Pipeline
Pipeline
Gene therapies

Safe and effective delivery is clearly a challenge for many types of active agent nucleic acids in development. There have been more than 2,900 gene therapy clinical trials initiated since 1989, with viral vectors used in around 2/3rds of trials (Source: The Journal of Gene Medicine - http://www.abedia.com/wiley/vectors.php).

Viral transfection vectors have a number of drawbacks as they can trigger immune responses and present potential safety issues, while their production processes are complex and expensive. Using standard manufacturing processes, LAMELLASOME™ formulations offer the ability to customise composition and characteristics to specific nucleic acids, as well as remarkably low immunogenicity.

We believe that our LAMELLASOME™ technology will play a key role in realising the potential of many of the nucleic acids in development, including microRNAs, siRNAs, mRNAs and plasmid DNA, potentially revolutionising the treatment of numerous rare and intractable diseases.

Lamellar is advancing a pipeline of products based on our gene therapy platform with novel drug candidates LAMELLASOME™ CF-NA and LAMELLASOME™ IPF-NA being developed for the treatment of Cystic Fibrosis and Idiopathic Pulmonary Fibrosis respectively

LAMELLASOME technology has demonstrated several key advantages for the delivery of nucleic acids:

  • Highly efficient cell entry (>80% of vesicles associated with cell surface are taken into cells vs <20% for standard cationic systems)
  • Very low cell toxicity in contrast to other gene therapy approaches
  • Protects nucleic acids cargos from extracellular enzymatic degradation and nebulisation forces
  • Low inflammatory response in contrast to cationic lipidic systems.
  • Does not modify host DNA
  • Cutting-edge processing systems that markedly improve the drug loading efficiency of anionic LAMELLASOME™ vesicles

 

LAMELLASOME™ IPF-NA

Overview

A LAMELLASOME™ treatment that delivers microRNAs to stop or slow IPF and other Interstitial Lung Diseases. May be used in combination with IPF-Lamellasome. It will be delivered topically on a monthly basis to the lung to regulate the alveolar inflammatory response and disrupt the progressive chronic fibrosis.

 

  Characteristic Existing evidence Type
Efficacy LAMELLASOME™ IPF-NA The active agent within LAMELLASOME™ IPF-NA balances the regulation of fibrosis within the lung

Pre-clinical

Safety LAMELLASOME™ respiratory safety Non-clinical respiratory safety program on LAMELLASOME™ completed demonstrating an excellent safety and tolerability profile.

Pre-clinical

 

Competitive edge

Existing treatments of IPF have sub-optimal efficacy with continued loss of quality of life and a median survival of 2-3 years after diagnosis. The pathology of the disease is driven by progressive chronic fibrosis. LAMELLASOME™ IPF-NA regulates the alveolar inflammatory response by disrupting progressive chronic fibrosis offering a significant advance in efficacy over existing treatments:

  • Interrupts disease progression by delivering microRNAs known to modulate IPF fibrosis.
  • Safer and low immunogenicity in relation to viral gene therapy vectors.
  • Once monthly instilled or nebulised treatment, possibly in combination with IPF Lamellasome.

Development route

Idiopathic Pulmonary Fibrosis (IPF) qualifies as an orphan disease indication in both the EU & US. Consequently, LAMELLASOME™ IPF-NA is expected to benefit from an accelerated development program and expedited regulatory review.

 

LAMELLASOME™ CF-NA

Overview

LAMELLASOME™ CF-NA is genotype-agnostic treatment that corrects the basic defect in the CFTR chloride ion channel which causes Cystic Fibrosis. A nebulised once monthly CFTR treatment to address the basic genetic defect in all CF patients by delivering a working copy of the CFTR gene.

 

  Characteristic Existing evidence Type
Efficacy LAMELLASOME™ CF-NA efficacy Lamellar and the Roslin Institute have shown that LAMELLASOME™ CF-NA is uniquely effective in a validated model.

Pre-clinical

Safety LAMELLASOME™ respiratory safety Non-clinical respiratory safety program on LAMELLASOME™ completed demonstrating an excellent safety and tolerability profile.

Pre-clinical

  LAMELLASOME™ CF-NA individual components safety The individual components of CF-NA have been shown to be safe and well tolerated in human studies.

Clinical testing

 

Competitive edge

Cystic Fibrosis is caused by mutation of the CFTR gene of which there are believed to be around 2,000 types. LAMELLASOME™ CF-NA addresses this basic defect in the CFTR chloride ion channel irrespective of the mutation genotype. Existing CFTR modifying medications tend to be genotype specific treating only a proportion of the Cystic Fibrosis population. Additionally, some existing treatments have notable side-effects whereas LAMELLASOME™ has been proven to be safe and well tolerated in the lung to date. LAMELLASOME™ CF-NA promises to provide a safer and more effective treatment. LAMELLASOME™ CF-NA also takes advantage of LAMELLASOME™ biophysical mucus penetrating system for delivery of CFTR gene therapy to the target respiratory epithelial cells.

Development route

Cystic Fibrosis qualifies as an orphan disease indication in both the EU & US. This is anticipated to provide the benefit of an accelerated development program and expedited regulatory review.

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