What trends will speed up the journey from clinic to bedside?
Feb 29, 2024
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Exploring 3D chemistry and AI for small molecule therapies, single-cell gene expression in next-gen sequencing, and CDMOs supporting mRNA developers. Advantages of AI in drug design, SC RNA sequencing for cancer research, and advancements in mRNA therapeutics manufacturing.
Utilizing 3D chemistry and AI in drug development improves specificity and potency of small molecule therapies.
Single Cell RNA Sequencing (SC RNA sec) reveals diverse cell behaviors, aiding in understanding drug response mechanisms.
Deep dives
Farm Enable's Approach to Small Molecule Drug Design
Farm Enable utilizes 3D chemistry and AI to develop small molecule therapies that target intracellular space, offering advantages over biologics. Their platform focuses on generating novel chemical entities with increased specificity and reduced off-target effects. By employing guided de novo molecular design and AI, they select specific and powerful small molecule drug candidates from the start.
Advantages of Synthesizable 3D Chemistry in Drug Design
Using 3D chemistry in small molecule drug design increases specificity, reduces toxicity, and enhances potency compared to 2D molecules. Success in clinical molecules indicates the benefits of tridimensionality for efficient drug development. Farm Enable's focus on oncology and neurodegenerative therapy showcases the advantages of their technology in creating efficient and specific drugs.
Potential of Single Cell RNA Sequencing in Understanding Diseases
Single Cell RNA Sequencing (SC RNA sec) allows high-resolution transcriptome sequencing of individual cells in a sample, revealing diverse cell behaviors and roles in tissues. Genentech's study on cancer cell resistance to EGFR inhibitors highlights SC RNA sec's application in identifying mechanisms of drug response. SC RNA sec offers insights into resistance mechanisms and cellular responses, particularly beneficial in cancer and immunology research.
Rise of mRNA Technologies in Therapeutic Development
mRNA technology's success in COVID-19 vaccines has propelled its prominence in drug development, offering flexibility in protein editing and disease targeting. The versatility of mRNA is exemplified by the increasing number of mRNA-based therapeutics in clinical trials. The technology's growth in addressing diseases like cancer, heart disease, and infectious diseases showcases its potential in revolutionizing nucleic acid therapies.
This is the latest episode of the free DDW narrated podcast, titled: What trends will speed up the journey from clinic to bedside? It covers three articles written for Volume 23 – Issue 4, Fall 2022 of DDW.
In the first article, Reece Armstrong speaks to Natalia Mateu, Co-Founder, Jelena Aleksic, Co-Founder and Peter Curran, Chemoinformatics Scientist, about utilising 3D chemistry and AI to get small molecule therapies from the clinic to the bedside.
In the second article, we hear from Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies.
In the third article, Samsung Biologics’ Senior Vice President, James Park, explores the opportunities emerging for CDMOs to support mRNA developers as they explore new therapeutic areas for patients.