Therapeutic Discovery for Laminopathies

A leading biotechnology company with a focus on rare disease therapeutics is seeking academic institutions or industry partners who are performing therapeutic discovery in the area of laminopathies, with a particular interest in Dilated Cardiomyopathy (DCM). The team is seeking therapeutic hypotheses, drug targets and novel modalities that can address more than one organ dysfunction (e.g. heart, brain, skeletal muscle). 

Approaches of Interest: 

• Therapeutics addressing organ dysfunction associated with all types of laminopathies
• Approaches targeting genes encoding proteins of the nuclear lamina
• Therapeutics targeting downstream pathways of lamin mutations
• Oligonucleotides (e.g. siRNA, ASO, mRNA) and biologic modalities are preferred, small molecules are also of potential interest

Out of Scope: 

• AAV and other viral approaches 
• Cell therapy and DNA delivery approaches 
• Therapeutics aimed at managing pain or other symptoms of laminopathies 

Developmental Stages of Interest: 

• Opportunities from preclinical research and beyond will be considered, provided that there is strong proof of concept data 
• Opportunities with in vivo validation are of highest interest, although opportunities with strong in vitro validation will also be considered 

Submission Information: 

Submission of 200–300-word briefs is encouraged, along with any supplementary information e.g. relevant publications, patents or slide decks. The team encourages including the proposed next steps in developing the research towards commercialization. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

Opportunity for Collaboration: 

Our client is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include funded research collaborations and agreements or licencing of assets.