I recently found myself catching up on tweets, and I came across this repost by Michael Gilman, CEO of Arrakis Therapeutics, and I was reminded that over the past 20 years we’ve seen an explosive redefinition of the edges of druggable space.
The nucleus of that explosion was the human genome project. In my own Forrest Gump moment, I found myself at ground zero of that explosion working on an effort to map out the 5000 most druggable genes in the human genome, through a programme called the Genome5000 project. The project used mouse knockout technology to delete druggable genes, study the phenotypic results, and use that knowledge to identify novel drug targets.
Beyond identifying new drug targets though, omics information has been used to expand druggable space from merely, the parts of the proteome that can be addressed by small molecules, to the parts of the genome and exome that can be addressed by gene therapy, monoclonal antibodies, antibody drug conjugates, bispecific antibodies, antisense drugs, CAR-T therapeutics, RNAi, cell-based therapeutics, mRNA vaccines and more.
Each one of these new modalities has played a role in redefining druggable space, and addressing previously unmet medical need.
We saw this same concept reflected in the stats of Pharm2Market communities. Although small molecules and antibodies continue to constitute the lion’s share of the drugs currently under development, it is heartening to see that in most communities nearly 50% of the projects in company pipelines are innovative new modalities.
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