Italian biotech Aptadir Therapeutics has launched with the promise that its pipeline of preclinical RNA inhibitors could crack intractable cancers.
The Milan-based company was founded by RNA leaders Annalisa Di Ruscio, M.D., Ph.D., of Harvard Medical School's Beth Israel Deaconess Medical Center and Vittorio De Franciscis, Ph.D., of the Italian Research National Council alongside leukemia expert Daniel Tenen, M.D., of the Cancer Science Institute of Singapore and oligonucleotide-focused Marcin Kortylewski, Ph.D., of the City of Hope National Medical Center.
At the center of this joint venture is a new class of RNA inhibitors called DNMTs interacting RNAs (DiRs), which are able to block aberrant DNA methylation at a single gene level. The theory is that this reactivates previously hypermethylated genes, considered to be a key feature in cancers as well as genetic disorders.
Reactivating specific genes offers the hope of reversing cancers and genetic conditions for which there are either no or limited curative options, such as the blood cancer myelodysplastic syndrome (MDS) in adults and the neurodevelopmental disorder fragile X syndrome in children.
Aptadir is hoping to get the most advanced of its DiRs, a MDS-focused candidate dubbed Ce-49, into clinical trials by the end of 2025. To help reach this milestone, the biotech has received $1.6 million in pre-seed funding from the Italian National Technology Transfer Hub’s EXTEND initiative. The hub was set up Italian VC manager CDP Venture Capital SGR.
Aptadir is the first biotech to come out the EXTEND initiative, which is partly funded by Rome-based VC firm Angelini Ventures as well as German biotech Evotec.
EXTEND’s goal is to “develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients,” CDP Venture Capital’s Claudia Pingue explained in the release.
Giovanni Amabile, entrepreneur in residence of EXTEND, has been appointed CEO of Aptadir, having previously helmed autoimmune biotech Enthera.
“Aptadir’s business is based on real innovation—a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions,” Amabile said in a Sept. 24 release.
“From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications,” Amabile added. “This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.”