Deep Dives
PROTACs eliminate, rather than inhibit, disease-driving proteins.They are bifunctional molecules that recruit an E3 ubiquitin ligase to tag the target protein for degradation by the proteasome. After the target is destroyed, the PROTAC is recycled to initiate further rounds of degradation.
US: Arvinas, C4 Therapeutics, Kymera Therapeutics Nurix Therapeutics, Monte Rosa, Starton Therapeutics, Biohaven, Parabilis Medicine, Frontier Medicines, BMS
Europe: Captor Therapeutics, Amphista Therapeutics, Bayer
Asia: BeOne Medicines, Ranok Therapeutics, Shanghai Leadingtac Pharmaceutical, GenFleet Therapeutics, Haisco Pharmaceutical Group, Ubix Therapeutics, Hinova Pharmaceuticals
The latest assets in development are:
BMS’ golcamide for NHL in Phase 3
BeOne Medicines’ BGB-16673 for NHL, CLL and CSU in Phase 3
For more on TPDs, click here
In chronic inflammation, TL1A overexpression drives persistent T-cell activation, cytokine release, and tissue fibrosis. Blocking TL1A turns down both inflammation and fibrosis simultaneously. Unlike IL-23/IL-12 or JAK inhibitors, which act on narrower or systemic pathways, TL1A inhibition offers a dual anti-inflammatory and anti-fibrotic mechanism, engaging Th1, Th2, and Th17 pathways while enabling biomarker-guided, precision treatment with reduced immunosuppression risk.
Prometheus Biosciences (acquired by Merck & Co), Sanofi, Roche, Spyre Therapeutics, Priovant Sciences, BIOCAD, Vial, Earendil Labs, Absci.
Merck & Co’s MK-7240 for IBD in Phase 3
Roche/Priovant Therapeutics’ afimkibart for IBD in Phase 3
For more on TL1A inhibitors, click here
MicroRNA therapeutics reprogram, rather than inhibit, disease biology.In many chronic and degenerative conditions, dysregulated microRNAs disrupt the cell’s natural gene-regulatory network, driving inflammation, fibrosis, and metabolic imbalance. By either restoring or silencing specific microRNAs, these therapies act upstream to rebalance entire gene networks, normalizing multiple disease pathways simultaneously. Unlike traditional RNA technologies such as mRNA or siRNA, which target single proteins or transcripts, microRNA therapeutics modulate coordinated gene networks, offering a systems-level approach to correcting disease.
Causeway Therapeutics, Biorchestra, Jaan Biotherapeutics, Curamir Therapeutics, Saverna Therapeutics, Chimerna Therapeutics, Nanokide Therapeutics, Sirana Pharma.
The most advanced asset in development are:
Novartis’ farabursen for PKD in Phase 1
For more on microRNA, click here
CircRNA molecules, due to their unique design, are able to produce more therapeutic protein inside the body than conventional mRNA, with the potential to engineer cells, replace damaged proteins, or add new beneficial proteins for patients with serious diseases.
US: Orna Therapeutics, Sail Biomedicines, Chimera Therapeutics
Europe: Curemed Pharmaceuticals, Torque Bio
China: RiboX Therapeutics, Therorna, Geneseed Biotech, Circode Bio
The latest asset in development is Ribox Therapeutics’ RXRG001 for radiation induced xerostomia and hyposalivation, which initiated its Phase I/II trial in Q1 2025.
For more on circRNA, click here
