Preclinical research of Recombinant Human Laminin-111

Recombinant Human Laminin-111 (rhLAM-111) is being developed for two therapeutic areas

1] Protein therapy for LAM-211 deficiency – LAMA2 congenital muscular dystrophy (CMD), also known as MDC1A

2] Upregulation of utrophin and alpha7beta1 integrin for treatment of DMD

Questions and Answers

What stage is this research?

This research is pre-clinical, we are developing our manufacturing capability but have not yet advanced to clinical trials.

Where is this research being done and who is funding this research?

LAM-111 research is performed at the University of Nevada, Reno and Prothelia Inc. in Massachusetts among other laboratories in the US, Canada and EU

Prothelia is funded by our company founders, and previous grants from NIH and advocacy groups such as the PPMD End Duchenne GAP program, Struggle Against Muscular Dystrophy (SAM) and Hope for Gus.

What is the goal or purpose of this research?

Manufacture of rhLAM-111 and clinical testing in patients with MDC1A and DMD.

What is the current state of this research?

This project is in preclinical development and we have demonstrated effectiveness in the mdx and dyW mouse models of Duchenne and MDC1A, respectively.

Current efforts are focused on MDC1A and positive clinical data for MDC1A will accelerate clinical development for DMD.

rhLAM-111 will help all DMD patients regardless of their mutation.

rhLAM-111 has 2 mechanisms of action important for DMD

  1. Potential to stabilize skeletal and cardiac muscle through upregulation (increased expression) of modifier proteins alpha7beta1 integrin and utrophin.
  2. Potential to direct stimulation of satellite cells and more efficient muscle regeneration.

What steps need to be completed before moving into a clinical trial?

  • Increase the scale of manufacturing
  • Pre-IND testing

Where would a clinical trial take place?

We have not made a decision but we anticipate clinical testing to occur at multiple sites.

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