Oligonucleotide-based therapeutics

Overproduction of proteins can lead to the development of many human diseases.

Oligonucleotide-based therapeutics, drugs designed on the basis of DNA/RNA sequence information, specifically and dramatically decrease the level of disease-associated proteins, including targets once considered un-druggable.

Complementary to the RNA encoding for a given protein, oligonucleotide therapeutics such as antisense oligonucleotides (ASO), upon binding, can elicit RNA degradation with consequent inhibition of the target protein expression.

Within the OLIGONUCLEOTIDES PLATFORM, Nogra Pharma works on three fronts:

  • Identification of target proteins where an increased level is associated with the development of Immune-Inflammatory-Mediated diseases.
  • Design and identification of oligonucleotides capable of down-regulating the expression of the target protein. This is achieved through in silico design of oligonucleotides complementary to the RNA encoding for the target protein, followed by nonclinical in vitro, ex vivo and in vivo screening.
  • Development of appropriate formulations aimed at delivering the oligonucleotide at the site of the disease.


Mongersen gastro-resistant delayed-release tablet for the oral treatment of Crohn’s Disease



Target ID

Lead ID & Preclinical

Formulation Development

Clinical Phase l

Clinical Phase lI

Clinical Phase lII





GED-0301 – Mongersen

GED-0301 (Mongersen) is an antisense oligonucleotide (ASO), complementary to Smad7 mRNA. Mongersen inhibits the expression of Smad7, a protein overexpressed in Inflammatory Bowel Diseases (IBD), thereby restoring the physiological immunosuppressive activity of TGF-β1 in the intestinal mucosa. For the oral treatment of Crohn’s Disease, Mongersen has been formulated as a gastro-resistant pH-dependent release tablet, designed to deliver the ASO in the terminal ileum and right colon (the areas of the intestine inflamed in the majority of Crohn’s Disease patients) and minimize systemic absorption.
Once delivered in the affected intestine, the ASO can elicit its pharmacological activity, by hybridization with Smad7 mRNA and decrease Smad7 protein levels.

New Oligonucleotides

NPD-1911 & NPD-1902

Status: Ongoing in vitro screening

Undisclosed Targets

Nogra Pharma is available to give comprehensive Project presentations, provide additional Project specific details/confidential information and discuss potential collaborations or out-licensing opportunities with interested companies.