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Scholar Rock Aims to Hit Disease-Causing Proteins in a “Niche”

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freely floating around in the body. Most of them are sitting “latently in depots” waiting to be activated. Springer, over the past few years, has been working to identify the mechanisms by which this happens. As it turns out, these growth factors can be switched on sometimes by other molecules, or “cofactors,” that are either on the surface of nearby cells, or floating in the extracellular matrix (the fluid between cells), he says.

Scholar’s plan, then, is to engineer drug candidates to “tweak” certain dormant growth factors—wake them up, or keep them sleeping—indirectly, by targeting these complexes of proteins. If a disease is caused by a deficiency of a specific growth factor, for instance, Scholar would try to switch it on. If a growth factor is inappropriately activated in a certain disease, Scholar would try to preemptively keep it dormant. These experimental drugs—likely initially antibodies, though Scholar may later try small molecules as well—would be Scholar’s so-called “niche” activators or inhibitors.

Scholar isn’t saying specifically which growth factors it’s looking at as of yet. It’s homing in on areas of disease biology where specific growth factors have a “very clear and known role,” but where systemically activating or inhibiting those growth factors hasn’t worked out well—either because of side effects at potent doses, or ineffectiveness at tolerable doses.

“That’s what creates the window of opportunity for [us], where we can come in with our therapeutic candidates and get a very potent and selective advantage in areas where the biology is well known,” he says.

For now, Scholar is narrowing that down to fibrotic, autoimmune and musculoskeletal diseases, though it won’t say which ones.

Still, it’s very early in the game. Scholar has worked in stealth over the past year figuring out which specific growth factors it’s interested in and exactly which complexes of proteins it should target to affect them, and making sure it can engineer those protein groups to test its scientific hypotheses. It’s also been developing assays to help it see if it can get the results it’s looking for. Just now, Scholar is moving on to the drug discovery phase.

Even so, Mahanthappa is talking boldly. Over the course of the year, Scholar plans to roughly double the size of its team—chiefly adding R&D positions— and gather initial proof-of-concept data. It’ll likely raise a Series A round to take its next step, but Mahanthappa wants to ink a partnership to validate the company’s approach before then.

“That’s the goal,” he says. “We have some very exciting discussions ongoing and I expect that some near-term partnering activity, in conjunction with the work that we’ve done with the seed funding, is going to create a very strong foundation for us before we actively seek the next round of financing.”

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