Biotechnology company Arkuda Therapeutics recently acquired multi-million dollar funding to continue its research program targeted at increasing progranulin protein production in persons diagnosed with FTD-GRN.

Researchers at Arkuda are continuing to develop their lead program to correct progranulin deficiencies that lead to neurodegenerative diseases, including genetic forms of FTD. Arkuda’s program will specifically target FTD-GRN, an inherited form of FTD stemming from a mutation in one of the copies of the GRN gene. The gene provides instructions for making progranulin, a protein that is involved in cell survival and the regulation of inflammation. FTD-causing variants in GRN lead to reduced progranulin levels.

Secondo a Feb. 10 article In MedCity News, Arkuda’s program is developing small molecules that get the healthy copy of the GRN gene to produce more progranulin. By correcting the protein deficiency, the intended goal is to restore the ability of lysosomes — the “digestive system” of cells — to clear neurons of excess materials.

Gerhard Koenig, Arkuda’s co-founder, president, and CEO, said in a comunicato stampa that the financing will enable his team to “accelerate our efforts to turn the promise of our emerging understanding of lysosomal biology into effective medicines with the potential to positively impact the trajectory of their disease.”

Read the full MedCity News article Qui.