New method for treating autoimmune diseases

 

112119
   
Technology Overview

Dr. Laura Conforti has discovered a way that uses lipid-derived nanovesicles functionalized with antibodies against membrane proteins expressed only on the surfaces of the subset of immune cells involved in the pathology to deliver small interfering RNAs (siRNAs) that knock down ion channels on these cells, inhibiting their immune activity and resulting in selective immunosuppression. Her invention for treating autoimmune disease provides a desired solution which eludes many therapies targeting in immune effector cells in that this approach will suppress the subset of immune cells responsible for progression of the disease without altogether shutting down the immune system, which is needed to fight acute infection. The invention promises to provide the basis for novel therapeutic agents in autoimmunity with limited side effects.

Dr. Conforti's group has developed lipid nanoparticles (NP) that target only a subset of T cells (memory T cells) which are critical players in the pathophysiology of SLE. These nano vesicles contain small interfering RNAs (siRNA, short double stranded RNA molecules that can be used to knock down a specific gene) against an ion channel (Kvl.3) that controls T cell function (Kvl.3-NP). Blockade of Kvl.3 suppresses immune cell function. They have fabricated these Kvl.3-NPs and tested them in vitro in human T cells.

Background

Up to 50 million Americans have autoimmune diseases such as multiple sclerosis, diabetes, lupus and rheumatoid arthritis.  Some anticipated clinical applications of this Kvl.3 ion channel targeting therapy include:
1. SLE
2. Rheumatoid arthritis
3. Multiple sclerosis
4. Crohn's disease
5. Psoriasis
6. Myasthenia gravis
7. Prevention of organ transplant rejection

The inventors have done research in SLE models and are planning work to investigate application to lupus nephritis. SLE affects about 1.5 million Americans and about 90% of persons with diagnosed SLE are women. Prevalence cases of lupus nephritis in the USA in 2016 are estimated to be 58,000. The health costs for lupus nephritis are significant ($33,500/patient/year); higher than patients with SLE.

Considering the broader universe of up to eighty autoimmune diseases as possible approachable indications these affect 50 million in the US and nearly the same abroad. Treatment costs are usually in the excess of $120 billion/year with 250,000 new cases diagnosed each year. Additionally, since immunosuppressive agents are indicated for patients who underwent an organ transplant, this market segment of $5 billion (as of 2007) within the overall $57 billion global sales of immunosuppressant in 2014 - with a CAGR of 6.4% from 2009 to 2014, could be nice upsides prompting further development interest for this UC platform technology.

The inventor's discovery also fits within the global market for RNAi drug delivery technologies (including RNAi drug delivery, RNAi-based therapeutics, and RNAi-based research applications) which reached nearly $9.3 billion in 2012 and was valued at approximately $11.7 billion in 2013. This market segment is expected to grow to $38.8 billion by 2018.

Advantages and Benefits
  • Selective immunosuppression
  • Broad clinical applications
  • Specificity to memory T Cells
  •  
  •  
  •  
Patents
Title Country Serial No. Patent No. File Date Issued Date
Methods and Compositions for Treating Autoimmune Disorders by Targeting Kv1.3 Ion Channels with Functionalized Lipid-Derived Nanovesicles United States 14/863,525 9,777,279 9/24/2015 10/3/2017
Methods and Compositions for Treating Autoimmune Disorders by Targeting Kv1.3 Ion Channels with Functionalized Lipid-Derived Nanovesicles United States 15/683,180 10,308,942 8/22/2017 6/4/2019
Other Media
Inventor(s)
Contact
Jill Uhl
Sr. Licensing Associate, Life Sciences
Lead Inventor
Laura Conforti
Keywords
Autoimmune Diseases Treatment, Healthcare, Kv1.3 Ion Channel Targeting, Lipid-Derived Nanovesicles, RNAi Drug Delivery,
Download as PDF
CONTACT US