Rapafusyn Pharmaceuticals develops RapaGlues™, a novel class of non-degrading molecular glues that facilitate protein-protein interactions by bringing the FKBP12 chaperone protein in proximity to previously undruggable intracellular targets. This technology addresses the lack of effective therapies for conditions like acute kidney injury and various cancers by enabling selective inhibition of critical protein functions.
Funding
$46M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
3BPVFounders
Product
Problem
Many intracellular proteins and protein-protein interactions (PPIs) are considered undruggable by conventional methods, leaving a significant gap in therapeutic options for diseases like acute kidney injury and various cancers. Traditional drug discovery approaches often fail to effectively modulate these challenging targets, hindering the development of new treatments.
Solution
Rapafusyn Pharmaceuticals is developing RapaGlues™, a novel class of non-degrading molecular glues designed to induce proximity between the FKBP12 chaperone protein and previously intractable intracellular targets. These cell-permeable macrocycles facilitate neo-protein-protein interactions, selectively blocking a target's native function. By extending nature's FKBP12-binding mechanism, RapaGlues™ offer a tunable platform for inhibiting a wide range of intracellular and transmembrane targets, including PPIs and intracellular domains of transmembrane proteins. This approach enables the development of therapeutics with desirable drug-like properties, such as permeability and exquisite selectivity, to address conditions with limited treatment options.
Target Audience
The primary target audience includes pharmaceutical companies, research institutions, and clinical investigators focused on developing novel therapies for diseases with limited treatment options, particularly those involving undruggable intracellular targets.
Features
- Non-degrading molecular glues that induce proximity between FKBP12 and target proteins
- Cell-permeable macrocycles for intracellular target engagement
- Tunable platform applicable to a wide range of intracellular and transmembrane targets
- Selective inhibition of protein-protein interactions and intracellular domains
- Potential for oral bioavailability and CNS penetration
- Applicable to targets in oncology, immunology, and acute kidney injury
- Lead program (RAP-0001) targeting ENT1 for the prevention and treatment of acute kidney injury