AAV Based Gene Therapy for Rare Genetic Disorders: Strategies and Challenges

Our mission is to help researchers and clinicians unlock single-cell biology to enable the discovery, development, and delivery of precision medicine.

Single cell cloning (SCC) is a critical and high value process for an increasing number of applications including cell engineering; stable cell lines for therapeutic mAbs and biosimilars; vector production for gene therapy; and many more. During this webinar, Tom Kelly from Janssen (part of Johnson & Johnson) will talk about his use of Solentim’s VIPS™ (Verified in-situ Plate Seeding) system, the world’s first All-in-One Single Cell Cloning System. He will explain why Janssen moved away from older generation CLD technology using Clonepix, which was based on detecting and picking high-producer clones in semi-solid media.

Therapeutic discovery and development rely on numerous analytical techniques in order to best position candidate drugs for commercial success. Different analytical technologies are utilized for their respective strengths in maximizing understanding of candidate properties.

Nucleic acids are known to be fragile payloads. To enable their safe and efficient delivery into cells, the RNA payload needs to be “packed” into a delivery vehicle, the lipid nanoparticles (LNP). This webinar gives insight into our capabilities for customized LNP formulation development for novel therapeutic modalities.