipids to greater than 500 g of each target compounds for use in toxicity studies. 
4 FTEs were eventually assigned to this research project, which resulted in hundreds of novel lipids being produced
in high purity for testing. Of particular importance for the success of the project was the use of uHPLC-CAD and
HPLC-ELSD for fraction analysis and semi-automated medium pressure chromatography equipment equipped
with ELSD fraction collectors.

A young biotech company engaged Organix to design an SAR (Structure Activity Relationship) study focused
on the discovery of novel lipid compounds that could be conjugated to GalNAc dendrimers to stabilize their propriety small single strand interfering DNA (ssiDNA) and small interfering RNA (siRNA.) Organix implemented a three-year synthesis program in which over 300 compounds involving multi-step synthetic sequences were prepared and evaluated. The lead compounds that emerged from the biological studies were scaled up at Organix to 25-50 g.
Several compounds were chosen for cGMP synthesis and technology was successfully transferred to a local cGMP CRO that resulted in clinical material being synthesized.

A multinational pharmaceutical company engaged the services of Organix for over 10 years. These services included basic research and custom synthesis projects of novel lipids for use in mRNA delivery. After successful collaboration for over
7 years the company spun off their mRNA division and it was acquired by a small biotech company.
Throughout this corporate restructuring Organix continued to perform novel research and development in the lipid/lipoid area and scaled up several novel lipids and lipiods to 100-250 g. This company then entered into a strategic partnership
with a multinational pharmaceutical company and again our services were retained and expanded resulting
in up to 8 FTEs being assigned to the research. Over this period Organix was involved in the design of new synthetic routes to novel lipid analogs of lead compounds and their preparation on milligram to hundreds of grams.
Parallel synthesis techniques were used successfully in this project that resulted in 20-30 compounds per month being delivered with a purity of over 85% each. Organix acquired the necessary equipment to allow for rapid parallel synthesis that resulted in a target that was brought to the clinic.