Advances in Discovery, Formulation, and Delivery of New Modalities
Sandeep Nema, PhD (he/him/his)
SVP
Saliogen Therapeutics
Lexington, Massachusetts
Much of the nucleic acid delivery has been focused on vaccine or targeting mRNA to liver. There is a significant gap in understanding and delivering DNA and RNAs to other organs.
This will be a symposium with 3-4 speakers with question and answer by the panel of speakers.
Scientists involved in pre-formulation, drug delivery, AI, process development
Success of mRNA-based vaccines for COVID has invigorated research and development of technologies to deliver RNA and DNA as therapeutic modalities for treating various genetic diseases. The field has however remained focused on liver as the target organ, since specificity in delivery to other tissues such as lung, muscle, spleen, cardiac system or brain is hard to achieve. The current mRNA LNPs also suffer from drawbacks of low percentage escape from endosomes, toxicity of lipids especially for chronic delivery, immune path stimulation either by nucleic acid or nucleic acid-lipid complex, as well as a complicated FTO environment. DNA delivery in-vivo, has also been extremely challenging as the payload not only has to escape from the endosome but transport across the nuclear membrane to reach the site of action. Many innovative technologies with therapeutic potential such as CRISPR, base and prime editing, gene coding etc. are on the threshold, but the barrier of delivery has prevented the full benefit of these efforts to reach the patients. This symposium will provide a few unique approaches that are being pursued (some of which are in clinic) to overcome these challenges and to allow improved delivery of nucleic acid to various tissues for correcting genetic diseases.
1. Selective Organ and cell targeting (SORT) for the precise delivery of genetic medicines – Daniel J. Siegwart, ReCode Therapeutics
2. Use of AI to screen and select novel lipids for tissue specific nucleic acid delivery – Yogev Debbi, Mana Bio
3. Avoiding the endosomal path for nucleic acid delivery via fusogenic vesicles – Arun Raturi, Entos
4. Polymeric delivery of DNA and mRNA: improving design via AI learning – Shashi Murthy, Nanite
Hot Topic Speaker: Shashi Murthy, PhD – Nanite Inc.
Hot Topic Speaker: Gaurav Sahay, PhD – Oregon State University
Hot Topic Moderator: Sandeep Nema, PhD (he/him/his) – Saliogen Therapeutics
Hot Topic Speaker: Shashi Murthy, PhD – Nanite Inc.
Hot Topic Speaker: Gaurav Sahay, PhD – Oregon State University