At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.

Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.

Aizen Therapeutics has emerged from stealth with a focus on early-stage programs powered by its novel drug discovery platform, Dax.

An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.

The development of new machine learning tools like Alphafold and Rfdiffusion has allowed scientists to predict the structure of proteins and design them for drug discovery purposes, among other uses. Now, scientists at the Arc Institute have created Evo, an AI model that generates DNA sequences and estimates their interaction with other molecules at single-nucleotide resolution, scalable to an entire genome.

As China rises to second place, next only to the U.S., for innovative new drugs, a new report by Clarivate – A Decade of Innovation, A Decade to Come – outlines key policy reforms and regulatory, R&D and investment trends driving past and future growth of Chinese biopharmaceuticals.

The development of new machine learning tools like Alphafold and Rfdiffusion has allowed scientists to predict the structure of proteins and design them for drug discovery purposes, among other uses. Now, scientists at the Arc Institute have created Evo, an AI model that generates DNA sequences and estimates their interaction with other molecules at single-nucleotide resolution, scalable to an entire genome.

The University of Texas MD Anderson Cancer Center has launched its Institute for Cell Therapy Discovery & Innovation, which seeks to build upon MD Anderson clinical and research expertise to advance impactful cell therapies.

A year out from Leqembi’s approval for Alzheimer’s disease (AD), ongoing research coupled with artificial intelligence is advancing both radiopharmaceuticals and small-molecule drugs for AD diagnostics and treatment, speakers at the 2024 KoNECT-MOHW-MFDS conference said.

A year out from Leqembi’s approval for Alzheimer’s disease (AD), ongoing research coupled with artificial intelligence is advancing both radiopharmaceuticals and small-molecule drugs for AD diagnostics and treatment, speakers at the 2024 KoNECT-MOHW-MFDS conference said.