Breakthrough in Gene Therapy: Andelyn Biosciences and Elly's Team Make History

Andelyn Biosciences, a leading Contract Development and Manufacturing Organization (CDMO), has successfully manufactured a novel viral vector gene therapy using its AAV Curator Platform to treat a baby with a rare neurodegenerative disease. The therapy was produced and released just 10 months from the start of development, a significant achievement in the field of gene therapy. Elly Krueger, the 8-month-old patient, was diagnosed with NEDAMSS, a disease with no known cures or treatments, and had been rapidly progressing. Her parents, Michelle and the team at Elly's Team, a faith-based foundation dedicated to finding treatments for NEDAMSS, worked closely with Andelyn Biosciences to develop and manufacture the gene therapy.

Key Takeaways:

• Andelyn Biosciences successfully manufactured a novel viral vector gene therapy using its AAV Curator Platform to treat a baby with NEDAMSS, a rare neurodegenerative disease.
• The therapy was produced and released just 10 months from the start of development, a significant achievement in the field of gene therapy.
• Elly Krueger, the 8-month-old patient, had been rapidly progressing with NEDAMSS, a disease with no known cures or treatments.
• The AAV Curator Platform utilizes Optimization-by-Design, a data-driven approach to correctly size processes for scale and speed to the clinic.
• The platform is highly predictable in terms of yield and quality, eliminating intellectual property concerns and streamlining review of investigational new drug (IND) applications.
• Andelyn Biosciences has over 20 years of experience in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials.

Statistics:

• 10 months: time from start of development to therapy production for Elly Krueger's case
• 8 months: the age of Elly Krueger when diagnosed with NEDAMSS
• 450 clinical batches: number of batches produced by Andelyn Biosciences
• 75 global clinical trials: number of trials supported by Andelyn Biosciences

Sources:

• Andelyn Biosciences, Inc.
• Elly's Team