Lacerta Therapeutics founders have made significant progress in identifying gene therapy solutions for central nervous system and lysosomal storage diseases. Lacerta is currently focusing on the following gene therapy pipeline:

  • Pompe Disease
  • Sanfilippo Syndrome Type B
  • Aromatic L-amino acid decarboxylase deficiency
  • Neurodegenerative Proteinopathies
  • Spinocerebellar Ataxias
  • Glioblastoma

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