Therapeutic Discovery for Laminopathies

A leading biotechnology company specializing in rare disease therapeutics seeks partners for therapeutic discovery in laminopathies, particularly Dilated Cardiomyopathy (DCM). They aim to address multiple organ dysfunctions (e.g., heart, brain, skeletal muscle) through therapeutic hypotheses, drug targets, and novel modalities.

Approaches of interest:

• Therapeutics for organ dysfunction in all laminopathies
• Approaches targeting nuclear lamina protein-encoding genes
• Therapeutics for downstream pathways of lamin mutations
• Oligonucleotides (siRNA, ASO, mRNA) and biologics (small molecules also considered)

Out of scope:

• AAV and other viral approaches
• Cell therapy and DNA delivery approaches
• Therapeutics for pain management or symptom control

Developmental stage of interest: Preclinical research and beyond with strong proof of concept data. In vivo validation is highly preferred, but strong in vitro validation will be considered.