The lysosome is not just the recycling center of the cell

For decades, Drug Discovery has focused on plasma membrane receptors and channels. But some of the most compelling therapeutic targets of the next generation sit deeper, inside, on the lysosomal membrane.

𝐇𝐞𝐫𝐞 𝐚𝐫𝐞 𝐤𝐞𝐲 𝐥𝐲𝐬𝐨𝐬𝐨𝐦𝐚𝐥 𝐭𝐚𝐫𝐠𝐞𝐭𝐬 𝐠𝐚𝐢𝐧𝐢𝐧𝐠 𝐭𝐫𝐚𝐜𝐭𝐢𝐨𝐧 𝐢𝐧 𝐭𝐡𝐞 𝐟𝐢𝐞𝐥𝐝:

✔️ 𝐓𝐑𝐏𝐌𝐋𝟏 – Ca²⁺ release channel. Linked to Parkinson’s disease and lysosomal storage disorders. First TRPML1 agonist now in Phase 1 clinical trial.

✔️ 𝐆𝐁𝐀𝟏 – Lysosomal enzyme glucocerebrosidase. The most common genetic risk factor for Parkinson’s disease. Multiple small molecule programs now in Phase 1b.

✔️ 𝐓𝐌𝐄𝐌𝟏𝟕𝟓 – K⁺/H⁺ channel and lysosomal pH gatekeeper. Loss-of-function mutations impair protein clearance and accelerate neurodegeneration. Selective modulators in active development.

✔️ 𝐓𝐏𝐂𝟏/𝐓𝐏𝐂𝟐 – Na⁺/Ca²⁺ channels. Implicated in neurodegeneration, cancer, and rare diseases.

✔️ 𝐂𝐋𝐂-𝟕 – Cl⁻/H⁺ transporter. Critical for lysosomal acidification. Loss-of-function mutations cause bone and neurological disorders.

And many others…

𝐒𝐨 𝐰𝐡𝐲 𝐚𝐫𝐞𝐧’𝐭 𝐭𝐡𝐞𝐬𝐞 𝐬𝐭𝐮𝐝𝐢𝐞𝐝 𝐦𝐨𝐫𝐞 𝐫𝐨𝐮𝐭𝐢𝐧𝐞𝐥𝐲?
Because lysosomes were difficult to access, isolate, and record from in a reproducible, scalable way.
That is precisely what ORIA Bioscience enables!

Each of these targets are available and can be expressed in our 𝐋𝐘𝐒𝐎-𝐏𝐫𝐞𝐩™ lysosomes, at expression levels tailored for your research needs.