Universitat de Lleida

New therapeutic candidate for Spinal Muscular Atrophy

Posted by Núria from Universitat de LleidaResponsive · Patents for licensing · Spain

Summary of the technology

We are looking for a licensing agreement with a company with expertise in the field of neurosciences. More particularly, we are willing to find a company involved in the development of therapeutic solutions for the rare disease Spinal Muscular Atrophy (SMA).

Universitat de Lleida

Description of the technology

Researchers in the University of Lleida have preclinical evidence for the positive effects of a calpain inhibitor in in vitro and in vivo models for SMA (severe model and mild model for SMA). When treated with this inhibitor, mice survival is substantially prolonged and the clinical course of the disease is improved by means of attenuating body weight loss and motor behavior (righting reflex and tube test). The treatment increases SMN protein level in motor neurons and also prevents neurite degeneration in SMA mutants MNs.

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease characterized by muscle atrophy and weakness. The disease generally manifests early in life and is the leading genetic cause of death in infants and toddlers. SMA is caused by defects in the Survival Motor Neuron 1 (SMN1) gene that encodes the SMN protein. The SMN protein is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons).

THE NEED

Disease modifying therapeutic approaches are currently aiming to increase SMN protein levels, using gene therapy to add an exogenous copy of SMN, or protecting motor neurons from degenerating. However, there is no effective treatment for SMA, particularly for the most severe forms (type I and type II).

SMA has generally been believed to affect as many as 10,000 to 25,000 children and adults in the United States, and therefore it is one of the most common rare diseases. One in 6,000 to one in 10,000 children are born with the disease. In Spain, there are around 60 new SMA diagnoses every year.

Spinraza© (Nusinersen, Biogen) is the only drug approved by the FDA and EMA, but there are other candidates in the pipeline. The SMA therapeutics pipeline is segmented into early stage candidates and late stage candidates under development, as Spinraza© long-term effects are still unknown.

Current development status

Experimental technologies - This project is currently in a preclinical stage of development. In vitro and in vivo assays have been performed with this potential therapeutic.

Desired business relationship

Patent licensing

Technology development

Intellectual property status

Patent already applied for
PCT/ES2018/070177

Technology Owner

Universitat de Lleida

Research & Technology Organization

Related keywords

  • Biological Sciences
  • Medicine, Human Health
  • Pharmaceutical Products / Drugs
  • Genetic Engineering / Molecular Biology
  • Microbiology Market
  • In vitro Testing, Trials Market
  • Medical Health related
  • Therapeutic
  • drug discovery
  • rare disease

About Universitat de Lleida

Research & Technology Organization from Spain

The Valorisation and Technology Transfer Unit (UViT) is the specialized unit for handling the IPR portfolio in the University of Lleida. The UViT works close to researchers in the fields of Agrifood, Biomedicine, Technology and Sustainability and Social Development to identify research results with market potential, to assess the community on the best IP protection strategy and to commercialise technologies through licensing-out agreements and the creation of technology-based companies.

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