Production of truncated immunoglobulins to induce cellular death

A novel therapeutic approach for the treatment of Multiple Myeloma and Lymphoproliferative disorders with an Antisense Drug.

Multiple Myeloma is a bone narrow disease characterized by the proliferation of a malignant B lymphocyte. This disease is still incurable despite the development of new therapeutic protocols including proteasome inhibitors and therapeutic antibodies. Antisense oligonucleotides have been studied since several years for modulates genomic expression and in most cases restore the functionality of an abnormal protein. In the original therapeutic approach described here after, the antisense oligonucleotides-mediated exon skipping strategy aims to induce the production of aberrantly rearranged immunoglobulins which cause cellular death by apoptosis.

Competitive Advantages

● Antisense drug in association with best in class treatments improves chemotherapy efficacy.

● Exon skipping is a new mechanism of action to treat multiple myeloma that meet one of the unmet needs for this disease.

● Exon skipping targets especially plasma cells and is expected to induce less side effects than usual chemotherapy.

Development Status

● In vitro POC on Multiple Myeloma cell lines and patients cells.

● In vitro POC for AL Amyloidosis (rare disease).

● In vitro validation of the synergic effect with existing

Chemotherapy

● In vivo POC on going

Business Opportunities

● New Adjuvant Treatment for Multiple Myeloma and other lymphoproliferative disorders like AL Amyloidosis (rare disease).

● New therapeutic solution for refractory Multiple Myeloma patients.

● First in class treatment : Antisense oligonucleotide inducing exon skipping is a new therapeutic strategy for B cell line disorders

● Strategy could be adapted to all B cell line disorders : allergies