Gene Therapy Approach

A Natural Evolution: Chaperones to Optimized ERT to Gene Therapy

Amicus’ Shift Towards Gene Therapy is Built Upon Years of Experience in Developing Genetic Medicines Designed to Deliver Deficient Proteins to Target Cells and Organelles

Pharmacologic Chaperones

Pharmacologic Chaperones

Next-Generation ERTs

Next-Generation ERTs

Gene Therapies

Gene Therapies

Validated Gene Therapy Platform

The core science for these neurological Lysosomal programs leverage the AAV technologies and platforms utilized in the neuromuscular space at NCH and have robust pre-clinical proof of concept for their application in Batten disease

  • Clinically validated AAV gene therapy approach by Dr. Brian Kaspar and team at NCH and Sanford
  • Preclinical safety and efficacy studies replicated across multiple diseases at NCH
  • Amicus applying platform to Multiple Types of Batten disease and other Neurologic LSDs

Amicus Approach: Engineered Transgenes for Optimal Cross-Correction

Amicus’ unique technologies for protein engineering in Gene Therapy represent a new major platform technology and a groundbreaking advancement in the field

Gene Therapy

Manufacturing and Process Science

Manufacturing and process science capabilities and capacity in Gene Therapy will be crucial for Amicus success. Our experience in complex biologics manufacturing and quality control provide a great competitive advantage here.

Manufacturing and Process Science
Manufacturing and Process Science
Manufacturing and Process Science
Manufacturing and Process Science

CMO Partnerships

Manufacturing Partnerships Secured with Industry Leading CMOs with Tech Transfer Process Underway

Manufacturing Partnerships